TOPLINE:

A cross‑sectional analysis found that few registration trials assessing immune checkpoint inhibitors (ICIs) cancer therapies report long-term overall survival data. Among nearly 90 FDA-approved ICIs indications in the metastatic setting, less than one third of trials had overall survival data at 3 years, and even fewer (about 11%) reported overall survival data at 5 years. 

METHODOLOGY:

• In the US, ICIs are widely used to treat a range of cancer types, but long-term overall survival benefit remains less clear.
• To assess the long-term benefit of these drugs, researchers identified 88 FDA-approved ICI indications in the metastatic cancer setting between 2011 and 2023, and conducted a cross-sectional analysis of long-term survival among patients taking ICIs, using data from recent supporting registration trials with the longest follow-up.
• Investigators analyzed the percentage of participants surviving at 12-, 24-, 36-, and 60-months follow-up intervals, and applied the American Society of Clinical Oncology (ASCO) Value Framework Tail of the Curve to help capture the long-term survival benefit. Achieving a tail-of-the-curve bonus indicated that the therapy was associated with a long-term or durable survival benefit.
• Of the 88 approved indications, most (61.4%) were for ICI monotherapy; the rest were combination therapies: 17% were indicated in combination with chemotherapy, 14% with another monoclonal antibody, and 5.7% with a TKI.

TAKEAWAY:

• Among trials evaluating the 88 approved ICI indications, only 22.7% (n = 20) of studies had data that qualified for ASCO’s tail-of-the-curve bonus, while 30.7% (n = 27) did not report overall survival.
• Half of approved indications had data on overall survival at 24 months, 32% had overall survival data at 36 months, and only about 11% had overall survival data at 60 months.
• Long-term overall survival benefit was limited to a few cancer types — most notably, non-small cell lung cancer (NSCLC) and melanoma.
• By contrast, trials on other cancer types, such as hepatobiliary and cervical cancers, had short follow-up or small survival differences between the intervention and control arms.

IN PRACTICE:

“Long-term benefit, in terms of sustained survival (5 or more years), which is the most desirable outcome for cancer therapy, was only able to be determined in just over 10% of trials,” the authors wrote.

SOURCE:

The study, led by Alyson Haslam, PhD, University of California San Francisco, was published online in Journal of Cancer Policy.

LIMITATIONS:

Long-term survivorship in oncology trials may have been underestimated. The maximal time with ≥ 10% of patients at risk was likely underestimated because the study relied on numbers at risk extracted from published Kaplan‑Meier curves, which omitted many timepoints and reported at widely spaced intervals.

DISCLOSURES:

The study was funded by Arnold Ventures. One author disclosed receiving research funding from Arnold Ventures through a grant made to University of California San Francisco and royalties from Johns Hopkins Press, MedPage, and the Free Press. He reported serving in consultancy roles with UnitedHealthcare and OptumRx, hosting podcasts, writing newsletters, and running a YouTube channel that generates revenue. No other disclosures were reported.

This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication.