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The professional advancement of drug and device innovation
I often say that there are both “guardrail” days and very good days when it comes to the ins and outs of health care builds and product launches. The process is much like starting down the path of a country road in the middle of a blizzard—unless you have dependable wipers and a good defrost system, that path can get murky very quickly. With this article I hope to offer my counsel to inventors, featuring a few of my prior launches as well as case studies of health care launches I was not involved with, and sharing the lessons learned and hurdles that were overcome. I encourage all entrepreneurs to act on their ideas because, in the world of health care startups, the only failure is not acting on an invention.
Case study 1: Cerezyme
Today, Cerezyme is indicated for patients with Gaucher, which is a lysosomal storage disorder. Cerezyme’s first-generation product, called Ceredase, was a human tissue-derived protein that we extracted from human placentas. At the time, the concept of moving this program forward was denied by the Board of Directors because they said that even if you could collect enough placentas to make the enzyme, it would be too expensive to manufacture. In fact, early scale-up modeling for manufacturing the protein demonstrated that Genzyme would need 4 tons of placentas per Gaucher patient per year.
Gaucher is a severe, early-onset disease that has a significant negative outcome for patients. Patients with Gaucher are in dire need of treatment. Genzyme went forward with the Ceredase program by financing it through the families of patients with the disease, by starting an LLC separate from the business and funding the initial clinical trial and the development of the protein through the families of Gaucher patients. That approach was a successful endeavor. A great example of a creative capital structure to advance a program.
This was in the late 1980s/early 1990s, and at the height of the AIDS challenge. Genzyme based the manufacturing in Lille, France, and we cryopreserved placentas in the United States and Europe and shipped them to Lille to be processed into therapy. Genzyme eventually received approval for Ceredase from the US Food and Drug Administration (FDA) and the European Medicines Agency. At the height of the placenta collection, we were gathering about 10% to 15% of the placentas in the United States and 30% to 40% of the placentas in Europe. Resources supply became an issue until we developed a recombinant form of the protein, accomplished by using a manufacturing system called a CHO cell line.
This is a very good success story: If this invention was not pursued, Gaucher patients would not benefit from the treatment today. In addition, there are a plethora of patients with different lysosomal storage disorders treated with additional proteins that have been aided by us going through the entire development, manufacturing, and global commercialization process. We figured out how to manufacture and deliver the treatment, working through multiple countries’ political systems, and today the therapy is paid for by insurance and government systems on a worldwide basis.
Continue to: Case study 2...
Case study 2: ThinPrep
I like to use the approval of ThinPrep as an example of avoiding a false negative—a stoppage in the development of the product or drug for the wrong reasons. False negatives, in my mind, occur when you are developing a technology and you run into issues during the clinical phase and/or with FDA approval, or with a technical failure or you run out of capital prior to knowing whether or not the innovation actually works. In the case of ThinPrep, a poorly run clinical trial almost resulted in a false negative.
The company at the time was Cytyc, and an initial clinical study presented to the FDA yielded a neutral-negative outcome. The FDA said that there were not enough data to show the differentiation from the current Pap smear standard of care.
The founders of the company at that time had inherited the study protocol from a prior leadership team, so they had to finish the trial with the initial protocol. Given the FDA’s advisement, they developed a new trial. It took the persistence of these two founders, who mortgaged their homes and spent their personal dollars to take this through the next wave of clinical development. In the end it was successful. The revised clinical trial yielded an approval for ThinPrep, which is now considered a standard of care.
The use of ThinPrep reduced cervical cancer deaths by 40% from preapproval. The challenging path from clinical development to eventual commercial launch and physician leadership in advancing patient care makes the story of ThinPrep a great example of not allowing an early false negative of a poorly designed and run clinical trial stop important innovation.
Case study 3: Cologuard
The development of Cologuard is a case study demonstrating that, sometimes, when your first attempt does not work, you need to have the persistence to raise additional capital and/or use a slightly different technical approach. The approval story of Cologuard is important to share because it is an important cancer screening diagnostic, using DNA from stool samples to test for colon cancer, giving access to important colon cancer screening to many patients. Currently, caregivers are only scraping the surface with Cologuard’s ability to screen the population. There are many more patients that need access to the test, and I believe they will get it in the years ahead.
