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A review of patient-reported outcomes used for regulatory approval of oncology medicinal products in the European Union between 2017 and 2020
INTRODUCTION: Cancer and corresponding available treatments are associated with substantial symptoms and functional limitations. In this context, collection of patient-reported outcomes (PRO) in clinical trials gained special interest and is recommended by regulatory authorities. Within clinical tri...
Autores principales: | , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Frontiers Media S.A.
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9411861/ https://www.ncbi.nlm.nih.gov/pubmed/36035431 http://dx.doi.org/10.3389/fmed.2022.968272 |
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author | Teixeira, Maria Manuel Borges, Fábio Cardoso Ferreira, Paula Sousa Rocha, João Sepodes, Bruno Torre, Carla |
author_facet | Teixeira, Maria Manuel Borges, Fábio Cardoso Ferreira, Paula Sousa Rocha, João Sepodes, Bruno Torre, Carla |
author_sort | Teixeira, Maria Manuel |
collection | PubMed |
description | INTRODUCTION: Cancer and corresponding available treatments are associated with substantial symptoms and functional limitations. In this context, collection of patient-reported outcomes (PRO) in clinical trials gained special interest and is recommended by regulatory authorities. Within clinical trials framework, PRO may provide evidence to support medicines approval, labeling and marketing claims. This study aims to analyze the existing evidence based on PRO as part of new oncology indications receiving positive opinions issued by the European Medicines Agency (EMA) between 2017 and 2020 and to identify PRO related label claims granted. METHODOLOGY: Oncology medicinal products and indications approved by the European Commission following a positive opinion from the EMA between 2017 and 2020 were identified. European Public Assessment Report (EPAR) and Summary of Product Characteristics (SmPC) were reviewed for each medicinal product to identify use of PRO and PRO label claims. RESULTS: A total of 128 oncology indications, corresponding to 76 medicines, were approved; of those, 100 (78.1%) included PRO in the confirmatory clinical trials. Thirty-seven indications were supported by double-blind randomized trials and the remainder 63 by open-label trials. Out of the 104 confirmatory trials analyzed, PRO were defined as a secondary endpoint in 60 studies (57.7%), exploratory in 31 (29.8%) and as both in 13 (12.5%). In total, 54 different PRO measures (PROM) were used, of those 41 (75.9%) were disease-specific measures. Nevertheless, PROM selected relied on the EORTC (41.3%), FACIT (17.1%) and EQ-5D (29.2%) measures. A total of 76 indications (59.4%) had PRO reviewers comments included in the EPAR, however only 22 indications (17.8%) included label claims in the SmPC. The reasons identified in the EMA assessment supporting the exclusion of PRO claims were described for 34 indications (44.7%). CONCLUSIONS: Despite growing recognition of the value of PRO data for the development of improved cancer therapies, PRO implementation remains challenging. The main reasons identified in our study are related with study design, missing data, study conduct and PROM selection. |
format | Online Article Text |
id | pubmed-9411861 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2022 |
publisher | Frontiers Media S.A. |
record_format | MEDLINE/PubMed |
spelling | pubmed-94118612022-08-27 A review of patient-reported outcomes used for regulatory approval of oncology medicinal products in the European Union between 2017 and 2020 Teixeira, Maria Manuel Borges, Fábio Cardoso Ferreira, Paula Sousa Rocha, João Sepodes, Bruno Torre, Carla Front Med (Lausanne) Medicine INTRODUCTION: Cancer and corresponding available treatments are associated with substantial symptoms and functional limitations. In this context, collection of patient-reported outcomes (PRO) in clinical trials gained special interest and is recommended by regulatory authorities. Within clinical trials framework, PRO may provide evidence to support medicines approval, labeling and marketing claims. This study aims to analyze the existing evidence based on PRO as part of new oncology indications receiving positive opinions issued by the European Medicines Agency (EMA) between 2017 and 2020 and to identify PRO related label claims granted. METHODOLOGY: Oncology medicinal products and indications approved by the European Commission following a positive opinion from the EMA between 2017 and 2020 were identified. European Public Assessment Report (EPAR) and Summary of Product Characteristics (SmPC) were reviewed for each medicinal product to identify use of PRO and PRO label claims. RESULTS: A total of 128 oncology indications, corresponding to 76 medicines, were approved; of those, 100 (78.1%) included PRO in the confirmatory clinical trials. Thirty-seven indications were supported by double-blind randomized trials and the remainder 63 by open-label trials. Out of the 104 confirmatory trials analyzed, PRO were defined as a secondary endpoint in 60 studies (57.7%), exploratory in 31 (29.8%) and as both in 13 (12.5%). In total, 54 different PRO measures (PROM) were used, of those 41 (75.9%) were disease-specific measures. Nevertheless, PROM selected relied on the EORTC (41.3%), FACIT (17.1%) and EQ-5D (29.2%) measures. A total of 76 indications (59.4%) had PRO reviewers comments included in the EPAR, however only 22 indications (17.8%) included label claims in the SmPC. The reasons identified in the EMA assessment supporting the exclusion of PRO claims were described for 34 indications (44.7%). CONCLUSIONS: Despite growing recognition of the value of PRO data for the development of improved cancer therapies, PRO implementation remains challenging. The main reasons identified in our study are related with study design, missing data, study conduct and PROM selection. Frontiers Media S.A. 2022-08-12 /pmc/articles/PMC9411861/ /pubmed/36035431 http://dx.doi.org/10.3389/fmed.2022.968272 Text en Copyright © 2022 Teixeira, Borges, Ferreira, Rocha, Sepodes and Torre. https://creativecommons.org/licenses/by/4.0/This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms. |
spellingShingle | Medicine Teixeira, Maria Manuel Borges, Fábio Cardoso Ferreira, Paula Sousa Rocha, João Sepodes, Bruno Torre, Carla A review of patient-reported outcomes used for regulatory approval of oncology medicinal products in the European Union between 2017 and 2020 |
title | A review of patient-reported outcomes used for regulatory approval of oncology medicinal products in the European Union between 2017 and 2020 |
title_full | A review of patient-reported outcomes used for regulatory approval of oncology medicinal products in the European Union between 2017 and 2020 |
title_fullStr | A review of patient-reported outcomes used for regulatory approval of oncology medicinal products in the European Union between 2017 and 2020 |
title_full_unstemmed | A review of patient-reported outcomes used for regulatory approval of oncology medicinal products in the European Union between 2017 and 2020 |
title_short | A review of patient-reported outcomes used for regulatory approval of oncology medicinal products in the European Union between 2017 and 2020 |
title_sort | review of patient-reported outcomes used for regulatory approval of oncology medicinal products in the european union between 2017 and 2020 |
topic | Medicine |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9411861/ https://www.ncbi.nlm.nih.gov/pubmed/36035431 http://dx.doi.org/10.3389/fmed.2022.968272 |
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