FDA Invites Patient Organizations to Take a Place at the Podium

By: Theresa M. Mullin, Ph.D.

Sometimes, the most valuable thing we can do as regulators at FDA is simply to listen. I’m reminded of that each time we hold a public meeting as part of the Patient-Focused Drug Development (PFDD) program.

Theresa MullinWe began PFDD to more systematically obtain the patient perspective on certain diseases and their treatments. The effort is part of an FDA commitment under the fifth authorization of the Prescription Drug User Fee Act (PDUFA V).

Each public meeting is focused on a specific disease area. Our commitment is to gain perspectives on at least 20 disease areas by the end of FY 2016. And having already held 17 meetings to hear from patients with diseases as varied as breast cancer, fibromyalgia and sickle cell disease, we are well on our way.

What have we learned so far? For one, thanks to PFDD we now have even more first-hand knowledge from those most affected by the diseases. We have heard directly from patients, their families, and care givers about the symptoms that matter most to them; the impact the disease has on patients’ daily lives; and their experiences with currently available treatments. For example, we’ve learned that for diseases that are progressive and severely disabling, patients and their families may consider an “ideal” treatment to be one that at minimum can halt disease progression.

These perspectives are critical to helping us understand the context in which we are making regulatory decisions for new drugs. And they’ll have ramifications for years to come. We believe that the long-term impact of PFDD will be better, more informed FDA decisions and oversight both during drug development and during our review of a marketing application.

Expanding the Benefits of the PFDD Meeting Model

This is a priority for FDA. To that end, we’ve committed to hold meetings for at least 20 disease areas, and are currently planning to hold 24 different disease-focused meetings by the end of FY2017, exceeding our commitment. We recognize, however, that there are many more disease areas than can be addressed in the planned FDA meetings where drug development and regulatory decision making would benefit from a meeting focused on obtaining the patient’s perspective.

To help expand the benefits of FDA’s PFDD initiative, FDA invites the independent efforts of patient organizations to identify and organize externally-led patient-focused collaborations to generate public input on other disease areas, using the process established through Patient-Focused Drug Development as a model. Given the tremendous number of diseases affecting the U.S. patient population and the effort required to conduct a successful PFDD meeting, externally led PFDD meetings should target disease areas where there is an identified need for patient input on topics related to drug development.

We recommend that patient organizations interested in conducting an externally-led PFDD meeting submit a letter of intent so that we are aware of their plans. Submission details and more information on considerations to take into account are outlined on FDA’s website.

Please note that an externally led PFDD meeting and any resulting products, such as surveys or reports, will not be considered FDA-sponsored or FDA-endorsed. And while we can’t guarantee FDA’s specific involvement at every meeting, FDA will be open to participating in a well-designed and well-conducted meeting.

And as the number of patient-focused forums continues to grow, we at FDA will continue to listen and look forward to gaining the additional insights that only patients, their families, and care givers can provide.

Theresa M. Mullin, Ph.D., is Director of FDA’s Office of Strategic Programs in the Center for Drug Evaluation and Research

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