Cologuard went through a first- and second-generational technical failure. They could not get the test’s specificity and sensitivity to be at the level of a screening tool; there were too many false-positive results. With the third iteration came the technical breakthrough, and a very large, expensive study was conducted—one the leadership team was criticized for. However, that study yielded the data that achieved a New England Journal of Medicine article, and reimbursement support across the country. The combination of the right technical team and the right leadership team, who planned a proper commercial launch, with a CEO that supported the extensive clinical study, has resulted in the fourth generation of Cologuard—an important breakthrough offering a very useful new standard of care in colon cancer detection and screening.
Continue to: Pearls for moving your innovations forward...
Pearls for moving your innovations forward
Because of my experience in undergoing health care start-ups, and contributing to several of those advancements of innovation, many inventors approach me for advice on their paths from idea to full-concept company. Here are a few of my lessons learned.
Consider purpose, not financial gain, first and foremost. Financial gain is typically the by-product or outcome of a standard-of-care breakthrough for inventors, but it’s a very hard road. Pursue your invention for advancing patient care and moving a new standard of care forward in health care versus financial gain at the end.
Determine whether your invention is a product or a company, or potentially, not capitalizable at all. Figure this out early. Analyze your idea to make sure it is sound and truly novel. Analyze the competition and to make sure it is sound and truly novel. Analyze the competition and the market dynamics to support a new product. Can the development path be defined very clearly to raise capital? Is your innovation a big enough breakthrough in the market with several current products to actually make a difference in patient outcomes (and eventually achieve product reimbursement)? The creation of a company may be the right strategy if the innovation can support a differentiated enough breakthrough where you can actually support all the infrastructure to build the business. If you find that the market is not there to support and develop your idea to eventual success, backing off early is important to preserve invested capital.
Protect early. Is your invention patentable, or has someone else already thought of the idea? What kind of patent(s) are appropriate? Where, geographically, do you want to protect your invention? Find a good patent attorney in your local area, early in the process, to help you answer all of these critical questions. Patents are expensive to file and maintain, but it is not expensive to do a literature search to find out if your idea is novel. A provisional patent, which would be your first step, is an important cost-effective step.
Capital is out there. If your invention or idea deserves capital, it is available. I will address raising capital in more detail in the next section.
Consider regulatory and manufacturing as achievable hurdles. Inventors often get tripped up here, considering the regulatory hurdles and manufacturing too challenging and abandoning their ideas because the risk is too great. Regulatory and manufacturing are very important aspects of health care standard-of-care builds. Cutting corners is not an option. That said, regulatory and manufacturing should not stop you. Challenges often can be worked through as long as the clinical need is there, and the clinical data support bringing that technology forward.
Consider corporate partnerships. I am a fan of corporate partners. But which ones should you target, and when and why? Corporate partnerships can bring significant capital, which is great, but there is enough investor capital out there that you should not pursue a corporate partner just for capital. The main benefit of a corporate partner is enterprise intellect. They typically know more about the field that you are entering than the investors or a small company leadership team.
Establish and listen to advisors. When thinking about who to trust, research their track record. Advisors who have gone through this process before, and specifically in your product area, are important to have access to.
Persistence is key. I have observed a tremendous “compression of innovation” in the health care areas that I have been involved with—human tissue-derived proteins, robotic surgery, stem cell therapy, and digital health (which is still in its infancy). For each of these breakthrough categories, early on, it appeared that it couldn’t be done. However, after the first 2 or 3 major breakthroughs in each one of these areas, a compression of innovation occurred. For instance, after approximately 15 years of protein development, we came out with the recombinant manufacturing systems for proteins. Very quickly, within 10 years, there were more than 70 proteins on the market. The persistence of the inventors to overcome early obstacles in each of these health care areas was critical to future success in each area.
Continue to: Raising capital...
