FDA Sets Inaugural Meeting of First-Ever Patient Engagement Advisory Committee

By: Kathryn O’Callaghan and Jeffrey Shuren, M.D., J.D.

Imagine checking your blood sugar levels several times a day with a glucose meter to keep your diabetes under control. Or maybe you’ve had a hip joint replaced or a stent inserted in your coronary artery to treat a heart blockage. Maybe you participated in a clinical trial to help researchers or a manufacturer better understand a new device to treat a condition.

Kathryn O'Callaghan

Kathryn O’Callaghan, Assistant Director for Strategic Programs, FDA’s Center for Devices and Radiological Health

It’s possible that you had some unexpected experiences with the device or clinical trial and you’d like to share what happened with your physician, the manufacturer, other patients or even FDA.

Now, there’s a way. For the first time, an FDA advisory committee will focus on patient-related issues. On Oct. 11-12, 2017, FDA will hold the inaugural meeting of the new Patient Engagement Advisory Committee (PEAC). The topic will be the challenges of clinical trial design, conduct, and reporting identified by patients.

FDA chose this subject because patients often have concerns about participating in clinical trials or drop out once they have enrolled in a trial. Inconsistent or minimal participation in clinical trials can make it difficult to reach reliable conclusions or to determine the level of benefit for patients. It also can take longer to bring technological advances to the patients who need them.

We also are excited to announce that the nine core voting members of our patient advisory committee, including the chair and the consumer representative, all have direct experience as a patient or as a care-partner for a patient.

Jeffrey Shuren

Jeffrey Shuren, M.D., J.D., Director of FDA’s Center for Devices and Radiological Health

They are experts in the field of patient engagement, and their experience extends beyond their personal disease or condition to the broader patient perspective, which is a critical piece of FDA’s work. While patient representatives currently participate in many FDA advisory committee meetings, we’ve never had a committee that was wholly focused on patients.

The PEAC is a forum for the voice of patients. It will be asked to advise on complex issues related to medical devices and their impact on patients. The goal of PEAC is to better understand and integrate patient perspectives into our oversight, to improve communications with patients about benefits, risks, and clinical outcomes related to medical devices, and to identify new approaches, unforeseen risks or barriers, and unintended consequences from the use of medical devices.

Federal legislation enacted in 2012 instructed FDA to solicit the views of patients during the medical product development process and consider the perspectives of patients during regulatory discussion. This gave us a welcome opportunity to establish the PEAC.

Our intent is to engage with and better understand the needs, views, and concerns of patients and other users of medical devices. After all, they are, along with their caregivers, the experts in terms of what they’re living with and what they have to deal with day-to-day.

We will also need better tools to accurately capture and characterize patient views on acceptable balances of benefits and risks. And, as the nascent science of patient preferences continues to evolve, we must adapt our policies, too.

Patients are at the heart of what we do. It makes sense to establish an advisory committee built just for them.

The nine core members of the PEAC are:

Paul Conway (Chair) – President of the American Association of Kidney Patients.

Katherine Seelman, Ph.D. – (Consumer Representative) – Retired from the University of Pittsburgh as Professor Emerita in 2016.

Cynthia Chauhan, M.S.W. – Served as a Patient Advocate for over 17 years. Served as an FDA patient representative for the National Mammography Quality Assurance Advisory Committee.

Amye Leong, M.B.A. – President and CEO of Health Motivations. Extensive patient advocacy history, communications specialist, health policy advisor, author.

Monica Willis-Parker, M.D. – Director, Minority Engagement Core, Emory Alzheimer’s Disease Research Center.

Frederick Downs, Jr., M.B.A. – Decorated Vietnam veteran, who retired from the Veterans Administration as the Senior Executive Service Chief Procurement and Logistics Officer.

Bennet Dunlap, M.S. – Health communicator and advocate for people with diabetes, with experience in social media and marketing programs.

Deborah Cornwall, M.B.A. – Patient advocacy expert. She is a longtime leadership volunteer for the American Cancer Society and the Cancer Action Network.

Suzanne Schrandt, J.D. – Director of Patient Engagement at the Arthritis Foundation.

