Exploring Novel Clinical Trial Designs for Gene-Based Therapies – A Workshop
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 name a few. Patients and clinicians may also face difficult decisions about enrollment in a gene therapy or trial in certain instances where the trial is considered to offer a “single chance,” possibly making the patients ineligible for future trials. To further explore the design complexities and ethical issues associated with clinical trials for these types of therapies, the Forum on Regenerative Medicine will hold a one-day workshop in Washington, DC on November 13, 2019. 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.