Commentary

Over the last decade, three series published in the Lancet on child development have increased global awareness of the importance of early brain development and highlighted the critical role nurturing care plays in the first 3 years of a child’s life. Yet, as practitioners and policy makers in low- and middle-income countries increasingly acknowledge the influence early development has on later developmental, educational, and socioeconomic trajectories, there has been less agreement regarding the most appropriate methods and measures for screening and monitoring child development over time.

Mr. Small is with the Oregon Research Institute, Eugene. Dr. Hix-Small is with Portland (Ore.) State University. The authors have stated that they had no interests that might be perceived as posing a conflict or bias. Dr. Hix-Small has worked as a paid ASQ trainer. Email them at [email protected].

Over the last decade, three series published in the Lancet on child development have increased global awareness of the importance of early brain development and highlighted the critical role nurturing care plays in the first 3 years of a child’s life. Yet, as practitioners and policy makers in low- and middle-income countries increasingly acknowledge the influence early development has on later developmental, educational, and socioeconomic trajectories, there has been less agreement regarding the most appropriate methods and measures for screening and monitoring child development over time.

Mr. Small is with the Oregon Research Institute, Eugene. Dr. Hix-Small is with Portland (Ore.) State University. The authors have stated that they had no interests that might be perceived as posing a conflict or bias. Dr. Hix-Small has worked as a paid ASQ trainer. Email them at [email protected].

Over the last decade, three series published in the Lancet on child development have increased global awareness of the importance of early brain development and highlighted the critical role nurturing care plays in the first 3 years of a child’s life. Yet, as practitioners and policy makers in low- and middle-income countries increasingly acknowledge the influence early development has on later developmental, educational, and socioeconomic trajectories, there has been less agreement regarding the most appropriate methods and measures for screening and monitoring child development over time.

Mr. Small is with the Oregon Research Institute, Eugene. Dr. Hix-Small is with Portland (Ore.) State University. The authors have stated that they had no interests that might be perceived as posing a conflict or bias. Dr. Hix-Small has worked as a paid ASQ trainer. Email them at [email protected].

As a physician you are the embodiment of delayed gratification. You spent more than 20 years in school before you earned a degree that then allowed you spend another 3-plus years in training before anyone would consider you a “real” doctor. Somewhere along that long and shallow trajectory someone may have said, “You must have done really well on the marshmallow test.”

Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Email him at [email protected].

As a physician you are the embodiment of delayed gratification. You spent more than 20 years in school before you earned a degree that then allowed you spend another 3-plus years in training before anyone would consider you a “real” doctor. Somewhere along that long and shallow trajectory someone may have said, “You must have done really well on the marshmallow test.”

Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Email him at [email protected].

As a physician you are the embodiment of delayed gratification. You spent more than 20 years in school before you earned a degree that then allowed you spend another 3-plus years in training before anyone would consider you a “real” doctor. Somewhere along that long and shallow trajectory someone may have said, “You must have done really well on the marshmallow test.”

Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Email him at [email protected].

The number of births in the United States has fallen for the second year in a row. Births in 2017 were down 2% from 2016, bringing the birth rate to a 30-year low at 60 births per 1,000 women aged 15-44 years. This decline spanned nearly all maternal age groups, including teenagers (Hamilton et al. NVSS Vital Statistics Rapid Release Report No 004, May 2018).

In the crudest terms, babies represent the raw material of pediatrics. Without children, we pediatricians would have to begin treating those whining adults. Most of us chose pediatrics because we enjoy being around children, and many of us were motivated to study medicine by a desire to help sick children. Is this decline in the supply stream of patients something we should be worrying about?

gpointstudio/Thinkstock

AleksandarNakic/E+/Getty Images

You can worry about job security if you like, but in a quirky kind of way pediatrics is following along its own rules of supply and demand. And, you should rest easy ... that is until the next panicked call from a parent wakes you in the middle of the night.


 

Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Email him at [email protected].

The number of births in the United States has fallen for the second year in a row. Births in 2017 were down 2% from 2016, bringing the birth rate to a 30-year low at 60 births per 1,000 women aged 15-44 years. This decline spanned nearly all maternal age groups, including teenagers (Hamilton et al. NVSS Vital Statistics Rapid Release Report No 004, May 2018).

In the crudest terms, babies represent the raw material of pediatrics. Without children, we pediatricians would have to begin treating those whining adults. Most of us chose pediatrics because we enjoy being around children, and many of us were motivated to study medicine by a desire to help sick children. Is this decline in the supply stream of patients something we should be worrying about?

gpointstudio/Thinkstock

AleksandarNakic/E+/Getty Images

You can worry about job security if you like, but in a quirky kind of way pediatrics is following along its own rules of supply and demand. And, you should rest easy ... that is until the next panicked call from a parent wakes you in the middle of the night.


 

Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Email him at [email protected].

The number of births in the United States has fallen for the second year in a row. Births in 2017 were down 2% from 2016, bringing the birth rate to a 30-year low at 60 births per 1,000 women aged 15-44 years. This decline spanned nearly all maternal age groups, including teenagers (Hamilton et al. NVSS Vital Statistics Rapid Release Report No 004, May 2018).

In the crudest terms, babies represent the raw material of pediatrics. Without children, we pediatricians would have to begin treating those whining adults. Most of us chose pediatrics because we enjoy being around children, and many of us were motivated to study medicine by a desire to help sick children. Is this decline in the supply stream of patients something we should be worrying about?

gpointstudio/Thinkstock

AleksandarNakic/E+/Getty Images

You can worry about job security if you like, but in a quirky kind of way pediatrics is following along its own rules of supply and demand. And, you should rest easy ... that is until the next panicked call from a parent wakes you in the middle of the night.


 

Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Email him at [email protected].

Once just a theory, gene therapies are now a therapeutic reality for some patients. These platforms may have the potential to treat and cure some of our most intractable and vexing diseases. The policy framework we construct for how these products should be developed, reviewed by regulators, and reimbursed, will help set the stage for the continued advancement of this new market. Last year, we announced our comprehensive policy framework for regenerative medicine, including a draft guidance that describes the expedited programs, such as the breakthrough therapy designation, and the regenerative medicine advanced therapy (RMAT) designation, that may be available to sponsors of these therapies. Today, we’re unveiling a complementary framework for the development, review and approval of gene therapies.

In the past 12 months, we’ve seen three separate gene therapy products approved by the FDA. This reflects the rapid advancements in this field. An inflection point was reached with the development of vectors that could reliably deliver gene cassettes in vivo, into cells and human tissue. In the future, we expect this field to continue to expand, with the potential approval of new treatments for many debilitating diseases. These therapies hold great promise. Our new steps are aimed at fostering developments in this innovative field.

Gene therapies are being studied in many areas, including genetic disorders, autoimmune diseases, heart disease, cancer and HIV/AIDS. We look forward to working with the academic and research communities to make safe and effective products a reality for more patients. But we know that we still have much to learn about how these products work, how to administer them safely, and whether they will continue to work properly in the body without causing adverse side effects over long periods of time. In contrast to traditional drug review, some of the more challenging questions when it comes to gene therapy relate to product manufacturing and quality, or questions about the durability of response, which often can’t be fully answered in any reasonably sized pre-market trial. For some of these products, we may need to accept some level of uncertainty around these questions at the time of approval. For example, in some cases the long-term durability of the effect won’t be fully understood at the time of approval. Effective tools for reliable post-market follow up, such as required post-market clinical trials, are going to be one key to advancing this field and helping to ensure that our approach fosters safe and innovative treatments.

Even when there may be uncertainty about some questions, we need to make certain we assure patient safety and adequately characterize the potential risks and demonstrated benefits of these products. In part because of the added questions that often surround a new technology like gene therapy, these products are initially being aimed at devastating diseases, many of which lack available therapies, including some diseases that are fatal. In such cases of devastating diseases without available therapies, we’ve traditionally been willing to accept more uncertainty to facilitate timely access to promising therapies. In such cases, drug sponsors are generally required to conduct post-marketing clinical trials, known as phase 4 confirmatory trials, to confirm clinical benefit of the drug. This is the direction Congress gave the FDA by creating vehicles like the accelerated approval pathway.

When it comes to novel technologies like gene therapy, the FDA is steadfastly committed to a regulatory path that maintains the agency’s gold standard for assuring safety and efficacy. As we develop this evidence-based framework, we’re going to have to modernize how we approach certain aspects of these products in order to make sure our approach is tailored to the unique challenges created by these new platforms.

Today, we’re taking a step toward shaping this modern structure for the regulation of gene therapy. The agency is issuing a suite of six scientific guidance documents intended to serve as the building blocks of a modern, comprehensive framework for how we’ll help advance the field of gene therapy while making sure new products meet the FDA’s gold standard for safety and effectiveness.

These policies are part of our efforts to communicate the steps we’re taking to provide clear recommendations to sponsors and researchers, so that we can better support innovation. The documents are being issued in draft form so that we can solicit public input on these new policies. As with all draft guidances, all of the comments we receive will be carefully considered prior to finalizing these documents. We’re committed to working with stakeholders to bring novel treatments to the market while ensuring the safety of patients.

Disease-Specific Gene Therapy Guidances

The FDA has issuing three new draft guidance documents on the development of gene therapy products for specific disease categories. These are the first three disease-specific guidances that the agency is issuing for gene therapy products. Our new commitment to develop disease-specific guidance documents reflects the increasing activity in this field, and its growing importance to advancing public health.

Human Gene Therapy for Hemophilia: Gene therapy products for hemophilia are now being developed as single-dose treatments that may enable long-term production of the missing or abnormal coagulation factor in patients. This may reduce or eliminate the need for coagulation factor replacement. To define the proper development pathway for such products, we’re issuing a new draft guidance on gene therapy products that are targeted to the treatment of hemophilia. Once finalized, this new guidance will provide recommendations on the FDA’s current thinking on clinical trial design and preclinical considerations to support the development of these gene therapy products. Among other elements, the draft guidance provides recommendations regarding surrogate endpoints that could be used by sponsors pursuing accelerated approval of gene therapy products that are intended for treatment of hemophilia.

Human Gene Therapy for Retinal Disorders: Another area of fast-paced activity is gene therapy products targeted to the treatment of retinal disorders. The Human Gene Therapy for Retinal Disorders guidance, once finalized, will assist those developing gene therapy products for a wide variety of retinal disorders affecting both adult and pediatric patients. Gene therapy products currently undergoing clinical trials in the United States for retinal disorders are commonly delivered by intravitreal injections (into the fluid portion of the eye), or by subretinal injections (beneath the retina). In some cases, the gene therapy products are encapsulated in a device to be implanted within the eye. This new guidance document will focus on issues that are specific to gene therapies for retinal disorders. The document provides recommendations related to product development, preclinical testing, and clinical trial design for such products.

Human Gene Therapy for Rare Diseases: Rare diseases are those that affect fewer than 200,000 people in the United States. The National Institutes of Health reports that nearly 7,000 rare diseases affect more than 25 million Americans. About 80 percent of rare diseases are caused by a single-gene defect, and about half of all rare diseases affect children. Since most rare diseases have no approved therapies, there is a significant unmet need. The Human Gene Therapy for Rare Diseases guidance, once finalized, will provide recommendations on preclinical, manufacturing and clinical trial design for all phases of the clinical development program for these types of gene therapies. The information is intended to assist sponsors in the design of clinical development programs, where there may be limited study population size, potential feasibility and safety issues, as well as issues relating to the interpretation of effectiveness.

Guidances on Manufacturing Gene Therapies

The FDA is also providing new and comprehensive updates to three existing guidances that address manufacturing issues related to gene therapy. These updates reflect input from many stakeholders. We encourage additional feedback on these documents during the comment period.

The first draft guidance, Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs), provides sponsors with recommendations on how to provide sufficient CMC information to assure safety, identity, quality, purity and strength/potency of investigational gene therapy products. The guidance applies to human gene therapies and to combination products that contain a human gene therapy in combination with a drug or device. In addition, this guidance is organized to follow the structure of the FDA guidance on the Common Technical Document.

The second draft guidance, Testing of Retroviral Vector-Based Gene Therapy Products for Replication Competent Retrovirus (RCR) during Product Manufacture and Patient Follow-up, provides additional recommendations regarding the proper testing for RCR during the manufacture of retroviral vector-based gene therapy products, as well as during the follow-up monitoring of patients who’ve received retroviral vector-based gene therapy products. Specifically, the draft guidance recommends the identification and amount of material to be tested. The guidance also provides advice on general testing methods.

The third draft guidance, Long Term Follow-Up After Administration of Human Gene Therapy Products, provides recommendations regarding the design of long-term follow-up (LTFU) observational studies for the collection of data on delayed adverse events following administration of a gene therapy product. Because of some of the additional uncertainty intrinsic to a novel platform like gene therapy -- including questions related to the durability of the treatment effects as well as the theoretical potential for off-target effects if the genes do not insert correctly -- there’s an increased need for robust long-term follow-up of patients in the post-market period. This guidance describes product characteristics, patient-related factors, and the preclinical and clinical data that should be considered when assessing the need for LTFU observations and describes the features related to effective post-market follow up.

Once finalized, these draft guidances will replace previous guidances issued by the FDA in April 2008 (CMC) and November 2006 (RCR and LTFU).

The field of gene therapy has progressed rapidly since these guidances were first issued. Therefore, the FDA is updating these guidances to provide sponsors with the agency’s most up-to-date thinking.

Our goal is to help promote safe and effective product development in this field. We’ll continue to work with the product sponsors to help make the development and approval of these innovative gene therapies more efficient, while putting in place the regulatory controls needed to ensure that the resulting therapies are both safe and effective. We’ll also make full use of our expedited programs such as breakthrough therapy designation and regenerative medicine advanced therapy designation whenever possible.