Kathryn O’Callaghan is Assistant Director for Strategic Programs, FDA’s Center for Devices and Radiological Health 

Jeffrey Shuren, M.D., J.D., is Director of FDA’s Center for Devices and Radiological Health

Why Partnerships are Key to the Science of Patient Input

By: Nina L. Hunter, Ph.D., and Robert M. Califf, M.D.

We recently announced the first FDA Patient Engagement Advisory Committee (PEAC), supported by the Center of Devices and Radiological Health (CDRH). The Committee will provide advice to the FDA Commissioner on complex issues relating to medical devices, the regulation of devices, and their use by patients. The PEAC will bring patients, patient advocacy groups, and experts together for a broader discussion of important patient-related issues, to increase integration of patient perspectives into the regulatory process, and to help drive more patient-centric medical device innovation, development, evaluation, and access.

Nina Hunter

Nina L. Hunter, Ph.D., FDA’s Associate Director for Science Policy in the Office of Medical Products and Tobacco

With the PEAC offering an important avenue for patient views to be incorporated in the assessment of new medical devices, complementary programs in the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER) are continuing to explore multiple approaches to patient involvement in development programs for drugs and biologic products, respectively. The Patient-Focused Drug Development (PFDD) Program, led by Dr. Theresa Mullin, provides a way for scientists from across the Agency to obtain patients’ input on specific disease areas, including their perspectives on their condition, its impact on daily life, and available therapies. As part of this program, FDA is holding a series of public meetings, each focused on a specific disease area. Outcomes of these meetings include detailed descriptions of patient perspectives on the most significant symptoms and treatments.

While FDA continues our work on patient engagement through our newly formed advisory committee and the PFDD Program, public-private partnerships (PPPs) are key to empowering patients across the spectrum of medical product development and evaluation. Here we will describe three such important partnerships.

FDA is a founding member of the Medical Device Innovation Consortium (MDIC), a PPP created with the objective of advancing medical device regulatory science. MDIC recently issued a catalog of available methods that can be used for collecting data on patient preferences, along with a framework for considering how to incorporate patient preferences across the total lifecycle of a device. The ultimate goal is to use these data to guide the development, assessment, and delivery of medical devices that better meet patients’ needs. As the scientific evidence and methodological approaches in this area mature, FDA will continue to collaborate with others on efforts to collect and use patient preference data for regulatory purposes.

Robert M. Califf, M.D., MACC, FDA's Commissioner of Food and Drugs

Robert M. Califf, M.D., Commissioner of the U.S. Food and Drug Administration

Like the MDIC, the Kidney Health Initiative (KHI) is a PPP that includes representatives from the FDA, healthcare professional societies, patient groups, and the medical products industry. Recently, KHI convened a workshop under the leadership of Dr. Frank Hurst and Ms. Carolyn Neuland, with patients, care partners, scientists, doctors, nurses, technicians, companies, and FDA, to hear discussions about the issues that patients with kidney diseases consider most important. More than 80 patients attended this workshop; many of these were not members of an organized patient advocacy group, but instead individuals truly driven to improve the plight of all patients with kidney disease. CDRH and CDER are working with the KHI to advance scientific understanding of the implications for patient health and safety posed by new and existing medical products, as well as fostering development of new therapies for kidney diseases. This PPP creates a transparent infrastructure and processes that facilitate collaboration and communication among the greater Nephrology community and FDA.

FDA has also held several meetings with the National Institutes of Health (NIH) throughout the PROMIS initiative, including the Patient Reported Outcome Consortium. PROMIS aims to provide clinicians and researchers access to efficient, precise, valid, and responsive patient-reported measures of health and well-being. PROMIS measures can be used as primary or secondary endpoints in clinical studies of the effectiveness of treatment, and PROMIS tools can be used across a wide variety of chronic diseases and conditions and in the general population. These tools pertain to all medical products, and they can be used to understand the burden of their disease and impacts of treatment on how patients feel and function in their daily lives, so that appropriate patient-centered outcome assessments can be developed and integrated into clinical trials to produce meaningful data to guide treatment decisions. Specifically at CDRH, the use of patient-reported outcome measures (PROMs) in regulatory submissions has increased significantly, with approximately 20 submissions per year citing PROMs prior to FDA’s guidance on the topic, to over 120 last year alone. This jump indicates significant interest by industry and clinical researchers in generating patient-centered evidence from studies done for regulatory purposes.

