FDA Commissioner Gottlieb Emerges as a Champion for Patient Engagement in 2018; Patient-Centric Guidance Development, Rare Disease Listening Sessions, and the Benefit-Risk Framework

April 11, 2018By James E. Valentine

While FDA’s medical product centers have been advancing programs and policies that support incorporating the voice of the patient into their regulatory decision-making for several years (see previous coverage of CDRH here and CDER/CBER here), in the first quarter of 2018 this work has been elevated as a central theme in Commissioner Scott Gottlieb’s public statements – it appears that the Commissioner of Food and Drugs made it a New Year’s resolution to be a champion for patient engagement.

This is said a bit tongue-in-cheek, as these are not Dr. Gottlieb’s first signals of being an advocate for patient-centricity. His administration previously established an FDA-wide Patient Affairs Staff to foster inter- and intra-Agency collaboration on issues of patient engagement, as well as the Patient Engagement Collaborative, a forum for patient advocacy organizations to discuss patient engagement at the FDA. However, as someone that has spent nearly a decade developing patient engagement policies and aiding patient communities engaging in medical product development and regulatory decision-making, it is refreshing to see the Nation’s top drug official further elevate progress in this area, as evidenced by his recent public statements.  (See our new “Patient Advocacy Organizations” industry page on our firm’s website.)

Advancing the Patient-Centric Development of Drug Development Guidance

On February 15th, Commissioner Gottlieb announced the development of five guidance documents on complex, serious neurological conditions – areas where he acknowledges FDA must “become more nimble, collaborative and patient-focused” to address these urgent unmet needs.  What is novel about these disease-specific drug development guidances is that three of them were developed in consultation with the respective patient communities.  In fact, two of FDA’s guidance documents were initially developed and proposed as draft guidance by a patient advocacy organization (The ALS Association’s & Parent Project Muscular Dystrophy’s), providing insights into those diseases that helped FDA advance their own draft guidances.  Then, according to Commissioner Gottlieb, the draft guidance on early Alzheimer’s disease drug development was a result of “working closely with patients” informing the innovative approaches to studying very early disease before onset of dementia.

Patient-centric guidance development brings the voice of the patient more central to informing FDA’s current thinking on clinical trial design and determinations of clinical meaningfulness, among other things. This type of activity was advanced at a March 19th public workshop, which was held to inform development of guidance, as required by the 21st Century Cures Act, on considerations for patient advocacy organizations to develop and submit proposed draft guidance, like was done for ALS and Duchenne.

Embracing the Role of Patients in Treating Rare Diseases

On February 26th, in observation of Rare Disease Day, Commissioner Gottlieb announced that FDA was entering into a Memorandum of Understanding with the National Organization for Rare Diseases “to conduct outreach with [the] new Patient Affairs Staff on ways to enhance the incorporation of patient experience into regulatory discussions.”  The MOU will do this, in part, through the planning of a series of listening sessions with rare disease patient communities to foster “early and iterative engagement” to establish an understanding of certain rare diseases and their unmet needs to inform medical product development programs.  This was reiterated by Gottlieb’s Acting Director of the Patient Affairs Staff, Andrea Furia-Helms, who previously led FDA’s Patient Representative Program for 10 years (her statement is available here).

Partnering with Patients to Incorporate Their Experience into FDA’s Benefit-Risk Decisions

Under PDUFA V, FDA held 20+ disease-specific meetings under the Patient-Focused Drug Development (PFDD) initiative, which generated the therapeutic, or clinical, context for those diseases directly from the experiences and perspectives of those patients who live with them. This input was intended to inform FDA’s benefit-risk decisions for investigational products intended to treat those diseases, and were to be articulated by Agency review staff in the “benefit-risk assessment framework”.   On March 30th, Commissioner Gottlieb announced an updated implementation plan for “Benefit-Risk Assessment in Drug Regulatory Decision-Making.”  A key tenant of this plan for fiscal years 2018-2022 is to continue to incorporate the patient voice into benefit-risk assessments under PDUFA VI and 21st Century Cures.  The plan highlights a number of efforts to enable more systematically gather and incorporate patient experience data, such as that which was collected during PFDD meetings:

  • Developing PFDD guidance on the collection of, submission to, and use of “patient experience data” by the Agency (see our firm’s comment with suggestions for the first PFDD guidance on this topic here);
  • Continuing to host PFDD meetings;
  • Encouraging patients stakeholders to conduct their own externally-led PFDD meetings (this has become a very productive venue for patent engagement; by our count, the 12th and 13th such meetings were held jointly on April 6th for two rare, severe dermatologic conditions);
  • Providing patient stakeholders more channels to provide input, such as by hosting Patient Engagement Advisory Committee meetings; and
  • Facilitating access to externally-submitted reports of patient experience data, such as Voice of the Patient reports from externally-led PFDD meetings.

Together, this plan and the activities discussed in Commissioner Gottlieb’s previous statements earlier this year will continue the shift to a more patient-centric and patient engaged regulatory framework. In the words of Commissioner Gottlieb:

Tools for capturing the patient experience…are transforming nearly every aspect of medical product development. Patients are teaching us about the benefits that matter most to them and the risks that they are most concerned about. Patients are, rightly so, becoming the driving force of the medical research enterprise.