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.
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 This includes studies to evaluate patient preferences in medical devices and a recently published draft guidance on patient preference information for PMAs, HDE applications, de novo requests, and inclusion in device labeling that describes how patient tolerance for risk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.
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