FDA of­fers more guid­ance on re­ly­ing on just one tri­al and con­fir­ma­to­ry ev­i­dence for a new drug ap­proval

Al­though FDA isn’t chang­ing what it re­quires for a new drug to win ap­proval (that hasn’t changed since 1998), the agency on Mon­day re­leased new draft guid­ance to bet­ter de­scribe how one ad­e­quate and well-con­trolled clin­i­cal in­ves­ti­ga­tion and con­fir­ma­to­ry ev­i­dence — in­clud­ing re­al-world ev­i­dence and even ex­pand­ed ac­cess da­ta — can be used to meet the agency’s sub­stan­tial ev­i­dence re­quire­ment.

Build­ing off pri­or guid­ance from 1998 and 2019, the agency says that the quan­ti­ty of con­fir­ma­to­ry ev­i­dence need­ed in any giv­en de­vel­op­ment pro­gram seek­ing to go this route “will be im­pact­ed by the fea­tures of, and re­sults from, the sin­gle ad­e­quate and well-con­trolled clin­i­cal in­ves­ti­ga­tion that the con­fir­ma­to­ry ev­i­dence is in­tend­ed to sub­stan­ti­ate,” ac­cord­ing to a Fed­er­al Reg­is­ter no­tice pub­lished Mon­day.

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