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Disease monitoring programs of rare genetic diseases: transparent data sharing between academic and commercial stakeholders
It has recently been suggested that registries for rare neuromuscular diseases should be formed and governed exclusively by physicians and patients in an effort to limit conflicts of interest. Enacting such an approach would not only be challenging logistically and financially, but it would also exc...
Autores principales: | , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
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BioMed Central
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7980582/ https://www.ncbi.nlm.nih.gov/pubmed/33743771 http://dx.doi.org/10.1186/s13023-021-01687-7 |
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author | Lochmüller, Hanns Ramirez, Antonio Nino Kakkis, Emil |
author_facet | Lochmüller, Hanns Ramirez, Antonio Nino Kakkis, Emil |
author_sort | Lochmüller, Hanns |
collection | PubMed |
description | It has recently been suggested that registries for rare neuromuscular diseases should be formed and governed exclusively by physicians and patients in an effort to limit conflicts of interest. Enacting such an approach would not only be challenging logistically and financially, but it would also exclude the involvement of sponsors, who are an integral component of drug development within the current compliance framework. Therefore, as an alternative to traditional registries, we propose the use of a better collaborative model for post-marketing follow-up that includes all stakeholders. We developed the concept of Disease Monitoring Programs (DMPs), which are designed to monitor disease manifestations over a 10-year period whether on a sponsored drug or not, and ensure consistent collection, ownership sharing and governance of data. |
format | Online Article Text |
id | pubmed-7980582 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | BioMed Central |
record_format | MEDLINE/PubMed |
spelling | pubmed-79805822021-03-22 Disease monitoring programs of rare genetic diseases: transparent data sharing between academic and commercial stakeholders Lochmüller, Hanns Ramirez, Antonio Nino Kakkis, Emil Orphanet J Rare Dis Letter to the Editor It has recently been suggested that registries for rare neuromuscular diseases should be formed and governed exclusively by physicians and patients in an effort to limit conflicts of interest. Enacting such an approach would not only be challenging logistically and financially, but it would also exclude the involvement of sponsors, who are an integral component of drug development within the current compliance framework. Therefore, as an alternative to traditional registries, we propose the use of a better collaborative model for post-marketing follow-up that includes all stakeholders. We developed the concept of Disease Monitoring Programs (DMPs), which are designed to monitor disease manifestations over a 10-year period whether on a sponsored drug or not, and ensure consistent collection, ownership sharing and governance of data. BioMed Central 2021-03-20 /pmc/articles/PMC7980582/ /pubmed/33743771 http://dx.doi.org/10.1186/s13023-021-01687-7 Text en © The Author(s) 2021 Open AccessThis article is licensed under a Creative Commons Attribution 4.0 International License, which permits 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/4.0/. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated in a credit line to the data. |
spellingShingle | Letter to the Editor Lochmüller, Hanns Ramirez, Antonio Nino Kakkis, Emil Disease monitoring programs of rare genetic diseases: transparent data sharing between academic and commercial stakeholders |
title | Disease monitoring programs of rare genetic diseases: transparent data sharing between academic and commercial stakeholders |
title_full | Disease monitoring programs of rare genetic diseases: transparent data sharing between academic and commercial stakeholders |
title_fullStr | Disease monitoring programs of rare genetic diseases: transparent data sharing between academic and commercial stakeholders |
title_full_unstemmed | Disease monitoring programs of rare genetic diseases: transparent data sharing between academic and commercial stakeholders |
title_short | Disease monitoring programs of rare genetic diseases: transparent data sharing between academic and commercial stakeholders |
title_sort | disease monitoring programs of rare genetic diseases: transparent data sharing between academic and commercial stakeholders |
topic | Letter to the Editor |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7980582/ https://www.ncbi.nlm.nih.gov/pubmed/33743771 http://dx.doi.org/10.1186/s13023-021-01687-7 |
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