PDUFA VI: Highlights on What to Expect

The US Food and Drug Administration (FDA) and stakeholders from industry and patient groups on Monday outlined the agreement they forged on the next reauthorization of the Prescription Drug User Fee Act (PDUFA) that begins in 2017, focusing on what’s to come on pre-market reviews, postmarket safety, regulatory decision tools and other ways FDA is preparing for the future of drug development.

The meeting follows the July release of the full details of the performance and procedural goals for fiscal years 2018 through 2022. The 46-page document offers some perspective on the direction FDA is headed for PDUFA VI in terms of incorporating the patient voice into drug development, enhancing the use of real-world evidence, improving benefit-risk assessments and expanding postmarket safety data and evaluations.

At the outset of Monday’s meeting, Janet Woodcock, director of FDA’s Center for Drug Evaluation and Research (CDER), highlighted the importance of enhancing patient input and integrating it into regulatory decision making, particularly on how to make their needs known and ensure they are met in the regulatory process.

Woodock also noted that fit for purpose tools to collect meaningful patient input on what really matters need to be addressed, in addition to what burden of disease means to patients and what different adverse events mean to them.

As far as real-world data, Woodcock said she believes everyone agrees that it can be a major source for improving the understanding of how drugs are used and their effects, though “the question is how to do it.”

Theresa Mullen, director of CDER’s Office of Strategic Programs, responded to concerns raised about FDA’s dissemination of guidance documents and the slow process by which they are finalized, noting FDA only has a handful of employees who have the expertise to write such documents, and a lot of these officials can be lured away from FDA by better paid industry jobs with more flexible schedules.

What’s Coming

Innovative clinical trial designs (particularly with regard to computerized simulations) is an area where sponsors that might have been previously hesitant to submit a unique idea to FDA can now make such a proposal and expect to hear feedback.

“Advancing Model-Informed Drug Development” is another hot topic likely to see much more depth from FDA in the coming five years, particularly as a new pilot project aims to gauge industry's interest.

New proposals on new molecular entity (NME) reviews are also expected to increase FDA's flexibility and ensure certain changes on NMEs “filed in protest” are codified:

Meanwhile, FDA and stakeholders took care to craft new types of meetings between pharmaceutical companies and FDA in PDUFA VI, as FDA receives thousands of meeting requests annually and both sides might not have enough time for internal deliberations.

Early consultations on new surrogate endpoints is also a major area of interest for FDA, particularly as FDA has to ensure it has the expertise to keep up with industry innovation. Other issues are outlined below:

Likewise, creating a qualification pathway for the use of new biomarkers is another major area drug companies will be keen to hear more details on, particularly as a public website from FDA is in the works on a list of biomarker qualification submissions.

In terms of postmarket safety, FDA is planning to improve processes and IT systems that track such data, as well as looking to integrate new sources of data into its Sentinel initiative, which is no longer in its pilot phase:

PDUFA VI: Fiscal Years 2018 - 2022