The Industry Hopes Congress Approves PDUFA Program in 2016

Pharma and biotech companies worked hard this past year to reach agreement with FDA this month on a new version of the Prescription Drug User Fee Act (PDUFA VI); now they’d like Congress to approve the next five-year fee program before leaving Washington at the end of the year. The aim is to reauthorize the program well before its September 2017 expiration date to avoid extensive delay if a new administration and new Congress take over. Broad and controversial measures for expanding and revising multiple FDA programs and policies await further consideration by Congress, and stakeholders fear that the usual strategy of linking PDUFA to such legislation will put off action on the must-pass PDUFA program.

The PDUFA VI performance goals letter, published July 15, 2016, makes some changes in how quickly FDA will review market applications and supplements for drugs and biologics and provide advice and assistance during the R&D process for new medicines. The Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER) highlight efforts to improve communication with sponsors and provide more consultation during product development. FDA specifies where a lack of data or analyses may delay the review of an application, with a particular emphasis on the importance of sponsors listing all drug manufacturing facilities that may need to be inspected prior to approval of an application or supplement.

Key initiatives are to better coordinate regulation of combination drugs, biologics, and medical devices; provide more assistance in vetting orphan drugs and treatments for rare diseases; utilize real-world evidence more broadly and support new initiatives to enhance drug development; authorize more complex and innovative clinical trial designs; and enhance postmarket safety evaluation.

The proposed new fee structure, as outlined in a Federal Register notice of July 19, 2016 aims to limit fee growth while also providing FDA with a more reliable funding stream. A new “program” fee that replaces current establishment and product fees will account for 80% of fee revenues, while application fees, which often vary from year to year, will total only 20% of fees (down from one-third of the total). There will be no fees for supplemental approvals to encourage sponsors to propose expanded and more informative product labels and to improve operations.

A prominent goal is to improve FDA’s ability to hire and retain talented employees. To this end, the program provides an additional $67 million to support 230 additional FDA staffers; they will provide added resources to manage the growing breakthrough therapy program, biomarker qualification initiatives, and efforts to further incorporate patient preferences in the development and review of new treatments.

FDA has scheduled a public meeting for August 15 at its White Oak campus to hear stakeholder views on the 46-page PDUFA proposal. While the usual plan is to send a final PDUFA letter to Congress in January, along with similar fee proposals for medical devices, generic drugs, biosimilars, and other regulated products supported by user fees, stakeholders are looking to move up the timeframe to gain Congressional action before the Obama administration steps down.