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 optimal 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 trials, 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, bioethicists, 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.

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