Lawmakers push for faster, conditional FDA approval pathway for rare disease drugs

As the FDA tight­ens its fo­cus on rare dis­ease treat­ments and speed­ing their en­try to mar­ket, law­mak­ers on Capi­tol Hill are putting se­ri­ous stock in a new ap­proval path­way that would speed new treat­ments for rare dis­eases to pa­tients even faster.

At a hear­ing Thurs­day be­fore the Sen­ate ag­ing com­mit­tee, Sen. Mike Braun (R-IN) tout­ed his Promis­ing Path­way Act, which would es­tab­lish a rolling pri­or­i­ty re­view path­way for drugs to treat life-threat­en­ing dis­eases or con­di­tions. The bill, in­tro­duced in June by a bi­par­ti­san group of sen­a­tors led by Braun and Kirsten Gilli­brand (D-NY), would cre­ate a new way for the FDA to grant time-lim­it­ed pro­vi­sion­al ap­proval to drugs that sub­stan­tial­ly demon­strate ear­ly ev­i­dence of safe­ty and ef­fi­ca­cy.

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