Setting the record straight on accelerated approval

Andrew Powaleny
Andrew Powaleny May 22, 2023

Setting the record straight on accelerated approval.

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As we’ve discussed on this blog before, the U.S. Food and Drug Administration’s (FDA) accelerated approval program has served as a critical lifeline for patients with serious and life-threatening conditions. The program was originally established at the urging of patient advocates during the height of the HIV/AIDS epidemic, in which patients waited years for the FDA to review and approve new treatments. From its inception three decades ago, the pathway’s purpose has always been to get medicines to patients most in need, especially those who lack any viable treatment options. Without accelerated approval, patients in need would have fewer or no treatment options.

Accelerated approval can be based on a surrogate endpoint which we’ve previously written about. A surrogate endpoint is a measure, such as laboratory measurement, that is expected to predict clinical benefit. When treating patients with life-threatening diagnoses, researchers may use surrogate endpoints in clinical trials when demonstrating a clinical outcome might take a long time to study, or when the severity or duration of a disease course would make extended or large studies unethical or impossible. This saves valuable time for patients with serious and life-threatening diseases without other treatment options, making new medicines available sooner.

One common misconception is medicines receiving accelerated approval are merely “conditional” and are not as safe and effective as medicines receiving traditional approval. This is not true.

Medicines with accelerated approval must adhere to the same standards for establishing safety and effectiveness as medicines receiving traditional approval. This includes requiring sponsors to provide substantial evidence of effectiveness based on “adequate” and “well-controlled” clinical investigations. Congress itself has made clear through statute accelerated approval does not alter the standards of evidence required for a new medicine to be approved.

To see how impactful the pathway is for patients, look no further than rare diseases.

From 1992 through about 2010, rare disease medicines made up 24.7% of the medicines with accelerated approval. Rare diseases are among some of the most difficult diseases to study for a range of factors, which include disease complexity, lack of timely diagnosis, and small or geographically dispersed patient populations that make trial recruitment and retainment a challenge. As such, rare diseases remain an area of significant unmet medical need with over 90% of the known rare diseases having no approved treatment option available. These factors combined make accelerated approval a critical tool for rare disease patients in urgent need of innovative medicines.

Policies that would penalize medicines with accelerated approval by imposing additional rebates or making other changes to reimbursement could mean that patients would have to wait an average of 3.2 years longer to access these innovative therapies. For patients with serious or life-threatening diseases, that is time they don’t have to waste. Since its creation, accelerated approval has been a resounding success in expediting patient access to lifesaving medicines and addressing unmet medical needs. Let’s make sure we don’t do anything to slow that progress.

To learn more about the importance of the accelerated approval pathway, click here.

Topics: FDA, Rare Diseases, Accelerated Approval