What Do Catering Requests and the FDA Patient Focused Drug Development Meetings Have in Common?

By Ronnie Tepp, Kim Cantor and Jenny McCue -

Preference and input not only matter, they are the key ingredients in a successful outcome.

When you cater a party, things can take a turn for the worse if you don’t account for party-goer preferences. It will not go well if you host a pig roast for a vegetarian club or serve shelled peanuts at an event attended by people with serious nut allergies.

Great ideas can go awry simply because preferences and input were not considered. It is true when planning a party and it is also true when designing a clinical trial or when reviewing drugs and devices.

The patient community recognized that this critical ingredient was missing in the regulatory review process at FDA and worked with Congress to address it. The result was FDA’s Patient-Focused Drug Development initiative (PFDD). PFDD is a commitment made in 2013 under the fifth authorization of the Prescription Drug User Fee Act (PDUFA V) that aims to systematically gather patients’ perspectives on their condition and available therapies to treat their condition. As part of this commitment, FDA was required to hold at least 20 public meetings, each focused on a specific disease area.

The FDA has followed through and from 2013 – 2016 hosted 20 PFDD meetings with four more on the way in 2017. Each of these meetings provided an opportunity for individuals to share their story with the FDA – the stories and experiences that can’t be found in textbooks and studies. What we saw happen from these meetings was critical. The groups, knowing the importance FDA places on data, began using data to package and tell stories in a way that FDA could use.

So what happens next? How will FDA collect and incorporate input from patient communities now that they’ve met the PFDD meeting-mandate? James Valentine wrote an excellent blog earlier this month questioning if FDA was passing the baton to patient organizations. He suggests that after 2017, perhaps PFDD meetings will be driven by patient organizations rather than the FDA. This raises a number of questions: will patient and disease organizations have the capacity to plan and execute these meetings; will FDA bring the same attention and focus to externally-led meetings as it did to the PFDD meetings; and how will the outcomes from the meetings be utilized?

There are indications that Valentine’s premise is correct and the FDA is looking to externally-organized meetings in the future. Already the FDA has recognized the significant interest from the patient community and green-lit external meetings in late 2014; they even published guidelines for groups that want to host a meeting. It is clear that patients want the FDA to hear from them, and patient organizations are responding by forming their own meeting.

Patient engagement with the FDA will continue, and most likely will be through externally driven meetings. With this model moving forward, it will be critical to ensure that every patient with a disease and condition has an opportunity to tell their story. There are lot more than 20 diseases and conditions out there. And patients need strategic engagement to drive the future of treatments that truly meet their needs.

At HCM, our priority is to ensure patients are at the center of the decision-making process, and we have a history of successfully connecting patients with the FDA. In 2014 we held a virtual town hall with FDA that included input from thousands of patients. Recently, we worked with the diabetes community to host a workshop with the FDA that had more than 900 individuals participate online or in-person. We know how important patient engagement is at FDA and look forward to working with patients and the FDA in this next phase.

And, as long as the FDA continues to engage and incorporate the patient preferences and input, they are on the right track to avoid parties filled with dangerous menus.

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