Cargando…

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...

Descripción completa

Detalles Bibliográficos
Autores principales: Teixeira, Maria Manuel, Borges, Fábio Cardoso, Ferreira, Paula Sousa, Rocha, João, Sepodes, Bruno, Torre, Carla
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2022
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
_version_ 1784775359953108992
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
work_keys_str_mv AT teixeiramariamanuel areviewofpatientreportedoutcomesusedforregulatoryapprovalofoncologymedicinalproductsintheeuropeanunionbetween2017and2020
AT borgesfabiocardoso areviewofpatientreportedoutcomesusedforregulatoryapprovalofoncologymedicinalproductsintheeuropeanunionbetween2017and2020
AT ferreirapaulasousa areviewofpatientreportedoutcomesusedforregulatoryapprovalofoncologymedicinalproductsintheeuropeanunionbetween2017and2020
AT rochajoao areviewofpatientreportedoutcomesusedforregulatoryapprovalofoncologymedicinalproductsintheeuropeanunionbetween2017and2020
AT sepodesbruno areviewofpatientreportedoutcomesusedforregulatoryapprovalofoncologymedicinalproductsintheeuropeanunionbetween2017and2020
AT torrecarla areviewofpatientreportedoutcomesusedforregulatoryapprovalofoncologymedicinalproductsintheeuropeanunionbetween2017and2020
AT teixeiramariamanuel reviewofpatientreportedoutcomesusedforregulatoryapprovalofoncologymedicinalproductsintheeuropeanunionbetween2017and2020
AT borgesfabiocardoso reviewofpatientreportedoutcomesusedforregulatoryapprovalofoncologymedicinalproductsintheeuropeanunionbetween2017and2020
AT ferreirapaulasousa reviewofpatientreportedoutcomesusedforregulatoryapprovalofoncologymedicinalproductsintheeuropeanunionbetween2017and2020
AT rochajoao reviewofpatientreportedoutcomesusedforregulatoryapprovalofoncologymedicinalproductsintheeuropeanunionbetween2017and2020
AT sepodesbruno reviewofpatientreportedoutcomesusedforregulatoryapprovalofoncologymedicinalproductsintheeuropeanunionbetween2017and2020
AT torrecarla reviewofpatientreportedoutcomesusedforregulatoryapprovalofoncologymedicinalproductsintheeuropeanunionbetween2017and2020