Gene therapy represents one of the most promising opportunities for developing highly effective and even curative treatments for many vexing disorders. Some of these products are almost certainly going to change the contours of medical practice, and the destiny of patients with some debilitating diseases.

The FDA’s goal is to help these innovations advance in a framework that assures the safety and effectiveness of these resulting treatments, and continues to build peoples’ confidence in this novel area of medicine.

–Scott Gottlieb, MD

Once just a theory, gene therapies are now a therapeutic reality for some patients. These platforms may have the potential to treat and cure some of our most intractable and vexing diseases. The policy framework we construct for how these products should be developed, reviewed by regulators, and reimbursed, will help set the stage for the continued advancement of this new market. Last year, we announced our comprehensive policy framework for regenerative medicine, including a draft guidance that describes the expedited programs, such as the breakthrough therapy designation, and the regenerative medicine advanced therapy (RMAT) designation, that may be available to sponsors of these therapies. Today, we’re unveiling a complementary framework for the development, review and approval of gene therapies.

In the past 12 months, we’ve seen three separate gene therapy products approved by the FDA. This reflects the rapid advancements in this field. An inflection point was reached with the development of vectors that could reliably deliver gene cassettes in vivo, into cells and human tissue. In the future, we expect this field to continue to expand, with the potential approval of new treatments for many debilitating diseases. These therapies hold great promise. Our new steps are aimed at fostering developments in this innovative field.

Gene therapies are being studied in many areas, including genetic disorders, autoimmune diseases, heart disease, cancer and HIV/AIDS. We look forward to working with the academic and research communities to make safe and effective products a reality for more patients. But we know that we still have much to learn about how these products work, how to administer them safely, and whether they will continue to work properly in the body without causing adverse side effects over long periods of time. In contrast to traditional drug review, some of the more challenging questions when it comes to gene therapy relate to product manufacturing and quality, or questions about the durability of response, which often can’t be fully answered in any reasonably sized pre-market trial. For some of these products, we may need to accept some level of uncertainty around these questions at the time of approval. For example, in some cases the long-term durability of the effect won’t be fully understood at the time of approval. Effective tools for reliable post-market follow up, such as required post-market clinical trials, are going to be one key to advancing this field and helping to ensure that our approach fosters safe and innovative treatments.

Even when there may be uncertainty about some questions, we need to make certain we assure patient safety and adequately characterize the potential risks and demonstrated benefits of these products. In part because of the added questions that often surround a new technology like gene therapy, these products are initially being aimed at devastating diseases, many of which lack available therapies, including some diseases that are fatal. In such cases of devastating diseases without available therapies, we’ve traditionally been willing to accept more uncertainty to facilitate timely access to promising therapies. In such cases, drug sponsors are generally required to conduct post-marketing clinical trials, known as phase 4 confirmatory trials, to confirm clinical benefit of the drug. This is the direction Congress gave the FDA by creating vehicles like the accelerated approval pathway.

When it comes to novel technologies like gene therapy, the FDA is steadfastly committed to a regulatory path that maintains the agency’s gold standard for assuring safety and efficacy. As we develop this evidence-based framework, we’re going to have to modernize how we approach certain aspects of these products in order to make sure our approach is tailored to the unique challenges created by these new platforms.

Today, we’re taking a step toward shaping this modern structure for the regulation of gene therapy. The agency is issuing a suite of six scientific guidance documents intended to serve as the building blocks of a modern, comprehensive framework for how we’ll help advance the field of gene therapy while making sure new products meet the FDA’s gold standard for safety and effectiveness.

These policies are part of our efforts to communicate the steps we’re taking to provide clear recommendations to sponsors and researchers, so that we can better support innovation. The documents are being issued in draft form so that we can solicit public input on these new policies. As with all draft guidances, all of the comments we receive will be carefully considered prior to finalizing these documents. We’re committed to working with stakeholders to bring novel treatments to the market while ensuring the safety of patients.

Disease-Specific Gene Therapy Guidances

The FDA has issuing three new draft guidance documents on the development of gene therapy products for specific disease categories. These are the first three disease-specific guidances that the agency is issuing for gene therapy products. Our new commitment to develop disease-specific guidance documents reflects the increasing activity in this field, and its growing importance to advancing public health.

Human Gene Therapy for Hemophilia: Gene therapy products for hemophilia are now being developed as single-dose treatments that may enable long-term production of the missing or abnormal coagulation factor in patients. This may reduce or eliminate the need for coagulation factor replacement. To define the proper development pathway for such products, we’re issuing a new draft guidance on gene therapy products that are targeted to the treatment of hemophilia. Once finalized, this new guidance will provide recommendations on the FDA’s current thinking on clinical trial design and preclinical considerations to support the development of these gene therapy products. Among other elements, the draft guidance provides recommendations regarding surrogate endpoints that could be used by sponsors pursuing accelerated approval of gene therapy products that are intended for treatment of hemophilia.

Human Gene Therapy for Retinal Disorders: Another area of fast-paced activity is gene therapy products targeted to the treatment of retinal disorders. The Human Gene Therapy for Retinal Disorders guidance, once finalized, will assist those developing gene therapy products for a wide variety of retinal disorders affecting both adult and pediatric patients. Gene therapy products currently undergoing clinical trials in the United States for retinal disorders are commonly delivered by intravitreal injections (into the fluid portion of the eye), or by subretinal injections (beneath the retina). In some cases, the gene therapy products are encapsulated in a device to be implanted within the eye. This new guidance document will focus on issues that are specific to gene therapies for retinal disorders. The document provides recommendations related to product development, preclinical testing, and clinical trial design for such products.

Human Gene Therapy for Rare Diseases: Rare diseases are those that affect fewer than 200,000 people in the United States. The National Institutes of Health reports that nearly 7,000 rare diseases affect more than 25 million Americans. About 80 percent of rare diseases are caused by a single-gene defect, and about half of all rare diseases affect children. Since most rare diseases have no approved therapies, there is a significant unmet need. The Human Gene Therapy for Rare Diseases guidance, once finalized, will provide recommendations on preclinical, manufacturing and clinical trial design for all phases of the clinical development program for these types of gene therapies. The information is intended to assist sponsors in the design of clinical development programs, where there may be limited study population size, potential feasibility and safety issues, as well as issues relating to the interpretation of effectiveness.

Guidances on Manufacturing Gene Therapies

The FDA is also providing new and comprehensive updates to three existing guidances that address manufacturing issues related to gene therapy. These updates reflect input from many stakeholders. We encourage additional feedback on these documents during the comment period.

The first draft guidance, Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs), provides sponsors with recommendations on how to provide sufficient CMC information to assure safety, identity, quality, purity and strength/potency of investigational gene therapy products. The guidance applies to human gene therapies and to combination products that contain a human gene therapy in combination with a drug or device. In addition, this guidance is organized to follow the structure of the FDA guidance on the Common Technical Document.

The second draft guidance, Testing of Retroviral Vector-Based Gene Therapy Products for Replication Competent Retrovirus (RCR) during Product Manufacture and Patient Follow-up, provides additional recommendations regarding the proper testing for RCR during the manufacture of retroviral vector-based gene therapy products, as well as during the follow-up monitoring of patients who’ve received retroviral vector-based gene therapy products. Specifically, the draft guidance recommends the identification and amount of material to be tested. The guidance also provides advice on general testing methods.

The third draft guidance, Long Term Follow-Up After Administration of Human Gene Therapy Products, provides recommendations regarding the design of long-term follow-up (LTFU) observational studies for the collection of data on delayed adverse events following administration of a gene therapy product. Because of some of the additional uncertainty intrinsic to a novel platform like gene therapy -- including questions related to the durability of the treatment effects as well as the theoretical potential for off-target effects if the genes do not insert correctly -- there’s an increased need for robust long-term follow-up of patients in the post-market period. This guidance describes product characteristics, patient-related factors, and the preclinical and clinical data that should be considered when assessing the need for LTFU observations and describes the features related to effective post-market follow up.

Once finalized, these draft guidances will replace previous guidances issued by the FDA in April 2008 (CMC) and November 2006 (RCR and LTFU).

The field of gene therapy has progressed rapidly since these guidances were first issued. Therefore, the FDA is updating these guidances to provide sponsors with the agency’s most up-to-date thinking.

Our goal is to help promote safe and effective product development in this field. We’ll continue to work with the product sponsors to help make the development and approval of these innovative gene therapies more efficient, while putting in place the regulatory controls needed to ensure that the resulting therapies are both safe and effective. We’ll also make full use of our expedited programs such as breakthrough therapy designation and regenerative medicine advanced therapy designation whenever possible.

Gene therapy represents one of the most promising opportunities for developing highly effective and even curative treatments for many vexing disorders. Some of these products are almost certainly going to change the contours of medical practice, and the destiny of patients with some debilitating diseases.

The FDA’s goal is to help these innovations advance in a framework that assures the safety and effectiveness of these resulting treatments, and continues to build peoples’ confidence in this novel area of medicine.

–Scott Gottlieb, MD

Once just a theory, gene therapies are now a therapeutic reality for some patients. These platforms may have the potential to treat and cure some of our most intractable and vexing diseases. The policy framework we construct for how these products should be developed, reviewed by regulators, and reimbursed, will help set the stage for the continued advancement of this new market. Last year, we announced our comprehensive policy framework for regenerative medicine, including a draft guidance that describes the expedited programs, such as the breakthrough therapy designation, and the regenerative medicine advanced therapy (RMAT) designation, that may be available to sponsors of these therapies. Today, we’re unveiling a complementary framework for the development, review and approval of gene therapies.

In the past 12 months, we’ve seen three separate gene therapy products approved by the FDA. This reflects the rapid advancements in this field. An inflection point was reached with the development of vectors that could reliably deliver gene cassettes in vivo, into cells and human tissue. In the future, we expect this field to continue to expand, with the potential approval of new treatments for many debilitating diseases. These therapies hold great promise. Our new steps are aimed at fostering developments in this innovative field.

Gene therapies are being studied in many areas, including genetic disorders, autoimmune diseases, heart disease, cancer and HIV/AIDS. We look forward to working with the academic and research communities to make safe and effective products a reality for more patients. But we know that we still have much to learn about how these products work, how to administer them safely, and whether they will continue to work properly in the body without causing adverse side effects over long periods of time. In contrast to traditional drug review, some of the more challenging questions when it comes to gene therapy relate to product manufacturing and quality, or questions about the durability of response, which often can’t be fully answered in any reasonably sized pre-market trial. For some of these products, we may need to accept some level of uncertainty around these questions at the time of approval. For example, in some cases the long-term durability of the effect won’t be fully understood at the time of approval. Effective tools for reliable post-market follow up, such as required post-market clinical trials, are going to be one key to advancing this field and helping to ensure that our approach fosters safe and innovative treatments.

Even when there may be uncertainty about some questions, we need to make certain we assure patient safety and adequately characterize the potential risks and demonstrated benefits of these products. In part because of the added questions that often surround a new technology like gene therapy, these products are initially being aimed at devastating diseases, many of which lack available therapies, including some diseases that are fatal. In such cases of devastating diseases without available therapies, we’ve traditionally been willing to accept more uncertainty to facilitate timely access to promising therapies. In such cases, drug sponsors are generally required to conduct post-marketing clinical trials, known as phase 4 confirmatory trials, to confirm clinical benefit of the drug. This is the direction Congress gave the FDA by creating vehicles like the accelerated approval pathway.

When it comes to novel technologies like gene therapy, the FDA is steadfastly committed to a regulatory path that maintains the agency’s gold standard for assuring safety and efficacy. As we develop this evidence-based framework, we’re going to have to modernize how we approach certain aspects of these products in order to make sure our approach is tailored to the unique challenges created by these new platforms.

Today, we’re taking a step toward shaping this modern structure for the regulation of gene therapy. The agency is issuing a suite of six scientific guidance documents intended to serve as the building blocks of a modern, comprehensive framework for how we’ll help advance the field of gene therapy while making sure new products meet the FDA’s gold standard for safety and effectiveness.

These policies are part of our efforts to communicate the steps we’re taking to provide clear recommendations to sponsors and researchers, so that we can better support innovation. The documents are being issued in draft form so that we can solicit public input on these new policies. As with all draft guidances, all of the comments we receive will be carefully considered prior to finalizing these documents. We’re committed to working with stakeholders to bring novel treatments to the market while ensuring the safety of patients.

Disease-Specific Gene Therapy Guidances

The FDA has issuing three new draft guidance documents on the development of gene therapy products for specific disease categories. These are the first three disease-specific guidances that the agency is issuing for gene therapy products. Our new commitment to develop disease-specific guidance documents reflects the increasing activity in this field, and its growing importance to advancing public health.

Human Gene Therapy for Hemophilia: Gene therapy products for hemophilia are now being developed as single-dose treatments that may enable long-term production of the missing or abnormal coagulation factor in patients. This may reduce or eliminate the need for coagulation factor replacement. To define the proper development pathway for such products, we’re issuing a new draft guidance on gene therapy products that are targeted to the treatment of hemophilia. Once finalized, this new guidance will provide recommendations on the FDA’s current thinking on clinical trial design and preclinical considerations to support the development of these gene therapy products. Among other elements, the draft guidance provides recommendations regarding surrogate endpoints that could be used by sponsors pursuing accelerated approval of gene therapy products that are intended for treatment of hemophilia.

Human Gene Therapy for Retinal Disorders: Another area of fast-paced activity is gene therapy products targeted to the treatment of retinal disorders. The Human Gene Therapy for Retinal Disorders guidance, once finalized, will assist those developing gene therapy products for a wide variety of retinal disorders affecting both adult and pediatric patients. Gene therapy products currently undergoing clinical trials in the United States for retinal disorders are commonly delivered by intravitreal injections (into the fluid portion of the eye), or by subretinal injections (beneath the retina). In some cases, the gene therapy products are encapsulated in a device to be implanted within the eye. This new guidance document will focus on issues that are specific to gene therapies for retinal disorders. The document provides recommendations related to product development, preclinical testing, and clinical trial design for such products.

