Familiar Principles In Gene Therapy Guidance For Rare Diseases
Use of historical controls and alternative study designs are allowed, but randomized control trial is preferred.
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US FDA's center directors chime in on topics such as antimicrobial drug development and public education at the 2019 Reagan-Udall annual meeting.
CBER Director Peter Marks says the "half-life" for the existing six gene therapy guidances likely is two to three years due to the fast pace of scientific discovery.
When firms are developing highly anticipated gene therapies, it’s important to establish GMP-controlled manufacturing processes earlier in development, as Sarepta discovered when US FDA placed a clinical hold on a promising Duchenne muscular dystrophy gene therapy.