Disease monitoring programs of rare genetic diseases: transparent data sharing between academic and commercial stakeholders.

Szczegóły
Abstrakt

Tytuł:: Disease monitoring programs of rare genetic diseases: transparent data sharing between academic and commercial stakeholders.
Autorzy:: Lochmüller H; Children's Hospital of Eastern Ontario Research Institute, Ottawa, Canada. .; Division of Neurology, Department of Medicine, The Ottawa Hospital, Ottawa, Canada. .; Brain and Mind Research Institute, University of Ottawa, Ottawa, Canada. .
Ramirez AN; Ultragenyx Pharmaceutical Inc., Novato, CA, USA.
Kakkis E; Ultragenyx Pharmaceutical Inc., Novato, CA, USA.
Źródło:: Orphanet journal of rare diseases [Orphanet J Rare Dis] 2021 Mar 20; Vol. 16 (1), pp. 141. Date of Electronic Publication: 2021 Mar 20.
Typ publikacji:: Letter; Research Support, Non-U.S. Gov't
Język:: English
Imprint Name(s):: Original Publication: [London] : BioMed Central, 2006-
MeSH Terms:: Distal Myopathies*
Information Dissemination*
Humans ; Rare Diseases/genetics ; Registries
References:: Adv Exp Med Biol. 2017;1031:97-124. (PMID: 29214567)
Eur J Hum Genet. 2017 Dec;25(12):1293-1302. (PMID: 29158551)
Neuromuscul Disord. 2018 Feb;28(2):158-168. (PMID: 29305133)
Orphanet J Rare Dis. 2020 Sep 3;15(1):235. (PMID: 32883346)
J Neuromuscul Dis. 2021;8(1):53-61. (PMID: 32925088)
J Neuromuscul Dis. 2021;8(2):225-234. (PMID: 33459658)
Eur J Hum Genet. 2017 Feb;25(2):162-165. (PMID: 27782107)
Orphanet J Rare Dis. 2019 Jan 21;14(1):18. (PMID: 30665421)
Grant Information:: Foundation Grant FDN-167281 Canada CIHR; CFI-JELF 38412 Canada Foundation for Innovation; (Network Catalyst Grant for NMD4C Canadian Institutes of Health Research and Muscular Dystrophy Canada; 950-232279 Canada Research Chairs program
Contributed Indexing:: Keywords: DMP; Data sharing; Disease monitoring program; GNE myopathy; GNE myopathy disease monitoring program; GNEM-DMP; Orphan drugs; Registries; Registry
Entry Date(s):: Date Created: 20210321 Date Completed: 20210618 Latest Revision: 20210618
Update Code:: 20240105
PubMed Central ID:: PMC7980582
DOI:: 10.1186/s13023-021-01687-7
PMID:: 33743771
: Raport

Pełny tekst

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.

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