Human Gene Therapy for Rare Diseases: Rare diseases are those that affect fewer than 200,000 people in the United States. The National Institutes of Health reports that nearly 7,000 rare diseases affect more than 25 million Americans. About 80 percent of rare diseases are caused by a single-gene defect, and about half of all rare diseases affect children. Since most rare diseases have no approved therapies, there is a significant unmet need. The Human Gene Therapy for Rare Diseases guidance, once finalized, will provide recommendations on preclinical, manufacturing and clinical trial design for all phases of the clinical development program for these types of gene therapies. The information is intended to assist sponsors in the design of clinical development programs, where there may be limited study population size, potential feasibility and safety issues, as well as issues relating to the interpretation of effectiveness.

Guidances on Manufacturing Gene Therapies

The FDA is also providing new and comprehensive updates to three existing guidances that address manufacturing issues related to gene therapy. These updates reflect input from many stakeholders. We encourage additional feedback on these documents during the comment period.

The first draft guidance, Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs), provides sponsors with recommendations on how to provide sufficient CMC information to assure safety, identity, quality, purity and strength/potency of investigational gene therapy products. The guidance applies to human gene therapies and to combination products that contain a human gene therapy in combination with a drug or device. In addition, this guidance is organized to follow the structure of the FDA guidance on the Common Technical Document.

The second draft guidance, Testing of Retroviral Vector-Based Gene Therapy Products for Replication Competent Retrovirus (RCR) during Product Manufacture and Patient Follow-up, provides additional recommendations regarding the proper testing for RCR during the manufacture of retroviral vector-based gene therapy products, as well as during the follow-up monitoring of patients who’ve received retroviral vector-based gene therapy products. Specifically, the draft guidance recommends the identification and amount of material to be tested. The guidance also provides advice on general testing methods.

The third draft guidance, Long Term Follow-Up After Administration of Human Gene Therapy Products, provides recommendations regarding the design of long-term follow-up (LTFU) observational studies for the collection of data on delayed adverse events following administration of a gene therapy product. Because of some of the additional uncertainty intrinsic to a novel platform like gene therapy -- including questions related to the durability of the treatment effects as well as the theoretical potential for off-target effects if the genes do not insert correctly -- there’s an increased need for robust long-term follow-up of patients in the post-market period. This guidance describes product characteristics, patient-related factors, and the preclinical and clinical data that should be considered when assessing the need for LTFU observations and describes the features related to effective post-market follow up.

Once finalized, these draft guidances will replace previous guidances issued by the FDA in April 2008 (CMC) and November 2006 (RCR and LTFU).

The field of gene therapy has progressed rapidly since these guidances were first issued. Therefore, the FDA is updating these guidances to provide sponsors with the agency’s most up-to-date thinking.

Our goal is to help promote safe and effective product development in this field. We’ll continue to work with the product sponsors to help make the development and approval of these innovative gene therapies more efficient, while putting in place the regulatory controls needed to ensure that the resulting therapies are both safe and effective. We’ll also make full use of our expedited programs such as breakthrough therapy designation and regenerative medicine advanced therapy designation whenever possible.

Gene therapy represents one of the most promising opportunities for developing highly effective and even curative treatments for many vexing disorders. Some of these products are almost certainly going to change the contours of medical practice, and the destiny of patients with some debilitating diseases.

The FDA’s goal is to help these innovations advance in a framework that assures the safety and effectiveness of these resulting treatments, and continues to build peoples’ confidence in this novel area of medicine.

–Scott Gottlieb, MD

On Friday, June 29, at 5:42 a.m., I stood with my family on a Florida shore overlooking Kennedy Space Center. We had gathered with about a hundred other people to watch a rocket launch and were overwhelmed with excitement as the coastline erupted in fire, and the spacecraft lifted off toward the heavens. Standing there watching the spectacle, I couldn’t help but be caught up in the irony of the moment. Here we were, at the place where NASA sent the first Americans into space – on the very shores where the Apollo astronauts set off for the moon to plant our nation’s flag in the lunar dust in July of 1969. Yet now, almost 50 years later, this launch was profoundly different. The rocket wasn’t built by NASA, and the intention of its builders wasn’t exploration. This was a Falcon 9, built by SpaceX, a for-profit company founded by an enterprising billionaire. Most surprisingly, this relatively routine launch was intended to accomplish something that NASA – the United States’ own space agency – currently can’t do on its own: Launch rockets.

Since retiring the Space Shuttle in 2011, the United States has had to rely on others – including even Roscosmos (the Russian space agency) – to ferry passengers, satellites, and cargo into space. Seeing this opportunity in a multibillion-dollar industry, private enterprise has risen to the challenge, innovating more quickly and at a lower cost than “the establishment” has ever been capable of. As a result, space travel has been disrupted by corporations competing in a new “space race.” Instead of national pride or scientific dominance, this race has been fueled by profit and is quite similar to one being run in another industry: health care.

Just 1 day prior to watching the launch – on June 28 – we learned that Amazon had purchased PillPack, a prescription drug home delivery service. The stock market responded to the news, and the establishment (in this case CVS, Walgreen’s, and WalMart, among others) collectively lost $17.5 billion in one day. This isn’t the first time Amazon has disrupted the health care world; in January of this year, they, along with Berkshire Hathaway and JPMorgan Chase, announced a health care partnership to cut costs and improve care delivery for their employees. This move also sent shivers through the market, as health insurers and providers such as Aetna and United Health lost big on expectations that Amazon et al. wouldn’t stop with their own employees. Those of us watching this play out from the sidelines realized we were witnessing a revolution that would mean the end of health care delivery as we know it – and that’s not necessarily a bad thing, especially in the world of Electronic Health Records.

As you’ve probably noticed, it is quite rare to find physicians nowadays who love computers. Once an exciting novelty in health care, PCs have become a burdensome necessity and providers often feel enslaved to the EHRs that run on them. There are numerous reasons for this, but one primary cause is that the hundreds of disparate EHRs currently available sprouted out of health care – a centuries-old and very provincial industry – prior to the development of technical and regulatory standards to govern them. As they’ve grown larger and larger from their primitive underpinnings, these EHRs have become more cumbersome to navigate, and vendors have simply “bolted-on” additional features without significant changes to their near-obsolete software architecture.

It’s worth noting that a few EHR companies purport to be true innovators in platform usability, such as industry giant, Epic. According to CEO Judy Faulkner, Epic pours 50% of their revenue back into research and development (though, as Epic is a privately held company, this number can’t be verified). If accurate, Epic is truly an exception, as most electronic record companies spend about 10%-30% on improving their products – far less than they spend on recruiting new customers. Regardless, the outcome is this: Physician expectations for user interface and user experience have far outpaced the current state of the art of EHRs, and this has left a gap that new players outside the health care establishment are apt to fill.

Like Amazon, other software giants have made significant investments in health care over the past several years. According to their website, Apple has been working with hospitals, scientists, and developers to “help health care providers streamline their work, deliver better care, and conduct medical research.” Similarly, Google claims to be “making a number of big bets in health care and life sciences,” by leveraging their artificial intelligence technology to assist in clinical diagnosis and scientific discovery. In spite of a few false starts in the past, these companies are poised to do more than simply disrupt health care. As experts in user interface and design, they could truly change the way physicians interact with health care technology, and it seems like it’s no longer a question of if, but when we’ll see that happen.

The effort of SpaceX and others to change the way we launch rockets tells a story that transcends space travel – It’s a story of how new thinking, more efficient processes, and better design can disrupt the establishment. It’s worth pointing out that NASA hasn’t given up – they are continuing to develop the Space Launch System, which, when completed, will be the most powerful rocket in the world and be capable of carrying astronauts into deep space. In the meantime, however, NASA is embracing the efforts of private industry to help pave a better way forward and make space travel safer and more accessible for everyone. We are hopeful that EHR vendors and other establishment health care institutions are taking note, adapting to meet the needs of the current generation of physicians and patients, and innovating a better way to launch health care into the future.


 

Dr. Notte is a family physician and associate chief medical information officer for Abington (Pa.) Jefferson Health. Follow him on twitter (@doctornotte). Dr. Skolnik is a professor of family and community medicine at Jefferson Medical College, Philadelphia, and an associate director of the family medicine residency program at Abington Jefferson Health.

On Friday, June 29, at 5:42 a.m., I stood with my family on a Florida shore overlooking Kennedy Space Center. We had gathered with about a hundred other people to watch a rocket launch and were overwhelmed with excitement as the coastline erupted in fire, and the spacecraft lifted off toward the heavens. Standing there watching the spectacle, I couldn’t help but be caught up in the irony of the moment. Here we were, at the place where NASA sent the first Americans into space – on the very shores where the Apollo astronauts set off for the moon to plant our nation’s flag in the lunar dust in July of 1969. Yet now, almost 50 years later, this launch was profoundly different. The rocket wasn’t built by NASA, and the intention of its builders wasn’t exploration. This was a Falcon 9, built by SpaceX, a for-profit company founded by an enterprising billionaire. Most surprisingly, this relatively routine launch was intended to accomplish something that NASA – the United States’ own space agency – currently can’t do on its own: Launch rockets.

Since retiring the Space Shuttle in 2011, the United States has had to rely on others – including even Roscosmos (the Russian space agency) – to ferry passengers, satellites, and cargo into space. Seeing this opportunity in a multibillion-dollar industry, private enterprise has risen to the challenge, innovating more quickly and at a lower cost than “the establishment” has ever been capable of. As a result, space travel has been disrupted by corporations competing in a new “space race.” Instead of national pride or scientific dominance, this race has been fueled by profit and is quite similar to one being run in another industry: health care.

Just 1 day prior to watching the launch – on June 28 – we learned that Amazon had purchased PillPack, a prescription drug home delivery service. The stock market responded to the news, and the establishment (in this case CVS, Walgreen’s, and WalMart, among others) collectively lost $17.5 billion in one day. This isn’t the first time Amazon has disrupted the health care world; in January of this year, they, along with Berkshire Hathaway and JPMorgan Chase, announced a health care partnership to cut costs and improve care delivery for their employees. This move also sent shivers through the market, as health insurers and providers such as Aetna and United Health lost big on expectations that Amazon et al. wouldn’t stop with their own employees. Those of us watching this play out from the sidelines realized we were witnessing a revolution that would mean the end of health care delivery as we know it – and that’s not necessarily a bad thing, especially in the world of Electronic Health Records.

As you’ve probably noticed, it is quite rare to find physicians nowadays who love computers. Once an exciting novelty in health care, PCs have become a burdensome necessity and providers often feel enslaved to the EHRs that run on them. There are numerous reasons for this, but one primary cause is that the hundreds of disparate EHRs currently available sprouted out of health care – a centuries-old and very provincial industry – prior to the development of technical and regulatory standards to govern them. As they’ve grown larger and larger from their primitive underpinnings, these EHRs have become more cumbersome to navigate, and vendors have simply “bolted-on” additional features without significant changes to their near-obsolete software architecture.

It’s worth noting that a few EHR companies purport to be true innovators in platform usability, such as industry giant, Epic. According to CEO Judy Faulkner, Epic pours 50% of their revenue back into research and development (though, as Epic is a privately held company, this number can’t be verified). If accurate, Epic is truly an exception, as most electronic record companies spend about 10%-30% on improving their products – far less than they spend on recruiting new customers. Regardless, the outcome is this: Physician expectations for user interface and user experience have far outpaced the current state of the art of EHRs, and this has left a gap that new players outside the health care establishment are apt to fill.

Like Amazon, other software giants have made significant investments in health care over the past several years. According to their website, Apple has been working with hospitals, scientists, and developers to “help health care providers streamline their work, deliver better care, and conduct medical research.” Similarly, Google claims to be “making a number of big bets in health care and life sciences,” by leveraging their artificial intelligence technology to assist in clinical diagnosis and scientific discovery. In spite of a few false starts in the past, these companies are poised to do more than simply disrupt health care. As experts in user interface and design, they could truly change the way physicians interact with health care technology, and it seems like it’s no longer a question of if, but when we’ll see that happen.

The effort of SpaceX and others to change the way we launch rockets tells a story that transcends space travel – It’s a story of how new thinking, more efficient processes, and better design can disrupt the establishment. It’s worth pointing out that NASA hasn’t given up – they are continuing to develop the Space Launch System, which, when completed, will be the most powerful rocket in the world and be capable of carrying astronauts into deep space. In the meantime, however, NASA is embracing the efforts of private industry to help pave a better way forward and make space travel safer and more accessible for everyone. We are hopeful that EHR vendors and other establishment health care institutions are taking note, adapting to meet the needs of the current generation of physicians and patients, and innovating a better way to launch health care into the future.


 

Dr. Notte is a family physician and associate chief medical information officer for Abington (Pa.) Jefferson Health. Follow him on twitter (@doctornotte). Dr. Skolnik is a professor of family and community medicine at Jefferson Medical College, Philadelphia, and an associate director of the family medicine residency program at Abington Jefferson Health.

On Friday, June 29, at 5:42 a.m., I stood with my family on a Florida shore overlooking Kennedy Space Center. We had gathered with about a hundred other people to watch a rocket launch and were overwhelmed with excitement as the coastline erupted in fire, and the spacecraft lifted off toward the heavens. Standing there watching the spectacle, I couldn’t help but be caught up in the irony of the moment. Here we were, at the place where NASA sent the first Americans into space – on the very shores where the Apollo astronauts set off for the moon to plant our nation’s flag in the lunar dust in July of 1969. Yet now, almost 50 years later, this launch was profoundly different. The rocket wasn’t built by NASA, and the intention of its builders wasn’t exploration. This was a Falcon 9, built by SpaceX, a for-profit company founded by an enterprising billionaire. Most surprisingly, this relatively routine launch was intended to accomplish something that NASA – the United States’ own space agency – currently can’t do on its own: Launch rockets.

