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BioCentury – Congress risks long-term damage to the FDA

BioCentury – Congress risks long-term damage to the FDA

As Congress returns from its annual August recess the familiar September task of reconciling the Federal budget before the end of the fiscal year is at hand. This year, however, during a compressed legislative session, a dire public health risk hangs in jeopardy. It is critical for Congress to complete the five-year reauthorization process of FDA’s user fee programs before they lapse at the end of the month, to avoid delays in patient access to new medicines and long-term detriment to FDA.

Predictable, consistent and robust user fee programs are a key component to efficient operation of the FDA. These funds allow some of the agency’s most important programs to function, including recently established programs designed to expedite review and access to new therapies.

If the user fee programs including the Prescription Drug User Fee Act (PDUFA) expire without reauthorization, it will not only hinder optimal functioning of FDA but grind certain activities to a halt.

In the short term, it will impede programs that enable efficient review and approval of medicines, delaying patient access to new therapies. In the long term, FDA will face massive personnel layoffs and be left hamstrung in hiring the necessary experts to fulfill the agency’s mission.

The impact of the expiration will be serious, affecting the lives of millions of U.S. citizens, slowing scientific progress, and fundamentally altering FDA for years to come.

Ahead of the reauthorization of PDUFA VII, Friends of Cancer Research developed an interactive dashboard to assess how provisions included in past PDUFA agreements have emerged as important tools to support timely development and regulatory decision-making.

Between 2013 and 2022, 98% (133/136) of original oncology drugs were approved on or ahead of their PDUFA goal date, indicating that the regulatory timelines established through PDUFA enable timely and predictable access to safe and effective new therapies.

Past reauthorizations have also established new policies and programs that expedited development and review of products that treat serious illness and alleviate unmet medical needs. Drugs using these expedited programs were approved in a median of 6.9 months compared with 11.6 months for traditional approvals. One hundred percent of the first-in-class oncology approvals granted since 2013 came out of an expedited program.

Since 2013, more than half of new oncology products received breakthrough therapy designation, which has been shown to shorten average development times by nearly three years — this has led to improvements in clinical outcomes.

PDUFA VII presents the opportunity to advance new programs and policies that enhance existing mechanisms to support efficient development and review of new products.

For example, a pilot program created by FDA’s Oncology Center of Excellence called Real-Time Oncology Review streamlines cancer product reviews by allowing applications to be submitted in parts. This enables reviews to begin in “real time” as sections are being completed. Since the program was expanded to original applications, Real-Time Oncology Review has helped streamline the 16 original oncology approvals and shortened review timelines to 6.4 months. This pilot program represents a new mechanism that can be implemented across the agency to optimize interactions with sponsors and review processes. Passage of PDUFA VII would establish a program similar to Real-Time Oncology Review, the Split Real-Time Application Review Pilot Program, that would make these benefits available outside of oncology.

Among the many new important policies proposed in the current user fee bills that have advanced through the House and the Senate Committee on Health, Education, Labor, and Pensions are provisions to enhance FDA’s accelerated approval pathway. This pathway has by and large been a success at providing new medicines to severely ill patients, in some cases years sooner. The bills currently under consideration would provide additional tools to ensure post-market requirements are completed in a reasonable timeframe. Our dashboard shows that while most accelerated approvals in oncology are converted or withdrawn within 3.2 years after approval, 30 have been on the market for greater than 3.2 years without fulfilling their post-market requirements. The challenge of these “dangling” accelerated approvals could be addressed through the enhancements proposed in the current bills.

Past improvements to FDA processes made possible through PDUFA reauthorizations have enabled access to the next generation of therapies and contributed to improved survival outcomes at the population level. Authorizing PDUFA VII is essential to continue building on these successes and to avoid disruptions in FDA activity that could delay access to life-saving therapies for patients with cancer and other serious diseases.

All who care about innovation, science and patients should feel the urgency of full passage of PDUFA and let Congress know that short-term extensions will result in long-term consequences.

We urge Congress to act swiftly and decisively in the coming weeks to protect PDUFA and the millions of U.S. citizens it impacts.

Jeff Allen is president and CEO of Friends of Cancer Research (Friends), an advocacy organization in Washington, D.C.