Ramping up comparative effectiveness research

The federal government and some private groups have been conducting comparative effectiveness research for years. Despite those efforts, physicians and patients still have many unanswered questions about how to choose between treatments and procedures.

The Affordable Care Act aims to answer some of those questions by funding new research into the comparative benefits and harms of treatments, tests, and care delivery approaches.

Section 6301 of the 2010 health law establishes the Patient-Centered Outcomes Research Institute (PCORI), an independent, nonprofit organization charged with funding research into the comparative health outcomes and clinical effectiveness of medical treatments, as well as studies into whether those treatments are appropriate in certain patient populations. PCORI is scheduled to receive an estimated $3.5 billion from a trust fund created by Congress to fund patient-centered outcomes research through Sept. 30, 2019.

PCORI’s executive director, Dr. Joe V. Selby, discussed how the organization’s work will help answer the questions that clinicians and patients say are most important to them.

Question: PCORI has awarded more than $150 million for research. How long will it be until we see the first round of results, and will that information be immediately useful to physicians in their practices?

Dr. Selby: As of May 7, PCORI has approved more than $129 million in funding for patient-centered comparative effectiveness research projects that address the first four of the institute’s five National Priorities for Research and Research Agenda. In addition, PCORI has committed another $30 million in funding for a series of pilot projects. These are 2- to 3-year studies, with results expected beginning in 2014.

To speed the adoption of useful results in clinical practice, PCORI is monitoring the projects to determine if actionable information arises before a project is completed. When that happens, we’ll report the findings to the community. PCORI also is developing strong dissemination processes that will enable us to communicate findings quickly and effectively to clinicians, patients, and other key stakeholders.

Question: How can physicians use the information from PCORI in their practices?

Dr. Selby: It’s not always easy to get new research findings adopted by practicing physicians. We’re specifically aiming to fund research that answers questions faced by physicians and patients, so we work hard, in collaboration with physicians, to be sure that we’re asking the right questions. We then encourage researchers to maintain close contact with physicians and other relevant stakeholder groups as the research is conducted so that they will be there when results emerge.

We think that will increase the chances that these studies’ findings will be picked up by physicians and, if warranted, adopted into practice.

For example, PCORI is funding a randomized controlled trial of three antiepilepsy medications frequently prescribed for children. The outcomes focus on cognitive effects – how these medications affect learning. The investigators are a network of pediatric neurologists who specialize in epilepsy. Prior to launching the study, they began engaging online communities of patients and parents, as well as two major professional societies dedicated to treatment of epilepsy. This active engagement increases the chances that the trial’s results will be noted and adopted.

Question: There are a lot of unanswered research questions in the area of clinical comparative effectiveness. How do you choose which studies to fund?

Dr. Selby: PCORI’s National Priorities for Research and Research Agenda provides a framework to guide the funding of studies that will give patients and those who care for them the ability to make better-informed health decisions. Through our new advisory panels, roundtables, and other activities, the institute is actively engaging patients, clinicians, caregivers, and other stakeholders in identifying the research topics of greatest value to them and guiding our efforts in designing and implementing studies. We are looking specifically for questions that have been raised by patients and clinicians, questions that, when answered, are likely to change decision-making and improve patient outcomes.

Applications for PCORI funding are reviewed by scientists, patients, caregivers, and other stakeholders, who assess the proposed projects’ methodological rigor, how well they engage patients and other stakeholders, and how well they fit within PCORI’s national research priorities. As you can see, we’re ensuring that the patient’s voice is represented throughout the research process.

Question: The ACA specifically bars PCORI from funding studies that compare cost effectiveness. Was that the right move?

Dr. Selby: The legislation that established PCORI called on us to pay close attention to disease incidence, prevalence, burden (including costs to individuals and society), chronic conditions, and disparities when choosing research questions. We focus on studying clinical outcomes – the information that is most frequently missing and that can be most difficult to obtain. With enhanced information on options and outcomes, doing the math on costs is something that patients, clinicians, and their health care systems can do from their own perspectives, taking into account the individual patient’s needs and preferences.

