Analysis reveals why head and neck cancer clinical trials end early

Head and neck cancer trials are frequently derailed before they can deliver answers. A new analysis suggests that the most common reasons are sponsor decisions related to safety or effectiveness and poor patient recruitment.

Researchers from Sylvester Comprehensive Cancer Center, part of the University of Miami Miller School of Medicine, and collaborators analyzed 692 clinical trials launched between 2000 and 2024. Alex Reznik, M.D./Ph.D. student at the University of Miami and co-author, described the study as "A retrospective query of head and neck cancer clinical trials in which we compared trial characteristics between failed clinical trials and completed clinical trials."

The findings, published Jan. 2, 2026, in JAMA Otolaryngology – Head & Neck Surgery, included 346 studies that ended early, either terminated or were withdrawn. The leading culprits?

• Strategic decisions by sponsors, often unrelated to safety or efficacy (29.5%)
• Poor patient recruitment (26%)

Early-phase trials and those testing immunotherapies or targeted therapies were more likely to fail because of sponsor-driven decisions. Later-phase trials investigating chemotherapy, radiation or combination treatments struggled with enrollment.

Understanding why trials fail is the first step toward designing studies that succeed. Every failure represents lost time for patients who urgently need better options." 

Elizabeth Franzmann, M.D., professor of otolaryngology and director of head and neck research at the Miller School and co-author of the study

Industry-sponsored trials were nearly three times more likely to fail compared with government-funded studies. Academic and network-funded trials fared better, mainly because they prioritized patient recruitment strategies and broader eligibility criteria.

Enrollment size also played a critical role. Trials with higher participant numbers were significantly more likely to reach completion. Conversely, restrictive eligibility criteria and logistical hurdles often doomed studies before they could deliver results.

"Beyond the science, study design plays a critical role in trial success, especially recruitment and eligibility. Recruitment challenges are not just statistics-they reflect real barriers for patients, and digital outreach can help improve access," said first author, Janice Huang, an M.D./Ph.D. student at the Miller School.

Clinical trial failure isn't just a research setback-it delays progress for patients and drives up costs. Developing a new cancer drug can cost more than $2 billion, and each terminated trial adds inefficiency to an already complex system. "Often, we think about clinical trial failure as not reaching primary endpoints (e.g. the clinical efficacy required to demonstrate the success of the intervention of interest), however, we decided to investigate the understudied and clinically significant pattern of early trial termination – studies that don't even make it to the finish line," said Reznik.

The study also found that failure rates have climbed steadily over the past two decades, underscoring the need for innovation in trial design. Solutions such as decentralized trials, adaptive protocols and nurse-led navigation could help overcome these barriers.

"Clinical trials are how hope becomes care," said Franzmann.