FDA is ready to advance the science of patient input and work with a wider community of patients, clinicians, and social science researchers in a collaborative way. We expect the number of partnerships with patients and their caregivers to grow, and the effort to become more effective as the underlying science and cultural understanding continues to develop.

Nina L. Hunter, Ph.D., is FDA’s Associate Director for Science Policy in the Office of Medical Products and Tobacco

Robert M. Califf, M.D., previously FDA’s Deputy Commissioner for Medical Products and Tobacco, became FDA’s Commissioner of Food and Drugs on Feb. 25, 2016

FDA’s Patient Preference Initiative: The Need for Evolving Tools and Policies

By: Nina L. Hunter, Ph.D., and Robert M. Califf, M.D.

Last week we announced FDA’s first-ever Patient Engagement Advisory Committee, which will provide advice to the FDA Commissioner on complex issues relating to medical devices, the regulation of devices, and their use by patients. We thought it would be good to step back and fill you in about our Patient Preference Initiative.

Nina Hunter

Nina L. Hunter, Ph.D., FDA’s Associate Director for Science Policy in the Office of Medical Products and Tobacco

In 2013, the FDA launched the Patient Preference Initiative, now led by Kathryn O’Callaghan, (Acting) Associate Director for Science and Strategic Partnerships at FDA’s Center for Devices and Radiological Health (CDRH). With this initiative, FDA’s CDRH expanded upon the current approach for capturing patient-centered perspectives in its structured benefit-risk framework, to outline a way of incorporating patients’ views on benefits and risks together with those of FDA’s health care professionals, scientists, and engineers during regulatory decision-making about certain medical devices.

This approach incorporates scientific, empirical evidence from different patients who, as a group, may have a range of views about the degree and types of risks associated with a medical device and how risks should be weighed against the anticipated benefits. When circumstances are appropriate and the data meets the requisite standard, device reviewers at the FDA can consider patient preference data in the overall evaluation of certain devices along with evidence from clinical and nonclinical testing.

If the device is ultimately cleared or approved, the product labeling could include a description of the range of patient preferences and characteristics described by those data, providing patients and healthcare practitioners with key information to make well-informed decisions. It is important to reiterate that FDA would not approve a device if it determines the device would expose patients to an unreasonable or significant risk of illness or injury, or that the benefits do not outweigh the risks for a defined target population.

Robert M. Califf, M.D., MACC, FDA's Commissioner of Food and Drugs

Robert M. Califf, M.D., Commissioner of the U.S. Food and Drug Administration

As we increasingly work to incorporate the perspectives of patients when evaluating technologies for regulatory approval, we will also need better tools in order to accurately capture and characterize patient views on acceptable balances of benefits and risks. And as this new science of patient preferences continues to evolve, policies must likewise continue to be adapted as approaches are refined.

The FDA, through CDRH and the Center for Biologics Evaluation and Research (CBER), released Draft Guidance on patient preference information this spring. When finalized, this will help device-makers and other stakeholders assess patient valuations of benefit and risk related to relevant device types, illnesses, and conditions. It will also facilitate more systematic consideration of patient views as part of structured benefit-risk assessments for new medical devices. In time, as patient groups, industry, and others conduct more patient preference studies, the FDA and others will better understand patients’ perspectives on benefits and risks, and tradeoffs they are willing to make. This research has the potential to drive more patient-centered device innovation, assessment and access.

The draft guidance provides a case study for such patient-centered device regulation. A recent study conducted by FDA scientists Drs. Martin Ho and Telba Irony and researchers at RTI Health Solutions demonstrated that robust empirical data can be successfully elicited from patients and used to inform deliberations surrounding the approval of a medical product—in this case, the first device for treating obesity to be approved by the FDA since 2007. This pioneering work was a fundamental step forward in our efforts to develop the best methods and practices for systematically incorporating patient preferences into device development and assessment.

As part of the Patient Preference Initiative and other activities to better integrate patient views into our decision making, the FDA is working with others to advance the science of patient input. In our next FDA Voice blog post we will expand on this growing dimension.

The FDA recognizes the potential benefit to be gained from understanding and applying patient input to spur patient-centered medical product innovation and inform patient-centered regulation. We believe that by better understanding patients’ experiences, needs, and views, we will be able to improve the development of medical products and enhance the safe and effective use of those products.

