Individualized Therapies on the RISE

The current drug development paradigm and associated regulations were developed to test single medicines that treat numerous patients, long before the concept of precision medicine became a reality in the form of individualized therapies [e.g., genome editing products and antisense oligonucleotides (ASOs)]. Individualized therapies, for the purposes of this meeting, are defined as therapies tailored to the unique genetic variants of one or a very small number of individuals. While ASOs are not novel, their application as individualized therapy is. The first individualized ASO treatment was administered in 2018 to treat a form of Batten disease, and since then, many more patients with various conditions have since received such treatments. More recently, individualized gene editing therapies have been developed, including one used to treat an infant with CPS1 deficiency.  Over recent years, the U.S. Food and Drug Administration (FDA) has developed numerous guidance documents for gene therapies and ASOs, including for individualized ASOs.  As individualized ASOs and gene editing products are entering the clinic, it is timely to evaluate whether current regulatory practices are optimal to support the development of individualized therapies. 

This public workshop, co-convened by the Duke-Margolis Institute for Health Policy and the Rare Disease Innovation Hub at the FDA, will examine the emerging science and regulatory environment for these individualized medicine programs, including nonclinical data recommendations, clinical assessments, regulatory submission structure, and additional information necessary to support the development, evaluation, and potential commercialization of these treatments.  

The workshop will also provide opportunities for members of the rare disease community, including researchers, sponsors, patients, patient advocates, and regulators, to share their experiences with individualized medicine approaches. The goal of this workshop is to enhance understanding of regulatory considerations for individualized treatments and to inform future policy around individualized treatments.   

This project is supported by the Food and Drug Administration (FDA) of the U.S. Department of Health and Human Services (HHS) as part of a financial assistance award U19FD006602 totaling $5,192,495 with 100 percent funded by FDA/HHS. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement, by FDA/HHS, or the U.S. Government.