The Role of Academic Medical Centers in Personalized Experimental Therapeutic Development: Key Considerations

The recent rapid rise in the number of identified genetic disorders that are potentially treatable has far outpaced the development of treatments, resulting in a worsening bottleneck in the translational pipeline. New therapeutics that target an individual's specific genetic variant, or "personalized" therapeutics, are being developed and tested at several academic medical centers. Personalized therapeutics represent a potentially transformative platform technology that could unlock new treatment models for rare disorders. At the same time, the field remains investigational, and developing and testing novel drugs in single individuals, particularly children, poses substantial sensitivities and challenges. In this review, we outline some important considerations for academic medical centers in determining their role in reviewing and supporting the development of personalized therapeutics for rare disorders, including oversight and resource allocation. We describe an institutional infrastructure and process for navigating this landscape, involving transparent oversight and communication processes.