Raising capital
There are different investors who specialize in different types of investment opportunities. The first phase of raising capital is the seed round—where there is typically early data, or even no data and just a concept. From this seed round forward, there is less risk as you develop your technology; thus, there are different investors that support different stages of development and that specialize in different types of investing. It is important to target the right investors and raise enough capital to be able to go achieve multiple operational milestones. Otherwise, when you go through your first round of capital, or the Series A or B financing rounds, there may not be a set of investors out there to fund the company moving forward. Health care investors will make it known that they invest in certain rounds of capital. You can determine who those investors are by doing a search online.
A mistake health care inventors can make is not taking enough capital from investors, because they are concerned about dilution. I advise investors not to focus on dilution but rather on, how big can you make “the pie” (value of the company) worth? The entire process is about bringing a true product through to a new standard-of-care curve.
Trust is the most important thing to earn with investors, and there is zero tolerance for a lack of trust. Share your vision as the inventor with investors, who want to know where this category could be in the next 5 or 10 years. Clinical data will always win, and health care investors and industry leaders should be focused on executing the most robust clinical data to demonstrate the clearest potential clinical outcome. Investors will follow a good plan that has been developed to achieve FDA approval, successful commercialization or “go to market” launch, and eventual reimbursement to support a true standard-of-care change.
Failure is defined by inaction
The 3 case studies that I have shared were success stories because the ideas and inventions were acted upon. When I was at Genzyme, we built the company up to more than $1 billion in revenue. We commercialized proteins in over 50 countries. Most importantly, many patients benefited from the innovation. If you have an invention and an idea, act on it—and surround yourself with great people in every discipline. Having the right people and team is extremely important. ●
I often say that there are both “guardrail” days and very good days when it comes to the ins and outs of health care builds and product launches. The process is much like starting down the path of a country road in the middle of a blizzard—unless you have dependable wipers and a good defrost system, that path can get murky very quickly. With this article I hope to offer my counsel to inventors, featuring a few of my prior launches as well as case studies of health care launches I was not involved with, and sharing the lessons learned and hurdles that were overcome. I encourage all entrepreneurs to act on their ideas because, in the world of health care startups, the only failure is not acting on an invention.
Case study 1: Cerezyme
Today, Cerezyme is indicated for patients with Gaucher, which is a lysosomal storage disorder. Cerezyme’s first-generation product, called Ceredase, was a human tissue-derived protein that we extracted from human placentas. At the time, the concept of moving this program forward was denied by the Board of Directors because they said that even if you could collect enough placentas to make the enzyme, it would be too expensive to manufacture. In fact, early scale-up modeling for manufacturing the protein demonstrated that Genzyme would need 4 tons of placentas per Gaucher patient per year.
Gaucher is a severe, early-onset disease that has a significant negative outcome for patients. Patients with Gaucher are in dire need of treatment. Genzyme went forward with the Ceredase program by financing it through the families of patients with the disease, by starting an LLC separate from the business and funding the initial clinical trial and the development of the protein through the families of Gaucher patients. That approach was a successful endeavor. A great example of a creative capital structure to advance a program.
This was in the late 1980s/early 1990s, and at the height of the AIDS challenge. Genzyme based the manufacturing in Lille, France, and we cryopreserved placentas in the United States and Europe and shipped them to Lille to be processed into therapy. Genzyme eventually received approval for Ceredase from the US Food and Drug Administration (FDA) and the European Medicines Agency. At the height of the placenta collection, we were gathering about 10% to 15% of the placentas in the United States and 30% to 40% of the placentas in Europe. Resources supply became an issue until we developed a recombinant form of the protein, accomplished by using a manufacturing system called a CHO cell line.
This is a very good success story: If this invention was not pursued, Gaucher patients would not benefit from the treatment today. In addition, there are a plethora of patients with different lysosomal storage disorders treated with additional proteins that have been aided by us going through the entire development, manufacturing, and global commercialization process. We figured out how to manufacture and deliver the treatment, working through multiple countries’ political systems, and today the therapy is paid for by insurance and government systems on a worldwide basis.
Continue to: Case study 2...
Case study 2: ThinPrep
I like to use the approval of ThinPrep as an example of avoiding a false negative—a stoppage in the development of the product or drug for the wrong reasons. False negatives, in my mind, occur when you are developing a technology and you run into issues during the clinical phase and/or with FDA approval, or with a technical failure or you run out of capital prior to knowing whether or not the innovation actually works. In the case of ThinPrep, a poorly run clinical trial almost resulted in a false negative.
