Impact of FDA Accelerated Approval in Oncology

 I'm Dr Maurie Markman, from City of Hope. I want to briefly discuss a very interesting, provocative, and timely article that recently appeared in the Journal of the National Comprehensive Cancer Network: "Life Years Gained From the FDA Accelerated Approval Program in Oncology: A Portfolio Model."

First off, the authors note that the paper was supported by a pharmaceutical company. Clearly, because we're talking about drugs, drug approvals, and benefits, this is important to recognize. 

That being said, the intent of this manuscript was to provide data to the question of the FDA's Accelerated Approval Program, which was designed and implemented many years ago to accelerate the delivery of drugs to patients — and oncology is in the forefront of this — based on what might be called surrogate models. Randomized phase 3 trials that we use for a survival endpoint, as important as that obviously is where possible, may delay for years a drug that might be a benefit for patients.

The idea would be a surrogate endpoint for what the FDA has called clinical benefit. Again, this process has been available for a long time; many of the drugs that are approved now in the oncology sphere are based on surrogate endpoints. 

A number of commentators have stated that the process is flawed and that drugs are being approved without evidence of clinical benefit. In fact, they go on to say that we don't know if there actually is any clinical benefit, or certainly clinical benefit based upon improvement in overall survival. 

What is the counterpoint to this? Is there a counterpoint? Again, the FDA, very appropriately, is approving, in my opinion, more drugs. As to the question of surrogacy, I think we're seeing a clinical benefit. Nevertheless, what about data? This particular analysis attempted to provide such data. 

They looked at oncology products that had been approved over a period of time. They state that the program gained — this is in the abstract — approximately 263,000 life-years across 69 products for which overall survival data were available for approximately 911,000 patients with cancer. This suggests that the process has benefited a large number of patients, improving their survival, with many products across many diseases.

Again, we're not talking about randomized controlled trial survival data here. We are talking about one way of looking at the impact of the FDA's Accelerated Approval Program which, in my opinion, has been incredibly important for our patients. 

For those of you interested in this topic, and certainly for those of you who've read the counterpoint to this saying that the program has not been as successful as perhaps it should be, I think this is an interesting counterpoint to that. I encourage you to read this very interesting paper. Thank you for your attention.

https://www.medscape.com/viewarticle/assessing-impact-fda-accelerated-approval-oncology-2024a1000g6w