Immuno-Oncology Resource Center
Immuno-Oncology Resource Center
May 10, 2018
2 min read

Immune checkpoint inhibitors reach most patients within 4 months after FDA approval

You've successfully added to your alerts. You will receive an email when new content is published.

Click Here to Manage Email Alerts

We were unable to process your request. Please try again later. If you continue to have this issue please contact

Most patients eligible for immune checkpoint inhibitors received treatment with new drugs within a few months of FDA approval, much sooner than is typical for new treatments, study data showed.

However, patients treated with new drugs in a real-world setting were often older than those who received the drugs in clinical trials.

“There are many reasons to be excited about the rapid adoption of these new treatments,” Jeremy O’Connor, MD, postdoctoral fellow at Yale School of Medicine, said in a press release. “But, it can be risky for rapid adoption to be based on early findings from trials, because drugs might be shown in later trials to be less effective or even 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.”

O’Connor and colleagues performed a retrospective cohort study of 3,089 patients (56.7% men, 66.9% white) from Flatiron Health Network — which consists of 233 academic and community oncology practices — who were eligible for anti-PD-1 treatment for melanoma (n = 555; mean age, 66 years), non-small cell lung cancer (n = 2,159; mean age, 67 years) and renal cell carcinoma (n = 375; mean age, 66 years).

The cumulative proportions of eligible patients who received the treatments and their ages served as the main outcomes.

More than two-thirds of patients (68.7%; n = 2,123) received anti-PD-1 treatment. These included 439 (79.1%) eligible patients with melanoma, 1,417 (65.6%) eligible patients with NSCLC and 276 (71.2%) eligible patients with renal cell carcinoma.

More than 60% of patients in each cohort received anti-PD-1 treatment within 4 months of the drugs’ FDA approval.

Researchers observed rapid uptake of pembrolizumab (Keytruda, Merck) for melanoma in the first 3 months of approval, with 70.4% of eligible patients receiving the treatment. However, during the next 12 months, nivolumab (Opdivo, Bristol-Myers Squibb) surpassed pembrolizumab as the treatment of choice for melanoma.

Researchers also reported rapid adoption of nivolumab, but not pembrolizumab, for NSCLC.

Similar proportions of older and younger patients received anti-PD-1 treatments within 9 months of FDA approval.

Cary Gross

However, more patients aged 65 years and older received the drugs in clinical practice (range, 60.6%-63.9%) than in pivotal clinical trials (range, 31.7%-41%; P < .001 for all).

“Our findings underscore the importance of a thorough FDA review of new cancer therapies,” Cary Gross, MD, professor at Yale School of Medicine, member of Yale Cancer Center and a co-author of the study, said in the press release. “We found that the standard of care can change virtually overnight when a new drug is brought to market. But, because these initial FDA approvals are frequently based on studies that may be small, have no comparison arm, or are restricted to younger and healthier patients, it is essential that we think of FDA approval as one step in the continuing evaluation of whether new drugs are indeed beneficial to the larger population.” – by Andy Polhamus

Disclosures: O’Connor reports no relevant financial disclosures. Gross reports research support from 21st Century Oncology, Johnson & Johnson, Medtronic Inc. and Pfizer. Please see the study for all other authors’ relevant financial disclosures.

More than 60% of patients in each cohort received anti-PD-1 treatment within 4 months of the drug's FDA approval.