Patient-reported outcomes in FDA-approved dermatologic malignancy trials: a systematic review (2006–2025)

Abstract Importance Patient-reported outcomes (PROs) have become central to clinical trials by characterizing changes in symptom burden and quality of life (QOL) in patients with dermatologic malignancies. However, their integration remains inconsistent, and the quality of their reporting is unclear. Objective To evaluate the quality of PRO reporting in clinical trials associated with FDA-approved therapies for dermatologic malignancies and to examine the rigor of PRO methodology and the incorporation of PROs into clinical trials. Evidence review From January 1, 2006, to December 31, 2025, a systematic review was conducted in which FDA drug approval notifications for dermatologic malignancies were identified along with their supporting trials. Manuscripts pertaining to the supporting trials were subsequently retrieved from PubMed. PRO data were extracted from trial protocols and retrieved manuscripts when reported. Manuscripts exclusively reporting PRO data were assessed using the Patient-Reported Outcome Evidence Assessment Score (PROEAS), which was derived from the Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints (SISAQOL) international reporting recommendations. Findings Forty-four approval notifications were linked to 43 unique FDA-registration trials, of which 22 (51%) published PRO data. The mean time to PRO data publication in secondary manuscripts was 26 months after publication of the primary manuscript. PROEAS scoring revealed significant gaps in the incorporation of PRO endpoints into trials, prespecification of statistical frameworks, and transparency in missing data reporting. Heterogeneity was also observed in statistical approaches, with only 12 of 26 (46%) secondary manuscripts meeting the requirement for at least one appropriate statistical test. Additional gaps included limited use of dermatology-specific QOL instruments and sensitivity analyses. Conclusion and relevance Opportunities remain to improve statistical methodology and prespecify frameworks for appropriate and consistent PRO reporting. Current clinical trials supporting FDA-approved therapies for dermatologic malignancies demonstrate substantial gaps in PRO reporting, including limited transparency in the handling of missing data and frequent use of exploratory analyses. These findings highlight opportunities to strengthen the rigor and interpretability of PRO evidence in this disease space.