Therapeutic innovation & regulatory science · 2026
Patient-Reported Outcomes Used for Regulatory Approval of Medicines Indicated for the Treatment of Dermatological Diseases Authorized in the European Union Between 1998 and 2024.
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Coenen Eléonore, Almeida Diogo, Sepodes Bruno, Torre Carla
Аннотация
BACKGROUND: Patient-reported outcomes (PROs) have gained increasing relevance in medicine approvals, particularly in dermatology, where traditional clinical and laboratory endpoints may not fully capture the patient's experience or treatment impact. This study aimed to assess the use of PROs in the European Commission's (EC) marketing authorizations for medicines indicated for dermatological diseases between 1998 and 2024.
METHODS: The European Public Assessment Reports (EPARs) and Summary of Product Characteristics (SmPCs) of approved medicines indicated for dermatological diseases approved between 1998 and 2024 were reviewed. For medicines including PROs, detailed information regarding their characteristics, applications and PRO label claims was collected.
RESULTS: Of the 21 medicines approved by the EC between 1998 and 2024, 14 (66.7%) incorporated PROs, and 9 (64.3%) of those received a PRO label claim. Across the 64 studies included in the EPARs, 45 distinct PRO Measures (PROMs) were identified. The most common purpose of PROMs was to capture signs/symptoms (n = 15; 33.3%). PRO use was identified in 314 instances, of which 114 (36.3%) were categorized as PRO-related secondary endpoints. Five (35.7%) medicines were not granted PRO label claims due to a lack of benefit, missing data, limited PROM validation, small sample size, or study design issues.
CONCLUSIONS: PRO use in dermatology regulatory submissions is evident and plays a valuable role in conveying the patient perspective. Overcoming operational and methodological challenges, ensuring stakeholder alignment, and striking a balance between instrument diversity and utility could strengthen the integration of PROs into patient-centric regulatory decision-making.
METHODS: The European Public Assessment Reports (EPARs) and Summary of Product Characteristics (SmPCs) of approved medicines indicated for dermatological diseases approved between 1998 and 2024 were reviewed. For medicines including PROs, detailed information regarding their characteristics, applications and PRO label claims was collected.
RESULTS: Of the 21 medicines approved by the EC between 1998 and 2024, 14 (66.7%) incorporated PROs, and 9 (64.3%) of those received a PRO label claim. Across the 64 studies included in the EPARs, 45 distinct PRO Measures (PROMs) were identified. The most common purpose of PROMs was to capture signs/symptoms (n = 15; 33.3%). PRO use was identified in 314 instances, of which 114 (36.3%) were categorized as PRO-related secondary endpoints. Five (35.7%) medicines were not granted PRO label claims due to a lack of benefit, missing data, limited PROM validation, small sample size, or study design issues.
CONCLUSIONS: PRO use in dermatology regulatory submissions is evident and plays a valuable role in conveying the patient perspective. Overcoming operational and methodological challenges, ensuring stakeholder alignment, and striking a balance between instrument diversity and utility could strengthen the integration of PROs into patient-centric regulatory decision-making.