Since retiring the Space Shuttle in 2011, the United States has had to rely on others – including even Roscosmos (the Russian space agency) – to ferry passengers, satellites, and cargo into space. Seeing this opportunity in a multibillion-dollar industry, private enterprise has risen to the challenge, innovating more quickly and at a lower cost than “the establishment” has ever been capable of. As a result, space travel has been disrupted by corporations competing in a new “space race.” Instead of national pride or scientific dominance, this race has been fueled by profit and is quite similar to one being run in another industry: health care.

Just 1 day prior to watching the launch – on June 28 – we learned that Amazon had purchased PillPack, a prescription drug home delivery service. The stock market responded to the news, and the establishment (in this case CVS, Walgreen’s, and WalMart, among others) collectively lost $17.5 billion in one day. This isn’t the first time Amazon has disrupted the health care world; in January of this year, they, along with Berkshire Hathaway and JPMorgan Chase, announced a health care partnership to cut costs and improve care delivery for their employees. This move also sent shivers through the market, as health insurers and providers such as Aetna and United Health lost big on expectations that Amazon et al. wouldn’t stop with their own employees. Those of us watching this play out from the sidelines realized we were witnessing a revolution that would mean the end of health care delivery as we know it – and that’s not necessarily a bad thing, especially in the world of Electronic Health Records.

As you’ve probably noticed, it is quite rare to find physicians nowadays who love computers. Once an exciting novelty in health care, PCs have become a burdensome necessity and providers often feel enslaved to the EHRs that run on them. There are numerous reasons for this, but one primary cause is that the hundreds of disparate EHRs currently available sprouted out of health care – a centuries-old and very provincial industry – prior to the development of technical and regulatory standards to govern them. As they’ve grown larger and larger from their primitive underpinnings, these EHRs have become more cumbersome to navigate, and vendors have simply “bolted-on” additional features without significant changes to their near-obsolete software architecture.

It’s worth noting that a few EHR companies purport to be true innovators in platform usability, such as industry giant, Epic. According to CEO Judy Faulkner, Epic pours 50% of their revenue back into research and development (though, as Epic is a privately held company, this number can’t be verified). If accurate, Epic is truly an exception, as most electronic record companies spend about 10%-30% on improving their products – far less than they spend on recruiting new customers. Regardless, the outcome is this: Physician expectations for user interface and user experience have far outpaced the current state of the art of EHRs, and this has left a gap that new players outside the health care establishment are apt to fill.

Like Amazon, other software giants have made significant investments in health care over the past several years. According to their website, Apple has been working with hospitals, scientists, and developers to “help health care providers streamline their work, deliver better care, and conduct medical research.” Similarly, Google claims to be “making a number of big bets in health care and life sciences,” by leveraging their artificial intelligence technology to assist in clinical diagnosis and scientific discovery. In spite of a few false starts in the past, these companies are poised to do more than simply disrupt health care. As experts in user interface and design, they could truly change the way physicians interact with health care technology, and it seems like it’s no longer a question of if, but when we’ll see that happen.

The effort of SpaceX and others to change the way we launch rockets tells a story that transcends space travel – It’s a story of how new thinking, more efficient processes, and better design can disrupt the establishment. It’s worth pointing out that NASA hasn’t given up – they are continuing to develop the Space Launch System, which, when completed, will be the most powerful rocket in the world and be capable of carrying astronauts into deep space. In the meantime, however, NASA is embracing the efforts of private industry to help pave a better way forward and make space travel safer and more accessible for everyone. We are hopeful that EHR vendors and other establishment health care institutions are taking note, adapting to meet the needs of the current generation of physicians and patients, and innovating a better way to launch health care into the future.


 

Dr. Notte is a family physician and associate chief medical information officer for Abington (Pa.) Jefferson Health. Follow him on twitter (@doctornotte). Dr. Skolnik is a professor of family and community medicine at Jefferson Medical College, Philadelphia, and an associate director of the family medicine residency program at Abington Jefferson Health.

R. Allan Purdy, MD, FRCPC

Dr. Purdy is Professor of Medicine (Neurology) at Dalhousie University, Halifax, Nova Scotia.

Although migraine has been with humans since antiquity, it is truly amazing that over the past 25 years and a bit longer there have been remarkable advances in our clinical understanding of migraine and its variations, along with sophisticated epidemiologic evidence and basic research into the neurobiological basis of migraine. These advances have brought this disorder/disease to the forefront as a serious neurologic condition deserving of attention. Migraine is a major cause of neurologic disability in the world. This fact has only been fully recognized in recent years.

From the 1980s through the 1990s, a series of events led to a seminal study of a medication that would truly alter the vector for migraine in the future. Studies coming out of Glaxo in the United Kingdom, under the direction of Pat Humphrey, OBE, DSc, PhD, led to the discovery of sumatriptan, the first truly designer medication for the treatment of acute migraine. The story of how this medication biologically affects the brain serotonin receptors on blood vessels and brain tissue to abort a migraine attack is well known today. Early observations on patients receiving subcutaneous sumatriptan clearly showed how powerful this agent was in shutting down the migraine attack. Patients having migraine in extremis, with severe throbbing headache, pallor, nausea and vomiting, appearing markedly distressed and ill, within minutes to an hour would return to a normal state. Nothing that preceded sumatriptan demonstrated such a remarkable clinical response in patients with headache.

In the past century, sumatriptan came to be one of the most important therapeutic advances in neurology. After its discovery, six other triptans entered the market over time. All of them had minor tweaks on the original molecule’s pharmacology and pharmacokinetics, including various modes of delivery with different results in subgroups of patients. Nevertheless, on balance, the triptans acted in a similar manner to produce similar outcomes. Today, many patients take triptans regularly for help with acute migraine attacks; however, other patients with migraine remain undiagnosed and undertreated and do not receive optimal care.

In the past 20 years, sophisticated laboratory and neuroimaging research allowed in-depth analysis of the migraine attack and spectrum of the migraine disorder. The brain areas that subserve the migraine attack have been mapped neuroanatomically throughout the nervous system, with input from the brainstem, hypothalamus, thalamus, and cortical structures. Cortical-initiated electrical events possibly trigger the trigeminal vascular system, and/or peripheral or central activation mechanisms produce the symptoms of migraine. The migraine story is not complete, but evidence clearly shows migraine to be a valid neurobiological disorder and disease. For some people with migraine, it is occasional aggravation, but for others it can be life-altering and, rarely, life-threatening.

In the past few years, new targets for migraine therapy have been pursued. A CGRP receptor antagonist (or “gepant”) showed benefit in early trials; however, because of potential hepatic side effects, other gepants and CGRP monoclonal antibodies have been studied in clinical trials.

Results of recent trials—one in episodic migraine and the other in chronic migraine—show that anti-CGRP monoclonal antibodies appear to be quite efficacious, have few side effects, and are well tolerated. Time will determine whether there are long-term consequences of their use, and what is the effectiveness of using these large molecules to treat migraine, but current results appear promising. Another triptan-like agent, a ditan, which activates receptors of serotonin without vasoconstrictor properties, has shown promise in acute migraine. Neuromodulation devices are also showing promise in migraine therapy and appear to be safe and well tolerated. Nonpharmacological therapies are more often utilized with benefits and help to avoid problems with medications and their side effects.

Increasing knowledge of migraine epidemiology has shown that there is a transition from acute to more frequent, and from high frequency to subsequent chronic migraine attacks. This transformation, or chronification, appears unique in patients with migraine. This process appears to be bidirectional and reversible in migraine. Whether migraine becomes chronic as a process over time or whether migraine is a chronic disease with episodic fluctuations is something to be further explored in research and clinical practice. Nevertheless, these concepts can only lead to better understanding and, hopefully, new therapeutic interventions that will reduce the frequency and severity of this unique neurologic disease.

As migraine progresses or evolves it can be associated with multiple comorbid disorders, including stroke, depression, seizures, and medication overuse. New preventive therapies in development can be modifications of medications for other neurologic conditions, such as the antiepileptic medications, for example. There are many other potential targets that will be explored for the management of migraine in this century. The future looks promising in that regard.

For decades, and now centuries, neurologists have been interested in migraine and related disorders. Sumatriptan jump-started the modern revolution and evolution of therapeutic options to manage migraine pharmacologically. Since its discovery and use in clinical medicine, the world of migraine has changed dramatically. As headache clinicians are being trained in the latest advances in migraine and other headache disorders, they are showing increasing interest and knowledge,which is provided by some of the most unique and relevant research involving the brain in the world. A cure for migraine may not be possible, but a better understanding and control of all of migraine’s myriad of symptoms is probably within reach in this century.

Suggested Reading

Bigal ME, Lipton RB. Clinical course in migraine: conceptualizing migraine transformation. Neurology. 2008;71(11):848-855.

Deen M, Correnti E, Kamm K, et al. Blocking CGRP in migraine patients – a review of pros and cons. J Headache Pain. 2017;18(1):96.

Dodick DW. Migraine. Lancet. 2018;391(10127):1315-1330.

Färkkilä M, Diener HC, Géraud G, et al. Efficacy and tolerability of lasmiditan, an oral 5-HT(1F) receptor agonist, for the acute treatment of migraine: a phase 2 randomised, placebo-controlled, parallel-group, dose-ranging study. Lancet Neurology. 2012;11(5):405-413.

Goadsby PJ, Reuter U, Hallström Y, et al. A controlled trial of erenumab for episodic migraine. N Engl J Med. 2017;377(22):2123-2132.

Humphrey PP. The discovery and development of the triptans, a major therapeutic breakthrough. Headache. 2008;48(5):685-687.

Puledda F, Goadsby PJ. An update on non-pharmacological neuromodulation for the acute and preventive treatment of migraine. Headache. 2017;57(4):685-691.

Silberstein SD, Dodick DW, Bigal ME, et al. Fremanezumab for the preventive treatment of chronic migraine. N Engl J Med. 2017;377(22):2113-2122.

Wietecha LA, Kuca B, Case MG, et al. Phase 3 study (SPARTAN) of lasmiditan compared to placebo for acute treatment of migraine. Cephalalgia. 2017;37 (suppl 1):367-68 (abstr).

R. Allan Purdy, MD, FRCPC

Dr. Purdy is Professor of Medicine (Neurology) at Dalhousie University, Halifax, Nova Scotia.

Although migraine has been with humans since antiquity, it is truly amazing that over the past 25 years and a bit longer there have been remarkable advances in our clinical understanding of migraine and its variations, along with sophisticated epidemiologic evidence and basic research into the neurobiological basis of migraine. These advances have brought this disorder/disease to the forefront as a serious neurologic condition deserving of attention. Migraine is a major cause of neurologic disability in the world. This fact has only been fully recognized in recent years.

From the 1980s through the 1990s, a series of events led to a seminal study of a medication that would truly alter the vector for migraine in the future. Studies coming out of Glaxo in the United Kingdom, under the direction of Pat Humphrey, OBE, DSc, PhD, led to the discovery of sumatriptan, the first truly designer medication for the treatment of acute migraine. The story of how this medication biologically affects the brain serotonin receptors on blood vessels and brain tissue to abort a migraine attack is well known today. Early observations on patients receiving subcutaneous sumatriptan clearly showed how powerful this agent was in shutting down the migraine attack. Patients having migraine in extremis, with severe throbbing headache, pallor, nausea and vomiting, appearing markedly distressed and ill, within minutes to an hour would return to a normal state. Nothing that preceded sumatriptan demonstrated such a remarkable clinical response in patients with headache.

In the past century, sumatriptan came to be one of the most important therapeutic advances in neurology. After its discovery, six other triptans entered the market over time. All of them had minor tweaks on the original molecule’s pharmacology and pharmacokinetics, including various modes of delivery with different results in subgroups of patients. Nevertheless, on balance, the triptans acted in a similar manner to produce similar outcomes. Today, many patients take triptans regularly for help with acute migraine attacks; however, other patients with migraine remain undiagnosed and undertreated and do not receive optimal care.

In the past 20 years, sophisticated laboratory and neuroimaging research allowed in-depth analysis of the migraine attack and spectrum of the migraine disorder. The brain areas that subserve the migraine attack have been mapped neuroanatomically throughout the nervous system, with input from the brainstem, hypothalamus, thalamus, and cortical structures. Cortical-initiated electrical events possibly trigger the trigeminal vascular system, and/or peripheral or central activation mechanisms produce the symptoms of migraine. The migraine story is not complete, but evidence clearly shows migraine to be a valid neurobiological disorder and disease. For some people with migraine, it is occasional aggravation, but for others it can be life-altering and, rarely, life-threatening.

In the past few years, new targets for migraine therapy have been pursued. A CGRP receptor antagonist (or “gepant”) showed benefit in early trials; however, because of potential hepatic side effects, other gepants and CGRP monoclonal antibodies have been studied in clinical trials.

Results of recent trials—one in episodic migraine and the other in chronic migraine—show that anti-CGRP monoclonal antibodies appear to be quite efficacious, have few side effects, and are well tolerated. Time will determine whether there are long-term consequences of their use, and what is the effectiveness of using these large molecules to treat migraine, but current results appear promising. Another triptan-like agent, a ditan, which activates receptors of serotonin without vasoconstrictor properties, has shown promise in acute migraine. Neuromodulation devices are also showing promise in migraine therapy and appear to be safe and well tolerated. Nonpharmacological therapies are more often utilized with benefits and help to avoid problems with medications and their side effects.

