FDA out­lines a laun­dry list of sur­ro­gate end­points with an eye to ex­pand­ing the ros­ter for drug de­vel­op­ers

The FDA on Wednes­day pub­lished a list of sur­ro­gate end­points to help in­form drug de­vel­op­er dis­cus­sions with rel­e­vant Cen­ter for Bi­o­log­ics Eval­u­a­tion and Re­search (CBER) or Cen­ter for Drug Eval­u­a­tion and Re­search (CDER) re­view di­vi­sions.

The list, which was cre­at­ed thanks to the 21st Cen­tu­ry Cures Act, in­cludes sur­ro­gate end­points that spon­sors have used as pri­ma­ry ef­fi­ca­cy clin­i­cal tri­al end­points for ap­proval of new drug ap­pli­ca­tions (NDAs) or bi­o­log­ics li­cense ap­pli­ca­tions (BLAs). It al­so in­cludes sur­ro­gate end­points that may be ap­pro­pri­ate for use as pri­ma­ry ef­fi­ca­cy clin­i­cal tri­al end­points for drug or bi­o­log­ic ap­provals, al­though they have not yet been used to sup­port an ap­proved NDA or BLA.

The list, which will be up­dat­ed every six months, fea­tures sur­ro­gate end­points for nu­mer­ous dis­eases in­clud­ing acromegaly, dif­fer­ent can­cers, chron­ic kid­ney dis­ease, cys­tic fi­bro­sis, he­pati­tis A, B and C, HIV, hy­per­ten­sion and os­teo­poro­sis, among oth­ers.

“The ac­cept­abil­i­ty of these sur­ro­gate end­points for use in a par­tic­u­lar drug or bi­o­log­ic de­vel­op­ment pro­gram will be de­ter­mined on a case-by-case ba­sis,” the FDA said.

The list, which sep­a­rates adult and pe­di­atric end­points, does not in­clude com­pos­ite end­points that are a com­bi­na­tion of bio­mark­er sur­ro­gate end­points and clin­i­cal end­points. The ta­ble al­so does not in­clude sur­ro­gate end­points that may have been ac­cept­ed for past drug de­vel­op­ment pro­grams but are no longer ac­cept­able as an end­point to sup­port reg­is­tra­tion.

And crit­ics of the list note a lack of speci­fici­ty.

Vinay Prasad

Vinay Prasad, as­so­ciate pro­fes­sor of med­i­cine at Ore­gon Health and Sci­ence Uni­ver­si­ty, told Fo­cus: “I would say, at least for can­cer, it is done at such a high lev­el with so lit­tle de­tail, it is pret­ty much use­less. A bet­ter sum­ma­ry of sur­ro­gates used to jus­ti­fy ap­provals and their strength can be found in our pa­per from the Mayo clin­ic pro­ceed­ings…We are show­ing the sur­ro­gate used by ap­proval, the path­way of ap­proval, and the strength of the sur­ro­gate in the bio­med­ical lit­er­a­ture. They just say what sur­ro­gates are used in what tu­mors, but do not ex­plain the line of ther­a­py, the drugs that uti­lized the path­way or the strength of cor­re­la­tion.”

Back­ground

Ac­cord­ing to sec­tion 507(e)(9) of the Food Drug & Cos­met­ic Act, “[t]he term ‘sur­ro­gate end­point’ means a mark­er, such as a lab­o­ra­to­ry mea­sure­ment, ra­di­ograph­ic im­age, phys­i­cal sign, or oth­er mea­sure, that is not it­self a di­rect mea­sure­ment of clin­i­cal ben­e­fit, and (A) is known to pre­dict clin­i­cal ben­e­fit and could be used to sup­port tra­di­tion­al ap­proval of a drug or bi­o­log­i­cal prod­uct; or (B) is rea­son­ably like­ly to pre­dict clin­i­cal ben­e­fit and could be used to sup­port the ac­cel­er­at­ed ap­proval of a drug or bi­o­log­i­cal prod­uct in ac­cor­dance with sec­tion 506(c).”


First pub­lished here. Reg­u­la­to­ry Fo­cus is the flag­ship on­line pub­li­ca­tion of the Reg­u­la­to­ry Af­fairs Pro­fes­sion­als So­ci­ety (RAPS), the largest glob­al or­ga­ni­za­tion of and for those in­volved with the reg­u­la­tion of health­care and re­lat­ed prod­ucts, in­clud­ing med­ical de­vices, phar­ma­ceu­ti­cals, bi­o­log­ics and nu­tri­tion­al prod­ucts. Email news@raps.org for more in­for­ma­tion. 

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