PDUFA VII: FDA, industry preview their reauthorization wish lists
At its first public meeting in the runup to the reauthorization of the Prescription Drug User Fee Act, the FDA, industry and other stakeholders on Thursday shed light on their goals for what will be the agency’s seventh PDUFA program.
The meeting, held fully virtually for the first time due to the COVID-19 pandemic, is the starting point for the negotiations with industry and discussions with stakeholders that will shape FDA’s new drug review program from FY2023-FY2027.
Going from VI to VII
FDA commissioner Stephen Hahn opened the meeting by touting the successes of the current PDUFA program. “Since FY2016, nearly five years after our previous meeting, CDER and CBER have approved over 150 new molecular entities, new drug applications and biologics license applications,” Hahn said, nearly half of which have been for orphan indications.
Hahn added that in the most recent fiscal year, FDA “reached a new high for priority applications filed — 72 — and total applications filed — 166 — and again are on track to meet or exceed most of our review performance goals.”
Outside of the review goals, Hahn said FDA has published more than 30 draft or revised guidances, held more than 20 public meetings and workshops and published nine public reports as part of its PDUFA VI commitments.
Andrew Kish, director, Office of Program and Strategic Analysis at the Center for Drug Evaluation and Research (CDER) gave an overview of the previous PDUFA programs and noted the modernized user fee structure in the latest agreement.
“PDUVA VI modernized the user fee structure to improve program funding predictability, stability and administrative efficiency. The new structure eliminated the supplement fees, replaced establishment and product fees with the program fee, which shifted a greater proportion of the target revenue to the new, more predictable, stable, annual program fee,” Kish said.
Kish also pointed out that user fees have accounted for an increasing portion of funding for FDA’s new drug review program. “User fee revenue has outpaced budget authority available for the program. As a point of comparison, PDUFA user fee revenue funded 7% of the program in 1993 and as of 2019 it funds 71% of the program,” he said.
For PDUFA VII, Kish laid out FDA’s four overarching priorities for the program: promote sustainable innovation in drug development; enhance regulatory predictability and postmarket safety; advance the regulatory infrastructure for digital technologies and new sources of data; and enhance operational capabilities, efficiency and agility.
Industry wish list
Representatives from both the Pharmaceutical Research and Manufacturers of America (PhRMA) and the Biotechnology Innovation Organization (BIO) presented their respective industries’ goals for the next user fee agreement that will be hashed out in meetings with FDA over the coming months.
Both Lucy Vereshchagina, vice president of science and regulatory advocacy at PhRMA, and Cartier Esham, executive vice president, emerging companies at BIO, stressed that PDUFA VII should glean from lessons learned during the COVID-19 pandemic.
“There is a pressing need for FDA, and industry, to identify actions taken during the COVID-19 pandemic and evaluate their effectiveness and applicability to innovative drug development beyond the public health emergency,” Vereshchagina said.
The pharmaceutical industry group would also like to see “More predictable and timely engagement and better communication during drug development,” Vereshchagina said, noting the group’s desire for more risk-based inspections and enhanced technological infrastructure at the agency.
Vereshchagina also said that, “PhRMA would support the establishment of a flexible and scalable global framework for digital technology development,” building on experience with a shift to telemedicine and digital health technologies during the pandemic.
Additionally, Vereshchagina stressed that PhRMA would like to see greater support for real world evidence (RWE) in FDA’s decision-making. “PhRMA believes that real world evidence, for example, can be used for demonstrating effectiveness either on its own in some circumstances or in combination with other data,” she said.
Speaking for BIO, Esham said the group has three main objectives, on top of its “evergreen position” supporting the agency’s ability to hire and retain “world-class personnel.”
Those objectives, Esham said, are to “continue to optimize the current program and processes; to ensure science-based and effective postapproval requirements and activities; and third, to develop new initiatives that will best prepare us for the future.”
For PDUFA VII, Esham said BIO would like to see more funding going to the Center for Biologics Evaluation and Research (CBER) to accommodate the increased workload around cell and gene therapies.
“With these products comes increased demand on CBER resources, including expert personnel, additional meetings and engagement with FDA,” Esham said.
BIO would also like further clarification on the evidentiary standards for FDA’s expedited pathways such as the agency’s new regenerative medicine advanced therapy (RMAT) designation, Esham said. FDA has granted more than 50 RMAT designations since the designation’s creation under the 21st Century Cures Act, but has yet to approve a single RMAT-designated product.
Esham also called for the establishment of processes and best practices to improve the efficiency and effectiveness of meetings between sponsors and FDA. “Best practices can ensure FDA-sponsor interactions are as efficient as possible and if properly done, could serve to decrease the amount of meeting requests,” she said. While FDA has generally met most of its PDUFA review performance goals over the last decade, it has fared less well with its meeting and communications goals.
Additionally, BIO would like to see FDA establish new mechanisms and timelines for communication with industry, such as providing an opportunity for sponsors to ask clarifying questions after milestone meetings and setting structured timelines for communication for labeling, postmarket requirement (PMR) and postmarket commitment (PMC) processes and pediatric study plans.
Esham added that it will be critically important to “continue to advance modern manufacturing quality and inspections methodology and processes,” and called for risk-based approaches to preapproval inspections.
RAPS: First published in Regulatory Focus™ by the Regulatory Affairs Professionals Society, the largest global organization of and for those involved with the regulation of healthcare products. Click here for more information.