PDUFA VII negotiations continue as industry presses for clear timelines

Regulatory NewsRegulatory News | 03 March 2021 |  By 

As meetings continue between the US Food and Drug Administration (FDA) and industry to negotiate the upcoming reauthorization of the Prescription Drug User Fee Amendments (PDUFA VII), cell and gene therapy demands and timelines continue to loom large. Minutes from this and other end-of-year PDUFA subgroup meetings continue to show how industry and FDA are working to balance commitments and resources for PDUFA VII.
In a 17 December 2020 breakout meeting, representatives from FDA’s Center for Biologics Evaluation and Research (CBER) met with industry to review proposed commitment language to support CBER’s capacity to develop and review the influx of cell and gene therapy products the agency will field in the coming years.
The meeting was led on the industry side by Brad Glasscock from BioMarin, representing BIO. BIO’s executive vice president for emerging companies, E. Cartier Esham, was also in attendance, as was PhRMA’s Lucy Vereshchagina.
According to the sparse meeting minutes, the parties talked through some specific areas within CBER’s cell and gene therapy work, including patient-focused drug development and looking at novel approaches in cell and gene therapy development. Additionally, the group looked at commitments for expedited programs to develop regenerative medicine therapies.
In addition to ongoing work in the cell and gene therapy area, the CBER working group will discuss commitment language for the inclusion of allergenics in future sessions, according to the minutes.
Pre-market subgroup
The PDUFA VII pre-market subgroup also met in December, continuing a dialog about speeding timely notification of post-marketing requirements during the process for review of the marketing application.
Attended by Esham, Glasscock and other representatives from BIO and PhRMA as well as officials from the Center for Drug Evaluation and Research (CDER) and CBER, the 16 December meeting saw industry asking for FDA timelines to conduct the sufficiency determination for Active Risk Identification and Analysis (ARIA). However, according to the minutes, “FDA noted that inclusion of ARIA timelines in a PDUFA commitment would most likely not be feasible due to variable safety evaluation timelines.” FDA has already agreed to a revision of all relevant internal operating, policy and procedure documents as well as relevant guidance documents. Industry representatives are considering whether they judge that these existing commitments will suffice to meet FDA’s obligations.
A real-world evidence (RWE) pilot is also being considered by the pre-market subgroup. Discussions are reaching the level of detail that had participants discussing which tracking and reporting metrics could be generated from databases already maintained by FDA.
Other topics addressed by the pre-market subgroup include use-related risk analysis (URRA) and human factor protocol review, especially during combination product development and review. With enough resources, FDA representatives said they could develop formal timelines for review of URRAs, with a planned guidance forthcoming. Human factor protocol reviews extending past 60 days are challenging for industry, according to the minutes. Here, though, FDA said that increasingly complex human factor protocol submissions are making the 60-day timelines infeasible to meet; more resources, said the agency, would not overcome this problem.
Looking at innovative approaches to review of submissions, the pre-market subgroup continued a dialog about FDA’s proposed Split Real-Time Application Review (STAR) pilot, bringing the split submission of marketing applications outside of the field of oncology to other therapeutic indications.  Negotiators worked on hashing out such details as a mid-program assessment, metrics and reporting, according to the minutes.
Meeting management was the final topic addressed in the pre-market subgroup’s December meeting. Industry is requesting a guidance and public workshop “to discuss process improvements for advisory committees.” Though FDA is conducting work internally to bolster how the Office of New Drugs prepares for advisory committee meetings, this work is “not focused on making changes to the [advisory committee] process nor would they affect industry’s role” in advisory committee meetings, according to the minutes. FDA is considering whether a public meeting and global look at advisory committee meetings is warranted.
FDA and industry are also looking at formalizing the INTERACT program within CBER, and starting a similar CDER-based program.
Regulatory Decision Tools subgroup
A 15 December meeting of the Regulatory Decision Tools subgroup saw industry representatives meeting with CDER And CBER officials to look at model-informed and patient-focused drug development and complex innovative designs.
With regard to complex innovative designs, FDA and industry worked on edits of a draft document, but also tackled a substantive discussion about how much resources are needed to support FDA’s commitments in this area, with FDA seeing a need for more staffing than industry is currently willing to support.
The meeting then turned to draft commitment language for model-informed drug development, with a discussion that was “somewhat parallel” to the complex innovative designs discussion. FDA expressed concern that timelines be achievable where paired meetings are concerned.
Turning to patient-focused drug development, negotiators discussed the possibility of a draft guidance on patient preference studies. End-of-year pressures prevented FDA from moving forward in considering this possibility, according to the minutes, so future negotiations will address this possibility.
CBER breakout minutes
Premarket subgroup minutes
Regulatory Decision Tools subgroup minutes


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