FDA looks to strength­en ex­pand­ed ac­cess pro­gram

The FDA on Thurs­day un­veiled plans to fur­ther strength­en its ex­pand­ed ac­cess pro­gram, as the con­tro­ver­sial Right to Try leg­is­la­tion has yet to help any pa­tients.

The ex­pand­ed ac­cess pro­gram al­lows pa­tients with se­ri­ous or im­me­di­ate­ly life-threat­en­ing dis­eases who are un­able to par­tic­i­pate in clin­i­cal tri­als to gain ac­cess to promis­ing in­ves­ti­ga­tion­al treat­ments.

Over the last five years, the FDA has au­tho­rized more than 9,000 ap­pli­ca­tions across drugs, bi­o­log­ics and de­vices through the agency’s ex­pand­ed ac­cess pro­gram, and the agency ex­pects about 800 more in­di­vid­ual re­quests next year. About 99% of all the re­quests the FDA has re­ceived, across all ap­pli­ca­tion types, have been grant­ed, though those re­quests are grant­ed af­ter the com­pa­ny of­fer­ing the treat­ment al­so signs off on the re­quest.

Fol­low­ing an in­de­pen­dent as­sess­ment of the ex­pand­ed ac­cess pro­gram last year, the agency says that it has now agreed to up­date the FDA’s Ex­pand­ed Ac­cess web­pages to im­prove us­abil­i­ty through the stream­lin­ing of con­tent and a more user-friend­ly or­ga­ni­za­tion. “This in­cludes a re­duc­tion in du­pli­ca­tion, as well as the ad­di­tion of new pages with com­mon­ly re­quest­ed in­for­ma­tion, such as forms and key­words,” the FDA said.

And as part of the re­cent­ly an­nounced pro­posed re­or­ga­ni­za­tion of the Of­fice of the Com­mis­sion­er, the FDA al­so in­tends to for­mal­ly es­tab­lish an agency-wide Pa­tient Af­fairs Staff and Health Care Provider Af­fairs Pro­gram, un­der the over­sight of the Of­fice of Clin­i­cal Pol­i­cy and Pro­grams.

“This will en­hance our en­gage­ment with these im­por­tant ex­ter­nal stake­hold­er groups. The Pa­tient Af­fairs Staff is al­ready in place and charged with serv­ing as the ‘home base’ and pri­ma­ry point of en­try for pa­tients and physi­cians start­ing the EA process and nav­i­gat­ing them through the steps,” the FDA said.

Mean­while, the FDA has es­tab­lished an agency-wide Ex­pand­ed Ac­cess Co­or­di­nat­ing Com­mit­tee, which fa­cil­i­tates cross-cen­ter com­mu­ni­ca­tion and pro­motes dis­cus­sion to ad­dress cross-cut­ting is­sues re­lat­ed to ex­pand­ed ac­cess to pro­mote con­sis­ten­cy and best prac­tices.

Right to Try

Mean­while, the FDA has re­leased a new web­site to help pa­tients un­der­stand Right to Try, and the agency has es­tab­lished a work group to con­sid­er what steps may be re­quired to im­ple­ment this leg­is­la­tion in a way that ad­vances Con­gress’ in­tent to pro­mote ac­cess and pro­tect pa­tients.

But such im­ple­men­ta­tion ef­forts from the FDA have al­ready proven con­tro­ver­sial, as Sen. Ron John­son (R-WI) made clear that the law “in­tends to di­min­ish the FDA’s pow­er over peo­ple’s lives.

Fo­cus reached out to two com­pa­nies ad­ver­tis­ing the use of Right to Try – Stem­Vacs and Ther­a­peu­tic So­lu­tions In­ter­na­tion­al – though nei­ther re­spond­ed.

A re­cent ar­ti­cle in Health Af­fairs on Right to Try not­ed, “In a way, pa­tients may have ben­e­fit­ed from RTT af­ter all: not be­cause it cre­at­ed a new path­way that cut the FDA out of the pic­ture, but be­cause it raised aware­ness that non-tri­al ac­cess was pos­si­ble, thus gal­va­niz­ing pa­tients and their doc­tors to re­quest it.”

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.


Zachary Brennan

managing editor, RAPS

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