Increasing knowledge of migraine epidemiology has shown that there is a transition from acute to more frequent, and from high frequency to subsequent chronic migraine attacks. This transformation, or chronification, appears unique in patients with migraine. This process appears to be bidirectional and reversible in migraine. Whether migraine becomes chronic as a process over time or whether migraine is a chronic disease with episodic fluctuations is something to be further explored in research and clinical practice. Nevertheless, these concepts can only lead to better understanding and, hopefully, new therapeutic interventions that will reduce the frequency and severity of this unique neurologic disease.

As migraine progresses or evolves it can be associated with multiple comorbid disorders, including stroke, depression, seizures, and medication overuse. New preventive therapies in development can be modifications of medications for other neurologic conditions, such as the antiepileptic medications, for example. There are many other potential targets that will be explored for the management of migraine in this century. The future looks promising in that regard.

For decades, and now centuries, neurologists have been interested in migraine and related disorders. Sumatriptan jump-started the modern revolution and evolution of therapeutic options to manage migraine pharmacologically. Since its discovery and use in clinical medicine, the world of migraine has changed dramatically. As headache clinicians are being trained in the latest advances in migraine and other headache disorders, they are showing increasing interest and knowledge,which is provided by some of the most unique and relevant research involving the brain in the world. A cure for migraine may not be possible, but a better understanding and control of all of migraine’s myriad of symptoms is probably within reach in this century.

Suggested Reading

Bigal ME, Lipton RB. Clinical course in migraine: conceptualizing migraine transformation. Neurology. 2008;71(11):848-855.

Deen M, Correnti E, Kamm K, et al. Blocking CGRP in migraine patients – a review of pros and cons. J Headache Pain. 2017;18(1):96.

Dodick DW. Migraine. Lancet. 2018;391(10127):1315-1330.

Färkkilä M, Diener HC, Géraud G, et al. Efficacy and tolerability of lasmiditan, an oral 5-HT(1F) receptor agonist, for the acute treatment of migraine: a phase 2 randomised, placebo-controlled, parallel-group, dose-ranging study. Lancet Neurology. 2012;11(5):405-413.

Goadsby PJ, Reuter U, Hallström Y, et al. A controlled trial of erenumab for episodic migraine. N Engl J Med. 2017;377(22):2123-2132.

Humphrey PP. The discovery and development of the triptans, a major therapeutic breakthrough. Headache. 2008;48(5):685-687.

Puledda F, Goadsby PJ. An update on non-pharmacological neuromodulation for the acute and preventive treatment of migraine. Headache. 2017;57(4):685-691.

Silberstein SD, Dodick DW, Bigal ME, et al. Fremanezumab for the preventive treatment of chronic migraine. N Engl J Med. 2017;377(22):2113-2122.

Wietecha LA, Kuca B, Case MG, et al. Phase 3 study (SPARTAN) of lasmiditan compared to placebo for acute treatment of migraine. Cephalalgia. 2017;37 (suppl 1):367-68 (abstr).

R. Allan Purdy, MD, FRCPC

Dr. Purdy is Professor of Medicine (Neurology) at Dalhousie University, Halifax, Nova Scotia.

Although migraine has been with humans since antiquity, it is truly amazing that over the past 25 years and a bit longer there have been remarkable advances in our clinical understanding of migraine and its variations, along with sophisticated epidemiologic evidence and basic research into the neurobiological basis of migraine. These advances have brought this disorder/disease to the forefront as a serious neurologic condition deserving of attention. Migraine is a major cause of neurologic disability in the world. This fact has only been fully recognized in recent years.

From the 1980s through the 1990s, a series of events led to a seminal study of a medication that would truly alter the vector for migraine in the future. Studies coming out of Glaxo in the United Kingdom, under the direction of Pat Humphrey, OBE, DSc, PhD, led to the discovery of sumatriptan, the first truly designer medication for the treatment of acute migraine. The story of how this medication biologically affects the brain serotonin receptors on blood vessels and brain tissue to abort a migraine attack is well known today. Early observations on patients receiving subcutaneous sumatriptan clearly showed how powerful this agent was in shutting down the migraine attack. Patients having migraine in extremis, with severe throbbing headache, pallor, nausea and vomiting, appearing markedly distressed and ill, within minutes to an hour would return to a normal state. Nothing that preceded sumatriptan demonstrated such a remarkable clinical response in patients with headache.

In the past century, sumatriptan came to be one of the most important therapeutic advances in neurology. After its discovery, six other triptans entered the market over time. All of them had minor tweaks on the original molecule’s pharmacology and pharmacokinetics, including various modes of delivery with different results in subgroups of patients. Nevertheless, on balance, the triptans acted in a similar manner to produce similar outcomes. Today, many patients take triptans regularly for help with acute migraine attacks; however, other patients with migraine remain undiagnosed and undertreated and do not receive optimal care.

In the past 20 years, sophisticated laboratory and neuroimaging research allowed in-depth analysis of the migraine attack and spectrum of the migraine disorder. The brain areas that subserve the migraine attack have been mapped neuroanatomically throughout the nervous system, with input from the brainstem, hypothalamus, thalamus, and cortical structures. Cortical-initiated electrical events possibly trigger the trigeminal vascular system, and/or peripheral or central activation mechanisms produce the symptoms of migraine. The migraine story is not complete, but evidence clearly shows migraine to be a valid neurobiological disorder and disease. For some people with migraine, it is occasional aggravation, but for others it can be life-altering and, rarely, life-threatening.

In the past few years, new targets for migraine therapy have been pursued. A CGRP receptor antagonist (or “gepant”) showed benefit in early trials; however, because of potential hepatic side effects, other gepants and CGRP monoclonal antibodies have been studied in clinical trials.

Results of recent trials—one in episodic migraine and the other in chronic migraine—show that anti-CGRP monoclonal antibodies appear to be quite efficacious, have few side effects, and are well tolerated. Time will determine whether there are long-term consequences of their use, and what is the effectiveness of using these large molecules to treat migraine, but current results appear promising. Another triptan-like agent, a ditan, which activates receptors of serotonin without vasoconstrictor properties, has shown promise in acute migraine. Neuromodulation devices are also showing promise in migraine therapy and appear to be safe and well tolerated. Nonpharmacological therapies are more often utilized with benefits and help to avoid problems with medications and their side effects.

Increasing knowledge of migraine epidemiology has shown that there is a transition from acute to more frequent, and from high frequency to subsequent chronic migraine attacks. This transformation, or chronification, appears unique in patients with migraine. This process appears to be bidirectional and reversible in migraine. Whether migraine becomes chronic as a process over time or whether migraine is a chronic disease with episodic fluctuations is something to be further explored in research and clinical practice. Nevertheless, these concepts can only lead to better understanding and, hopefully, new therapeutic interventions that will reduce the frequency and severity of this unique neurologic disease.

As migraine progresses or evolves it can be associated with multiple comorbid disorders, including stroke, depression, seizures, and medication overuse. New preventive therapies in development can be modifications of medications for other neurologic conditions, such as the antiepileptic medications, for example. There are many other potential targets that will be explored for the management of migraine in this century. The future looks promising in that regard.

For decades, and now centuries, neurologists have been interested in migraine and related disorders. Sumatriptan jump-started the modern revolution and evolution of therapeutic options to manage migraine pharmacologically. Since its discovery and use in clinical medicine, the world of migraine has changed dramatically. As headache clinicians are being trained in the latest advances in migraine and other headache disorders, they are showing increasing interest and knowledge,which is provided by some of the most unique and relevant research involving the brain in the world. A cure for migraine may not be possible, but a better understanding and control of all of migraine’s myriad of symptoms is probably within reach in this century.

Suggested Reading

Bigal ME, Lipton RB. Clinical course in migraine: conceptualizing migraine transformation. Neurology. 2008;71(11):848-855.

Deen M, Correnti E, Kamm K, et al. Blocking CGRP in migraine patients – a review of pros and cons. J Headache Pain. 2017;18(1):96.

Dodick DW. Migraine. Lancet. 2018;391(10127):1315-1330.

Färkkilä M, Diener HC, Géraud G, et al. Efficacy and tolerability of lasmiditan, an oral 5-HT(1F) receptor agonist, for the acute treatment of migraine: a phase 2 randomised, placebo-controlled, parallel-group, dose-ranging study. Lancet Neurology. 2012;11(5):405-413.

Goadsby PJ, Reuter U, Hallström Y, et al. A controlled trial of erenumab for episodic migraine. N Engl J Med. 2017;377(22):2123-2132.

Humphrey PP. The discovery and development of the triptans, a major therapeutic breakthrough. Headache. 2008;48(5):685-687.

Puledda F, Goadsby PJ. An update on non-pharmacological neuromodulation for the acute and preventive treatment of migraine. Headache. 2017;57(4):685-691.

Silberstein SD, Dodick DW, Bigal ME, et al. Fremanezumab for the preventive treatment of chronic migraine. N Engl J Med. 2017;377(22):2113-2122.

Wietecha LA, Kuca B, Case MG, et al. Phase 3 study (SPARTAN) of lasmiditan compared to placebo for acute treatment of migraine. Cephalalgia. 2017;37 (suppl 1):367-68 (abstr).

Documentation got you down? Feel like you’re turning into a data entry clerk?

Sorry, but I can’t help you. What I can do is tell you you’re not alone. Datamania is now an endemic malady. Everybody has it and everybody’s doing it, even some you’d never imagine. If misery loves company, you should soon be head over heels.

1. Tiers for Tots

PRImageFactory/iStock/Getty Images

“What are the parents like?” I ask.

“They’re great!” Tracy says. “They want their kids to be creative and play.”

She frowns. “But my boss insists I give him data.”

“Data? What data?”

“Studies show that letter recognition in kindergarten correlates with reading ability in third grade,” she says. “So I have to test the kids and provide him with the data.”

“And what if the kids flub letter recognition?”

Tracy’s smile is now rueful. “Then they might need a Tier 2 intervention.”

“Good grief! What is a Tier 2 intervention?”

“It’s time consuming,” she says. “It takes a lot of one-on-one work, me and the kid.”

Less play all around, I guess. But documentation must be done, and data delivered. By the kindergarten teacher!

2. Filing for firefighters

Bruce has been a firefighter for 30 years, and it’s starting to wear him down. The physical exertion? The stress? Nah.

“The paperwork is driving me crazy,” he says.

“What paperwork?”

“In between calls, we spend hours filling out forms,” he says.

“Which forms?”

“At the scene, you go to work on the fire and help people get to safety. Then you see how they’re doing, and refer the ones who need it for medical help.

“Used to be,” says Bruce, “that you’d eyeball someone, ask them how they felt and if they needed to go to the hospital. If they said they were OK, they were good to leave.”

“And now?”

“Now we have to cover ourselves. We need to document how they look, what they say, what we asked them, what they answered. They have to sign a release that we asked them what we needed to ask and they answered what we needed to hear, that they said they were OK and didn’t need to go to the ER and signed off on it. Takes a lot of time.”

And paper. Maybe little kids who used to dream of being firefighters will start to dream that they’ll be file clerks with big red hats.

3. Your personal banker doesn’t know you!

Marina looks frazzled. “Stress at work,” she says. “It gets worse all the time.”

I know Marina works at a community bank. “What’s the problem?” I ask. “More restrictions on lending?”

“Oh sure,” she says, “but that’s an old story. Now there are new regulations to prevent money laundering. We have to know the identity of anybody who makes a deposit.”

“Sounds reasonable.”

“In principle sure,” she says. “But in practice what happens is this: Somebody wants to make any change – to add a relative, upgrade to a newer checking account. Even if they’ve been our depositors for 20 years, we have to ask them to produce all kinds of personal information for us to show regulators if they ask if we know people we’ve known forever.”

“Do the regulators ever ask?”

“Of course not,” says Marina. “But we have to fill out the forms, which take all day.”

Dr. Rockoff practices dermatology in Brookline, Mass., and is a longtime contributor to Dermatology News. He serves on the clinical faculty at Tufts University, Boston, and has taught senior medical students and other trainees for 30 years. His second book, “Act Like a Doctor, Think Like a Patient,” is available at amazon.com and barnesandnoble.com. Write to him at [email protected].

Documentation got you down? Feel like you’re turning into a data entry clerk?

Sorry, but I can’t help you. What I can do is tell you you’re not alone. Datamania is now an endemic malady. Everybody has it and everybody’s doing it, even some you’d never imagine. If misery loves company, you should soon be head over heels.

1. Tiers for Tots

PRImageFactory/iStock/Getty Images

“What are the parents like?” I ask.

“They’re great!” Tracy says. “They want their kids to be creative and play.”

She frowns. “But my boss insists I give him data.”

“Data? What data?”

“Studies show that letter recognition in kindergarten correlates with reading ability in third grade,” she says. “So I have to test the kids and provide him with the data.”

“And what if the kids flub letter recognition?”

Tracy’s smile is now rueful. “Then they might need a Tier 2 intervention.”

“Good grief! What is a Tier 2 intervention?”

“It’s time consuming,” she says. “It takes a lot of one-on-one work, me and the kid.”

Less play all around, I guess. But documentation must be done, and data delivered. By the kindergarten teacher!

2. Filing for firefighters

Bruce has been a firefighter for 30 years, and it’s starting to wear him down. The physical exertion? The stress? Nah.

“The paperwork is driving me crazy,” he says.

“What paperwork?”

“In between calls, we spend hours filling out forms,” he says.

“Which forms?”

“At the scene, you go to work on the fire and help people get to safety. Then you see how they’re doing, and refer the ones who need it for medical help.

“Used to be,” says Bruce, “that you’d eyeball someone, ask them how they felt and if they needed to go to the hospital. If they said they were OK, they were good to leave.”

