CVS Health January, 2020
Troy Brennan, M.D., Sree Chaguturu, M.D., Daniel Knecht, M.D.


After many years of scientific promise but few real results, gene therapies are now a reality. These exciting new interventions have the potential to relieve tremendous suffering for patients with genetic disorders. Making sure these treatments are accessible for all those who need them requires innovative partnerships, as well as new ways of providing health insurance and pharmacy benefit design.

Generally speaking, gene therapies use a target gene that expresses protein products at a sufficient level to cure, or at least ameliorate, a disease caused by a genetic defect. So far, the U.S. Food and Drug Administration (FDA) has approved a handful of gene therapies to treat serious diseases that were previously untreatable except through supportive measures. The European...