Moreover, many studies suggest that, when patients have good evidence on clinical effectiveness, they tend to make different decisions about what they think is the best course, including choices that, in some cases, can lead to lower utilization.

Question: Comparative effectiveness research was controversial when the ACA was being debated. Has that died down now that PCORI is up and running?

Dr. Selby: Comparative effectiveness research, or CER, has been carried out in the United States for years, mainly through the sponsorship of the National Institutes of Health, the Agency for Healthcare Research and Quality, and the Department of Veterans Affairs. CER has traditionally had bipartisan support. We are mindful of concerns that the results of these studies not be used to inappropriately limit patient or physician choices. The expansive involvement of stakeholders in PCORI’s activities and its independence from government as a private nonprofit make it ideally suited to lead the way in this important field of health research.

Since PCORI has been increasing its activities, we’ve received a lot of interest and feedback from many stakeholders who have become actively involved in our work. We’re hearing a lot of enthusiasm from health care professionals, the pharmaceutical and biotechnology industries, patients and patient advocates, clinical researchers, and many others.

I believe that, as people follow PCORI’s ongoing work and the efforts of our many partners across the health community, they will gain ever greater assurance that patient-centered CER is providing data and evidence that will help them make better decisions about their health and health care.

Dr. Selby is a family physician, clinical epidemiologist, and health services researcher who has worked on comparative effectiveness research for many years. Before PCORI, he was the director of the division of research at Kaiser Permanente, Northern California.

The federal government and some private groups have been conducting comparative effectiveness research for years. Despite those efforts, physicians and patients still have many unanswered questions about how to choose between treatments and procedures.

The Affordable Care Act aims to answer some of those questions by funding new research into the comparative benefits and harms of treatments, tests, and care delivery approaches.

Section 6301 of the 2010 health law establishes the Patient-Centered Outcomes Research Institute (PCORI), an independent, nonprofit organization charged with funding research into the comparative health outcomes and clinical effectiveness of medical treatments, as well as studies into whether those treatments are appropriate in certain patient populations. PCORI is scheduled to receive an estimated $3.5 billion from a trust fund created by Congress to fund patient-centered outcomes research through Sept. 30, 2019.

PCORI’s executive director, Dr. Joe V. Selby, discussed how the organization’s work will help answer the questions that clinicians and patients say are most important to them.

Question: PCORI has awarded more than $150 million for research. How long will it be until we see the first round of results, and will that information be immediately useful to physicians in their practices?

Dr. Selby: As of May 7, PCORI has approved more than $129 million in funding for patient-centered comparative effectiveness research projects that address the first four of the institute’s five National Priorities for Research and Research Agenda. In addition, PCORI has committed another $30 million in funding for a series of pilot projects. These are 2- to 3-year studies, with results expected beginning in 2014.

To speed the adoption of useful results in clinical practice, PCORI is monitoring the projects to determine if actionable information arises before a project is completed. When that happens, we’ll report the findings to the community. PCORI also is developing strong dissemination processes that will enable us to communicate findings quickly and effectively to clinicians, patients, and other key stakeholders.

Question: How can physicians use the information from PCORI in their practices?

Dr. Selby: It’s not always easy to get new research findings adopted by practicing physicians. We’re specifically aiming to fund research that answers questions faced by physicians and patients, so we work hard, in collaboration with physicians, to be sure that we’re asking the right questions. We then encourage researchers to maintain close contact with physicians and other relevant stakeholder groups as the research is conducted so that they will be there when results emerge.

We think that will increase the chances that these studies’ findings will be picked up by physicians and, if warranted, adopted into practice.

For example, PCORI is funding a randomized controlled trial of three antiepilepsy medications frequently prescribed for children. The outcomes focus on cognitive effects – how these medications affect learning. The investigators are a network of pediatric neurologists who specialize in epilepsy. Prior to launching the study, they began engaging online communities of patients and parents, as well as two major professional societies dedicated to treatment of epilepsy. This active engagement increases the chances that the trial’s results will be noted and adopted.

Question: There are a lot of unanswered research questions in the area of clinical comparative effectiveness. How do you choose which studies to fund?

