Is the Rapid Uptake of Immunotherapies Sacrificing Patient Safety?

In a study published in JAMA Oncology, researchers found that a majority of patients eligible for immune checkpoint inhibitors started to receive the treatment within a few months of the drugs approval. The study also found that cancer immunotherapies are adopted as treatments at a faster rate than has traditionally been seen for newly approved medical treatments.

In a collaboration with Yale researchers and investigators from Flatiron Health, study authors looked to evaluate the speed in which programmed death 1 (PD-1) inhibitors reach eligible patients and sought to compare the ages of patients treated in clinical practice with the ages of those treated in clinical trials.

Another study found breakthrough therapies might need more rigorous evaluation.

A total of 3089 patients (1742 men and 1347 women) were enrolled in the study. The mean age of patients was 66 for melanoma and renal cell carcinoma, and 67 for non—small cell lung cancer (NSCLC). The results found that 68.7% of patients received anti-PD-1 treatment within 4 months of FDA approval. These rates were notably high for those with melanoma (79.1%), followed by renal cell carcinoma (71.2%) and NSCLC (65.6%).

“There are many reasons to be excited about the rapid adoption of these new treatments,” Jeremy O’Connor, MD, lead author and postdoctoral fellow at Yale School of Medicine, said in a statement. “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.”

Additionally, the majority of patients treated with checkpoint inhibitors in clinical practice were aged 65 years and older. Conversely, patients treated in trials had median ages that ranged from late 50s to early 60s. The researchers noted that recognizing patients in clinical trials differ from those treated in routine clinical practice emphasized the importance of studying real-world populations.

“We found that the standard of care can change virtually overnight, when a new drug is brought to market,” said Cary Gross, MD, senior author and Yale School of Medicine professor of medicine in a statement. “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 1 step in the continuing evaluation of whether new drugs are indeed beneficial in the larger population."

Reference

O'Connor JM, Fessele KL, Steiner J, et al. Speed of adoption of immune checkpoint inhibitors of programmed cell deth 1 protein and comparison of patient ages in clinical practice vs pivotal clinical trials. JAMA Oncol. 2018;4(8):e180798. doi:10.1001/jamaoncol.2018.0798.