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FDA’s Patient-Focused Drug Development: The view from the other side of the table

04/07/2015

Since the Patient-Focused Drug Development (PFDD) initiative was launched in 2012, the Food and Drug Administration (FDA) has held or scheduled 14 disease-specific meetings to better understand patients’ experience with symptoms, impacts on daily living, and available therapies. With more meetings on the docket for 2015, and growing interest among patient groups in applying the PFDD model to developing their own data, FasterCures examines the experience of organizations engaged in this process.

Tune in to hear insights from patient groups about how they prepared for their PFDD meetings, what lessons they learned, and how their activities have since been impacted. And find out how groups that do not have a scheduled meeting with FDA are adapting the PFDD model to develop their own information about patient preference and seeking to have it integrated into FDA decision-making.

Summary

Traditionally, the opportunities to infuse patient input into regulatory decision-making have been very limited and relegated to the late stage of the new drug application review process, noted Kim McCleary, FasterCures’ director of strategic initiatives, at the beginning of FasterCures’ April 7 Webinar. During the hour-long discussion, “FDA’s Patient-Focused Drug Development: The view from the other side of the table,” hundreds of attendees tuned in to hear insights from patient groups engaged in the U.S. Food and Drug Administration’s (FDA) Patient-Focused Drug Development (PFDD) initiative – a commitment under the fifth authorization of the Prescription Drug User Fee Act (PDUFA) to hold at least 20 disease-specific meetings to better understand patients’ experiences with symptoms, impacts on daily living, and available therapies.

PFDD was launched in 2012 and reflects a larger movement to ensure that patients’ perspectives are more meaningfully captured and applied to product development and regulatory decision-making. It has opened up a new dialogue among patients, pharmaceutical and device developers, and regulatory gatekeepers about expected benefits and tolerable risks of new therapies, as well as the outcomes that matter most to patients and caregivers.  

More than half of the 20 disease meetings have already been completed, and with more on the docket for 2015, the broader patient community has already begun looking for ways to apply early learnings to the development of their own patient preference data.

Along with McCleary, panelists were Kimberly Beer of Susan G. Komen and Andrea Ferris of LUNGevity, leaders in patient organizations for diseases that have completed PFDD meetings, and Jennifer Sheridan Palute of Parkinson’s Action Network, speaking on behalf of a community whose disease will be the focus of a meeting to take place later this year. In addition to sharing experiences and lessons learned from their PFDD engagement, they also explained how it has impacted other activities at their organizations.

How do diseases get selected?

The announcement of the initial 16 of the 20 specified diseases left many patient groups asking how decisions were made and how their condition might be selected for one of the final four spots. The FDA’s PFDD Web site lists the following criteria for the diseases selected:

  • chronic, symptomatic, or affect functioning and activities of daily living;
  • aspects of the disease are not formally captured in clinical trials;
  • currently no therapies or very few therapies, or available therapies do not directly affect how patient feels, functions, or survives; and
  • has a severe impact on identifiable subpopulations (such as children or the elderly).

 Meeting preparation and recruitment

Webinar slide on recruitmentThe PFDD meeting on lung cancer, in which Ferris participated, was among one of the first PFDD sessions to be held. It took place in June 2013 and brought together more than 50 patients and representatives both in-person and via Webcast. Held earlier this month, on April 2, the breast cancer meeting was the most recent PFDD session. Beer was one of nearly 100 attendees at this session, though she noted that given the scope and impact of breast cancer on the population she had hoped to see more patients and their families in attendance, of which there were about 25-30 individuals who self-identified in this role.

Both Ferris and Beer cited the lack of diversity as a challenge, noting that the attendees at their meetings did not reflect the full scope of their patient populations. “I was hoping that the panel would be representative of racial and ethnic diversity, as well as different levels of health literacy, stage of disease, treatment experience, time since diagnosis, and where they were in their overall patient journey,” Beer remarked.

“Compared to the overall lung cancer population, the in-person participants represented a high proportion of women, patients diagnosed more than five years ago, and patients whose lung cancer was currently in remission,” Ferris weighed in, which was not representative of the overall patient population.

Ferris and Beer offered these tips about recruiting for PFDD meetings:

  • Reach out to your patient community early, and be clear about how they can participate.
  • Work with other organizations in your disease space.
  • Identify different subsets of your patient population to ensure diversity in experience.
  • If possible, identify resources to allow patients to travel and participate in person.
  • Make sure to underscore the exponential value patients bring to these meetings and the impact they can have on FDA policies and decisions.

