The rapid adoption of new cancer immunotherapies raises some concerns.
A recent study conducted by Yale University researchers using data collected by Flatiron Health, an oncology software and services company that develops real-world datasets from a national network of oncology clinics and academic research centers, found that cancer immunotherapies based on checkpoint inhibitors are being used by the majority of eligible patients within under four months of their approval by the US Food and Drug Administration (FDA).
This timeframe is much shorter than for most newly approved drugs. The researchers looked at patients being treated for melanoma, non-small cell lung cancer (NSCLC) and renal cell carcinoma. Immune checkpoint inhibitors have been shown to have the potential to extend the lives of terminal cancer patients with no treatment options.
However, often the clinical studies on which the approvals of checkpoint inhibitors are based only enroll a limited number of patients, include no comparison data or are conducted in younger and healthier patients than the patients receiving the drugs in practice. These differences are a cause for concern, according to the researchers, because they suggest that clinical research is disconnected from practical medicine.
For instance, clinical studies generally enroll patient in their late 50s to early 60s, while most patients receiving checkpoint inhibitors are 65 or older. In addition, in some cases drugs approved based on early clinical trials are found in later studies to be less effective or potentially harmful.
“This is why it's so important for the FDA to use strong evidence for its approvals and for trials to test drugs among patients who are similar to those being treated in everyday practice,” says Jeremy O'Connor, M.D., first author and a postdoctoral fellow at Yale School of Medicine (YSM).
Adds Cary Gross, M.D., YSM professor of medicine and a member of the Yale Cancer Center: "Our findings underscore the importance of a thorough FDA review of new cancer therapies. We found that the standard of care can change virtually overnight when a new drug is brought to market. But … it is essential that we think of FDA approval as one step in the continuing evaluation of whether new drugs are indeed beneficial in the larger population."