Setting the record straight on accelerated approval

Is the accelerated approval pathway helping patients achieve better health outcomes or imposing on society medicines that don’t work? In BIO’s view, the pathway provides incredible benefit to patients with serious unmet medical need, and there’s data to back it up.

The pathway relies on surrogate endpoints that can reasonably predict whether a treatment will result in clinical benefit, such as tumor shrinkage or a lab result. For more than 30 years, the pathway has successfully sped up the development of new medicines for people with life-threatening conditions and few treatment options. But that hasn’t stopped critics from spinning misleading studies meant to cast doubt on the pathway.

The most recent example – published in JAMA Network – claims that nearly half of cancer medicines granted accelerated approval lack clinical benefit because they don’t improve overall survival. Recent commentary and new research demonstrate why this assertion is wrong and accelerated approval is worth supporting.

• A “drive-by analysis.” Steve Usdin, Washington editor for BioCentury, takes aim at the JAMA study, calling it “irresponsible” and “intellectual malpractice” for suggesting nearly half of accelerated approved cancer medicines are useless. Usdin points out that a lack of data on whether a drug has extended lives is different than proving it doesn’t extend lives. He also explains the practical – and ethical – challenges of studying overall survival, which is why it’s often not required by the Food and Drug Administration (FDA) to confirm a medicine’s benefit. Other experts – including former FDA official Peter Pitts and Dr. Timothy Franson – were also critical of the JAMA paper for making “sweeping generalizations” that distort the reality of accelerated approval.
• Extending lives for people with cancer. If the authors of the JAMA study think there isn’t data to show accelerated approval extends patients’ lives, a study just published in the Journal of the National Comprehensive Cancer Network proves otherwise. The study looked at 69 cancer therapies that received accelerated approval. Researchers found that across the 69 therapies, between 2006 and 2022, the earlier access to the treatments added roughly 263,000 life years across 911,000 cancer patients. With these benefits in mind, the authors conclude that “any major change to the program should consider the impact on patient outcomes for groups with a high unmet need.”
• Helping disadvantaged communities. A new Avalere analysis found accelerated approval helps Medicare beneficiaries in underserved communities gain access to life-changing medicines. According to the analysis, accelerated approval led to a significant increase in new treatments for patients with blood cancers, other cancers, and iron overload disorders. The pathway is providing new treatments for people disproportionately impacted by certain life-threatening conditions, such as Black and Asian communities impacted by blood disorders, leading to improved outcomes and fewer disparities in care.

The facts clearly show the accelerated approval pathway is providing new hope and meaningful new treatments for patients to fight their disease, and why we shouldn’t be misled by biased studies that distort reality.