“And now?”

“Now we have to cover ourselves. We need to document how they look, what they say, what we asked them, what they answered. They have to sign a release that we asked them what we needed to ask and they answered what we needed to hear, that they said they were OK and didn’t need to go to the ER and signed off on it. Takes a lot of time.”

And paper. Maybe little kids who used to dream of being firefighters will start to dream that they’ll be file clerks with big red hats.

3. Your personal banker doesn’t know you!

Marina looks frazzled. “Stress at work,” she says. “It gets worse all the time.”

I know Marina works at a community bank. “What’s the problem?” I ask. “More restrictions on lending?”

“Oh sure,” she says, “but that’s an old story. Now there are new regulations to prevent money laundering. We have to know the identity of anybody who makes a deposit.”

“Sounds reasonable.”

“In principle sure,” she says. “But in practice what happens is this: Somebody wants to make any change – to add a relative, upgrade to a newer checking account. Even if they’ve been our depositors for 20 years, we have to ask them to produce all kinds of personal information for us to show regulators if they ask if we know people we’ve known forever.”

“Do the regulators ever ask?”

“Of course not,” says Marina. “But we have to fill out the forms, which take all day.”

Dr. Rockoff practices dermatology in Brookline, Mass., and is a longtime contributor to Dermatology News. He serves on the clinical faculty at Tufts University, Boston, and has taught senior medical students and other trainees for 30 years. His second book, “Act Like a Doctor, Think Like a Patient,” is available at amazon.com and barnesandnoble.com. Write to him at [email protected].

Documentation got you down? Feel like you’re turning into a data entry clerk?

Sorry, but I can’t help you. What I can do is tell you you’re not alone. Datamania is now an endemic malady. Everybody has it and everybody’s doing it, even some you’d never imagine. If misery loves company, you should soon be head over heels.

1. Tiers for Tots

PRImageFactory/iStock/Getty Images

“What are the parents like?” I ask.

“They’re great!” Tracy says. “They want their kids to be creative and play.”

She frowns. “But my boss insists I give him data.”

“Data? What data?”

“Studies show that letter recognition in kindergarten correlates with reading ability in third grade,” she says. “So I have to test the kids and provide him with the data.”

“And what if the kids flub letter recognition?”

Tracy’s smile is now rueful. “Then they might need a Tier 2 intervention.”

“Good grief! What is a Tier 2 intervention?”

“It’s time consuming,” she says. “It takes a lot of one-on-one work, me and the kid.”

Less play all around, I guess. But documentation must be done, and data delivered. By the kindergarten teacher!

2. Filing for firefighters

Bruce has been a firefighter for 30 years, and it’s starting to wear him down. The physical exertion? The stress? Nah.

“The paperwork is driving me crazy,” he says.

“What paperwork?”

“In between calls, we spend hours filling out forms,” he says.

“Which forms?”

“At the scene, you go to work on the fire and help people get to safety. Then you see how they’re doing, and refer the ones who need it for medical help.

“Used to be,” says Bruce, “that you’d eyeball someone, ask them how they felt and if they needed to go to the hospital. If they said they were OK, they were good to leave.”

“And now?”

“Now we have to cover ourselves. We need to document how they look, what they say, what we asked them, what they answered. They have to sign a release that we asked them what we needed to ask and they answered what we needed to hear, that they said they were OK and didn’t need to go to the ER and signed off on it. Takes a lot of time.”

And paper. Maybe little kids who used to dream of being firefighters will start to dream that they’ll be file clerks with big red hats.

3. Your personal banker doesn’t know you!

Marina looks frazzled. “Stress at work,” she says. “It gets worse all the time.”

I know Marina works at a community bank. “What’s the problem?” I ask. “More restrictions on lending?”

“Oh sure,” she says, “but that’s an old story. Now there are new regulations to prevent money laundering. We have to know the identity of anybody who makes a deposit.”

“Sounds reasonable.”

“In principle sure,” she says. “But in practice what happens is this: Somebody wants to make any change – to add a relative, upgrade to a newer checking account. Even if they’ve been our depositors for 20 years, we have to ask them to produce all kinds of personal information for us to show regulators if they ask if we know people we’ve known forever.”

“Do the regulators ever ask?”

“Of course not,” says Marina. “But we have to fill out the forms, which take all day.”

Dr. Rockoff practices dermatology in Brookline, Mass., and is a longtime contributor to Dermatology News. He serves on the clinical faculty at Tufts University, Boston, and has taught senior medical students and other trainees for 30 years. His second book, “Act Like a Doctor, Think Like a Patient,” is available at amazon.com and barnesandnoble.com. Write to him at [email protected].

We are fortunate to have plentiful acne treatment options available that cater to each patient’s clinical examination, predispositions, and triggers, but the choices are daunting amidst the vast prescription and over-the-counter (OTC) topicals available along with disparate insurance and cost nuances. In addition, when prescribing generic oral therapies, it is complicated to parse out regional differences in price, supply, and insurance coverage to advocate best for each patient and land upon the delicate balance between efficacy, safety, and financial stewardship, both at an individual and community level. I will outline some challenges and solutions to the management of acne amidst these complicated factors.

Oral Therapies

For isotretinoin, generic choices, cost, and tiering within insurance plans are perpetual moving targets despite the drug being the only member of its class.¹ Prescriber resources include tandem searches of electronic medical record price estimates within each insurance formulary, individual pharmacy search engines, and compilation mobile applications such as GoodRx to select the most affordable version for each patient. As an example of a regional trend, isotretinoin generic coverage by one provider in Pennsylvania shifted earlier this year so that every patient, whether new to isotretinoin or midcourse, required a new prior authorization with more stringent coverage requirements including failure of 2 oral antibiotics. Swiftly thereafter, efforts across the state driven by the Pennsylvania Academy of Dermatology and Dermatologic Surgery and its members and fueled by poignant patient vignettes about fragmented and substandard patient care helped to reverse this policy and remove the prior authorization mandate.²

Tetracyclines have experienced broad cost swings, mostly based on disruption of manufacturing at the limited number of distribution sites in the United States. In 2011, a tetracycline shortage arose due to a major manufacturer’s recall³ and persisted with subsequent material shortages, as doxycycline became the preferred and cheaper member of the class. Doxycycline price tag hikes then occurred following Hurricane Sandy when an East Coast manufacturing site was damaged.³ Spironolactone backorder also has been frequent due to recent raw material shortages and delays in production,³ forcing pharmacies to refill small amounts of medication in various dosage forms despite patients owing the same copayment per prescription.

Topical Therapies

Topical retinoid prescription affordability has always been fraught with difficulty owing to age cutoffs because it is often restricted to patients younger than 25 or 40 years, depending on the plan,^4,5 but the availability of adapalene gel 0.1% as an OTC preparation in 2016 has broadened retinoid access and use.⁶ Prescription benzoyl peroxide (BPO) products alone or in combination with retinoids or topical antibiotics (or other combination topical therapies) comprise a large number of branded prescriptions often not covered by insurance or are only affordable using proprietary and intermittently available coupon cards (eg, BPO-clindamycin, clindamycin-tretinoin, BPO-adapalene); therefore, prescribers tend to dispense the individual ingredients and instruct the patient to compound them at home. Furthermore, BPO products can be purchased in effective concentrations as OTC products, and patients looking to procure more affordable, albeit less effective, topical retinoids that also have less irritation potential reach for OTC nightly retinol creams nestled in the antiaging section of the pharmacy.⁷

Opportunities to be involved in the larger scaffold of patient advocacy also are plentiful at the state and national levels. For example, with the support of dermatology state societies and the American Academy of Dermatology Association, Pennsylvania House Bill 2211⁸ and similar bills in other states call for reversal of the gag clause that prevents pharmacists from disclosing the best medication price to patients. Also guided by the American Academy of Dermatology Association, prior authorization model legislation to promote transparency across insurers in this haphazard process is emerging across the country.^9,10

Final Thoughts

These examples of acne medication access and cost quandaries serve to embody the daily problem-solving that dermatologists execute as part of their growing administrative and economic duties. They also represent worthy efforts to consider on behalf of patients, their pocketbooks, and the prudent use of their dermatologists’ time.

We are fortunate to have plentiful acne treatment options available that cater to each patient’s clinical examination, predispositions, and triggers, but the choices are daunting amidst the vast prescription and over-the-counter (OTC) topicals available along with disparate insurance and cost nuances. In addition, when prescribing generic oral therapies, it is complicated to parse out regional differences in price, supply, and insurance coverage to advocate best for each patient and land upon the delicate balance between efficacy, safety, and financial stewardship, both at an individual and community level. I will outline some challenges and solutions to the management of acne amidst these complicated factors.

Oral Therapies

For isotretinoin, generic choices, cost, and tiering within insurance plans are perpetual moving targets despite the drug being the only member of its class.¹ Prescriber resources include tandem searches of electronic medical record price estimates within each insurance formulary, individual pharmacy search engines, and compilation mobile applications such as GoodRx to select the most affordable version for each patient. As an example of a regional trend, isotretinoin generic coverage by one provider in Pennsylvania shifted earlier this year so that every patient, whether new to isotretinoin or midcourse, required a new prior authorization with more stringent coverage requirements including failure of 2 oral antibiotics. Swiftly thereafter, efforts across the state driven by the Pennsylvania Academy of Dermatology and Dermatologic Surgery and its members and fueled by poignant patient vignettes about fragmented and substandard patient care helped to reverse this policy and remove the prior authorization mandate.²

Tetracyclines have experienced broad cost swings, mostly based on disruption of manufacturing at the limited number of distribution sites in the United States. In 2011, a tetracycline shortage arose due to a major manufacturer’s recall³ and persisted with subsequent material shortages, as doxycycline became the preferred and cheaper member of the class. Doxycycline price tag hikes then occurred following Hurricane Sandy when an East Coast manufacturing site was damaged.³ Spironolactone backorder also has been frequent due to recent raw material shortages and delays in production,³ forcing pharmacies to refill small amounts of medication in various dosage forms despite patients owing the same copayment per prescription.

Topical Therapies

Topical retinoid prescription affordability has always been fraught with difficulty owing to age cutoffs because it is often restricted to patients younger than 25 or 40 years, depending on the plan,^4,5 but the availability of adapalene gel 0.1% as an OTC preparation in 2016 has broadened retinoid access and use.⁶ Prescription benzoyl peroxide (BPO) products alone or in combination with retinoids or topical antibiotics (or other combination topical therapies) comprise a large number of branded prescriptions often not covered by insurance or are only affordable using proprietary and intermittently available coupon cards (eg, BPO-clindamycin, clindamycin-tretinoin, BPO-adapalene); therefore, prescribers tend to dispense the individual ingredients and instruct the patient to compound them at home. Furthermore, BPO products can be purchased in effective concentrations as OTC products, and patients looking to procure more affordable, albeit less effective, topical retinoids that also have less irritation potential reach for OTC nightly retinol creams nestled in the antiaging section of the pharmacy.⁷

Opportunities to be involved in the larger scaffold of patient advocacy also are plentiful at the state and national levels. For example, with the support of dermatology state societies and the American Academy of Dermatology Association, Pennsylvania House Bill 2211⁸ and similar bills in other states call for reversal of the gag clause that prevents pharmacists from disclosing the best medication price to patients. Also guided by the American Academy of Dermatology Association, prior authorization model legislation to promote transparency across insurers in this haphazard process is emerging across the country.^9,10

Final Thoughts

These examples of acne medication access and cost quandaries serve to embody the daily problem-solving that dermatologists execute as part of their growing administrative and economic duties. They also represent worthy efforts to consider on behalf of patients, their pocketbooks, and the prudent use of their dermatologists’ time.

We are fortunate to have plentiful acne treatment options available that cater to each patient’s clinical examination, predispositions, and triggers, but the choices are daunting amidst the vast prescription and over-the-counter (OTC) topicals available along with disparate insurance and cost nuances. In addition, when prescribing generic oral therapies, it is complicated to parse out regional differences in price, supply, and insurance coverage to advocate best for each patient and land upon the delicate balance between efficacy, safety, and financial stewardship, both at an individual and community level. I will outline some challenges and solutions to the management of acne amidst these complicated factors.

Oral Therapies

For isotretinoin, generic choices, cost, and tiering within insurance plans are perpetual moving targets despite the drug being the only member of its class.¹ Prescriber resources include tandem searches of electronic medical record price estimates within each insurance formulary, individual pharmacy search engines, and compilation mobile applications such as GoodRx to select the most affordable version for each patient. As an example of a regional trend, isotretinoin generic coverage by one provider in Pennsylvania shifted earlier this year so that every patient, whether new to isotretinoin or midcourse, required a new prior authorization with more stringent coverage requirements including failure of 2 oral antibiotics. Swiftly thereafter, efforts across the state driven by the Pennsylvania Academy of Dermatology and Dermatologic Surgery and its members and fueled by poignant patient vignettes about fragmented and substandard patient care helped to reverse this policy and remove the prior authorization mandate.²

Tetracyclines have experienced broad cost swings, mostly based on disruption of manufacturing at the limited number of distribution sites in the United States. In 2011, a tetracycline shortage arose due to a major manufacturer’s recall³ and persisted with subsequent material shortages, as doxycycline became the preferred and cheaper member of the class. Doxycycline price tag hikes then occurred following Hurricane Sandy when an East Coast manufacturing site was damaged.³ Spironolactone backorder also has been frequent due to recent raw material shortages and delays in production,³ forcing pharmacies to refill small amounts of medication in various dosage forms despite patients owing the same copayment per prescription.

