After more than two years of negotiations, the Senate has voted almost unanimously to re-authorize the Food and Drug Administration’s (FDA) user-fee programs. These programs, which were due to expire at the end of September, cover prescription drugs, generic drugs, medical devices and biosimilars.

The Prescription Drug User Fee Act (PDUFA), created by Congress in 1992, is the oldest of these user-fee programs and is in its sixth iteration (PDUFA VI). It focuses on pre-market reviews, post-market safety, regulatory tools and the future of drug development.

Why reauthorization matters

For 25 years, PDUFA has allowed the FDA to collect fees from drug companies and use that money to speed up the approval process.  Advocates of PDUFA VI argue it will speed up the pace of medical innovation – and the speed at which patients can be treated. Its critics, however, say with faster approvals comes a greater compromise to medical safety.

The reauthorization of PDUFA will allow the FDA to collect an estimated $9 billion in fees over the next five years – more than a quarter of all FDA funding – in return for processing drug applications. Andrew Powaleny, director of public affairs at industry group PhRMA says “reauthorization of PDUFA is critical to ensuring America’s biopharmaceutical companies can continue scientific innovation and bring new treatments to help patients live longer, healthier lives.”

Before the PDUFA came into effect, delays were almost inevitable. Drug companies relied on the FDA to approve new drugs, but the FDA lacked the resources (both staff and budgetary) to get the job done.

No one expects the drug approval process to be straight-forward, but it is in everyone’s best interests to bring new drugs to market as quickly as possible. For 25 years, PDUFA has allowed the FDA to collect fees from drug companies and use that money to speed up the approval process.

4 key themes of PDUFA VI

PDUFA has helped the US remain at the forefront of regulatory review, pioneering in the development of effective, safe drugs. The Act has already contributed to more than 1,500 new drugs and biologics (including medications for cancer, cardiovascular, neurological, rare, and infectious diseases).

PDUFA VI will be a significant step forward in modernizing the FDA’s review of drugs. It deals with four recurring themes in the drug approval process:

1. Incorporating the patient voice into drug development

By gathering patient input by disease group rather than individual treatment, the FDA hopes to incorporate the patient voice into clinical development. Patients are able to give a unique perspective on their disease and the treatment they receive. This information informs drug development and establishes the context in which regulatory decisions are made.

Related Reading: Benefits of Focusing on Patient-Centered Outcomes

2. Enhancing the use of real-world evidence

Greater use of real-world evidence (RWE) could mean more efficient drug development programs, greater insight into the benefits and risks of new drugs, and quicker patient access to safe drugs. By clarifying the context of RWE through public workshops with stakeholders, the FDA can boost scientific rigor, improve regulatory decision-making, define appropriate use of information, and create a more efficient drug development process.

3. Improving benefit-risk assessments

Benefit-risk assessment is the foundation of the FDA’s drug approval process. The FDA has established a structured approach to benefit-risk assessment in regulatory decision-making. PDUFA VI will take the existing benefit-risk assessment framework, develop it further in the drug review process, and apply it to all regulatory decisions related to drug applications.

4. Expanding post-market safety data

PDFUA VI will continue to support the review, management, monitoring, and communication of post-market drug safety issues. By documenting product safety in the post-market setting, notification will be more timely and consistent.

Keep Reading3 Ways Big Data Affects Biomedical Research


Author: Ryan Clark

Ryan Clark is a strategic client director working on partnerships between CCC and commercial clients. Ryan has been with CCC over five years and has worked in rightsholder relations and business development prior to joining the strategic client group. Ryan enjoys spending time with his family, and following Bruins hockey and Ireland rugby in his time away from work. 
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