Patient-Reported Outcomes Used for Regulatory Approval of Oncology Medicinal Products in the European Union Between 2021 and 2024: A Review Update
Author(s)
Maria Manuel Teixeira, MSc1, Diogo Manuel Monteiro, PharmD2, Fábio Cardoso Borges, PharmD1, Bruno Sepodes, PharmD MSc PhD MPH2, Madeline Pe, PhD1, Carla Torre, PharmD MSc PhD2.
1EORTC, Brussels, Belgium, 2Faculdade de Farmácia, Universidade de Lisboa, Portugal, Lisbon, Portugal.
1EORTC, Brussels, Belgium, 2Faculdade de Farmácia, Universidade de Lisboa, Portugal, Lisbon, Portugal.
OBJECTIVES: Building on a study of patient-reported outcomes (PROs) on the European Union (EU) regulatory approvals (2017-2020), this study aims to characterise the implementation, assessment, and translation of PRO data into labelling claims of subsequent approvals of oncological medicinal products between 2021-2024.
METHODS: A retrospective review of the oncology medicinal products and indications (including line extensions) approved on the EU following a positive opinion from the EMA between 2021-2024 was conducted. European Public Assessment Reports (EPAR) and Summary of Product Characteristics (SmPC) were reviewed for each medicine to identify PROs use and PRO label claims.
RESULTS: A total of 134 oncology indications were approved, and 111 (82.8%) included PROs in the confirmatory clinical trials. Of these, 31 indications were supported by randomized double-blinded trials, 79 by open-label trials (22 of which were single-arm trials), and 1 by both. PROs were defined as a secondary endpoint in 56 studies (49.6%), exploratory in 27 (23.9%) and as both in 27 (23.9%). This variable is unknown for 3 (2.6%) studies. The selected PRO measures (PROMs) relied on the EORTC (43.9%), FACIT (8.6%) and EQ-5D (27.6%) instruments. Of the 66 different PROMs identified, 50 (75.7%) were oncology-specific, 7 of which were symptom-specific. PROs reviewer’s comments were included in the EPAR for 80 indications (59.7%), however only 1 (0.7%) included a Health-related quality of life claim in the SmPC. Reasons for excluding PRO claims were identified for 54 indications (67.5%).
CONCLUSIONS: Despite increased PRO inclusion in confirmatory trials, translating this evidence into label claims remains challenging. Although EPARs appear to provide more detailed PRO data, label claims continue to be granted cautiously. The main reasons identified are related to the exploratory nature of the data and its limited methodological robustness, limitations in the trial design and conduct, missing data, selection bias and limited clinical relevance.
METHODS: A retrospective review of the oncology medicinal products and indications (including line extensions) approved on the EU following a positive opinion from the EMA between 2021-2024 was conducted. European Public Assessment Reports (EPAR) and Summary of Product Characteristics (SmPC) were reviewed for each medicine to identify PROs use and PRO label claims.
RESULTS: A total of 134 oncology indications were approved, and 111 (82.8%) included PROs in the confirmatory clinical trials. Of these, 31 indications were supported by randomized double-blinded trials, 79 by open-label trials (22 of which were single-arm trials), and 1 by both. PROs were defined as a secondary endpoint in 56 studies (49.6%), exploratory in 27 (23.9%) and as both in 27 (23.9%). This variable is unknown for 3 (2.6%) studies. The selected PRO measures (PROMs) relied on the EORTC (43.9%), FACIT (8.6%) and EQ-5D (27.6%) instruments. Of the 66 different PROMs identified, 50 (75.7%) were oncology-specific, 7 of which were symptom-specific. PROs reviewer’s comments were included in the EPAR for 80 indications (59.7%), however only 1 (0.7%) included a Health-related quality of life claim in the SmPC. Reasons for excluding PRO claims were identified for 54 indications (67.5%).
CONCLUSIONS: Despite increased PRO inclusion in confirmatory trials, translating this evidence into label claims remains challenging. Although EPARs appear to provide more detailed PRO data, label claims continue to be granted cautiously. The main reasons identified are related to the exploratory nature of the data and its limited methodological robustness, limitations in the trial design and conduct, missing data, selection bias and limited clinical relevance.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HPR155
Topic
Health Policy & Regulatory, Patient-Centered Research
Topic Subcategory
Approval & Labeling
Disease
Oncology