• Author
    Andre Naguib Guirguis
  • Discovery PI

    Chandra Smart MD

  • Project Co-Author

    Amanda Ruci; Yvonne Chung

  • Abstract Title

    Patient-Reported Outcomes in Dermatologic Malignancy Clinical Trials: A Systematic Review

  • Discovery AOC Petal or Dual Degree Program

    Basic, Clinical, & Translational Research

  • Abstract

    Keywords: Patient-reported outcomes, Dermatologic oncology, Clinical trial reporting

    Background: Patient-reported outcomes are endpoints that capture the quality of life and disease burden in patients. Despite recent increases in their incorporation, they are inconsistently integrated into clinical trials. There has also been limited standardization in the analysis and reporting of these endpoints.

    Objective: To examine the reporting quality of patient-reported outcomes in clinical trails tied to FDA approved drugs for dermatologic malignancies.

    Methods: A systematic review was conducted of FDA drug approvals for dermatologic malignancies from 2006 to 2025. Each approval was linked to its supporting clinical trial, and associated primary and secondary publications were identified through PubMed. PRO data were extracted from trial protocols and manuscripts. Reporting quality was assessed using a structured scoring framework (PROEAS) informed by international recommendations. Descriptive analyses and exploratory statistical comparisons were performed to evaluate reporting patterns and associations with trial characteristics.

    Results: Among 43 trials supporting FDA approvals, 22/43 (51.2%) reported PRO data, with limited inclusion in primary publications (8/42, 19.0%) and more reliance on secondary, PRO-focused manuscripts (26 total). PROs were commonly designated as secondary (18/34, 52.9%) or exploratory (15/34, 44.1%) endpoints, with no studies including PROs as primary outcomes (0/34). Use of instruments was variable, with general measures such as EORTC QLQ-C30 (25/34, 73.5%) more frequently used than dermatology-specific tools (e.g., Skindex-29: 4/34, 11.8%). Methodological reporting was inconsistent: only 10/26 (38.5%) studies prespecified a missing data strategy, 3/26 (11.5%) defined missingness, and 4/26 (15.4%) adjusted for multiple comparisons despite multiple endpoints. Sensitivity analyses were performed in 7/26 (26.9%) studies. Overall reporting quality was moderate (mean PROEAS score 12.1/24) with no improvement observed over time.

    Conclusions: Despite international reporting initiatives, PRO reporting in dermatologic malignancy trails remains inconsistent. Variations are found in statistical methodology, poor missing data handling, and limited incorporation.

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