Exploring novel clinical trial designs for gene-based therapies proceedings of a workshop

Recognizing the potential design complexities and ethical issues associated with clinical trials for gene therapies, the Forum on Regenerative Medicine of the National Academies of Sciences, Engineering, and Medicine held a 1-day workshop in Washington, DC, on November 13, 2019. Speakers at the work...

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Bibliographic Details
Main Author: Addie, Siobhan
Corporate Authors: National Academies of Sciences, Engineering, and Medicine (U.S.) Forum on Regenerative Medicine, Exploring Novel Clinical Trial Designs for Gene-Based Therapies: a Workshop (2019, Washington, D.C.)
Other Authors: Hackmann, Meredith ([rapporteur]), Alper, Joe ([rapporteur]), Beachy, Sarah H. ([rapporteur])
Format: eBook
Language:English
Published: Washington, D.C. National Academies Press [2020], 2020
Subjects:
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Collection: National Center for Biotechnology Information - Collection details see MPG.ReNa
Description
Summary:Recognizing the potential design complexities and ethical issues associated with clinical trials for gene therapies, the Forum on Regenerative Medicine of the National Academies of Sciences, Engineering, and Medicine held a 1-day workshop in Washington, DC, on November 13, 2019. Speakers at the workshop discussed patient recruitment and selection for gene-based clinical trials, explored how the safety of new therapies is assessed, reviewed the challenges involving dose escalation, and spoke about ethical issues such as informed consent and the role of clinicians in recommending trials as options to their patients. The workshop also included discussions of topics related to gene therapies in the context of other available and potentially curative treatments, such as bone marrow transplantation for hemoglobinopathies. This publication summarizes the presentation and discussion of the workshop
Physical Description:1 PDF file (xx, 106 pages) illustrations
ISBN:9780309672986
0309672988