Why So Many Head and Neck Cancer Clinical Trials Fail
Article Summary
- Sylvester Comprehensive Cancer Center research shows a significant fraction of head and neck cancer trials fail, most often due to strategic decisions or poor recruitment.
- Industry-funded trials face a higher risk of failure. Academic funding and larger enrollment improve the odds of success.
- Two University of Miami Miller School of Medicine students contributed to the study and said recruitment challenges are a significant factor in clinical trial success.
Head and neck cancer ranks as the seventh most common cancer worldwide, and its incidence continues to rise. In the United States alone, 2025 projections estimate more than 72,000 new cases and 16,000 deaths. More than 90% of these cancers are head and neck squamous cell carcinomas, which originate in the mucosal lining of the oral cavity, pharynx and larynx.
While immunotherapy and precision chemoradiation have improved outcomes for some patients, survival rates for recurrent or metastatic head and neck squamous cell carcinomas remain dismal. Clinical trials are essential to finding better treatments, but many fail before completion.
A Troubling Trend for Head and Neck Cancer Clinical Trials
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, an M.D./Ph.D. student at the Miller School and co-author of the study, 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The study included 346 studies that ended early, either terminated or were withdrawn.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,” said Elizabeth Franzmann, M.D., a professor of otolaryngology—head and neck surgery and director of head and neck research at the Miller School and co-author of the study.
Funding Matters
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 of the study, Janice Huang, an M.D./Ph.D. student at the Miller School.
Clinical Significance
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—the clinical efficacy required to demonstrate the success of the intervention of interest,” said Reznik. “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.”
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 Dr. Franzmann.
Tags: cancer research, cancer screening, clinical trials, Dr. Elizabeth Franzmann, head and neck cancers, Newsroom, Sylvester Comprehensive Cancer Center