04 May Cancer Drug Approval: FDA Acceptance of Surrogate End Points
MedicalResearch.com Interview with:
Emerson Y. Chen, MD
Assistant Professor of Medicine, Medical Oncology
Knight Cancer Institute, Oregon Health & Science University
Portland, OR 97239
MedicalResearch.com: What is the background for this study?
Response: Our research group had previously studied how oncology drugs are approved in these two previous papers listed below. One is focused on the time delay trade-off from surrogate endpoints (i.e. response rate and progression-free survival) over definite endpoints (i.e. overall survival and quality of life). The other is focused on how promising the response rate of a drug candidate have to be to be considered for oncology drug approval.
MedicalResearch.com: What are the main findings?
Response: Because of these two works, we decided to look earlier into the 1990s and 2000s to see at what point we transitioned from use of definite endpoints to more convenient endpoints like response rate and progression-free survival. And we found that, more and more, the regulatory agency has favored the use of convenient endpoints to allow new drugs to be approved in the United States. Such practice is common across many cancer types.
MedicalResearch.com: What should readers take away from your report?
Response: Many people have mistakenly think that the FDA takes too long or is too strict in making a decision about new drug candidates. We actually show that the FDA accepts convenient endpoints in many cancer types in this article. While we recognize many patients with cancer urgently need to go onto the next-best treatment, it is also unfair to recommend any therapy with little or uncertain benefit to patients. Their regulatory decisions have a lasting medical, financial, and societal impact. And we maintain that a well-structured assessment of a cancer drug’s benefit either upfront prior to approval or verified shortly after accelerated approval is important in the oncology drug approval process.
MedicalResearch.com: What recommendations do you have for future research as a result of this work?
Response: All of our studies suggest that use of randomized controlled trials as study design and clear assessment of quantity and quality of life either upfront or shorting after accelerated drug approval are ways to ensure our oncology drug evaluation process is truly valid. We hope that more investigators and drug developers will be encouraged to use high-quality trial designs. We also hope drug regulators would be more willing to recommend and enforce these guidelines for future oncology trials.
MedicalResearch.com: Is there anything else you would like to add?
Response: We hope more researchers would be interested in such healthy policy questions, and we encourage others to scrutinize the publicly available documents available from the FDA and make their own interpretations and opinions.
Chen EY, Haslam A, Prasad V. FDA Acceptance of Surrogate End Points for Cancer Drug Approval: 1992-2019. JAMA Intern Med. Published online April 27, 2020. doi:10.1001/jamainternmed.2020.1097
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