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The Food and Drug Administration’s approval of the Alzheimer’s drug Aduhelm has sparked a broader debate on the merits and limitations of the FDA’s accelerated approval program. This debate — and its timing — are important.

The FDA issued its accelerated approval regulation nearly 30 years ago, and has since applied this approach for more than 200 new drug indications. This program allows for faster approval of drugs for severe conditions based on markers predicting clinical benefit, on the rationale that patients with no alternatives need options sooner, not later.

Writing in STAT, pharmaceutical CEO Emil Kakkis described how the program transformed HIV/AIDS from a death sentence into a more manageable condition and how it offers a promising road map for rare neurological diseases. As I show here, accelerated approval has also transformed cancer care.


Because this program has had a dramatic impact in several disease areas and holds promise for others, Congress should strengthen it next year, during the program’s 30th anniversary.

Over the years, the accelerated approval program has been questioned by some who prefer to see a drug’s impact on long-term clinical outcomes, such as overall survival, before it is approved and available to patients. While extending survival and improving patient quality of life is the goal of cancer treatments, the impact on intermediate endpoints such as tumor shrinkage may be a clear indicator of effect and measuring overall survival could unjustifiably delay access to new therapies.


Accelerated approval undoubtedly provides earlier access to therapies for individual patients. But it can also provide a significant benefit on the population level. For the first time in history, recent studies show an overall improvement in lung cancer survival due to the availability of new therapies, the majority of which have been approved via the accelerated approval pathway. Without accelerated approval, these lifesaving effects would still be years in the making with thousands of patients deprived of treatment.

Congress should clearly recognize the totality of what the accelerated approval program has achieved. It has not, of course, been perfect. That’s because the program was designed to be rigorous and flexible, not perfect. If the surrogate is persuasive, keep working on any residual uncertainty but don’t make patients wait. With this explicit policy choice in mind, lawmakers now have the opportunity to strengthen the accelerated approval program for the next 30 years.

As a first step, Congress should use its oversight powers to conduct a gap analysis: Does Congress still believe in the fundamental objective of the accelerated approval program, which is earlier approval of drugs that treat serious conditions and fill unmet needs based on surrogate endpoints? If so, how well is the program achieving these objectives?

Congress should then develop constructive, bipartisan fixes. There will be considerable overlap across academic and consumer groups, patient and industry groups, and the FDA itself concerning two buckets of emerging reforms. The first bucket is improvements to pathway mechanics such as front-loading confirmatory study design to help ensure that studies will be feasible and illuminating, or modernizing the process for withdrawing an approval when the evidence doesn’t support its continued use. The second bucket is program management enhancements to promote appropriate consistency, flexibility, and transparency for accelerated approval across disease areas and FDA organizational units.

The good news is that the House and Senate committees that write FDA laws have a strong track record of developing constructive, bipartisan FDA reforms. In FDA legislation, it’s often feasible to find a sweet spot between competing objectives. There aren’t a lot of us-against-them political issues where each side digs in and gets stuck. Accelerated approval, in fact, is not a partisan issue — optimizing the means to provide timely access to medicines for people with limited options while safeguarding their safety and continuing to robustly monitor the effects of these medicines is surely a goal everyone can agree on.

The timing is auspicious. The FDA’s drug, generic drug, biosimilar, and device user fee programs expire in 2022. Congress must reauthorize these programs in order for FDA reviewers to keep working and ensure the availability of safe and effective treatments in years to come. Because the reauthorizing legislation is considered “must pass,” it is a natural vehicle for other FDA legislative reforms.

Keith Flanagan served as senior health counsel on the U.S. Senate’s Health, Education, Labor, and Pensions (HELP) Committee; founded the offices of new and generic drug policy in the FDA’s Center for Drug Evaluation and Research; and now consults for patient groups, biopharmaceutical innovators, and investors.

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