No political tantrums upstaged the importance of passing PDUFA VI in a timely manner when a House subcommittee met Wednesday to discuss the user fee agreement that the FDA, industry and patient groups have negotiated over the past two years.
Rather, members of the Energy and Commerce Health Subcommittee and witnesses reflected on the consequences of not passing the sixth version of the prescription drug agreement by July, positioning PDUFA VI as a top priority and an enhancement to the subcommittee's 21st Century Cures Act that Congress passed overwhelmingly last year.
If PDUFA isn't reauthorized on time, the implications would be "Titanic in nature," Kay Holcombe, senior vice president of science policy for the Biotechnology Innovation Organization, testified.
Throughout the hearing, PDUFA – the oldest of the FDA user fee agreements – was lauded for halving drug review times and making the agency the global gold standard of drug approvals. But despite its 25 years of success and ardent support from the agency and stakeholders, the agreement faces a fresh challenge as, for the first time, it comes up for renewal in the first year of a new administration.
Witnesses and lawmakers stressed the importance of the agreement, its statutory mandates, the effort put into its negotiation and the support it has from all sectors. The legislators, both Republicans and Democrats, expressed concern as they alluded to President Donald Trump's budget blueprint that suggested an unwinding of the negotiations to shift more of the FDA's financial burden to drug companies. (See BioWorld Today, March 17, 2017.)
Rep. G.K. Butterfield (D-N.C.) said the budget proposal shows a lack of understanding of the good faith effort the FDA and industry put into the negotiations every five years.
Since the user fees are negotiated among stakeholders rather than dictated by Congress or the administration, any move to change the agreement could lead to months, if not years, of new negotiations and congressional hearings. In the meantime, the current agreement will expire Sept. 30. If no new fees have been approved by then, the FDA would be forced to lay off much of its staff, including 70 percent of the review staff at the Center for Drug Evaluation and Research (CDER).
The impact of any hint that PDUFA might not be renewed in time would hit the agency as early as July, when the FDA, by law, would have to start identifying and warning staff about possible layoffs, said CDER Director Janet Woodcock. The loss at CDER would be mostly scientists and doctors who are always being heavily recruited by the private sector.
In the past, the renewal of PDUFA has come down to the wire once, Woodcock said. That time, the agency saw a slowdown in productivity in the months leading up to the deadline, as staff began to pursue other offers because of the uncertainty. Woodcock predicted it could take at least a year for the agency to restore its staffing levels should that happen again.
Noting the impact to CDER staffing, Rep. Fred Upton (R-Mich.), one of the lead sponsors of the 21st Century Cures Act, stressed the urgency of getting the user fee agreement signed into law ahead of the expiration of the current one. "PDUFA VI is very important," he said.
Staffing issues already are a concern at the FDA due to the hiring freeze Trump imposed soon after taking office. The agency is working with the administration to exclude selective hires from the freeze, and Upton indicated he's asked that the FDA be allowed to hire to fulfill its Cures mandates, but he has yet to hear back from the White House on his request.
MEETING NEEDS, ADVANCING CURES
Ironically, PDUFA VI addresses staffing needs at CDER more so than any other iteration of the agreement, providing funding to help the center attract scientists with specific expertise in areas such as patient outcome tools, real-world evidence and rare diseases. The science has come a long way in recent years, Woodcock said, and, in addition to its existing staff, the FDA needs experts with a background in these new areas.
Failure to approve PDUFA VI on time would have ramifications for the agency's device center as well, as the agreement would provide funding to help with its review of drug-device combination products. Rep. Leonard Lance (R-N.J.) said that funding would enhance the recent progress the FDA has made in its review of such products.
"Everyone agrees that it's going better," Woodcock said of the agency's improved coordination of combination product reviews, adding that the products are the future of medical care.
Unlike Tuesday's user fee agreement hearing before the Senate Health, Education, Labor and Pensions Committee, the House subcommittee hearing not only steered clear of the political posturing on efforts to amend parts of the Affordable Care Act, it also avoided questions and speeches about drug pricing. Such comments have become a staple at recent hearings dealing with prescription drugs. (See BioWorld Today, March 22, 2017.)
Instead, the hearing delved into how the agreement would advance the 21st Century Cures Act with its emphasis on patient engagement in every phase of drug development, use of real-world evidence and the qualification of more biomarkers for clinical trials.
The hearing also provided some insight into issues lawmakers may try to address through additions to PDUFA VI, including prohibitions on using risk evaluation and mitigation strategies to block competition, more incentives for drug development for rare diseases and pediatric use, and a possible breakthrough therapy approach to incentivize the development of generics of older drugs that face no competition.
Woodcock cautioned lawmakers about getting carried away with incentives, especially those granting new exclusivities. Reminding them of the Hippocratic Oath – "first, do no harm" – she urged them to consider the unintended consequences of some of their proposals.