Trump Administration Abandoning User Fee Agreements
FDA testimony delivered by CDER director Janet Woodcock during a 3/22 House Energy and Commerce Committee health subcommittee hearing on reauthorizing the Prescription Drug User Fee Act (PDUFA 6) indicated that the Trump Administration does not stand behind the agency- and industry-negotiated user fee agreements and that new agreements should be re-negotiated to double fees as outlined in last week’s budget blueprint. Immediately opposed by FDA constituencies, the 3/16-released fiscal year 2018 budget blueprint would boost user fees on regulated industry’s medical product marketing applications to about $2 billion annually. Such a jump “replaces the need for new budget authority to cover pre-market review costs,” the budget plan said. “In a constrained budget environment, industries that benefit from FDA’s approval can and should pay for their share.”
In Woodcock’s official testimony, the Trump Administration noted that the reauthorization proposal that was submitted to Congress 12/2016 under the previous Administration “reflects a different approach to the federal budget. The Blueprint Budget supports many of the goals of the reauthorization proposal but proposes a different way of financing these goals. The Administration looks forward to working with Congress, with industry input, to develop a reauthorization proposal that speeds the development and approval of vital drugs and biologics that are safe and effective.”
Rep. Gene Green (D-TX) said he was disappointed with the Administration’s stance to not support the current drug user fee proposal, which was the “result of months of work between FDA and stakeholders to examine the program, figure out what is working and what can work better, and come to an agreement on how the program should be for the next five years through a public, drawn out process. This progress is a long one, and the statutory deadline for reauthorization is coming up quickly,” he said.
Also expressing budget concerns, Friends of Cancer Research president Jeff Allen said during the hearing that “holding the FDA budget authority at stagnant levels prevents progress on agency functions that are not applicable to user fees. These include critical functions of the agency such as drug-safety surveillance programs, oversight of drug compounding facilities, review of product advertising material, oversight of over-the-counter medicines and conduct of scientific programming. Compounded with the constraints of the federal hiring freeze, the FDA will be hamstrung. The ramifications will be felt for years to come... Finally, for the programs of this proposed user fee agreement to succeed, the full budget for the FDA must be robust, and the capacity at which the agency can maintain and hire the best scientific minds must be unencumbered.”
During questioning, Green asked Woodcock what would happen if Congress failed to reauthorize the user fee program by 9/30. Woodcock said if not reauthorized, the agency would have to initiate a “reduction in force process where we would prepare to let go 70% of the staff working on the reviews of human drugs.” She noted that CDER would need to begin working on that in July because there are “complicated personnel rules that have to do with who has to be notified first and so forth.”
Health subcommittee chairman Michael Burgess (R-TX) said that the user fee reauthorization legislation will be “shepherded” through the committee and onto the House floor for a vote with ample time to spare before the current fee programs expire 9/30.
In her opening remarks, Woodcock reviewed enhancements that PDUFA 6 currently contains, including a provision on advancing clinical trial designs, “which is something dear to my heart.” The next user fee agreement “promises to encourage future efforts by advancing Model-Informed Drug Development (MIDD) and the use of complex innovative and adaptive clinical trial designs,” her testimony said. “The development and application of exposure-based, biological, and statistical models derived from preclinical and clinical data sources can be used to inform regulatory decision making, for example, in determining patient selection in clinical trials, individualized dosing for specific populations, or the need for post-marketing studies.”
To advance these approaches during PDUFA 6, FDA proposes to convene a series of workshops to identify best practices for MIDD, to conduct a pilot program, to develop guidance, and to update policies and procedures, as appropriate, to incorporate guidelines for the evaluation of MIDD approaches, Woodcock said in her testimony. “To facilitate the advancement and use of complex adaptive, Bayesian, and other novel clinical trial designs during PDUFA VI, FDA proposes to convene a public workshop on complex innovative trial designs, publish guidance on complex innovative trial designs, to conduct a pilot program, and to update policies and procedures as appropriate to incorporate guidelines on evaluating complex innovative trial designs.”
Responding to a question by Rep. Morgan Griffith (R-VA) about FDA struggling with exclusivity decisions, Woodcock said that many of the laws on exclusivity that Congress passed long ago did not foresee some of the current situations. “We struggle all the time with trying to figure out how to apply exclusivity fairly and justly and yet not disadvantage public health goals that we may have,” she said. Asked whether new legislation around exclusivity is needed, Woodcock declined to answer directly, but hinted it would help because “times have changed.”