FDA’s Patient-Focused Drug Development Initiative: What Have We Learned?
One of the high-profile provisions of the 2012 Food and Drug Administration Safety and Innovation Act was the Patient-Focused Drug Development (PFDD) initiative. It required FDA to hold 20 meetings with the patient community over five years to hear directly from those with lived experience about the symptoms that most impact their lives and the variety of measures they take to manage their condition.
This series of meetings, which numbered 24 by the end of FY2017, has now been expanded to include externally-led meetings, for a total of 34 sessions to date. There are more externally-led meetings on the calendar, plus a robust pipeline of requests before the agency.
Our panelists will share their experiences in working with patient communities to plan for these often-intense listening sessions, as well as what they’ve learned from them and how they are applying this information. Please join us for an update and a forecast for what’s next in patient-focused drug development.
• Meghana Chalasani, Analyst, Center for Drug Evaluation and Research, U.S. Food and Drug Administration
• Annie Kennedy, Senior Vice President for Legislation & Public Policy, Parent Project Muscular Dystrophy
• Colleen Rye, Director, Research and Regulatory Policy, FasterCures, a center of the Milken Institute
• Cynthia Grossman, Director, Science of Patient Input, FasterCures, a center of the Milken Institute (moderator)
“The FDA Patient-Focused Drug Development meetings can be a powerful platform to capture the voice of patients and their caregivers and make the information available to all.” Moderator Cynthia Grossman, director, FasterCures, shared these words during her opening remarks for the June 7 webinar, “FDA’s Patient-Focused Drug Development Initiative: What Have We Learned?”
In 2012, the Food and Drug Administration (FDA) established the Patient-Focused Drug Development (PFDD) initiative. While it was unknown then how the novel initiative would fare, it has come to have an outsized impact on the inclusion of the voice of patients in drug development, review, and evaluation. The June 7 webinar featured perspectives from the FDA, the patient community, and FasterCures on the impact of PFDD and how it might continue to shape drug development and review.
What is Patient-Focused Drug Development?
The event began with some definitions by panelists of what PFDD is and how it is intended to be utilized.
PFDD is the FDA’s initiative to ensure that “patients’ experience, perspectives, needs, and priorities are captured and meaningfully incorporated into drug development and evaluation,” according to webinar panelist Meghana Chalasani, analyst at the FDA’s Center for Drug Evaluation and Review. More specifically, PFDD aims to identify the best approaches for collecting and using patient input, best practices for clinical trials, best ways to communicate with patients to understand their preferences for benefits and risks of treatments, and identify unaddressed treatment targets for patients. Put another way by Colleen Rye, director at FasterCures, “Scientists can’t work on problems when they don’t know those problems exist.”
History of Patient-Focused Drug Development
Rye shared that a focus on patients hasn’t always been the M.O. for medical product development. The history of PFDD begins with the Prescription Drug User Fee Act V (PDUFA V; 2012 to 2017) for the FDA. In PDUFA V, the FDA agreed to host 20 condition-specific PFDD meetings to gather patients’ perspectives on their conditions and available therapies, and to increase the use of FDA’s patient representatives as government employee consults. The FDA’s meetings, which surpassed the 20 required, were a “wonderful success” according to Rye. As a result, the FDA has voluntarily continued to host select meetings with an additional meeting planned for this July.
In the past year and a half, PDUFA VI and the 21st Century Cures Act were passed. Both acts had complementary language for strengthening the FDA’s PFDD initiative. Under these acts, the FDA was committed to strengthening its staff capacity for PFDD, conducting workshops to garner new ideas about PFDD from patients, and issuing a series of guidance documents for PFDD.
As Rye mentioned in the conclusion of her presentation, interested parties can follow progress on the implementation of 21st Century Cures, including its patient engagement efforts, via FasterCures’ online tracker.
Current State of PFDD at the FDA
Now that efforts to collect patient perspectives are underway, Chalasani shared that the FDA is working on incorporating patient experience data in several ways. These include:
- Incorporating patient experience data into clinical review documentation
- Creating a PFDD glossary to standardize definitions
- Drafting methodological guidance documents for the collection and use of patient experience data
- Revising internal documents, such as standard operating procedures
- Creating a repository of PFDD tools
- Continuing to conduct PFDD meetings
Chalasani also encouraged other stakeholders to contribute to PFDD, sharing multiple ways that organizations can do so. These include supporting research, conducting natural history studies of disease, forming centers of excellence for the study and treatment of disease, participating in policy discussions, convening, and contributing to guidance documents.
