TOPLINE:

A retrospective analysis found that strategic decisions and recruitment challenges were leading contributors to head and neck squamous cell carcinoma trial failures, accounting for 29.5% and 26.0% of cases, respectively.

METHODOLOGY:

• Clinical trials evaluating therapies for head and neck squamous cell carcinoma have high failure rates, though factors driving this trend remain unclear.
• To better understand this issue, researchers conducted a retrospective cross-sectional case control study of interventional clinical trials for treating head and neck squamous cell carcinoma, comparing completed and failed trials.
• The analysis included 692 matched clinical trials, identified on ClinicalTrials.gov between January 1, 2000, and December 31, 2024. Half were completed control trials, and half were trial failures (277 terminated and 69 withdrawn).
• Researchers compared trial characteristics between failed and completed trials, with trial failures defined as early termination or withdrawal.

TAKEAWAY:

• The main reasons for trial failures were strategic decisions in 29.5% (n = 102), defined as nonscientific, sponsor-driven choices, as well as poor recruitment in 26% of trials (n = 90).
• Failure trends varied by trial stage. Strategic decisions were the leading reason for trail failures in phase 1 studies, accounting for 42.3% of failures, whereas poor recruitment was the chief reason for later-stage trial failures, accounting for 30% of phase 2 failures, 22% of phase 3 failures, and 100% of phase 4 failures.
• Strategic decisions were also the leading cause for immunotherapy trials (54.8%) and targeted therapy trial (27.4%).
• Compared with government-funded trials, industry-sponsored trials were more likely to be terminated (odds ratio [OR], 2.84), as were trials involving a procedure or surgery (OR, 1.98). In contrast, trials that included a drug, device, biologic/vaccine, or radiation therapy had lower odds of being terminated.

IN PRACTICE:

Clinical trials evaluating therapies for head and neck squamous cell carcinoma “were terminated early or withdrawn for a variety of reasons, most commonly due to strategic decisions or poor recruitment,” the authors wrote, adding that strategically assessing trial characteristics associated with failure could help improve failure rates.

SOURCE:

The study, led by Janice J. Huang, MS, and Alex S. Reznik, Medical Scientist Training Program, University of Miami Miller School of Medicine, was published online in JAMA Otolaryngology-Head & Neck Surgery.

LIMITATIONS:

The analysis was limited by publicly accessible data and inconsistent reporting of reasons for failure of trials. The analysis also did not account for site-level or protocol-specific factors.

DISCLOSURES:

The study was supported by the Medical Scientist Training Program at the University of Miami Miller School of Medicine through a National Institutes of Health grant. Sonis reported owning stock in InnoPath Therapeutics and Vigilant Laboratories, receiving nonfinancial support from Aveta Biomics, and having a patent for early detection test licensed to Vigilant Laboratories. Villa reported receiving grants from Merck, Lipella, Mureva, and MeiraGTx, as well as personal fees from Primary Endpoint Solutions, WSK Medical, Lipella, Amgen, and K Pharmaceuticals.

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