Rare Disease Report

Patient's Voice Becomes Official Part of FDA Review Process

OCTOBER 11, 2017
James Radke
Today, Scott Gottlieb, M.D, Commissioner of the U.S. Food and Drug Administration (FDA) reaffirmed the agency’s commitment to have patients more involved in the drug development process.

In a news release coinciding with today’s first meeting of the Patient Engagement Advisory Committee (PEAC) at the FDA, Gottlieb expanded on the growing role patients will have in the development and approval of medicines and medical devices.

PEAC is the first advisory committee consisting of only patients, caregivers, and patient advocates, and the initial meeting will be held October 11 and 12. On the docket, the committee intends to discuss and make recommendations regarding patient input in clinical trials. Although the meeting is not specifically related to orphan drugs or rare disease treatment, it should be considered a milestone as it signifies a change in how the FDA will review medicines and devices.

In a news release, Gottlieb wrote: “Patients are increasingly collecting much of this real world data. Patients are spearheading the creation of novel registry platforms that collect health data to track their progress, and facilitate medical product evaluations. We’re fostering interactions with these patient-led registries to help them become vehicles for regulatory change.”

Additionally, the FDA is recognizing that advances in technology are making it easier for patients to monitor their health records and provide data that was previously unobtainable.

Gottlieb wrote: “Rapid advances in smartphones enable the creation of platforms that make it easier for patients to take ownership of their health information. It also allows us to use more of that information to advance regulatory decision-making and product science. This is especially relevant as more information is being collected in real-world settings, and more closely reflects the patient experience. This gives us a new and valuable perspective on the performance of technologies.”

“The bottom line is this,” he continued, “when assessing whether valid scientific evidence shows that a device’s probable benefit outweighs its likely risks, the FDA can also consider rigorous, systematically gathered patient preference information as a part of the totality of the evidence from clinical and nonclinical testing. One of the mandates of this committee will be to help us develop the parameters for assessing these questions. When does this preference information meet the right standard, and address the right criteria, to be relevant to inform our decisions?”

Gottlieb hopes this first PEAC meeting is the first of many and that the agency’s long term goal to work with the patient community to develop assessment tools to collect and tabulate patient-reported outcomes that can help the FDA in their regulatory process.

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