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Theresa M. Mullin

FDA Compliance

The Patient’s Voice Enhances FDA’s Approach to Drug Review and Development

Public meetings helped fulfill an FDA commitment one year ahead of schedule

Published: Thursday, October 20, 2016 - 15:51

Since the launch of the Patient-Focused Drug Development (PFDD) program as part of the fifth authorization of the Prescription Drug User Fee Act, the U.S. Food and Drug Administration (FDA) has worked intensively to explore ways to enhance the patient’s voice in drug development. Recently the FDA reached a particularly gratifying milestone in this important work with the 20th Patient-Focused Drug Development public meeting.

The Prescription Drug User Fee Act provides much needed funding from the pharmaceutical industry to support the FDA’s premarket review activities and the agency’s work to encourage drug development. Under this act, the FDA committed to obtain patients’ views in at least 20 disease areas over the course of the five-year period when the Prescription Drug User Fee Act must be reauthorized in September 2017. That means conducting a public meeting for each disease area to obtain patient perspectives on current treatment approaches and the impact of the disease on daily life. The FDA fulfilled this commitment one year ahead of schedule at the closing of the 20th PFDD meeting, which was with patients who have received organ transplants.

The PFDD meetings have provided the FDA opportunity to hear directly from patients, their families, and caregivers about the symptoms that matter most to them, the impact of disease on daily life, and how they view the benefits, risks, and burdens of current disease treatments. Hearing what patients care about is extremely valuable in understanding targeted disease areas and determining how best to facilitate drug development for particular diseases.

The FDA plays a critical role in drug development, but it is only one player in the process. Other stakeholders attending PFDD meetings (i.e., healthcare providers and industry sponsors) are also gaining valuable information. These meetings have helped identify and raise awareness of patients’ unmet needs, and focus engagement within the patient community (e.g., in preparation for the narcolepsy meeting patient groups collaborated to form a coalition called United Narcolepsy). This and more has been chronicled in Voice of the Patient reports, which provide a detailed account of the input heard at each meeting.

The Voice of the Patient reports are intended to be useful to FDA colleagues conducting reviews and the broader community. These reports summarize what was learned through patient speaker panels, audience participation, webcasts, and submissions to the public docket. Each report faithfully captures this information, which is distributed internally to the relevant review divisions for reference when advising sponsors on their drug development programs and when assessing products under review.

As drug development advances in the 21st century, sponsors are using increasingly sophisticated and vital forms of technology to generate medicines of the future. But there is a critical part of drug development that has little to do with advanced technology. Instead, it has to do with listening to patients about their personal experiences living with diseases and treatments, and determining the best ways to reliably capture their perspectives to better integrate them into decision-making.

I believe that the long-term impact of PFDD will be better, more informed FDA decisions and oversight during drug development and during the review of marketing applications. We at the FDA are extremely grateful to the hundreds of patients and their loved ones who have so generously and, in some cases, courageously, participated in the PFDD meetings and shared personal stories, experiences, and perspectives.

The FDA may have met the latter of its Prescription Drug User Fee Act commitment, but it isn’t finished yet. Patient-focused drug development is a priority for the FDA. Beyond the 20 PFDD meetings already held, the FDA plans to hold four more by the end of fiscal year 2017. Additionally, the FDA recognizes that there are many more disease areas to address. To help expand the benefits of the PFDD initiative, the FDA welcomes similar patient-focused meetings organized by the patient groups themselves. Interested patient groups can submit a letter of intent, which is outlined in “Externally-led Patient-Focused Drug Development Meetings” on the FDA’s website.

One of the most valuable things the regulators at the FDA can do is simply listen. I’m reminded of that each time a PFDD public meeting is held. The FDA regulators will continue to listen—and learn—and look forward to continuing to gain the additional insights that only patients, their families, and caregivers can provide.


About The Author

Theresa M. Mullin

Theresa M. Mullin, Ph.D., is the director of the Food and Drug Administration’s (FDA) Office of Strategic Programs in the Center for Drug Evaluation and Research (CDER). Mullin currently leads the CDER Patient Focused Drug Development Initiative and the FDA delegation to the International Conference on Harmonization.