Today, FDA Commissioner, Scott Gottlieb, MD, spoke at our event, a dialogue on FDA’s first draft guidance on patient-focused drug development. Meeting participants engaged and interacted with people from various patient organizations and companies from the health care community.
This dialogue was important to the patient community because it provided an opportunity to discuss and provide feedback on the FDA’s first Draft Guidance for Patient Engagement in Drug Development.
NHC Chief Executive Officer Marc Boutin kicked-off the meeting with a fireside chat with Dr. Gottlieb discussing patient engagement issues in drug development and ways to change the system for the better.
Dr. Gottlieb made it clear that the FDA is committed to getting this right. The Agency aims to go beyond just releasing these guidelines but restructuring FDA processes and procedures to ensure they offer certainty for drug companies that want to improve the way they develop medicines by seeking and incorporating the views of people who will use their products.
Watch the livestream of their conversation:
The Patient Perspective
Boutin and Gottlieb’s fireside chat was followed by a panel that provided the patient perspective. NHC Senior Vice President for Strategic Initiatives, Eleanor Perfetto, moderated the conversation between Suzanne Schrandt from the Arthritis Foundation, Bari Talente from the National Multiple Sclerosis Society, and Annie Kennedy from Parent Project Muscular Dystrophy.
The panelists expressed the importance of engaging patients “early and often” throughout the drug research and development process. This includes: choosing outcomes that matter to patients, better understanding patient views on benefits and risks, how to design clinical trials, and the importance of informing other decision makers of this information.
Throughout the panel, participants discussed how there are multiple purposes for patient engagement and it is essential to include perspectives from a wide range of their patient populations. Additionally, the panelists stressed that the patient perspective is crucial for decision makers such as insurance companies, value framework developers, and health technology assessors.
The Industry Perspective
Next, a panel comprised of pharmaceutical industry representatives was moderated by Kay Holcombe from the Milken Institute Center for Public Health. Panelists included Cartier Esham from the Biotechnology Innovation Organization, Kristin Van Goor from Biogen, and Stacy Holdsworth from Eli Lilly and Company.
The participants discussed how the guidance documents should provide the roadmap they need to engage patients in meaningful ways and incorporate their findings into their research and development processes. They expressed optimism that the FDA has laid a solid foundation for their work in patient engagement. They also mentioned that Commissioner Gottlieb’s recognition that the FDA needs to improve its processes is an important point. They look forward to working with the FDA to ensure a system is created for them to speak with FDA as they develop their patient-engagement strategies.
Throughout the meeting, the panelists highlighted their positive reactions to the first draft and how the guideline is on the right track. All recognized there have been recent, substantive achievements by the FDA and other stakeholders toward inclusion of the patient perspective to better understand the wants and needs of patients, patient organizations, and caregivers. However, participants recognized there is still more work done by the FDA and other stakeholders to make drug development more patient focused. It also was recognized by the panelists that the language in the FDA’s guidance reflects recommendations from the NHC and other patient organizations.
The NHC would like to thank those who participated in this dialogue. We will be using the information learned from this meeting to inform our comments on the FDA’s draft guidance, due to the agency on Sept. 11.