FDA Ponders Trimming PDUFA Programs

Acting Commissioner Ostroff considers whether all aspects of the user fee program are delivering maximum added value.

By FDA’s own admission, its Prescription Drug User Fee Act program could be somewhat bloated and require rebalancing.

Acting Commissioner Stephen Ostroff said during opening remarks at a July 15 public meeting on the prescription drug user fee reauthorization that the program now includes 30 measurable review and procedural goals as well as other commitments like guidance development and public meetings.

Ostroff said PDUFA might require a review to determine whether all fee-supported programs are delivering the maximum return on investment.

Formal PDUFA VI negotiations with industry are expected to begin in the fall (Also see "PDUFA VI: Price Tag For Patient Experience Data Validation May Go Up" - Pink Sheet, 18 Jun, 2015.).

While building on past success, FDA “should also examine whether aspects of the program should be scaled back, whether they can be modified, or whether they can be discontinued to be able to better focus on those things that are of the most value-added,” he said.

The agency did not offer examples of programs that could be cut or adjusted. “It is the beginning of the process,” a spokesperson said.

Ostroff also did not suggest fees might be lowered.

But it is notable FDA could enter negotiations wanting to refocus the PDUFA program on a smaller number of topics.

Ostroff may want to communicate to negotiators as well as stakeholders that even though fee revenue increases each year, it still is a limited resource.

In addition to renewing the traditional fee programs, the PDUFA V agreement made in 2011 included other items such as increasing staff for rare disease development, adding patient views to benefit-risk decision-making and creating a dedicated meta-analysis team (Also see "PDUFA V: Final Recommendations Fund Proposals In Two Of Three Tiers" - Pink Sheet, 2 May, 2011.).

PDUFA V also made changes to the new molecular entity and biologic license application review systems, allowing more time for a filing review and adding mid- and late-cycle communications between the agency and drug application sponsors (Also see "Buying Time: Industry Sacrifices Early To Gain Later With PDUFA V Review Model" - Pink Sheet, 1 Oct, 2012.).

A number of other topics were not scheduled for funding under PDUFA V, but FDA at the time said it would use existing resources to pay for them, including better planning for optimum dose selection, improving human subject protection and ensuring the quality of non-inferiority and adaptive trial designs (Also see "FDA's Un-(User Fee) Funded Priorities" - Pink Sheet, 13 Jul, 2011.).

Are Priorities Shifting?

It is possible a number of these topics or others, while all laudable, may no longer be immediate priorities.

FDA also may have determined that the cost of its primary function, drug review, may require more user fees and treasury dollars to be the most effective.

Programs like the breakthrough therapy designation, which technically are not part of the PDUFA agreement per se, remain connected to it because they affect product reviews.

Breakthrough, which was included in the 2012 FDA Safety and Innovation Act along with the last PDUFA reauthorization, has been determined to require many more resources than previously thought.

FDA has received many more breakthrough requests and granted more designations than expected (Also see "‘Breakthrough Therapy’ Designations" - Pink Sheet, 4 May, 2015.).

The idea of a breakthrough designation application fee has been proposed in part to deter frivolous requests for access to the enhanced FDA involvement that characterizes the pathway (Also see "‘Breakthrough’ Fee Eyed As Deterrent To Frivolous Requests" - Pink Sheet, 4 May, 2015.).

Recognizing Budget Reality

Ostroff’s comments build on his previous remarks that straddle the line between advocating for more funding while recognizing existing resources are adequate to complete the mission.

In one of his first public speeches as acting commissioner, Ostroff said the agency is better resourced than ever, but still warned it needed to keep up with emerging scientific advances (Also see "FDA Staying The Course Under Ostroff; Hamburg’s Concerns Will Remain Focus" - Pink Sheet, 20 Apr, 2015.).

He also has warned that program cuts may be necessary if the 21st Century Cures legislation includes too many unfunded mandates (Also see "FDA Program Cuts Loom If ‘Cures’ Bill Isn’t Fully Funded, Ostroff Warns" - Pink Sheet, 3 Jun, 2015.).

In some ways, FDA could already be carrying out a strategic realignment. In its fiscal 2016 budget request, FDA said it wanted to trim some field operations to focus on its highest priorities (Also see "FDA Field Operations Would Endure Reductions Under Budget" - Pink Sheet, 9 Feb, 2015.).

Steps like that could resonate with lawmakers. The House Appropriations Committee approved a funding plan that would give FDA an increase in fiscal 2016 compared to the current enacted level (Also see "Biosimilar Naming, Interchangeability Policies Must Be Done Soon, House Tells FDA" - Pink Sheet, 8 Jul, 2015.).

Stakeholders Ask For More

In contrast to FDA's tone of potential retrenchment, stakeholder groups spoke almost entirely about additions to the program during the hearing.

A number of groups suggested further enhancing the patient-focused drug development program created in PDUFA V.

With more than 20 disease-specific meetings scheduled or conducted to gather risk tolerance and other comments from patients and advocates, several organizations now want to include the data in product reviews.

Several stakeholders have called for a systematic way to incorporate patient opinions (Also see "Systematizing Patient Views Unlikely, FDA’s Temple Says" - Pink Sheet, 24 Feb, 2014.).

[Editor's note: “The Pink Sheet” also published this article. "The Tan Sheet" brings selected complementary coverage from our sister publications to our subscribers.]