The company at the time was Cytyc, and an initial clinical study presented to the FDA yielded a neutral-negative outcome. The FDA said that there were not enough data to show the differentiation from the current Pap smear standard of care.
The founders of the company at that time had inherited the study protocol from a prior leadership team, so they had to finish the trial with the initial protocol. Given the FDA’s advisement, they developed a new trial. It took the persistence of these two founders, who mortgaged their homes and spent their personal dollars to take this through the next wave of clinical development. In the end it was successful. The revised clinical trial yielded an approval for ThinPrep, which is now considered a standard of care.
The use of ThinPrep reduced cervical cancer deaths by 40% from preapproval. The challenging path from clinical development to eventual commercial launch and physician leadership in advancing patient care makes the story of ThinPrep a great example of not allowing an early false negative of a poorly designed and run clinical trial stop important innovation.
Case study 3: Cologuard
The development of Cologuard is a case study demonstrating that, sometimes, when your first attempt does not work, you need to have the persistence to raise additional capital and/or use a slightly different technical approach. The approval story of Cologuard is important to share because it is an important cancer screening diagnostic, using DNA from stool samples to test for colon cancer, giving access to important colon cancer screening to many patients. Currently, caregivers are only scraping the surface with Cologuard’s ability to screen the population. There are many more patients that need access to the test, and I believe they will get it in the years ahead.
Cologuard went through a first- and second-generational technical failure. They could not get the test’s specificity and sensitivity to be at the level of a screening tool; there were too many false-positive results. With the third iteration came the technical breakthrough, and a very large, expensive study was conducted—one the leadership team was criticized for. However, that study yielded the data that achieved a New England Journal of Medicine article, and reimbursement support across the country. The combination of the right technical team and the right leadership team, who planned a proper commercial launch, with a CEO that supported the extensive clinical study, has resulted in the fourth generation of Cologuard—an important breakthrough offering a very useful new standard of care in colon cancer detection and screening.
Continue to: Pearls for moving your innovations forward...
Pearls for moving your innovations forward
Because of my experience in undergoing health care start-ups, and contributing to several of those advancements of innovation, many inventors approach me for advice on their paths from idea to full-concept company. Here are a few of my lessons learned.
Consider purpose, not financial gain, first and foremost. Financial gain is typically the by-product or outcome of a standard-of-care breakthrough for inventors, but it’s a very hard road. Pursue your invention for advancing patient care and moving a new standard of care forward in health care versus financial gain at the end.
Determine whether your invention is a product or a company, or potentially, not capitalizable at all. Figure this out early. Analyze your idea to make sure it is sound and truly novel. Analyze the competition and to make sure it is sound and truly novel. Analyze the competition and the market dynamics to support a new product. Can the development path be defined very clearly to raise capital? Is your innovation a big enough breakthrough in the market with several current products to actually make a difference in patient outcomes (and eventually achieve product reimbursement)? The creation of a company may be the right strategy if the innovation can support a differentiated enough breakthrough where you can actually support all the infrastructure to build the business. If you find that the market is not there to support and develop your idea to eventual success, backing off early is important to preserve invested capital.
Protect early. Is your invention patentable, or has someone else already thought of the idea? What kind of patent(s) are appropriate? Where, geographically, do you want to protect your invention? Find a good patent attorney in your local area, early in the process, to help you answer all of these critical questions. Patents are expensive to file and maintain, but it is not expensive to do a literature search to find out if your idea is novel. A provisional patent, which would be your first step, is an important cost-effective step.
Capital is out there. If your invention or idea deserves capital, it is available. I will address raising capital in more detail in the next section.
Consider regulatory and manufacturing as achievable hurdles. Inventors often get tripped up here, considering the regulatory hurdles and manufacturing too challenging and abandoning their ideas because the risk is too great. Regulatory and manufacturing are very important aspects of health care standard-of-care builds. Cutting corners is not an option. That said, regulatory and manufacturing should not stop you. Challenges often can be worked through as long as the clinical need is there, and the clinical data support bringing that technology forward.
