Successful Applications of Real-World Data and Real-World Evidence in Rare Disease Programs
The Premier Consulting Blog
OCTOBER 11, 2022
Case Study 1: Using RWD and RWE to support an ultra-rare orphan drug program Recently, Premier Consulting was engaged by a sponsor who had developed a potential treatment for an ultra-rare, life-threatening congenital disease. The FDA also granted a second meeting for review.
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