Nina L. Hunter, Ph.D., is FDA’s Associate Director for Science Policy in the Office of Medical Products and Tobacco

Robert M. Califf, M.D., previously FDA’s Deputy Commissioner for Medical Products and Tobacco, became FDA’s Commissioner of Food and Drugs on Feb. 25, 2016

FDA Announces First-ever Patient Engagement Advisory Committee

By: Nina L. Hunter, Ph.D., and Robert M. Califf, M.D.

Although it may seem odd in retrospect, the development of new technologies intended to improve patients’ lives has largely relied upon expert opinions rather than asking patients and families directly what they consider most important.

Nina Hunter

Nina L. Hunter, Ph.D., FDA’s Associate Director for Science Policy in the Office of Medical Products and Tobacco

But that’s changing. We are entering an era of “patient-centered” medicine in which patients and their care partners participate actively in decision-making and priority-setting about all aspects of health care. Americans are becoming increasingly active consumers of health care, making choices about their doctors, diagnostics, treatments, and healthcare experiences rather than simply allowing health care providers to make the decisions for them. Moreover, FDA believes that patients can and should bring their own experiences to bear in helping the Agency define meaningful benefits or unreasonable risks for certain new devices.

Today we are excited to announce FDA’s first-ever Patient Engagement Advisory Committee (PEAC). This body will provide advice to the FDA Commissioner on a range of complex issues relating to medical devices, the regulation of devices, and their use by patients. It will give FDA the opportunity to obtain expertise on various patient-related topics, with the goal of improving communication of benefits and risks and increasing integration of patient perspectives into the regulatory process. Some questions that the PEAC may discuss include where and how best to engage patients across the device development and assessment lifecycle as well as how FDA and sponsors should communicate patient preference information to patients. The PEAC represents a new and exciting opportunity to foster patient partnerships with FDA, and it complements other efforts at FDA to bring the patient into the medical device regulatory process. This includes studies to evaluate patient preferences in medical devices and a recently published draft guidance on patient preference information for PMAs, HDE applications, de novo requests, and inclusion in device labeling that describes how patient tolerance for risk and perspective on benefit, in addition to clinical data and other information, may be considered in FDA’s assessment of the benefit-risk profile of certain devices. While it’s important to consider patient perspectives, we understand that patients still expect FDA to do our primary job — namely, ensuring the safety and effectiveness of FDA-regulated medical devices. This is primarily accomplished at FDA through regulation at the Center for Devices and Radiological Health and the Center for Biologics Evaluation and Research. When assessing whether valid scientific evidence shows that a device’s probable benefit outweighs its likely risks, FDA may consider rigorous, systematically gathered patient preference information as a part of the totality of the evidence from clinical and nonclinical testing. However, patient preference information will not be used to justify approval of unsafe or ineffective devices: if FDA determines the device would expose patients to an unreasonable or significant risk of illness or injury, or that the benefits do not outweigh the risks for a defined target population, FDA would not approve such a device.

Robert M. Califf, M.D., MACC, FDA's Commissioner of Food and Drugs

Robert M. Califf, M.D., Commissioner of the U.S. Food and Drug Administration

When is patient preference information most useful? These data can be used in several major ways:

  • to help identify the most important benefits and risks of a technology from a patient’s perspective;
  • to assess the relative importance to patients of different attributes of benefit and risk, and clarify how patients think about the tradeoffs of these benefits and risks for a given technology; and
  • to help understand how patient preferences vary across a population.

As part of the Patient Preference Initiative and other activities to better integrate patient views into our decision-making, FDA is working with others to advance the science of patient input. We will discuss these extensive partnerships in an upcoming FDA Voice blog.

FDA’s sharpened focus on patient-centered technology development, evaluation, and use has already begun to positively affect the development of innovative therapies and clinical solutions. These efforts are helping to drive a more patient-centered medical product development and assessment process. We’re excited to invite the patient, industry, and academic communities to join in and help us accelerate this important work.

Nina L. Hunter, Ph.D., is FDA’s Associate Director for Science Policy in the Office of Medical Products and Tobacco

Robert M. Califf, M.D., previously FDA’s Deputy Commissioner for Medical Products and Tobacco, became FDA’s Commissioner of Food and Drugs on Feb. 25, 2016