Consider corporate partnerships. I am a fan of corporate partners. But which ones should you target, and when and why? Corporate partnerships can bring significant capital, which is great, but there is enough investor capital out there that you should not pursue a corporate partner just for capital. The main benefit of a corporate partner is enterprise intellect. They typically know more about the field that you are entering than the investors or a small company leadership team.
Establish and listen to advisors. When thinking about who to trust, research their track record. Advisors who have gone through this process before, and specifically in your product area, are important to have access to.
Persistence is key. I have observed a tremendous “compression of innovation” in the health care areas that I have been involved with—human tissue-derived proteins, robotic surgery, stem cell therapy, and digital health (which is still in its infancy). For each of these breakthrough categories, early on, it appeared that it couldn’t be done. However, after the first 2 or 3 major breakthroughs in each one of these areas, a compression of innovation occurred. For instance, after approximately 15 years of protein development, we came out with the recombinant manufacturing systems for proteins. Very quickly, within 10 years, there were more than 70 proteins on the market. The persistence of the inventors to overcome early obstacles in each of these health care areas was critical to future success in each area.
Continue to: Raising capital...
Raising capital
There are different investors who specialize in different types of investment opportunities. The first phase of raising capital is the seed round—where there is typically early data, or even no data and just a concept. From this seed round forward, there is less risk as you develop your technology; thus, there are different investors that support different stages of development and that specialize in different types of investing. It is important to target the right investors and raise enough capital to be able to go achieve multiple operational milestones. Otherwise, when you go through your first round of capital, or the Series A or B financing rounds, there may not be a set of investors out there to fund the company moving forward. Health care investors will make it known that they invest in certain rounds of capital. You can determine who those investors are by doing a search online.
A mistake health care inventors can make is not taking enough capital from investors, because they are concerned about dilution. I advise investors not to focus on dilution but rather on, how big can you make “the pie” (value of the company) worth? The entire process is about bringing a true product through to a new standard-of-care curve.
Trust is the most important thing to earn with investors, and there is zero tolerance for a lack of trust. Share your vision as the inventor with investors, who want to know where this category could be in the next 5 or 10 years. Clinical data will always win, and health care investors and industry leaders should be focused on executing the most robust clinical data to demonstrate the clearest potential clinical outcome. Investors will follow a good plan that has been developed to achieve FDA approval, successful commercialization or “go to market” launch, and eventual reimbursement to support a true standard-of-care change.
Failure is defined by inaction
The 3 case studies that I have shared were success stories because the ideas and inventions were acted upon. When I was at Genzyme, we built the company up to more than $1 billion in revenue. We commercialized proteins in over 50 countries. Most importantly, many patients benefited from the innovation. If you have an invention and an idea, act on it—and surround yourself with great people in every discipline. Having the right people and team is extremely important. ●
I often say that there are both “guardrail” days and very good days when it comes to the ins and outs of health care builds and product launches. The process is much like starting down the path of a country road in the middle of a blizzard—unless you have dependable wipers and a good defrost system, that path can get murky very quickly. With this article I hope to offer my counsel to inventors, featuring a few of my prior launches as well as case studies of health care launches I was not involved with, and sharing the lessons learned and hurdles that were overcome. I encourage all entrepreneurs to act on their ideas because, in the world of health care startups, the only failure is not acting on an invention.
Case study 1: Cerezyme
Today, Cerezyme is indicated for patients with Gaucher, which is a lysosomal storage disorder. Cerezyme’s first-generation product, called Ceredase, was a human tissue-derived protein that we extracted from human placentas. At the time, the concept of moving this program forward was denied by the Board of Directors because they said that even if you could collect enough placentas to make the enzyme, it would be too expensive to manufacture. In fact, early scale-up modeling for manufacturing the protein demonstrated that Genzyme would need 4 tons of placentas per Gaucher patient per year.
Gaucher is a severe, early-onset disease that has a significant negative outcome for patients. Patients with Gaucher are in dire need of treatment. Genzyme went forward with the Ceredase program by financing it through the families of patients with the disease, by starting an LLC separate from the business and funding the initial clinical trial and the development of the protein through the families of Gaucher patients. That approach was a successful endeavor. A great example of a creative capital structure to advance a program.
