All Times EDT
Keywords: rare disease, gene therapy, cell therapy
Clinical trials for rare diseases, including gene therapies and cell therapies, call for the same basic principles of all clinical trials; however, there are challenges to designing, conducting and analyzing such trials which need special consideration. Aspects that may require special consideration include smallness of disease populations, potential lack of a relevant control arm, difficulty with randomization and blinding, absence of validated endpoints in some disease settings, use of surrogate outcomes, retaining participants for long term safety follow, compressed development timeline engendered by high unmet need and so on. Making therapy development even more challenging in this area is the fact that sponsors and regulators have limited experience and most existing guidelines were developed in the context of treatments for diseases affecting large population. Despite these challenges, emerging science in this area provide opportunity to make life-changing difference for patients who may otherwise have no treatment options. This round table is intended to facilitate discussions on the issues and challenges regulators and sponsors have faced with rare disease programs, including gene therapy and cell therapy programs and to generate discussion on potential solutions. Topics to consider including choice of control, validating endpoints in new disease settings and/or surrogate endpoints that may facilitate early approval, reconciling feedback across regulatory agencies, and how to ensure long term follow data are adequately captured.