Topical Therapies

Topical retinoid prescription affordability has always been fraught with difficulty owing to age cutoffs because it is often restricted to patients younger than 25 or 40 years, depending on the plan,^4,5 but the availability of adapalene gel 0.1% as an OTC preparation in 2016 has broadened retinoid access and use.⁶ Prescription benzoyl peroxide (BPO) products alone or in combination with retinoids or topical antibiotics (or other combination topical therapies) comprise a large number of branded prescriptions often not covered by insurance or are only affordable using proprietary and intermittently available coupon cards (eg, BPO-clindamycin, clindamycin-tretinoin, BPO-adapalene); therefore, prescribers tend to dispense the individual ingredients and instruct the patient to compound them at home. Furthermore, BPO products can be purchased in effective concentrations as OTC products, and patients looking to procure more affordable, albeit less effective, topical retinoids that also have less irritation potential reach for OTC nightly retinol creams nestled in the antiaging section of the pharmacy.⁷

Opportunities to be involved in the larger scaffold of patient advocacy also are plentiful at the state and national levels. For example, with the support of dermatology state societies and the American Academy of Dermatology Association, Pennsylvania House Bill 2211⁸ and similar bills in other states call for reversal of the gag clause that prevents pharmacists from disclosing the best medication price to patients. Also guided by the American Academy of Dermatology Association, prior authorization model legislation to promote transparency across insurers in this haphazard process is emerging across the country.^9,10

Final Thoughts

These examples of acne medication access and cost quandaries serve to embody the daily problem-solving that dermatologists execute as part of their growing administrative and economic duties. They also represent worthy efforts to consider on behalf of patients, their pocketbooks, and the prudent use of their dermatologists’ time.

I write this editorial at the end of June as summer officially begins. Much of the country—my New Mexico home included—is suffering under an unbearable heat wave in which even those without belief pray for rain. Summer for many is associated with vacations, family trips, and happy hours in the swimming pool among other enjoyable activities that provide a welcome and much deserved break from routine and relief from the grind of work and school. In the words of the George Gershwin tune, “Summertime, and the livin’ is easy.”

In stark contrast to this season, where there is more lightness in being, is the heaviness of the news reports about the Department of Veteran Affairs (VA) that have been featured in the media and the federal press. I suspect I am not alone in having a hard time opening those e-mails; feeling once more the weight of failure on the VA and the employees who have dedicated a good part of their careers to its mission. Even for the VA, June has seen an exceptional string of bad press. I ask as you read this column to think about what the adjective bad means in this context. In the conclusion to this column, I will suggest that the meaning is multivalent.

Among the most distressing stories was the USA Today and Boston Globe headline, “Secret VA nursing home ratings hide poor quality of care from the public.”² In an all too predictable sequence, this led justifiably to a cascade of demands from the fifth estate, congressional representatives, the administration, veterans and their families, and watchdog organizations for release of the data, investigation of the allegedly deplorable conditions, and rapid fixes to the problems along with the punishment of the guilty.

As an ethicist I am committed to the principles of transparency and accountability that these entities rightly adjure in the wake of any disclosure of a breach of duty to treat each veteran with the best we have—especially the disabled, elderly, and vulnerable. But I have come to believe that the way in which this cycle of scandal and reaction plays out over and over again in VA facilities across the country, what I call “caring under the microscope,” is actually undermining the righteous goals it seeks to achieve.

I encourage you to try this online. Search for the phrase, “VA under microscope” and see what you get. Briefly read the summary, or the entire story if you have the inclination, and then take a few minutes to reflect on the emotional impact of what you read. Under a microscope is an idiom coined to capture the experience of being the object of close inspection and intense scrutiny. As most everyone knows from their own science education, microscopes magnify images that cannot normally be seen with the human eye, allowing us to observe a more detailed and focused image. The microscope surely helped revolutionize medicine and science. But what effect does such amplified and constant observation have on VA employees?³

For the thousands of staff members who do their job every day with all the empathy and skill, integrity, and dedication they can muster, there is demoralization. Researchers in the health professions describe it as “a feeling state of dejection, hopelessness, and a sense of personal ‘incompetence’ that may be tied to a loss of or threat to one’s own goals or values. It has an existential dimension when beliefs and values about oneself are disconfirmed.”⁴ If you are a nurse assigned to one of VA’s nursing homes, daily striving to ensure patients are clean and comfortable, or a therapist in a continuing living center using all your training to maximize an elder’s mobility and participation in activities, you might well begin to doubt your ability as a professional and question the worth of your work. This is exactly the opposite outcome that the microscopic oversight is intended to attain.

The impact of demoralization on health professionals directly contributes to unprecedented burnout and turnover. Were this not damaging enough, it also has an insidious rippling effect—like contaminated groundwater that poisons where it should be reviving. The humanistic, even spiritual, heart of all the health professions is the relationship between the practitioner and the patient, ideally a relationship of mutual respect and trust. Waves of negative news triggering harsh and unyielding criticism distort even the strongest, purest therapeutic alliances with fear and distrust, just as a microscope not properly focused changes a beautiful image into a blurred muddle.

Worried families of veterans staring at this picture invariably are drawn into the hyper media focus, feeling alarmed and betrayed, even when their loved one may be receiving excellent VA care. In 20 years as a physician and ethicist in VA hospitals, clinics, and community living centers, I know well that bad things happen to good people (both patients and staff). Yet VA patients, families, and staff are seldom offered the wider corrective vision that would note that bad things also happen in other health care institutions and good care is delivered in the VA. Acting Secretary of Veterans Affairs Peter O’Rourke crisply summarized in his response to the nursing home story.⁵

No veteran or any other human being in a VA or any other nursing home should ever be medicated into a zombie state or left alone in pain like those patients reported in the news story. And if the USA Today story improves the care of a single VA patient, then good has been done at least in the short run. Yet we must also take the long view and consider the moral and psychological outcome of prolonged demoralization on the very staff who must carry out the congressional mandates.

In the same time frame as the nursing home scandal, the VA Office of Inspector General also issued a report on the continued understaffing in the VA.⁶ This may be the most concerning aftermath of demoralization. One of my best residents had thought about the VA but in the end made a different choice when he completed his training. When I asked him why he told me, “I am afraid to end up in the newspaper.”

Summer will go by far too quickly. Enjoy it while you can so that with renewed strength we may all search for a better way that the light of truth and heat of power can do what they must while also not withering the spirit of caring that animates the people of the VA.

I write this editorial at the end of June as summer officially begins. Much of the country—my New Mexico home included—is suffering under an unbearable heat wave in which even those without belief pray for rain. Summer for many is associated with vacations, family trips, and happy hours in the swimming pool among other enjoyable activities that provide a welcome and much deserved break from routine and relief from the grind of work and school. In the words of the George Gershwin tune, “Summertime, and the livin’ is easy.”

In stark contrast to this season, where there is more lightness in being, is the heaviness of the news reports about the Department of Veteran Affairs (VA) that have been featured in the media and the federal press. I suspect I am not alone in having a hard time opening those e-mails; feeling once more the weight of failure on the VA and the employees who have dedicated a good part of their careers to its mission. Even for the VA, June has seen an exceptional string of bad press. I ask as you read this column to think about what the adjective bad means in this context. In the conclusion to this column, I will suggest that the meaning is multivalent.

Among the most distressing stories was the USA Today and Boston Globe headline, “Secret VA nursing home ratings hide poor quality of care from the public.”² In an all too predictable sequence, this led justifiably to a cascade of demands from the fifth estate, congressional representatives, the administration, veterans and their families, and watchdog organizations for release of the data, investigation of the allegedly deplorable conditions, and rapid fixes to the problems along with the punishment of the guilty.

As an ethicist I am committed to the principles of transparency and accountability that these entities rightly adjure in the wake of any disclosure of a breach of duty to treat each veteran with the best we have—especially the disabled, elderly, and vulnerable. But I have come to believe that the way in which this cycle of scandal and reaction plays out over and over again in VA facilities across the country, what I call “caring under the microscope,” is actually undermining the righteous goals it seeks to achieve.

I encourage you to try this online. Search for the phrase, “VA under microscope” and see what you get. Briefly read the summary, or the entire story if you have the inclination, and then take a few minutes to reflect on the emotional impact of what you read. Under a microscope is an idiom coined to capture the experience of being the object of close inspection and intense scrutiny. As most everyone knows from their own science education, microscopes magnify images that cannot normally be seen with the human eye, allowing us to observe a more detailed and focused image. The microscope surely helped revolutionize medicine and science. But what effect does such amplified and constant observation have on VA employees?³

For the thousands of staff members who do their job every day with all the empathy and skill, integrity, and dedication they can muster, there is demoralization. Researchers in the health professions describe it as “a feeling state of dejection, hopelessness, and a sense of personal ‘incompetence’ that may be tied to a loss of or threat to one’s own goals or values. It has an existential dimension when beliefs and values about oneself are disconfirmed.”⁴ If you are a nurse assigned to one of VA’s nursing homes, daily striving to ensure patients are clean and comfortable, or a therapist in a continuing living center using all your training to maximize an elder’s mobility and participation in activities, you might well begin to doubt your ability as a professional and question the worth of your work. This is exactly the opposite outcome that the microscopic oversight is intended to attain.

The impact of demoralization on health professionals directly contributes to unprecedented burnout and turnover. Were this not damaging enough, it also has an insidious rippling effect—like contaminated groundwater that poisons where it should be reviving. The humanistic, even spiritual, heart of all the health professions is the relationship between the practitioner and the patient, ideally a relationship of mutual respect and trust. Waves of negative news triggering harsh and unyielding criticism distort even the strongest, purest therapeutic alliances with fear and distrust, just as a microscope not properly focused changes a beautiful image into a blurred muddle.

Worried families of veterans staring at this picture invariably are drawn into the hyper media focus, feeling alarmed and betrayed, even when their loved one may be receiving excellent VA care. In 20 years as a physician and ethicist in VA hospitals, clinics, and community living centers, I know well that bad things happen to good people (both patients and staff). Yet VA patients, families, and staff are seldom offered the wider corrective vision that would note that bad things also happen in other health care institutions and good care is delivered in the VA. Acting Secretary of Veterans Affairs Peter O’Rourke crisply summarized in his response to the nursing home story.⁵

No veteran or any other human being in a VA or any other nursing home should ever be medicated into a zombie state or left alone in pain like those patients reported in the news story. And if the USA Today story improves the care of a single VA patient, then good has been done at least in the short run. Yet we must also take the long view and consider the moral and psychological outcome of prolonged demoralization on the very staff who must carry out the congressional mandates.

In the same time frame as the nursing home scandal, the VA Office of Inspector General also issued a report on the continued understaffing in the VA.⁶ This may be the most concerning aftermath of demoralization. One of my best residents had thought about the VA but in the end made a different choice when he completed his training. When I asked him why he told me, “I am afraid to end up in the newspaper.”

Summer will go by far too quickly. Enjoy it while you can so that with renewed strength we may all search for a better way that the light of truth and heat of power can do what they must while also not withering the spirit of caring that animates the people of the VA.

I write this editorial at the end of June as summer officially begins. Much of the country—my New Mexico home included—is suffering under an unbearable heat wave in which even those without belief pray for rain. Summer for many is associated with vacations, family trips, and happy hours in the swimming pool among other enjoyable activities that provide a welcome and much deserved break from routine and relief from the grind of work and school. In the words of the George Gershwin tune, “Summertime, and the livin’ is easy.”

In stark contrast to this season, where there is more lightness in being, is the heaviness of the news reports about the Department of Veteran Affairs (VA) that have been featured in the media and the federal press. I suspect I am not alone in having a hard time opening those e-mails; feeling once more the weight of failure on the VA and the employees who have dedicated a good part of their careers to its mission. Even for the VA, June has seen an exceptional string of bad press. I ask as you read this column to think about what the adjective bad means in this context. In the conclusion to this column, I will suggest that the meaning is multivalent.

Among the most distressing stories was the USA Today and Boston Globe headline, “Secret VA nursing home ratings hide poor quality of care from the public.”² In an all too predictable sequence, this led justifiably to a cascade of demands from the fifth estate, congressional representatives, the administration, veterans and their families, and watchdog organizations for release of the data, investigation of the allegedly deplorable conditions, and rapid fixes to the problems along with the punishment of the guilty.

As an ethicist I am committed to the principles of transparency and accountability that these entities rightly adjure in the wake of any disclosure of a breach of duty to treat each veteran with the best we have—especially the disabled, elderly, and vulnerable. But I have come to believe that the way in which this cycle of scandal and reaction plays out over and over again in VA facilities across the country, what I call “caring under the microscope,” is actually undermining the righteous goals it seeks to achieve.

I encourage you to try this online. Search for the phrase, “VA under microscope” and see what you get. Briefly read the summary, or the entire story if you have the inclination, and then take a few minutes to reflect on the emotional impact of what you read. Under a microscope is an idiom coined to capture the experience of being the object of close inspection and intense scrutiny. As most everyone knows from their own science education, microscopes magnify images that cannot normally be seen with the human eye, allowing us to observe a more detailed and focused image. The microscope surely helped revolutionize medicine and science. But what effect does such amplified and constant observation have on VA employees?³

For the thousands of staff members who do their job every day with all the empathy and skill, integrity, and dedication they can muster, there is demoralization. Researchers in the health professions describe it as “a feeling state of dejection, hopelessness, and a sense of personal ‘incompetence’ that may be tied to a loss of or threat to one’s own goals or values. It has an existential dimension when beliefs and values about oneself are disconfirmed.”⁴ If you are a nurse assigned to one of VA’s nursing homes, daily striving to ensure patients are clean and comfortable, or a therapist in a continuing living center using all your training to maximize an elder’s mobility and participation in activities, you might well begin to doubt your ability as a professional and question the worth of your work. This is exactly the opposite outcome that the microscopic oversight is intended to attain.

