Trends in Otolaryngology Clinical Trials and Their Alignment with Clinical Practice

Introduction: Evaluating the scope of otolaryngology research activity in relation to disease prevalence in clinical practice may help identify evidence gaps and inform strategies to enhance the relevance and impact of future research.

Methods: Clinical trial data was extracted from ClinicalTrials.gov using MeSH and non-MeSH disease condition terms for studies submitted from 1999 to 2025. Studies were subcategorized for descriptive analysis into the following groups: otology, rhinology, allergy, sleep, head and neck cancer, thyroid, facial, and mouth. To characterize conditions and interventions encountered in routine clinical practice, the most frequent diagnoses and procedures were identified using ICD-9 and ICD-10 codes queried from the AAO-HNSF Reg-ENT Registry. These data were analyzed to evaluate the extent to which research activity reflects real-world clinical care patterns.

Results: A subset of 1,969 otolaryngology-related clinical trials submitted between 1999 and 2025 was selected from an initial cohort of 28,218 studies for a snapshot analysis. Otology and neurotology represented the majority of studies (1,160), followed by mixed or general ENT studies (549). Sleep medicine and rhinology accounted for a smaller proportion of trials, while laryngology/airway and head and neck oncology were least represented. Although the overall number of registered ENT clinical trials increased over time, the relative distribution across condition categories remained uneven, with limited evidence of representation of real-world clinical encounter patterns.

Conclusions: Gaps exist between the focus of clinical trials and the relative distribution of conditions encountered in everyday practice. Expanding this snapshot study to the full cohort of trials will further elucidate gaps and trends of importance to patients, physicians, funders and policy makers.