Do Accelerated Approvals Really Benefit Patients? With Ravi Parikh, MD, MPP

Recently, Ravi Parikh, MD, MPP, an Assistant Professor of Medical Ethics and Health Policy and Medicine at the University of Pennsylvania, Perelman School of Medicine, released a research letter titled, "Exposure to US Cancer Drugs With Lack of Confirmed Benefit After US Food and Drug Administration Accelerated Approval." In this interview with OncData, he further discusses his research and gives a thought-provoking analysis of what these findings could mean for the future of the accelerated approval program, as well as how the multidisciplinary team could better advocate and let their voices be heard for more quality clinical trials.

Oncology Data Advisor: Welcome to Oncology Data Advisor, an expert resource for the multidisciplinary cancer team. Today, I am joined by Dr. Ravi Parikh, an Assistant Professor in the Department of Medical Ethics and Health Policy and Medicine at the University of Pennsylvania, to talk about his research letter regarding lack of benefit from accelerated approvals and negative confirmatory trial data.

Thank you for joining us today, Dr. Parikh. Would you like to introduce yourself and tell us a bit about your interests?

Ravi Parikh, MD, MPP: Sure. I'm Ravi Parikh. I'm a Medical Oncologist and a Health Policy and Clinical Researcher at the University of Pennsylvania. A lot of my research focuses on applications of real-world data to understand oncology prescribing patterns and particularly how we can encourage high-value utilization of drugs and discourage low-value utilization of drugs in clinical practice.

Oncology Data Advisor: Incredible. Again, great to have you here. So, we are here today to talk about your research regarding the lack of benefit of accelerated approvals from the FDA. Would you like to give us a brief overview of your research?

Dr. Parikh: Sure. So, the FDA accelerated approval program, which began many years ago, is intended to speed up access to novel therapies that show promise in non-randomized, largely phase 2 trials and accelerate market access to those drugs prior to them undergoing standard of care confirmatory phase 3 testing in comparison to a standard of care. This program has been a good thing; it's been associated with the entry of drugs into the market earlier, and usually, those drugs are confirmed to have benefit in phase 3 testing. So, it really is earlier access to drugs that can be beneficial to patients—and I've seen this in my own clinical practice.

One of the issues that's come up with the accelerated approval program is that about a quarter of indications that are approved in the oncology setting are subsequently withdrawn, meaning that they don't show benefit in confirmatory phase 3 trials. This is, in some ways, a problem, because it exposes patients to drugs that may not be beneficial over standard of care and may, in fact, be either harmful in comparison to a standard of care, or pose adverse events and pose cost to the health care system. Trying to redesign the accelerated approval program so that it can still maintain its benefits of speeding up access to effective drugs, while figuring out ways to discourage access to ineffective drugs, is a big policy interest of the FDA, of federal legislators, and to our research group.