FDA’s plans for revamping rare disease regulation

Eager to demonstrate tangible successes in speeding reviews of new medicines and clearing away outmoded regulatory barriers, FDA Commissioner Marty Makary and senior FDA leaders are prioritizing reforms to the regulatory framework for therapies to treat rare diseases.

Vinay Prasad, the newly appointed director of the Center for Biologics Evaluation and Review (CBER), joined senior agency career staff from CBER and the Center for Drug Evaluation and Research (CDER) this week in reaffirming the agency’s commitment to the rare disease community in remarks to the National Organization for Rare Disorders (NORD) scientific symposium. ...