This was in the late 1980s/early 1990s, and at the height of the AIDS challenge. Genzyme based the manufacturing in Lille, France, and we cryopreserved placentas in the United States and Europe and shipped them to Lille to be processed into therapy. Genzyme eventually received approval for Ceredase from the US Food and Drug Administration (FDA) and the European Medicines Agency. At the height of the placenta collection, we were gathering about 10% to 15% of the placentas in the United States and 30% to 40% of the placentas in Europe. Resources supply became an issue until we developed a recombinant form of the protein, accomplished by using a manufacturing system called a CHO cell line.
This is a very good success story: If this invention was not pursued, Gaucher patients would not benefit from the treatment today. In addition, there are a plethora of patients with different lysosomal storage disorders treated with additional proteins that have been aided by us going through the entire development, manufacturing, and global commercialization process. We figured out how to manufacture and deliver the treatment, working through multiple countries’ political systems, and today the therapy is paid for by insurance and government systems on a worldwide basis.
Continue to: Case study 2...
Case study 2: ThinPrep
I like to use the approval of ThinPrep as an example of avoiding a false negative—a stoppage in the development of the product or drug for the wrong reasons. False negatives, in my mind, occur when you are developing a technology and you run into issues during the clinical phase and/or with FDA approval, or with a technical failure or you run out of capital prior to knowing whether or not the innovation actually works. In the case of ThinPrep, a poorly run clinical trial almost resulted in a false negative.
The company at the time was Cytyc, and an initial clinical study presented to the FDA yielded a neutral-negative outcome. The FDA said that there were not enough data to show the differentiation from the current Pap smear standard of care.
The founders of the company at that time had inherited the study protocol from a prior leadership team, so they had to finish the trial with the initial protocol. Given the FDA’s advisement, they developed a new trial. It took the persistence of these two founders, who mortgaged their homes and spent their personal dollars to take this through the next wave of clinical development. In the end it was successful. The revised clinical trial yielded an approval for ThinPrep, which is now considered a standard of care.
The use of ThinPrep reduced cervical cancer deaths by 40% from preapproval. The challenging path from clinical development to eventual commercial launch and physician leadership in advancing patient care makes the story of ThinPrep a great example of not allowing an early false negative of a poorly designed and run clinical trial stop important innovation.
Case study 3: Cologuard
The development of Cologuard is a case study demonstrating that, sometimes, when your first attempt does not work, you need to have the persistence to raise additional capital and/or use a slightly different technical approach. The approval story of Cologuard is important to share because it is an important cancer screening diagnostic, using DNA from stool samples to test for colon cancer, giving access to important colon cancer screening to many patients. Currently, caregivers are only scraping the surface with Cologuard’s ability to screen the population. There are many more patients that need access to the test, and I believe they will get it in the years ahead.
Cologuard went through a first- and second-generational technical failure. They could not get the test’s specificity and sensitivity to be at the level of a screening tool; there were too many false-positive results. With the third iteration came the technical breakthrough, and a very large, expensive study was conducted—one the leadership team was criticized for. However, that study yielded the data that achieved a New England Journal of Medicine article, and reimbursement support across the country. The combination of the right technical team and the right leadership team, who planned a proper commercial launch, with a CEO that supported the extensive clinical study, has resulted in the fourth generation of Cologuard—an important breakthrough offering a very useful new standard of care in colon cancer detection and screening.
Continue to: Pearls for moving your innovations forward...
Pearls for moving your innovations forward
Because of my experience in undergoing health care start-ups, and contributing to several of those advancements of innovation, many inventors approach me for advice on their paths from idea to full-concept company. Here are a few of my lessons learned.
Consider purpose, not financial gain, first and foremost. Financial gain is typically the by-product or outcome of a standard-of-care breakthrough for inventors, but it’s a very hard road. Pursue your invention for advancing patient care and moving a new standard of care forward in health care versus financial gain at the end.
