Kennedy cited the successful treatment ... therapy at the Children’s Hospital of Philadelphia. “Every child facing a rare genetic disease deserves the same kind of treatment that KJ got,” Kennedy said. Under the new framework, a disease with 100 mutations of the same gene would no longer require 100 clinical trials, Kennedy said, ... Kennedy cited the successful treatment of KJ Muldoon, also known as Baby KJ, who was born in August 2024 with a fatal metabolic disorder and survived after being successfully treated with a customized gene-editing therapy at the Children’s Hospital of Philadelphia. “Every child facing a rare genetic disease deserves the same kind of treatment that KJ got,” Kennedy said. Under the new framework, a disease with 100 mutations of the same gene would no longer require 100 clinical trials, Kennedy said, adding that the guidelines require long-term safety monitoring. The FDA’s Center for Drug Evaluation and Research will conduct the reviews of so-called investigational new drugs, clustering treatments that work with similar mechanisms, such as gene editing, into a single clinical trial. “Our system is built for common diseases not for rare ones,” Makary said Monday. “Historically rare diseases have been at the FDA an afterthought. Rigorous, elaborate standards are so high that they’re just not practical for many of the therapies for rare diseases.” About 30 million Americans have a rare disease, according to FDA Commissioner Dr. Marty Makary Called the plausible mechanism framework, the draft FDA guidelines establish a new approval process for individualized therapies for rare disorders. “This framework aligns regulations with biology for ultra-rare conditions,” Health and Human Services Secretary Robert F. Kennedy Jr. said at an event where he was joined by FDA Commissioner Dr. Marty Makary and the parents of children with rare diseases. “Randomized control trials are mostly just not feasible.”

Original title: “FDA says it will speed up rare disease treatment approvals”