Dr. Selby: PCORI’s National Priorities for Research and Research Agenda provides a framework to guide the funding of studies that will give patients and those who care for them the ability to make better-informed health decisions. Through our new advisory panels, roundtables, and other activities, the institute is actively engaging patients, clinicians, caregivers, and other stakeholders in identifying the research topics of greatest value to them and guiding our efforts in designing and implementing studies. We are looking specifically for questions that have been raised by patients and clinicians, questions that, when answered, are likely to change decision-making and improve patient outcomes.

Applications for PCORI funding are reviewed by scientists, patients, caregivers, and other stakeholders, who assess the proposed projects’ methodological rigor, how well they engage patients and other stakeholders, and how well they fit within PCORI’s national research priorities. As you can see, we’re ensuring that the patient’s voice is represented throughout the research process.

Question: The ACA specifically bars PCORI from funding studies that compare cost effectiveness. Was that the right move?

Dr. Selby: The legislation that established PCORI called on us to pay close attention to disease incidence, prevalence, burden (including costs to individuals and society), chronic conditions, and disparities when choosing research questions. We focus on studying clinical outcomes – the information that is most frequently missing and that can be most difficult to obtain. With enhanced information on options and outcomes, doing the math on costs is something that patients, clinicians, and their health care systems can do from their own perspectives, taking into account the individual patient’s needs and preferences.

Moreover, many studies suggest that, when patients have good evidence on clinical effectiveness, they tend to make different decisions about what they think is the best course, including choices that, in some cases, can lead to lower utilization.

Question: Comparative effectiveness research was controversial when the ACA was being debated. Has that died down now that PCORI is up and running?

Dr. Selby: Comparative effectiveness research, or CER, has been carried out in the United States for years, mainly through the sponsorship of the National Institutes of Health, the Agency for Healthcare Research and Quality, and the Department of Veterans Affairs. CER has traditionally had bipartisan support. We are mindful of concerns that the results of these studies not be used to inappropriately limit patient or physician choices. The expansive involvement of stakeholders in PCORI’s activities and its independence from government as a private nonprofit make it ideally suited to lead the way in this important field of health research.

Since PCORI has been increasing its activities, we’ve received a lot of interest and feedback from many stakeholders who have become actively involved in our work. We’re hearing a lot of enthusiasm from health care professionals, the pharmaceutical and biotechnology industries, patients and patient advocates, clinical researchers, and many others.

I believe that, as people follow PCORI’s ongoing work and the efforts of our many partners across the health community, they will gain ever greater assurance that patient-centered CER is providing data and evidence that will help them make better decisions about their health and health care.

Dr. Selby is a family physician, clinical epidemiologist, and health services researcher who has worked on comparative effectiveness research for many years. Before PCORI, he was the director of the division of research at Kaiser Permanente, Northern California.

The federal government and some private groups have been conducting comparative effectiveness research for years. Despite those efforts, physicians and patients still have many unanswered questions about how to choose between treatments and procedures.

The Affordable Care Act aims to answer some of those questions by funding new research into the comparative benefits and harms of treatments, tests, and care delivery approaches.

Section 6301 of the 2010 health law establishes the Patient-Centered Outcomes Research Institute (PCORI), an independent, nonprofit organization charged with funding research into the comparative health outcomes and clinical effectiveness of medical treatments, as well as studies into whether those treatments are appropriate in certain patient populations. PCORI is scheduled to receive an estimated $3.5 billion from a trust fund created by Congress to fund patient-centered outcomes research through Sept. 30, 2019.

PCORI’s executive director, Dr. Joe V. Selby, discussed how the organization’s work will help answer the questions that clinicians and patients say are most important to them.

Question: PCORI has awarded more than $150 million for research. How long will it be until we see the first round of results, and will that information be immediately useful to physicians in their practices?

Dr. Selby: As of May 7, PCORI has approved more than $129 million in funding for patient-centered comparative effectiveness research projects that address the first four of the institute’s five National Priorities for Research and Research Agenda. In addition, PCORI has committed another $30 million in funding for a series of pilot projects. These are 2- to 3-year studies, with results expected beginning in 2014.

