Reata’s Skyclarys Approval Aided By Natural History And Delayed-Start Analyses
Executive Summary
The first treatment for the rare neurodegenerative disorder Friedreich’s ataxia relied on a single placebo-controlled efficacy trial.
You may also be interested in...
Natural History Studies: Launch With Care And Consider Alternative Evidence For Drug Approval, FDA’s Marks Says
A different evidence-generation approach may lead to a quicker approval than leveraging a natural history study, CBER Director Peter Marks says; EveryLife Foundation's Annie Kennedy says participation in natural history studies can empower patients who are not eligible for drug-specific clinical trials.
Keeping Track: Opdivo, Keytruda Continue Adjuvant Push As Cancer Dominates Recent Applications
The latest drug development news and highlights from the Pink Sheet’s US FDA Performance Tracker
RWE On The Runway: Trio Of Upcoming Approval Decisions Will Test US FDA’s Thinking On RWE For Efficacy
Real-world evidence provides external controls for bluebird bio’s eli-cel and Ipsen’s palovarotene, while Reata used RWE to address FDA concerns about the pivotal single-arm study of omaveloxolone. In the post-market, Intercept is gearing up to seek full approval of Ocaliva relying in part on claims database.