Determine whether your invention is a product or a company, or potentially, not capitalizable at all. Figure this out early. Analyze your idea to make sure it is sound and truly novel. Analyze the competition and to make sure it is sound and truly novel. Analyze the competition and the market dynamics to support a new product. Can the development path be defined very clearly to raise capital? Is your innovation a big enough breakthrough in the market with several current products to actually make a difference in patient outcomes (and eventually achieve product reimbursement)? The creation of a company may be the right strategy if the innovation can support a differentiated enough breakthrough where you can actually support all the infrastructure to build the business. If you find that the market is not there to support and develop your idea to eventual success, backing off early is important to preserve invested capital.
Protect early. Is your invention patentable, or has someone else already thought of the idea? What kind of patent(s) are appropriate? Where, geographically, do you want to protect your invention? Find a good patent attorney in your local area, early in the process, to help you answer all of these critical questions. Patents are expensive to file and maintain, but it is not expensive to do a literature search to find out if your idea is novel. A provisional patent, which would be your first step, is an important cost-effective step.
Capital is out there. If your invention or idea deserves capital, it is available. I will address raising capital in more detail in the next section.
Consider regulatory and manufacturing as achievable hurdles. Inventors often get tripped up here, considering the regulatory hurdles and manufacturing too challenging and abandoning their ideas because the risk is too great. Regulatory and manufacturing are very important aspects of health care standard-of-care builds. Cutting corners is not an option. That said, regulatory and manufacturing should not stop you. Challenges often can be worked through as long as the clinical need is there, and the clinical data support bringing that technology forward.
Consider corporate partnerships. I am a fan of corporate partners. But which ones should you target, and when and why? Corporate partnerships can bring significant capital, which is great, but there is enough investor capital out there that you should not pursue a corporate partner just for capital. The main benefit of a corporate partner is enterprise intellect. They typically know more about the field that you are entering than the investors or a small company leadership team.
Establish and listen to advisors. When thinking about who to trust, research their track record. Advisors who have gone through this process before, and specifically in your product area, are important to have access to.
Persistence is key. I have observed a tremendous “compression of innovation” in the health care areas that I have been involved with—human tissue-derived proteins, robotic surgery, stem cell therapy, and digital health (which is still in its infancy). For each of these breakthrough categories, early on, it appeared that it couldn’t be done. However, after the first 2 or 3 major breakthroughs in each one of these areas, a compression of innovation occurred. For instance, after approximately 15 years of protein development, we came out with the recombinant manufacturing systems for proteins. Very quickly, within 10 years, there were more than 70 proteins on the market. The persistence of the inventors to overcome early obstacles in each of these health care areas was critical to future success in each area.
Continue to: Raising capital...
Raising capital
There are different investors who specialize in different types of investment opportunities. The first phase of raising capital is the seed round—where there is typically early data, or even no data and just a concept. From this seed round forward, there is less risk as you develop your technology; thus, there are different investors that support different stages of development and that specialize in different types of investing. It is important to target the right investors and raise enough capital to be able to go achieve multiple operational milestones. Otherwise, when you go through your first round of capital, or the Series A or B financing rounds, there may not be a set of investors out there to fund the company moving forward. Health care investors will make it known that they invest in certain rounds of capital. You can determine who those investors are by doing a search online.
A mistake health care inventors can make is not taking enough capital from investors, because they are concerned about dilution. I advise investors not to focus on dilution but rather on, how big can you make “the pie” (value of the company) worth? The entire process is about bringing a true product through to a new standard-of-care curve.
Trust is the most important thing to earn with investors, and there is zero tolerance for a lack of trust. Share your vision as the inventor with investors, who want to know where this category could be in the next 5 or 10 years. Clinical data will always win, and health care investors and industry leaders should be focused on executing the most robust clinical data to demonstrate the clearest potential clinical outcome. Investors will follow a good plan that has been developed to achieve FDA approval, successful commercialization or “go to market” launch, and eventual reimbursement to support a true standard-of-care change.
Failure is defined by inaction
The 3 case studies that I have shared were success stories because the ideas and inventions were acted upon. When I was at Genzyme, we built the company up to more than $1 billion in revenue. We commercialized proteins in over 50 countries. Most importantly, many patients benefited from the innovation. If you have an invention and an idea, act on it—and surround yourself with great people in every discipline. Having the right people and team is extremely important. ●
