In a Viewpoint article published online this week in JAMA, researchers from McGill University in Montreal and the Memorial Sloan Kettering Cancer Center in New York caution that researchers, physicians, and regulators may be overestimating the value of many new drugs because they fail to consider all the evidence available to them.
“Drug companies often run dozens of trials for a new drug against different diseases,” says McGill bioethicist Jonathan Kimmelman, lead author of the JAMA article. “Some of those trials will suggest efficacy by chance alone. Unless you adjust for the effects of chance by considering all the trials a drug developer has run, doctors who pick out a single trial that shows a big treatment effect may be making bad treatment decisions.”
The viewpoint is especially relevant for physicians who treat patients with serious illnesses and have exhausted proven treatment options. Often, such patients are given drugs for which there is only a small amount of trial evidence suggesting usefulness.
Kimmelman and co-authors also argue that, if physicians and researchers considered all trials run for a new drug, patients in trials could be better protected from harms.
“When deciding whether to use a drug against a disease, physicians are used to looking at evidence testing the same exact drug against the same disease, says Kimmelman. “That makes sense if you have a lot of good evidence. Our point – which is probably surprising for many- is that if you lack definitive evidence of efficacy, physicians and others need to consider trials testing the drug against other diseases.”
The article is co-authored by McGill doctoral student Benjamin Carlisle and Mithat Gönen, chief of Memorial Sloan Kettering’s biostatistics service.
To read the full article: http://jamanetwork.com/journals/jama/fullarticle/2652428
“Drug Development at the Portfolio Level Is Important for Policy, Care Decisions and Human
Protections,” Jonathan Kimmelman, Benjamin Carlisle, Mithat Gönen, JAMA, published online Aug. 24, 2017.