Study Finds Rapid Uptake of Anti–PD-1 Agents Following FDA Approval

Published on: 

In the 4 months following their approvals, pembrolizumab and nivolumab reached more than 60% of eligible patients with melanoma, non-small cell lung cancer, and renal cell carcinoma.

Following their approval, anti—PD-1 agents reach a significant proportion of patients within a few months, according to new study findings.

Anticancer agents, including anti—PD-1 agents, continue to enter the market at an accelerated rate through the breakthrough designation. While traditional estimates suggest it often takes more than 10 years for innoations to change patient care, little is known about how quickly novel therapies reach the hands of patients.

To answer this question, researchers conducted a retrospective cohort study of patients receiving nivolumab or pembrolizumab for previously treated or untreated melanoma, nivolumab or pembrolizumab for previously treated non—small cell lung cancer (NSCLC), or nivolumab for previously treated renal cell carcinoma (RCC) from January 1, 2011 through August 31, 2016. The results were published in JAMA Oncology.


Of the 3089 patients eligible for anti—PD-1 treatment, 555 had melanoma, 2159 had NSCLC, and 375 had RCC. The median age at diagnosis of advanced stage disease for patients with melanoma and RCC was 66 and for patients with NSCLC was 67.

More than 60% of patients in each cohort received anti—PD-1 treatment within 4 months of the FDA approval. By the end of the study period, 439 patients with melanoma (79.1%), 1417 patients with NSCLC (65.6%), and 267 patients with RCC (71.2%) had received anti–PD-1 treatment.

The authors observed rapid uptake of pembrolizumab for melanoma in the first 3 months following FDA approval, with 31 of 44 patients receiving the treatment. However, in the following 12 months, nivolumab became the preferred anti—PD-1 treatment for melanoma. In NSCLC, there was rapid uptake of nivolumab, but not pembrolizumab.

“Such rapid adoption stands in contrast to older estimates that suggest it takes years or even decades for new treatments to be adopted, including evidence that highly effective treatments, such as tamoxifen for breast cancer, can take more than 10 years to reach most eligible patients,” wrote the authors.

The swift uptake of these treatments may be attributable to high disease severity, overall preference for novelty, perceived gains over existing treatments, and promotional activities, such as direct to consumer advertisements, they explained.

Of note, there were larger proportions of patients treated with anti—PD-1 drugs aged 65 and older in real-world cohorts than in pivotal trials. However, with up to 9 months of followup, the uptake of each treatment was similar among younger and older patients, which suggests that patient age is unlikely to be associated with differences in clinical adoption of the treatments, according to the authors.


O'Connor J, Fessele K, 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. doi:10.1001/jamaoncol.2018.0798.