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Suggested Citation:"Appendix C: Statement of Task." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
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Page 99
Suggested Citation:"Appendix C: Statement of Task." National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25712.
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Page 100

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Appendix C Statement of Task Designing clinical trials to test the safety and efficacy of regenerative medicine therapies, such as gene- and gene-editing-based therapies, can be complex for several reasons, including challenges with determining an opti- mal dosage, delivering the product effectively, and successfully recruiting patients to what may be “single chance” trials, to name a few. To explore the design complexities and ethical issues associated with clinical trials for these types of therapies, an ad hoc planning committee will hold a 1-day public workshop in Washington, DC. Speakers at the workshop may be asked to discuss patient recruitment and selection for gene-based clinical tri- als, assessing the safety of new therapies, dose escalation, and ethical issues such as informed consent and the role of clinicians in recommending trials as options to their patients. The concept of repeat dosing and sensitization treatments may also be explored. A broad array of stakeholders may take part in the workshop, including academic and industry researchers, regulatory officials, clinicians, bioethi- cists, and individuals/patients and patient advocacy groups. The planning committee will develop the workshop agenda, select and invite speakers and discussants, and may moderate the discussions. A proceedings of the workshop will be prepared by a designated rapporteur in accordance with institutional policy and procedures. 99 PREPUBLICATION COPY—Uncorrected Proofs

PREPUBLICATION COPY—Uncorrected Proofs

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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.

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