The impact of demoralization on health professionals directly contributes to unprecedented burnout and turnover. Were this not damaging enough, it also has an insidious rippling effect—like contaminated groundwater that poisons where it should be reviving. The humanistic, even spiritual, heart of all the health professions is the relationship between the practitioner and the patient, ideally a relationship of mutual respect and trust. Waves of negative news triggering harsh and unyielding criticism distort even the strongest, purest therapeutic alliances with fear and distrust, just as a microscope not properly focused changes a beautiful image into a blurred muddle.

Worried families of veterans staring at this picture invariably are drawn into the hyper media focus, feeling alarmed and betrayed, even when their loved one may be receiving excellent VA care. In 20 years as a physician and ethicist in VA hospitals, clinics, and community living centers, I know well that bad things happen to good people (both patients and staff). Yet VA patients, families, and staff are seldom offered the wider corrective vision that would note that bad things also happen in other health care institutions and good care is delivered in the VA. Acting Secretary of Veterans Affairs Peter O’Rourke crisply summarized in his response to the nursing home story.⁵

No veteran or any other human being in a VA or any other nursing home should ever be medicated into a zombie state or left alone in pain like those patients reported in the news story. And if the USA Today story improves the care of a single VA patient, then good has been done at least in the short run. Yet we must also take the long view and consider the moral and psychological outcome of prolonged demoralization on the very staff who must carry out the congressional mandates.

In the same time frame as the nursing home scandal, the VA Office of Inspector General also issued a report on the continued understaffing in the VA.⁶ This may be the most concerning aftermath of demoralization. One of my best residents had thought about the VA but in the end made a different choice when he completed his training. When I asked him why he told me, “I am afraid to end up in the newspaper.”

Summer will go by far too quickly. Enjoy it while you can so that with renewed strength we may all search for a better way that the light of truth and heat of power can do what they must while also not withering the spirit of caring that animates the people of the VA.

During the early days of the American Academy of Nurse Practitioners (incorporated in 1985), I spotted a full-page ad by the Campaign Consultants of America addressed to professional fund­raisers. What caught my eye was a photograph of a mother with the tagline, “There’s only one person who understands you better than we do, and she still doesn’t understand what you do for a living.” I pulled the page from the magazine and made a note to consider using it to promote the NP profession. What we needed at the time, despite being an established profession, was to publicly market our role as experts in health promotion and disease prevention. What we needed was brand recognition.

Historically, branding has been a task undertaken by a company’s marketing department or an advertising agency to identify elements that differentiate their product from the competition’s. Designing a logo, creating a jingle (Oscar-Mayer, anyone?), or recording a sound bite are the means to emphasize the difference. It paints the mental picture people have of a company, a product, or a provider. These cues remind the consumer about the product. So, how does this apply to the NP (and PA) profession?

The importance of establishing a “brand”—of distinguishing ourselves as competent clinicians with a specific skillset to offer the primary care community—cannot be overstated. Personal branding is a key component of fostering patient loyalty, building your reputation, and increasing referrals to your practice. Understanding the needs and desires of patients, their families, and the community is crucial. Our personal brand emphasizes our assets and expertise. While it can be difficult to look at yourself objectively (especially your assets), it is necessary in today’s competitive world of health care.

NPs constitute the fastest-growing segment of the primary care workforce in the US. More than 50 years of transforming health care as we know it has made us indispensable as health care providers. The literature has long supported the position that NPs provide care that is effective, patient-centered, and evidenced-based. Who we are, what we do, and how well we do it has been documented in myriad reports, surveys, and publications. Yet in many ways, we continue to struggle with an in-between identity. Despite our increasing responsibility in the clinical realm, some are still confused as to who we are.

We are known as nurses first, yet much of the health care we now provide was traditionally in the “physician-only” domain. And because of that history, our ability to function to the fullest extent of our education has been hobbled. These practice restrictions are counterproductive at a time when our nation is facing serious public health challenges.

Over the years, barriers to practice have slowly been whittled away, but full appreciation and recognition of our professional excellence and our contribution to improve the nation’s health is lacking. The fact that much of the research on health status and health ranking fails to include NPs and PAs is testimony that we remain somewhat invisible. And that, my friends, is exactly why it is time to revisit that aforementioned advertisement—not because our mothers don’t know what we do, but because, to some degree, we have eased off the belief that there are still obstacles to full access to NPs as primary care providers. And that is the origin of the need to establish your own brand.

Creating and maintaining your personal brand necessitates that you be multi-functional. You must be a role model, a mentor, and a voice that is respected and reliable. Your brand should advertise what you are known for and what motivates people to seek you, specifically, for their health care needs. Be relentlessly focused on what you do that adds value. As NPs, we have a unique blend of nursing and medicine that allows us to provide the patient-centered care that is central to meeting the existing and future primary care needs of our nation. From our roots in nursing, we offer patients high-quality care and a provider to partner with them in developing their plan of care.

Continue to: A foundational component of building your brand is...

A foundational component of building your brand is positioning yourself as a credible expert and leader. We each have a unique collection of experiences in preventive and primary care. Share that experience by getting involved in your community: participate in health fairs, interact with local news media, or volunteer to serve on your local health board. Emphasize the quality, flexibility, and continuity of care that you can provide. Share any survey findings that demonstrate your ability to anticipate, meet, and even exceed patients’ needs. Demonstrate your ability to deliver quality, accessible health care in a diverse society with increasingly complex medical needs.

As the nation continues to face a shortage of primary care providers and services—a gap that NPs and PAs are equipped to fill—it’s time for us to promote ourselves and advertise all that we can do. This isn’t just for our own sakes, but for our patients’ as well. Give some serious thought (and even more serious effort) to imagining and developing yourself as a brand. Define your brand’s attributes and the qualities or characteristics that make you distinctive from your competitors (or even your colleagues). You are the CEO of brand YOU!

If you have examples of how you promote your personal brand, please share them with me at [email protected].

During the early days of the American Academy of Nurse Practitioners (incorporated in 1985), I spotted a full-page ad by the Campaign Consultants of America addressed to professional fund­raisers. What caught my eye was a photograph of a mother with the tagline, “There’s only one person who understands you better than we do, and she still doesn’t understand what you do for a living.” I pulled the page from the magazine and made a note to consider using it to promote the NP profession. What we needed at the time, despite being an established profession, was to publicly market our role as experts in health promotion and disease prevention. What we needed was brand recognition.

Historically, branding has been a task undertaken by a company’s marketing department or an advertising agency to identify elements that differentiate their product from the competition’s. Designing a logo, creating a jingle (Oscar-Mayer, anyone?), or recording a sound bite are the means to emphasize the difference. It paints the mental picture people have of a company, a product, or a provider. These cues remind the consumer about the product. So, how does this apply to the NP (and PA) profession?

The importance of establishing a “brand”—of distinguishing ourselves as competent clinicians with a specific skillset to offer the primary care community—cannot be overstated. Personal branding is a key component of fostering patient loyalty, building your reputation, and increasing referrals to your practice. Understanding the needs and desires of patients, their families, and the community is crucial. Our personal brand emphasizes our assets and expertise. While it can be difficult to look at yourself objectively (especially your assets), it is necessary in today’s competitive world of health care.

NPs constitute the fastest-growing segment of the primary care workforce in the US. More than 50 years of transforming health care as we know it has made us indispensable as health care providers. The literature has long supported the position that NPs provide care that is effective, patient-centered, and evidenced-based. Who we are, what we do, and how well we do it has been documented in myriad reports, surveys, and publications. Yet in many ways, we continue to struggle with an in-between identity. Despite our increasing responsibility in the clinical realm, some are still confused as to who we are.

We are known as nurses first, yet much of the health care we now provide was traditionally in the “physician-only” domain. And because of that history, our ability to function to the fullest extent of our education has been hobbled. These practice restrictions are counterproductive at a time when our nation is facing serious public health challenges.

Over the years, barriers to practice have slowly been whittled away, but full appreciation and recognition of our professional excellence and our contribution to improve the nation’s health is lacking. The fact that much of the research on health status and health ranking fails to include NPs and PAs is testimony that we remain somewhat invisible. And that, my friends, is exactly why it is time to revisit that aforementioned advertisement—not because our mothers don’t know what we do, but because, to some degree, we have eased off the belief that there are still obstacles to full access to NPs as primary care providers. And that is the origin of the need to establish your own brand.

Creating and maintaining your personal brand necessitates that you be multi-functional. You must be a role model, a mentor, and a voice that is respected and reliable. Your brand should advertise what you are known for and what motivates people to seek you, specifically, for their health care needs. Be relentlessly focused on what you do that adds value. As NPs, we have a unique blend of nursing and medicine that allows us to provide the patient-centered care that is central to meeting the existing and future primary care needs of our nation. From our roots in nursing, we offer patients high-quality care and a provider to partner with them in developing their plan of care.

Continue to: A foundational component of building your brand is...

A foundational component of building your brand is positioning yourself as a credible expert and leader. We each have a unique collection of experiences in preventive and primary care. Share that experience by getting involved in your community: participate in health fairs, interact with local news media, or volunteer to serve on your local health board. Emphasize the quality, flexibility, and continuity of care that you can provide. Share any survey findings that demonstrate your ability to anticipate, meet, and even exceed patients’ needs. Demonstrate your ability to deliver quality, accessible health care in a diverse society with increasingly complex medical needs.

As the nation continues to face a shortage of primary care providers and services—a gap that NPs and PAs are equipped to fill—it’s time for us to promote ourselves and advertise all that we can do. This isn’t just for our own sakes, but for our patients’ as well. Give some serious thought (and even more serious effort) to imagining and developing yourself as a brand. Define your brand’s attributes and the qualities or characteristics that make you distinctive from your competitors (or even your colleagues). You are the CEO of brand YOU!

If you have examples of how you promote your personal brand, please share them with me at [email protected].

During the early days of the American Academy of Nurse Practitioners (incorporated in 1985), I spotted a full-page ad by the Campaign Consultants of America addressed to professional fund­raisers. What caught my eye was a photograph of a mother with the tagline, “There’s only one person who understands you better than we do, and she still doesn’t understand what you do for a living.” I pulled the page from the magazine and made a note to consider using it to promote the NP profession. What we needed at the time, despite being an established profession, was to publicly market our role as experts in health promotion and disease prevention. What we needed was brand recognition.

Historically, branding has been a task undertaken by a company’s marketing department or an advertising agency to identify elements that differentiate their product from the competition’s. Designing a logo, creating a jingle (Oscar-Mayer, anyone?), or recording a sound bite are the means to emphasize the difference. It paints the mental picture people have of a company, a product, or a provider. These cues remind the consumer about the product. So, how does this apply to the NP (and PA) profession?

The importance of establishing a “brand”—of distinguishing ourselves as competent clinicians with a specific skillset to offer the primary care community—cannot be overstated. Personal branding is a key component of fostering patient loyalty, building your reputation, and increasing referrals to your practice. Understanding the needs and desires of patients, their families, and the community is crucial. Our personal brand emphasizes our assets and expertise. While it can be difficult to look at yourself objectively (especially your assets), it is necessary in today’s competitive world of health care.

NPs constitute the fastest-growing segment of the primary care workforce in the US. More than 50 years of transforming health care as we know it has made us indispensable as health care providers. The literature has long supported the position that NPs provide care that is effective, patient-centered, and evidenced-based. Who we are, what we do, and how well we do it has been documented in myriad reports, surveys, and publications. Yet in many ways, we continue to struggle with an in-between identity. Despite our increasing responsibility in the clinical realm, some are still confused as to who we are.

We are known as nurses first, yet much of the health care we now provide was traditionally in the “physician-only” domain. And because of that history, our ability to function to the fullest extent of our education has been hobbled. These practice restrictions are counterproductive at a time when our nation is facing serious public health challenges.

Over the years, barriers to practice have slowly been whittled away, but full appreciation and recognition of our professional excellence and our contribution to improve the nation’s health is lacking. The fact that much of the research on health status and health ranking fails to include NPs and PAs is testimony that we remain somewhat invisible. And that, my friends, is exactly why it is time to revisit that aforementioned advertisement—not because our mothers don’t know what we do, but because, to some degree, we have eased off the belief that there are still obstacles to full access to NPs as primary care providers. And that is the origin of the need to establish your own brand.

Creating and maintaining your personal brand necessitates that you be multi-functional. You must be a role model, a mentor, and a voice that is respected and reliable. Your brand should advertise what you are known for and what motivates people to seek you, specifically, for their health care needs. Be relentlessly focused on what you do that adds value. As NPs, we have a unique blend of nursing and medicine that allows us to provide the patient-centered care that is central to meeting the existing and future primary care needs of our nation. From our roots in nursing, we offer patients high-quality care and a provider to partner with them in developing their plan of care.

Continue to: A foundational component of building your brand is...

A foundational component of building your brand is positioning yourself as a credible expert and leader. We each have a unique collection of experiences in preventive and primary care. Share that experience by getting involved in your community: participate in health fairs, interact with local news media, or volunteer to serve on your local health board. Emphasize the quality, flexibility, and continuity of care that you can provide. Share any survey findings that demonstrate your ability to anticipate, meet, and even exceed patients’ needs. Demonstrate your ability to deliver quality, accessible health care in a diverse society with increasingly complex medical needs.

As the nation continues to face a shortage of primary care providers and services—a gap that NPs and PAs are equipped to fill—it’s time for us to promote ourselves and advertise all that we can do. This isn’t just for our own sakes, but for our patients’ as well. Give some serious thought (and even more serious effort) to imagining and developing yourself as a brand. Define your brand’s attributes and the qualities or characteristics that make you distinctive from your competitors (or even your colleagues). You are the CEO of brand YOU!

If you have examples of how you promote your personal brand, please share them with me at [email protected].