There are several questions that stakeholders are best-positioned to address, and their responses could significantly help the FDA utilize the PFDD process to improve research, development, and regulation across the medical field.
Stakeholders can start by asking themselves what disease impacts matter most to patients, how preference can vary by patient subgroup, how to increase the likelihood of patient enrollment in a study, as well as what leads to their retention, and other questions that get to the heart of what matters most within patient communities.
Considerations for Externally Led PFDD Meetings
Many of these questions can be supported through another effort that Chalasani promoted: externally led PFDD meetings. Externally led PFDD meetings are similar to the FDA’s internal meetings in that they convene health-care stakeholders to hear directly from patients about their experiences with their conditions and current therapies. These meetings can draw from the FDA meeting structure but should not be bound by it. Each organization should tailor the meeting to meet the needs of its community and stakeholders.
Chalasani offered several pieces of advice for organizations that plan to host externally led PFDD meetings. First, organizations should submit a letter of intent to the FDA so that the FDA is aware of the meeting and can serve as a resource. Also, organizations should not be daunted by resource constraints – externally led PFDD meetings don’t have to be resource intensive, she emphasized. Organizations should work together to ensure efforts aren’t duplicative and should also be considerate of patients’ time. It may be best to coincide externally led PFDD meetings with already planned events, such as annual meetings.
PFDD from the Patient Group Perspective
There have been early innovators and adopters as patient organizations engage with the FDA, as Annie Kennedy, senior vice president for legislation and public policy of Parent Project Muscular Dystrophy (PPMD), pointed out. PFDD created a framework for patient organizations to formally engage with the FDA in a way that wasn’t possible before.
For PPMD, the establishment of PDUFA V came at the right time to influence research and clinical trials on Duchenne muscular dystrophy. Kennedy said that the establishment of PFDD “created a roadmap” for her organization to develop tools for patient engagement.
In reaction to PFDD, PPMD has engaged with the FDA, conducted five patient-focused benefit-risk studies, published two whitepapers on patient engagement, established a patient registry, created patient-reported outcomes that reflect the needs of the patient community, and written a draft guidance for Duchenne muscular dystrophy that the FDA later adopted. Notably, PPMD had not until recently conducted a PFDD meeting under the existing framework developed by the FDA.
Since the establishment of PDUFA V in 2012, two new therapies for Duchenne have been developed. Recognizing that the therapy context has changed but wanting to continue its patient-engagement efforts, PPMD used the PFDD framework to host the Duchenne Patient-Focused Compass Meeting. This meeting brought a diverse set of stakeholders into a conversation about Duchenne, including payers, several federal agencies, medical product developers, and providers. At the meeting, PPMD established a community advisory board, hosted a live webcast, and incorporated a diverse set of panels, including a panel for adults with Duchenne and a health economics panel to understand the economic burden of the disease.
Kennedy highlighted a new platform, NCATS Patient-Focused Therapy Development Toolkit, to assist groups wanting to advance patient engagement in therapy development. Drawing on the experience of a group of organizations, including FasterCures, this recent collection is one effort to share lessons learned to the broader community.
Big Hopes for the Future, Next Steps, and How to Stay Engaged
When asked how the FDA intends to use the voice of the patient information, Chalasani believes the PFDD is having substantial impacts in the FDA’s product evaluation, specifically for conducting benefit-risk assessments and understanding the therapeutic context. For sponsors, PFDD can inform target product profiles and endpoints for clinical trials, identify unmet needs, and enhance patient engagement.
With the hope that many different types of patient groups, large and small, will be able to participate in PFDD, Kennedy encouraged organizations to conduct internal needs assessments to understand and articulate what they are hoping to accomplish and let that drive their activities under PFDD. While the experience of PPMD can be an example of successful interactions with the FDA and sponsors, it is only one example, and each disease area has different needs. The good news is that the PFDD initiative includes multiple pathways of engagement and a suite of activities that aim at including the voice of patients and caregivers at many different points along the drug development lifecycle.
Resources to Support Your Involvement with PFDD: