FDA Brings Patients Into the Process

By: Michelle McMurry-Heath, M.D., Ph.D. 

What do patients really want? 

At the FDA, we ask that question every day and in all kinds of contexts. Earlier this year, the Center for Devices and Radiological Health (CDRH) began to explore new ways to identify and incorporate the patient voice into our decision-making on medical devices. 

We’re calling this effort the Patient Preference Initiative. As part of this effort, we held a public workshop the past two days with patients, caregivers, health care providers, researchers, and industry to discuss ways to incorporate patient preferences as we weigh the risks and benefits of the products we regulate both before and after the product goes to market. 

In trying to incorporate patient preferences into our regulatory decisions, it’s important to know how to accurately and reliably measure their preferences for treating and diagnosing their conditions. So, we’re also trying to advance the tools and methods that could be used to do so. 

For example, there is a risk of injury with many medical devices. How much of a risk is acceptable to a patient in the context of the potential benefits of the device? If patients enter a clinical trial, how much of a chance are they willing to take on an unproven treatment? If a device could greatly improve a patient’s health, but is not portable or cannot be used at home, would a patient find this too limiting? If a device has new-found risks but is the only one of its kind on the market, should it be recalled? 

In appropriate cases, we foresee approving devices for which a fully-informed subset of patients would accept the risks as weighed against the benefits, if patients and their practitioners can be provided with the information they need to make their own well-informed decision and the information can be presented in a manner that can be understood by the practitioners and patients. 

At the workshop, we invited attendees to actively explore such issues as clinical trial design and ways to facilitate getting new, safe and effective, innovative devices out to the patients who may need them. Our panels weighed in on such questions as: 

  • How do we integrate patient preferences into clinical trial design?
  • How can we build partnerships to collect this information?
  • How can we use data on patient preference for post-market and compliance issues?
  • What disease areas or device types are best suited for the patient preference approach?
  • Which diseases or device types are best suited for patient preference input?
  • How can we ensure that patients, families and caregivers are well informed so that they understand the choices they have and the decisions they are making? 

We also announced that we will be establishing a new Patient Engagement Panel as part of our Medical Device Advisory Committee to provide advice on issues important to patients, such as more understandable labeling and the use of medical devices at home. 

Throughout the workshop, it was clear that determining the benefits and risks of medical devices is one of the most important things we do. 

In 2012, the FDA published a document to help industry understand the key factors we consider when making benefit-risk determinations for certain medical devices. Importantly, it discusses collecting patient-centric metrics to measure benefit and ways of measuring a patient’s tolerance for risks. 

But the last two days have been all about the patients who may need these products, the caregivers who would be helping patients use them, and the health care professionals who may prescribe them. What do they want? What do they need? 

The FDA is committed to giving patients in the United States access to safe and effective medical devices of high quality and we work hard to improve the predictability, consistency and transparency of the pre-market review process. We’re pleased at the progress we’ve made over the last two days as part of our effort to invite patients into the regulatory process, and we look forward to more close collaboration with patients in the future. 

Patients, after all, are at the core of our mission and the focus of our vision. 

Michelle McMurry-Heath, M.D., Ph.D., is the Associate Director for Science at the FDA’s Center for Devices and Radiological Health

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