To speed the adoption of useful results in clinical practice, PCORI is monitoring the projects to determine if actionable information arises before a project is completed. When that happens, we’ll report the findings to the community. PCORI also is developing strong dissemination processes that will enable us to communicate findings quickly and effectively to clinicians, patients, and other key stakeholders.

Question: How can physicians use the information from PCORI in their practices?

Dr. Selby: It’s not always easy to get new research findings adopted by practicing physicians. We’re specifically aiming to fund research that answers questions faced by physicians and patients, so we work hard, in collaboration with physicians, to be sure that we’re asking the right questions. We then encourage researchers to maintain close contact with physicians and other relevant stakeholder groups as the research is conducted so that they will be there when results emerge.

We think that will increase the chances that these studies’ findings will be picked up by physicians and, if warranted, adopted into practice.

For example, PCORI is funding a randomized controlled trial of three antiepilepsy medications frequently prescribed for children. The outcomes focus on cognitive effects – how these medications affect learning. The investigators are a network of pediatric neurologists who specialize in epilepsy. Prior to launching the study, they began engaging online communities of patients and parents, as well as two major professional societies dedicated to treatment of epilepsy. This active engagement increases the chances that the trial’s results will be noted and adopted.

Question: There are a lot of unanswered research questions in the area of clinical comparative effectiveness. How do you choose which studies to fund?

Dr. Selby: PCORI’s National Priorities for Research and Research Agenda provides a framework to guide the funding of studies that will give patients and those who care for them the ability to make better-informed health decisions. Through our new advisory panels, roundtables, and other activities, the institute is actively engaging patients, clinicians, caregivers, and other stakeholders in identifying the research topics of greatest value to them and guiding our efforts in designing and implementing studies. We are looking specifically for questions that have been raised by patients and clinicians, questions that, when answered, are likely to change decision-making and improve patient outcomes.

Applications for PCORI funding are reviewed by scientists, patients, caregivers, and other stakeholders, who assess the proposed projects’ methodological rigor, how well they engage patients and other stakeholders, and how well they fit within PCORI’s national research priorities. As you can see, we’re ensuring that the patient’s voice is represented throughout the research process.

Question: The ACA specifically bars PCORI from funding studies that compare cost effectiveness. Was that the right move?

Dr. Selby: The legislation that established PCORI called on us to pay close attention to disease incidence, prevalence, burden (including costs to individuals and society), chronic conditions, and disparities when choosing research questions. We focus on studying clinical outcomes – the information that is most frequently missing and that can be most difficult to obtain. With enhanced information on options and outcomes, doing the math on costs is something that patients, clinicians, and their health care systems can do from their own perspectives, taking into account the individual patient’s needs and preferences.

Moreover, many studies suggest that, when patients have good evidence on clinical effectiveness, they tend to make different decisions about what they think is the best course, including choices that, in some cases, can lead to lower utilization.

Question: Comparative effectiveness research was controversial when the ACA was being debated. Has that died down now that PCORI is up and running?

Dr. Selby: Comparative effectiveness research, or CER, has been carried out in the United States for years, mainly through the sponsorship of the National Institutes of Health, the Agency for Healthcare Research and Quality, and the Department of Veterans Affairs. CER has traditionally had bipartisan support. We are mindful of concerns that the results of these studies not be used to inappropriately limit patient or physician choices. The expansive involvement of stakeholders in PCORI’s activities and its independence from government as a private nonprofit make it ideally suited to lead the way in this important field of health research.

Since PCORI has been increasing its activities, we’ve received a lot of interest and feedback from many stakeholders who have become actively involved in our work. We’re hearing a lot of enthusiasm from health care professionals, the pharmaceutical and biotechnology industries, patients and patient advocates, clinical researchers, and many others.

I believe that, as people follow PCORI’s ongoing work and the efforts of our many partners across the health community, they will gain ever greater assurance that patient-centered CER is providing data and evidence that will help them make better decisions about their health and health care.

Dr. Selby is a family physician, clinical epidemiologist, and health services researcher who has worked on comparative effectiveness research for many years. Before PCORI, he was the director of the division of research at Kaiser Permanente, Northern California.