New FDA regulatory initiatives improving rare disease drug development
Listen to Parexel's SVP of Global Regulatory Strategy Mwango Kashoki, M.D., M.P.H., who is also a former FDA regulator, on how the FDA's new "plausible mechanism pathway" aims to advance individualized therapies like gene editing for serious rare diseases with no adequate treatments.
Building on recent CRISPR approvals and Rare Disease Evidence Principles, this promising development for drug innovation has the healthcare community eagerly awaiting further details to fully realize its potential for patients with very rare genetic conditions.
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