“Celebrating 10 years of Patient-Focused Drug Development” with Dr. Theresa Mullins, CDER Associate Director for Strategic Initiatives.

On Friday, September 16, the Alliance for a Stronger FDA continued its series of FDA webinars, hosting Dr. Theresa Mullin, the Associate Director - Strategic Initiatives at FDA’s Center for Drug Evaluation and Research (CDER). Dr. Mullin discussed the impressive history and development of the Patient-Focused Drug Development (PFDD) meetings program and its plans for the future. She credited the program’s success to the active engagement by the many patient groups that have participated as well as the PFDD staff and the other FDA personnel. She emphasized PFDD’s role in bringing the patient’s voice into its prominent position in the regulatory process. She also acknowledged the importance of Budget Authority (BA) appropriations, which is the Alliance’s focus, in sustaining PFDD and all FDA programs.

PFDD is a systematic approach to help ensure that the experiences, perspectives, needs, and priorities of patients -- the experts in what it is like to live with their condition -- are captured and meaningfully incorporated into drug development and regulatory evaluation. Dr. Mullin described how the FDA began with patient consultation sessions in early 2012, well ahead of the PDUFA V reauthorization that mandated the PFDD program, to obtain the view of patients on how to design PFDD. The 24 disease-specific PFDD meetings in the program’s first five years, and the Voice of the Patient Report from each of them amplified the patients’ voice and helped make it an integral part of the regulatory process.

The agency extended the program after the success of these initial PFDD meetings, encouraging patient groups to conduct their own, externally led PFDD meetings and to invite FDA participation. Over the past five years, 55 of these externally led meetings have enabled the FDA to listen to the voices of these additional patients and include them as important parts of the regulatory process.

Following PDUFA VI, FDA is in the process of developing four key guidance documents for systematic approaches to the collection and use of patient data:

• Collecting Comprehensive and Representative Input (final);

• Methods to Identify What is Important to Patients (final);

• Selecting, Developing or Modifying Fit-for-Purpose Clinical Outcome Assessments (draft); and

• Incorporating Clinical Outcome Assessments into Endpoints for Regulatory Decision Making (to be issued in the coming months).

Looking forward, Dr. Mullin called for a sustained effort to better interpret patient experiences and improve the quality and reliability of patient data. She wants to ensure that industry is aware of the pertinent guidance documents and that patient groups are involved in more of the development of new measures and tools. She wants to see standard core medical outcomes and assessments, as variable quality of tools limits their effectiveness. She pointed to the Standard Core Clinical Outcome Assessments Grant Program as a resource to help in the development of these tools. She also said there could be a national catalogue that would apply reliable measures to all efforts.

During a very interactive question and answer period, Dr. Mullin addressed how patient groups can engage with the FDA, how to strengthen patient information for use in the regulatory process, and the importance of diversity and engagement of underrepresented populations.

The transcript and video of the webinar will be available shortly on the Alliance website.