Di­min­ish­ing the FDA’s pow­er was my in­tent: Right-to-try au­thor scolds Scott Got­tlieb as agency im­ple­ments new law

In a bizarre twist for the new­ly signed “right-to-try” law, the FDA — cut out of the process of sign­ing off on cer­tain us­es of ex­per­i­men­tal drugs for ter­mi­nal­ly ill pa­tients by that law — now has to fig­ure out how to im­ple­ment it. And al­ready, the bill’s main au­thor is un­hap­py with com­mis­sion­er Scott Got­tlieb in the af­ter­math and has point­ed­ly made his feel­ings known in a let­ter.

“This law in­tends to di­min­ish the FDA’s pow­er over peo­ple’s lives, not in­crease it,” Sen­a­tor Ron John­son, Re­pub­li­can from Wis­con­sin, wrote to Got­tlieb yes­ter­day, dis­pleased over tweets and state­ments the com­mis­sion­er has made re­cent­ly around im­ple­men­ta­tion of the law. The state­ment is re­mark­able be­cause the is­sue has long been framed by “right-to-try” sup­port­ers as a win for ter­mi­nal­ly ill pa­tients and not an end-around to weak­en the FDA.

As we have re­port­ed, “right-to-try” is a land­mark win for the lib­er­tar­i­an Gold­wa­ter In­sti­tute and its po­lit­i­cal al­lies.

The FDA says it will re­view and di­rect­ly re­ply to Sen­a­tor John­son’s let­ter.

Be­fore the sen­a­tor’s let­ter was pub­lished yes­ter­day, the agency said in an emailed state­ment that it “is con­ven­ing an in­ter­nal group to as­sess how to ef­fec­tive­ly and ef­fi­cient­ly im­ple­ment the new law. We will re­port on our im­ple­men­ta­tion steps reg­u­lar­ly.”

In an email to FDA’s Cen­ter for Drug Eval­u­a­tion and Re­search (CDER) staff yes­ter­day, CDER Di­rec­tor Janet Wood­cock said: “We are re­view­ing the leg­is­la­tion and will work to im­ple­ment it in a man­ner con­sis­tent with Con­gres­sion­al in­tent and with FDA’s pub­lic health mis­sion. We re­al­ize that you may re­ceive ques­tions about this process. We will be work­ing ex­pe­di­tious­ly to de­vel­op fur­ther in­for­ma­tion on how to re­spond to such in­quiries. How­ev­er, we be­lieve that spon­sors are in the best po­si­tion to pro­vide in­for­ma­tion on the de­vel­op­ment sta­tus of their prod­ucts (which is crit­i­cal to de­ter­min­ing whether a drug or bi­o­log­i­cal prod­uct is el­i­gi­ble for use un­der Right to Try) and whether a spon­sor in­tends to make an in­ves­ti­ga­tion­al prod­uct avail­able un­der Right to Try.”

Pres­i­dent Don­ald Trump signed in­to law the Trick­ett Wendler, Frank Mongiel­lo, Jor­dan McLinn, and Matthew Bel­li­na Right to Try Act of 2017 (Right to Try Act) Wednes­day to amend the Fed­er­al Food, Drug, and Cos­met­ic Act.

Wood­cock said the law “is in­tend­ed to in­crease ac­cess to cer­tain un­ap­proved, in­ves­ti­ga­tion­al treat­ments (drugs or bi­o­log­i­cal prod­ucts) for pa­tients di­ag­nosed with life-threat­en­ing dis­eases or con­di­tions who have ex­haust­ed ap­proved treat­ment op­tions and who are un­able to par­tic­i­pate in a clin­i­cal tri­al in­volv­ing the in­ves­ti­ga­tion­al drug, as cer­ti­fied by a physi­cian.‎”

She al­so ex­plained what el­i­gi­ble pa­tients and treat­ments are for ‘right-to-try’:

An el­i­gi­ble pa­tient is one with a life-threat­en­ing dis­ease as de­fined in 21 CFR 312.81 and who meets cer­tain oth­er con­di­tions set forth in the statute. An in­ves­ti­ga­tion­al drug or bi­o­log­i­cal prod­uct can on­ly be pro­vid­ed un­der Right to Try if it:

  • Is not ap­proved or li­censed for any use;
  • Has com­plet­ed a Phase 1 tri­al;
  • Ei­ther (1) is the sub­ject of an NDA or BLA filed with FDA or (2) is the sub­ject of an ac­tive IND, and is un­der in­ves­ti­ga­tion in a clin­i­cal tri­al that ‘is in­tend­ed to form the pri­ma­ry ba­sis of a claim of ef­fec­tive­ness’; and
  • Is ac­tive­ly be­ing de­vel­oped (‘ac­tive de­vel­op­ment…is on­go­ing’), not dis­con­tin­ued, and not on clin­i­cal hold.

“In the near term,” Wood­cock told staff, “if you re­ceive in­quiries about the leg­is­la­tion from pa­tients or physi­cians about a spe­cif­ic prod­uct, please re­fer them to the spon­sor of the in­ves­ti­ga­tion­al drug or bi­o­log­i­cal prod­uct. If spon­sors con­tact you re­gard­ing their oblig­a­tions un­der this law, we sug­gest that you re­fer them to the statute. If you re­ceive more de­tailed ques­tions re­gard­ing Right to Try, please re­fer those in­quiries to Drug­in­fo@fda.hhs.gov or by phone: (855) 543-3784.”

Hol­ly Fer­nan­dez Lynch of the De­part­ment of Med­ical Ethics and Health Pol­i­cy at Perel­man School of Med­i­cine, Uni­ver­si­ty of Penn­syl­va­nia, told Fo­cus: “I think there is lots of room for FDA to pro­tect pa­tients from po­ten­tial­ly dan­ger­ous ef­fects of the law, which I hope they will im­ple­ment through rule­mak­ing and guid­ance, al­though that will take time.”

She al­so of­fered a few pos­si­bil­i­ties for im­ple­men­ta­tion:

  1. Clar­i­fy that it is not enough to have a life-threat­en­ing ill­ness, but rather that the ill­ness should be im­me­di­ate­ly life-threat­en­ing.
  2. Re­quire ad­verse event re­port­ing not via the right to try law, but rather un­der the terms of the over­ar­ch­ing IND for the drug (this may be tricky, but not en­tire­ly im­plau­si­ble).
  3. Clar­i­fy what it means for a Phase 1 tri­al to be com­plet­ed, i.e., what is suc­cess?
  4. Re­quire that spon­sors de­vel­op con­tracts with cer­ti­fy­ing physi­cians that in or­der to ac­cess the drug, the physi­cian must col­lect and re­port safe­ty da­ta.
  5. Spec­i­fy the sorts of ad­verse event da­ta that needs to be col­lect­ed in or­der to sat­is­fy the re­quire­ments of the an­nu­al sum­ma­ry.
  6. Post as much in­for­ma­tion pub­licly as pos­si­ble, to help pa­tients and their physi­cians un­der­stand that the un­like­li­hood that right to try will be help­ful.
  7. En­cour­age pa­tients and com­pa­nies to uti­lize the ex­pand­ed ac­cess path­way rather than right to try.
  8. En­cour­age spon­sors and in­ves­ti­ga­tors to al­low for the broad­est clin­i­cal tri­al in­clu­sion con­sis­tent with par­tic­i­pant safe­ty and sci­en­tif­ic in­tegri­ty.

Ar­salan Arif con­tributed re­port­ing to this sto­ry.

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. 

Author

Zachary Brennan

managing editor, RAPS

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