MedicalResearch.com Interview with:
Jeremy O’Connor, MD
Section of General Internal Medicine
Department of Internal Medicine
Postdoctoral Fellow, National Clinician Scholars Program
MedicalResearch.com: What is the background for this study?
Response: There has been a lot of enthusiasm for the use of novel therapies in cancer care, and in particular for novel anticancer agents known as immune checkpoint inhibitors. But very little is known about how quickly providers have adopted immune checkpoint inhibitors into clinical practice. Existing studies suggest, in fact, that the process of clinical adoption is slow, with conventional wisdom holding that it takes an average of 17 years for new evidence to change practice.
Our study evaluated whether the adoption of novel therapies might be much faster in certain contexts with the early use of immune checkpoint inhibitors as a notable example.
MedicalResearch.com: What are the main findings?
Response: There were two main findings in our study.
First, we found rapid adoption of immune checkpoint inhibitors among eligible patients across 3 cancer types. Instead of it taking years for the drugs to reach the majority of eligible patients, we found that it took less than 4 months. This is great news for patients who have had rapid access to drugs that are safe and effective in certain contexts.
Second, we found that patients receiving checkpoint inhibitors in clinical practice tended to be substantially older than patients receiving the drugs in the pivotal clinical trials. This suggests that checkpoint inhibitors are rapidly changing practice on the basis of evidence from trial participants that are not representative of the patients who are treated in actual practice. This raises concerns about the degree to which the clinical outcomes following treatment will be different in the real world vs in the pivotal clinical trials.
MedicalResearch.com: What should readers take away from your report?
Response: We know that clinicians, in some settings, can adopt novel treatments very quickly. But in some cases, the initial FDA approvals are granted on the basis of relatively small studies that offer a limited understanding of risks and benefits. That is why we need to align the rapid adoption with rigorous and prompt studies ofnovel drugs after they enter the market. We should think of a new approval as the beginning of a period of evaluation of whether it works in the real world setting.
Such evaluations have greater relevance during periods when the rates of adoption are fast. This is because rapid adoption may lead to many patients being exposed to a new drug at a time when there is a high level of uncertainty about its risks and benefits. It is encouraging that much of the recent data about the use of checkpoint inhibitors confirm the positive early findings from clinical trials. But in the future, we might not be so fortunate.
Another key point is that we need to move away from conceptualizing FDA approval as a singular stamp of approval for widespread clinical use. As more and more agents are evaluated through accelerated pathways, it’s important for clinicians to consider the body of evidence supporting these new agents as still in development. Post-marketing studies, using larger samples and more real-world patients, may show the agents to be beneficial — or in some cases, not beneficial. It’s not reasonable to consider an agent to be “experimental” and then overnight have it be considered “proven standard of care”. Science doesn’t work that way.
MedicalResearch.com: What recommendations do you have for future research as a result of this work?
Response: In addition to what is stated above, it is very important for the FDA and others to continue to encourage the enrollment of more representative populations of patients into clinical trials. This is a problem that we have known about for some time, but our study suggests that we have made little of any progress towards improving the degree to which the clinical trials are representative of the patients with cancer who are receiving the newest types of therapies in clinical practice.
O’Connor JM, Fessele KL, Steiner J, et al. Speed of Adoption of Immune Checkpoint Inhibitors of Programmed Cell Death 1 Protein and Comparison of Patient Ages in Clinical Practice vs Pivotal Clinical Trials. JAMA Oncol. Published online May 10, 2018. doi:10.1001/jamaoncol.2018.0798
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