Cargando…

Capturing Data in Rare Disease Registries to Support Regulatory Decision Making: A Survey Study Among Industry and Other Stakeholders

INTRODUCTION: In rare diseases, registry-based studies can be used to provide natural history data pre-approval and complement drug efficacy and/or safety knowledge post-approval. OBJECTIVE: The objective of this study was to investigate the opinion of stakeholders about key aspects of rare disease...

Descripción completa

Detalles Bibliográficos
Autores principales: Jonker, Carla J., de Vries, Sieta T., van den Berg, H. Marijke, McGettigan, Patricia, Hoes, Arno W., Mol, Peter G. M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer International Publishing 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8279983/
https://www.ncbi.nlm.nih.gov/pubmed/34091881
http://dx.doi.org/10.1007/s40264-021-01081-z
_version_ 1783722557266984960
author Jonker, Carla J.
de Vries, Sieta T.
van den Berg, H. Marijke
McGettigan, Patricia
Hoes, Arno W.
Mol, Peter G. M.
author_facet Jonker, Carla J.
de Vries, Sieta T.
van den Berg, H. Marijke
McGettigan, Patricia
Hoes, Arno W.
Mol, Peter G. M.
author_sort Jonker, Carla J.
collection PubMed
description INTRODUCTION: In rare diseases, registry-based studies can be used to provide natural history data pre-approval and complement drug efficacy and/or safety knowledge post-approval. OBJECTIVE: The objective of this study was to investigate the opinion of stakeholders about key aspects of rare disease registries that are used to support regulatory decision making and to compare the responses of employees from industry to other stakeholders. METHODS: A web-based survey was used to gauge the importance of (1) common data elements (including safety outcomes), (2) data quality and (3) governance aspects that are generic across different rare diseases. The survey included 47 questions. The data were collected in the period April-October 2019. RESULTS: Seventy-three respondents completed ≥ 80% of the survey. Most of the respondents were from the industry (n = 42, 57%). For safety data, 31 (42%) respondents were in favour of collecting all adverse events. For data quality, the respondents found a level of 30% reasonable for source data verification. For missing data, a level of 20% was considered acceptable. Compared to responders from industry, the other stakeholders found it less relevant to share data with industry and found it less acceptable if the registry is financed by industry. CONCLUSIONS: This study showed that the opinion towards data and governance is well aligned across parties, and issues of data and governance on their own should not pose a barrier to collaboration. This finding is supportive of the European Medicines Agency’s efforts to encourage stakeholders to work with existing registries when collecting data to support regulatory decision making. SUPPLEMENTARY INFORMATION: The online version contains supplementary material available at 10.1007/s40264-021-01081-z.
format Online
Article
Text
id pubmed-8279983
institution National Center for Biotechnology Information
language English
publishDate 2021
publisher Springer International Publishing
record_format MEDLINE/PubMed
spelling pubmed-82799832021-07-20 Capturing Data in Rare Disease Registries to Support Regulatory Decision Making: A Survey Study Among Industry and Other Stakeholders Jonker, Carla J. de Vries, Sieta T. van den Berg, H. Marijke McGettigan, Patricia Hoes, Arno W. Mol, Peter G. M. Drug Saf Original Research Article INTRODUCTION: In rare diseases, registry-based studies can be used to provide natural history data pre-approval and complement drug efficacy and/or safety knowledge post-approval. OBJECTIVE: The objective of this study was to investigate the opinion of stakeholders about key aspects of rare disease registries that are used to support regulatory decision making and to compare the responses of employees from industry to other stakeholders. METHODS: A web-based survey was used to gauge the importance of (1) common data elements (including safety outcomes), (2) data quality and (3) governance aspects that are generic across different rare diseases. The survey included 47 questions. The data were collected in the period April-October 2019. RESULTS: Seventy-three respondents completed ≥ 80% of the survey. Most of the respondents were from the industry (n = 42, 57%). For safety data, 31 (42%) respondents were in favour of collecting all adverse events. For data quality, the respondents found a level of 30% reasonable for source data verification. For missing data, a level of 20% was considered acceptable. Compared to responders from industry, the other stakeholders found it less relevant to share data with industry and found it less acceptable if the registry is financed by industry. CONCLUSIONS: This study showed that the opinion towards data and governance is well aligned across parties, and issues of data and governance on their own should not pose a barrier to collaboration. This finding is supportive of the European Medicines Agency’s efforts to encourage stakeholders to work with existing registries when collecting data to support regulatory decision making. SUPPLEMENTARY INFORMATION: The online version contains supplementary material available at 10.1007/s40264-021-01081-z. Springer International Publishing 2021-06-06 2021 /pmc/articles/PMC8279983/ /pubmed/34091881 http://dx.doi.org/10.1007/s40264-021-01081-z Text en © The Author(s) 2021 https://creativecommons.org/licenses/by-nc/4.0/Open AccessThis article is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License, which permits any non-commercial use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by-nc/4.0/ (https://creativecommons.org/licenses/by-nc/4.0/) .
spellingShingle Original Research Article
Jonker, Carla J.
de Vries, Sieta T.
van den Berg, H. Marijke
McGettigan, Patricia
Hoes, Arno W.
Mol, Peter G. M.
Capturing Data in Rare Disease Registries to Support Regulatory Decision Making: A Survey Study Among Industry and Other Stakeholders
title Capturing Data in Rare Disease Registries to Support Regulatory Decision Making: A Survey Study Among Industry and Other Stakeholders
title_full Capturing Data in Rare Disease Registries to Support Regulatory Decision Making: A Survey Study Among Industry and Other Stakeholders
title_fullStr Capturing Data in Rare Disease Registries to Support Regulatory Decision Making: A Survey Study Among Industry and Other Stakeholders
title_full_unstemmed Capturing Data in Rare Disease Registries to Support Regulatory Decision Making: A Survey Study Among Industry and Other Stakeholders
title_short Capturing Data in Rare Disease Registries to Support Regulatory Decision Making: A Survey Study Among Industry and Other Stakeholders
title_sort capturing data in rare disease registries to support regulatory decision making: a survey study among industry and other stakeholders
topic Original Research Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8279983/
https://www.ncbi.nlm.nih.gov/pubmed/34091881
http://dx.doi.org/10.1007/s40264-021-01081-z
work_keys_str_mv AT jonkercarlaj capturingdatainrarediseaseregistriestosupportregulatorydecisionmakingasurveystudyamongindustryandotherstakeholders
AT devriessietat capturingdatainrarediseaseregistriestosupportregulatorydecisionmakingasurveystudyamongindustryandotherstakeholders
AT vandenberghmarijke capturingdatainrarediseaseregistriestosupportregulatorydecisionmakingasurveystudyamongindustryandotherstakeholders
AT mcgettiganpatricia capturingdatainrarediseaseregistriestosupportregulatorydecisionmakingasurveystudyamongindustryandotherstakeholders
AT hoesarnow capturingdatainrarediseaseregistriestosupportregulatorydecisionmakingasurveystudyamongindustryandotherstakeholders
AT molpetergm capturingdatainrarediseaseregistriestosupportregulatorydecisionmakingasurveystudyamongindustryandotherstakeholders