Parkinson’s Action Network is preparing for its meeting later this year, which will be focused both on Parkinson’s disease and Huntington’s disease. Palute sees the meeting as an invaluable opportunity to educate the FDA about current treatment for Parkinson’s disease and the unmet medical needs faced by those living with the disease. She noted a challenge with this meeting, and perhaps others, is the heterogeneity of the disease. “Parkinson’s symptoms are different from patient to patient – some have really severe motor symptoms – and others, their symptoms don’t really impair them. It’s really the depression of non-motor symptoms that impact their day-to-day life.”

Impact on programs

All three panelists expressed gratitude to the FDA for organizing these meetings and allowing patients’ voices to be heard. As McCleary highlighted at the beginning of the Webinar, this type of opportunity is a step in the right direction for patient experience to inform R&D. “There was a clear desire [from the FDA] to hear the patient perspective, and they were very respectful of that,” Beer said.

Based on the recommendations coming from the 2013 lung cancer PFDD meeting, LUNGevity launched a new program, Project Transform, together with a researcher at Johns Hopkins University, to engage patients and integrate their experiences into lung cancer policy, treatment, and research. The goal of Project Transform is to “change the paradigm in lung cancer from assumptions being made about patient wishes to evidence-based conclusions about patient desires – to ensure that their preferences are recognized, their voices are valued, and that living well with lung cancer can ultimately become the norm,” explained Ferris.

What can you do if you don’t have a PFDD meeting?

040715 PFDD external meetings slidePatients and disease organizations not represented at one of the 20 PFDD meetings have been vocal about their desire to leverage this process and its takeaways to better communicate the needs and preferences of their own patient communities with the FDA. The agency suggests the following to help draft an organization’s roadmap for externally led PFDD meetings:

  1. Coordinate with other patient groups.
  2. Submit a Letter of Intent (LOI) to the FDA.
    • FDA will then review the letter and identify internal stakeholders.
    • The FDA will respond to LOI with recommendations.
  3. Hold a pre-meeting teleconference with the FDA.
  4. Prepare your meeting by identifying facilities [preferably close to FDA’s campus in Silver Spring, Md., since the agency has tightly restricted travel capability], agenda, logistics, and attendee outreach plans.
  5. Hold your meeting.
  6. Hold a post-meeting teleconference with the FDA.
  7. Finally, publish a report with your findings.

FDA’s key takeaways from the first 12 PFDD meetings

Borrowing from comments made by Theresa Mullin, the director of the Office of Strategic Programs at FDA’s Center for Drug Evaluation and Research, at a conference in March, McCleary underlined some of the key takeaways FDA has had from the PFDD process, and underscored how far the agency has come in its appreciation for the role of patient input over the past few years:

  • Patients with chronic serious disease are experts on what it’s like to live with their condition.
  • Patients’ “chief complaints” may not be factored explicitly into drug development plans, including measures of drug benefit planned in trials.
  • Patients want to be as active as possible in the work to develop and evaluate new treatments.
  • Patients want their experience described using their own words.
  • Patients aren’t expecting the FDA to address all the gaps in current treatment or current approaches to drug development, but do want the FDA to help identify the most effective pathways for them to play a major contributing role.

Going forward

These meetings have been a clear catalyst for many disease organizations to start productive conversations with the FDA about not only how to engage patients in the approval process, but also how to engage throughout the entire development process. The FDA has released a series of “Voice of the Patients” reports to summarize the input provided by patients and patient representatives at each of the PFDD meetings. So far, nine of the 12 meetings have published reports. McCleary noted that, having attended eight of the meetings held so far, the reports are “a very authentic, accurate record of the patient testimony given, as well as comments submitted in writing through the docket process.”

FasterCures is excited and hopeful that this process will help advance a culture of patient engagement and empowerment throughout the regulatory decision making process.

To learn more about the work we’re doing to advance patient-centered benefit-risk assessment, please visit our Web site at fastercures.org/benefitrisk.

 

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Speakers

Beer

Kimberly Beer,
Director, Public Policy, Susan G. Komen for the Cure

Ferris

Andrea Ferris,
President and Chairman, LUNGevity

Sheridan

Jennifer Sheridan Palute,
Director of Policy, Parkinson's Action Network

Moderator

McCleary2014

Kim McCleary,
Director, Strategic Initiatives, FasterCures