The first pa­tient has been treat­ed un­der the con­tro­ver­sial 'right to try' law

A pa­tient di­ag­nosed with an ag­gres­sive form of brain can­cer be­came the first per­son in the US to ac­cess an ex­per­i­men­tal treat­ment un­der Right to Try. Signed in­to law by Pres­i­dent Don­ald Trump in May 2018, it has been tout­ed as a new way to help peo­ple with ter­mi­nal ill­ness­es and few op­tions, al­though un­til now, no pa­tients had ever used it.

ERC-USA and the Uni­ver­si­ty of Cal­i­for­nia, Irvine ini­ti­at­ed treat­ment with the com­pa­ny’s in­ves­ti­ga­tion­al com­pound ERC-1671 — known as Glio­vac in Eu­rope — and which is in Phase 2 clin­i­cal tri­als in the US. The pa­tient’s treat­ment with ERC-1671 be­gan at the uni­ver­si­ty in late No­vem­ber 2018.

The pa­tient re­sort­ed to Right to Try af­ter fail­ing to qual­i­fy for en­roll­ment in the on­go­ing tri­al. But rather than work around the agency that may even­tu­al­ly ap­prove the ex­per­i­men­tal treat­ment, the com­pa­ny said in Au­gust that it in­formed the FDA that it in­tend­ed to make the ex­per­i­men­tal treat­ment avail­able to this one pa­tient. FDA ac­knowl­edged ac­cep­tance of the com­pa­ny’s no­ti­fi­ca­tion on 13 Ju­ly, ERC said.

Oth­er com­pa­nies, in­clud­ing Ther­a­peu­tics So­lu­tions In­ter­na­tion­al, have an­nounced their in­ten­tions to use Right to Try, though ac­cord­ing to Gold­wa­ter In­sti­tute, no oth­er pa­tients have yet to use the new law.

The law has proven to be a thorn in the side of FDA, which has said it will work to im­ple­ment it in a man­ner con­sis­tent with con­gres­sion­al in­tent and with FDA’s pub­lic health mis­sion, but the agency still has its own process for help­ing ter­mi­nal­ly ill pa­tients re­ceive ex­per­i­men­tal treat­ments, known as ex­pand­ed ac­cess. FDA grants about 99% of the ex­pand­ed ac­cess re­quests it re­ceives.

Ac­cord­ing to Bio­Cen­tu­ry, FDA’s On­col­o­gy Cen­ter of Ex­cel­lence is al­so work­ing on a new ini­tia­tive, known as Pro­ject Fa­cil­i­tate, un­der which the agency will pro­vide a tele­phone num­ber that pa­tients and physi­cians seek­ing ex­pand­ed ac­cess to an ex­per­i­men­tal treat­ment can call. FDA staff will an­swer calls and fill out the form re­quired to ap­ply for a sin­gle-pa­tient IND re­quest. The pa­per­work will be for­ward­ed to the man­u­fac­tur­er. A pi­lot ver­sion of this ini­tia­tive is ex­pect­ed to launch in the first half of 2019.


First pub­lished here and syn­di­cat­ed in part­ner­ship with RAPS.

Author

Zachary Brennan

managing editor, RAPS

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