Julia M. Y. Pian, Nana Owusu, Julia Vitarello, Winston X. Yan, Andrew W. Lo &
Timothy W. Yu (Nature Medicine)
June 19, 2024
For precision genetic medicines to fulfill their potential as treatments for ultra-rare diseases, fresh approaches to academic–industry partnerships and data sharing are needed, together with regulatory change and adaptation of reimbursement models.
Advances in gene therapy and gene editing technologies could revolutionize the ability to treat individuals with genetic disease, allowing treatments to be devised that target specific genetic mutations in people with even the rarest of disease indications. In 2018, a seven-year-old child with Batten disease received attention for becoming the first recipient of a customized antisense oligonucleotide (ASO) therapy specifically designed for her unique mutation. Since then, multiple patients with ultra-rare genetic conditions have been treated with precision ASOs through academic-investigator-initiated programs.
Development of these ASOs has been rapid, justified by the severity of the conditions being treated (for example, rapidly progressive neurologic degeneration), following streamlined regulatory processes. Here we discuss possible models for drug development, regulation and reimbursement that could allow these tailored genetic interventions to be scaled.
Pian, J.M.Y., Owusu, N., Vitarello, J. et al. How to pay for individualized genetic medicines.Nat Med (2024). https://doi.org/10.1038/s41591-024-03071-x
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