miércoles, 19 de octubre de 2016

FDA Law Blog: As the Patient-Focused Drug Development “Pilot” under PDUFA V Concludes, Is FDA Passing the Baton to Patient Organizations?

FDA Law Blog: As the Patient-Focused Drug Development “Pilot” under PDUFA V Concludes, Is FDA Passing the Baton to Patient Organizations?



Posted: 07 Oct 2016 09:53 AM PDT
By James E. Valentine –

In what can only be described as a success story, FDA’s Patient-Focused Drug Development (“PFDD”) initiative has opened the doors to hundreds of patients, caregivers, and other patient representatives to share their experiences with their diseases and conditions with FDA. This series of disease-specific meetings has helped set what FDA refers to as the “therapeutic context” for its regulatory decision-making for drugs and biologics.  PFDD gives patients an opportunity to help CDER and CBER review staff better understand the burden and impacts of their condition and their experiences with treatment options.  This amplification of the patient perspective allows for the evaluation and inclusion of information that is not conveyed by reading the medical literature and textbooks.  This input is not limited to the day of the meeting itself, but is memorialized in a “Voice of the Patient” report that is in turn made available to FDA review staff.  This report importantly includes a draft benefit-risk assessment framework, which provides patient input in a format that can be used during drug approval decisions.

Four years into what FDA refers to as its “pilot,” the Agency announced a milestone for the program: it held its 20th PFDD meeting – the number of meetings FDA committed to host under the fifth authorization of the Prescription Drug User Fee Act (“PDUFA V”).  While that was the minimum required number, FDA will hold four more PFDD meetings by the end of FY2017, which is when PDUFA V expires.

While the PFDD meetings were underway, FDA negotiated a draft commitment letter for PDUFA VI.  As proposed, FDA will not be committing to host any additional PFDD meetings.  This begs the question: what is the future of PFDD?

(This is not a new question; see one of our previous discussions here.)

The Future: Externally-Led PFDD Meetings?

The most obvious answer is that PFDD meetings are not going to stop, but will instead be carried on by patient organizations instead. Because there are many more disease areas than can be covered by the 20+ FDA meetings under PDUFA V, in December 2015 FDA invited patient organizations to host their own PFDD meetings.  This parallel effort involves patient organizations submitting a Letter of Intent (“LOI”) to FDA, after which point they may proceed with planning and hosting an externally-led PFDD meeting.

While this program is still getting off the ground, early examples have demonstrated that well-planned and executed externally-hosted meetings can be a successful alternative to FDA-hosted meetings:

  • On November 16, 2015, the Amyloidosis Research Consortium (ARC) in collaboration with the Amyloidosis Foundation and Amyloidosis Support Groups hosted an “Amyloidosis Patient Forum with FDA.” Although this meeting occurred before FDA formally announced a process for hosting externally-led PFDD meetings, the agenda mirrored that of FDA’s PFDD meetings. As a result of the meeting, on June 7, 2016, ARC submitted a “Voice of the Patient” report to FDA, which follows the same format as the FDA reports (the report can be found here).
  • On September 15, 2016, the Myotonic Dystrophy Foundation (MDF) hosted the first official externally-led PFDD meeting, which also had an agenda that followed the format of FDA’s PFDD meeting. MDF has committed to develop a “Voice of the Patient” report to submit to FDA.
Both the amyloidosis and myotonic dystrophy meetings had the key elements that made the FDA meetings so successful: large turnouts by their patient communities; a well-constructed agenda that included a mixture of patient panels, polling questions, and moderated audience discussion; webcast/livestream participation; and, attendance by key FDA officials, including remarks from some. In addition, the externally-drafted “Voice of the Patient” report will serve as an important resource to FDA review staff.

So do Externally-Led PFDD Meetings Fit Within PDUFA VI?

While PDUFA VI does not explicitly address whether FDA will continue to accept LOIs for externally-led PFDD meetings, there are a number of activities under the “Enhancing the Incorporation of the Patient’s Voice in Drug Development and Decision-Making” commitment that could accommodate the program. Alternatively, the draft commitment letter indicates that FDA may be moving away from the PFDD meeting model:

FDA will develop a series of guidance documents to focus on approaches and methods to bridge from initial patient-focused drug development meetings, like those piloted under PDUFA V, to fit-for-purpose tools to collect meaningful patient and caregiver input for ultimate use in regulatory decision making.
This could include more qualitative methodological approaches, such as surveys of patient communities and other technologies to capture patient experiences (e.g., Patient-Reported Outcome measures).

Could PFDD Meetings Reemerge Under FDASIA Section 1137?

On February 19, 2016, FDA published a report on publicly-submitted stakeholder views on potential “strategies to solicit the views of patients during the medical product development process and consider the perspectives of patients during regulatory discussions,” which the Agency is required to develop and implement under the Food and Drug Administration Safety and Innovation Act (“FDASIA”) Section 1137, “Patient Participation in Medical Product Discussions.”  The report states that several comments recommended the facilitation of more systematic patient engagement by the Office of the Commissioner across FDA Centers responsible for human medical product regulation.  If there is a demand by patient stakeholders for the initiative to continue, one plausible possibility would be for PFDD to transition to the Office of the Commissioner where patients could share their experiences with CDER, CBER, and CDRH.

If that happens, I guess the initiative would have to be renamed Patient-Focused Medical Product Development or PFMPD. Or just keep it as PFDD…