TY - JOUR
T1 - Data sharing to advance gene-targeted therapies in rare diseases
AU - Lekstrom-Himes, Julie
AU - Augustine, Erika F.
AU - Brower, Amy
AU - Defay, Thomas
AU - Finkel, Richard S.
AU - McGuire, Amy L.
AU - Skinner, Mark W.
AU - Yu, Timothy W.
N1 - Funding Information:
At this writing, the WFH GTR is ready to accept data. Data collection will commence upon licensure of the first gene therapy for hemophilia anticipated in early 2023. Numerous national registries and hemophilia treatment centers have already completed the necessary ethical review and signed participation agreements. The database infrastructure, registry interface and patient app have been developed. A global steering, research review, industry and patient advisory committees have been established. Governance protocols have been developed. The WFH GTR is funded through multi‐year commitments from gene‐therapy sponsoring manufacturers.
Publisher Copyright:
© 2023 The Authors. American Journal of Medical Genetics Part C: Seminars in Medical Genetics published by Wiley Periodicals LLC.
PY - 2023/3
Y1 - 2023/3
N2 - Recent advancements in gene-targeted therapies have highlighted the critical role data sharing plays in successful translational drug development for people with rare diseases. To scale these efforts, we need to systematize these sharing principles, creating opportunities for more rapid, efficient, and scalable drug discovery/testing including long-term and transparent assessment of clinical safety and efficacy. A number of challenges will need to be addressed, including the logistical difficulties of studying rare diseases affecting individuals who may be scattered across the globe, scientific, technical, regulatory, and ethical complexities of data collection, and harmonization and integration across multiple platforms and contexts. The NCATS/NIH Gene-Targeted Therapies: Early Diagnosis and Equitable Delivery meeting series held during June 2021 included data sharing models that address these issues and framed discussions of areas that require improvement. This article describes these discussions and provides a series of considerations for future data sharing.
AB - Recent advancements in gene-targeted therapies have highlighted the critical role data sharing plays in successful translational drug development for people with rare diseases. To scale these efforts, we need to systematize these sharing principles, creating opportunities for more rapid, efficient, and scalable drug discovery/testing including long-term and transparent assessment of clinical safety and efficacy. A number of challenges will need to be addressed, including the logistical difficulties of studying rare diseases affecting individuals who may be scattered across the globe, scientific, technical, regulatory, and ethical complexities of data collection, and harmonization and integration across multiple platforms and contexts. The NCATS/NIH Gene-Targeted Therapies: Early Diagnosis and Equitable Delivery meeting series held during June 2021 included data sharing models that address these issues and framed discussions of areas that require improvement. This article describes these discussions and provides a series of considerations for future data sharing.
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U2 - 10.1002/ajmg.c.32028
DO - 10.1002/ajmg.c.32028
M3 - Comment/debate
C2 - 36594517
AN - SCOPUS:85145491242
SN - 1552-4868
VL - 193
SP - 87
EP - 98
JO - American Journal of Medical Genetics, Part C: Seminars in Medical Genetics
JF - American Journal of Medical Genetics, Part C: Seminars in Medical Genetics
IS - 1
ER -