Kicking off the PDUFA VI Reauthorization Process

By: Theresa M. Mullin, Ph.D.

The Prescription Drug User Fee Act (PDUFA) authorizes FDA to collect fees from pharmaceutical companies to help fund the agency’s drug review work. PDUFA’s intent is to provide additional funding for FDA to hire staff, improve systems, and establish a better-managed review process that enables us to do more timely reviews of human drug applications.

Theresa MullinAs a result, many important new drug therapies have been made available to patients sooner without compromising FDA’s high standards for safety, efficacy, and quality.

As part of FDA’s agreement with industry during each reauthorization of the Act, the agency agrees to certain performance goals to enhance the process of drug review. The goals, now 30 in total, apply to many review processes, including the review of original new drug applications, resubmissions, and supplemental applications. Since the first user fee law was passed in 1992, PDUFA has been reauthorized four times. The current legislation, PDUFA V, is set to expire in September 2017.

On July 15, 2015, FDA gathered stakeholder perspectives during a meeting on what features the agency should propose in the reauthorization of PDUFA for fiscal years 2018 – 2022. Attendees included patient advocates, consumer advocates, representatives of professional health care associations, biopharmaceutical industry representatives, academic researchers, policy analysts, and others. FDA received mostly positive feedback on our progress under PDUFA V and helpful input about the future for PDUFA VI.

Highlights of progress noted during this meeting include:

  • FDA continues to meet or exceed most review goals.
  • The program is experiencing high rates of approvals for novel products during their first submission. This includes a historically high number of approvals for novel products treating rare diseases (17 orphan drug approvals in 2014).
  • There are improved and increased communication functions and practices between FDA and new drug companies, or sponsors. This includes implementation of a structured risk-benefit framework within the review process.
  • The Patient-Focused Drug Development program has been successful in systematically obtaining patient perspectives on certain diseases and related treatments.

Meeting participants also contributed a number of ideas for further enhancements, including:

  • Further efforts to involve the patient perspective in drug development processes;
  • Building on FDA’s Sentinel System for active surveillance of safety issues for medical products, including expanding its use as a source of data; and
  • Enhancing regulatory science initiatives, including the use of patient-reported outcomes and biomarkers.

More detailed information about the meeting is available at PDUFA Meetings, which includes a webcast of the one-day meeting, the agenda, access to the docket for online public comments, and (soon to follow) a complete written transcript of the public meeting’s proceedings.

PDUFA was designed to enable FDA to fulfill its mission to protect and promote public health by helping to more effectively bring to market critical new medicines for patients. FDA is strongly committed to the components, enhancements, and initiatives that constitute this program and have made it so successful. The public feedback received during the July 2015 meeting indicates that we are on the right track in achieving our goals under PDUFA V, and FDA looks forward to PDUFA VI.

Theresa M. Mullin, Ph.D., is Director of FDA’s Office of Strategic Programs in the Center for Drug Evaluation and Research

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