Study Advises Caution When Setting Expectations for Novel Cancer Drugs Interview with:
Alyson Haslam, PhD
Nutritional Epidemiologist
Center for Indigenous Health Research and Policy
Oklahoma State University What is the background for this study?

Response: Checkpoint inhibitor drugs for the treatment of cancers have received a lot of attention in recent years because of their ability to induce responses in certain tumors.

To quantify the eligibility and response of these drugs in the US population, we published an article about a year ago ( Since that publication, there were several confirmatory studies that failed to show a benefit in important outcomes such as overall survival or progression-free survival, and the US FDA made some revisions to certain checkpoint inhibitor drug labels. This prompted us to re-evaluate the eligibility of these drugs. What are the main findings?

Response: We found that because of label changes and failure to improve other important endpoints for urothelial, gastric, and hepatocellular cancers, about 9% of patients in the US with cancer were exposed to checkpoint inhibitor drugs that failed to show improvement in confirmatory studies. While we did see an improvement in eligibility to checkpoint inhibitor drugs for triple negative breast cancer, the overall eligibility in checkpoint inhibitor drugs decreased from 43.6% in our prior estimates to as low as 36.1% in our current estimates. What should readers take away from your report?

Response: The take away is that we need to be cautious about our expectations for novel drugs in oncology.  While we do not want good drugs to be withheld from patients because of lengthy trials and approval processes, we also want to make sure that the drugs that are approved have actual benefit, in terms of the number of patients who are both eligible and have response to them. What recommendations do you have for future research as a result of this work?

Response: Future research should evaluate ways of maximizing the benefit of checkpoint inhibitor drugs by identifying individuals who are most likely to respond, and therefore refine who should really be eligible for these drugs. 

I have no disclosures to report.


Haslam A, Gill J, Prasad V. Estimation of the Percentage of US Patients With Cancer Who Are Eligible for Immune Checkpoint Inhibitor Drugs. JAMA Netw Open. 2020;3(3):e200423. doi:10.1001/jamanetworkopen.2020.0423



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Mar 13, 2020 @ 3:45 pm




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