A new analysis indicates that head and neck cancer trials are frequently terminated prematurely, primarily due to sponsor decisions related to safety or effectiveness, and insufficient patient recruitment. Researchers from Sylvester Comprehensive Cancer Center, part of the University of Miami Miller School of Medicine, and collaborators conducted a retrospective study of 692 clinical trials launched between 2000 and 2024.
Key Findings
- Of the trials analyzed, 346 ended early (terminated or withdrawn).
- The most common reasons for early termination were strategic decisions by sponsors (29.5%) and poor patient recruitment (26%).
- Early-phase trials, especially those testing immunotherapies or targeted therapies, were more likely to fail due to sponsor-driven decisions.
- Later-phase trials involving chemotherapy, radiation, or combination treatments frequently experienced difficulties with enrollment.
- Industry-sponsored trials were nearly three times more likely to fail compared to government-funded studies.
- Academic and network-funded trials showed better outcomes, attributed to a focus on patient recruitment strategies and broader eligibility criteria.
- Trials with higher participant numbers demonstrated a greater likelihood of completion, while restrictive eligibility criteria and logistical challenges often led to termination.
Elizabeth Franzmann, M.D., professor of otolaryngology and director of head and neck research at the Miller School, noted that understanding trial failure is crucial for designing successful studies. First author Janice Huang highlighted that recruitment challenges reflect real patient barriers and suggested digital outreach could improve access. Alex Reznik, a co-author, emphasized that the study focused on early trial termination rather than just not meeting primary endpoints.
Implications
Clinical trial failures contribute to delays in patient progress and increased costs, with new cancer drug development potentially exceeding $2 billion. The study, published January 2, 2026, in JAMA Otolaryngology – Head & Neck Surgery, also found that failure rates have risen over the past two decades. Proposed solutions to these barriers include decentralized trials, adaptive protocols, and nurse-led navigation.