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. 

Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

Amid mon­key­pox fears, biotechs spring to ac­tion; Mod­er­na’s CFO trou­ble; Cuts, cuts every­where; Craft­ing the right pro­teins; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

It’s always a bittersweet moment saying goodbye, but as Josh Sullivan goes off to new adventures we are grateful for the way he’s built up the Endpoints Manufacturing section — which the rest of the team will now carry forward. If you’re not already, this may be a good time to sign up for your weekly dose of drug manufacturing news. Thank you for reading and wish you a restful weekend.

Bay­er sounds re­treat from a $670 mil­lion CAR-T pact in the wake of a pa­tient death

Two months after Atara Biotherapeutics hit the hold button on its lead CAR-T 2.0 therapy following a patient death, putting the company under the watchful eye of the FDA, its Big Pharma partners at Bayer are bowing out of a $670 million global alliance. And the move is forcing a revamp of Atara’s pipeline plans, even as research execs vow to continue work on the two drugs allied with Bayer 18 months ago, which delivered a $60 million cash upfront.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 142,600+ biopharma pros reading Endpoints daily — and it's free.

Sanofi and Re­gen­eron clear the fin­ish line in an in­flam­ma­to­ry esoph­a­gus dis­ease, leav­ing Take­da in the dust

With atopic dermatitis rivals breathing down Dupixent’s neck, Sanofi and Regeneron on Friday secured a first win in new territory in what Sanofi’s head of immunology and inflammation Naimish Patel called the fastest approval he’s ever seen.

The FDA approved Dupixent on Friday to treat patients 12 years and older with eosinophilic esophagitis (EoE), an inflammatory condition that causes swelling and scarring of the esophagus. The approval came just a couple months after regulators granted Dupixent priority review, and months ahead of its PDUFA date on Aug. 3.

Fu­ji­film con­tin­ues its biotech build­ing spree with new fa­cil­i­ty in Chi­na

A Japanese conglomerate is making a big play in China with the opening of a new facility, as it continues to expand.

Fujifilm Irvine Scientific has opened its new Innovation and Collaboration Center in Suzhou New District, China, an area in Jiangsu province specifically designated for technological and industrial development.

According to Fujifilm, the 12,000-square-foot site will be responsible for the company’s cell culture media optimization, analysis and design services. Cell culture media itself often requires customization of formulas and protocols to achieve the desired quantity and quality of therapeutic desired. Fujifilm Irvine Scientific is offering these services from its headquarters in California and Japan to its customers globally, as well as in China now.

Rob Etherington, Clene CEO

Mary­land of­fers loan to Clene de­spite ALS tri­al bumps

Even after Utah-based Clene failed to hit its primary endpoints for its ALS drug last year, the state of Maryland is putting its money at least behind Clene’s manufacturing facility.

The Maryland Board of Public Works has finalized a $3 million, 60-month loan facility with Clene Nanomedicine. The loan was provided by the state’s Neighborhood BusinessWorks program within the Maryland Department of Housing and Community Development.

Armon Sharei, SQZ founder and CEO

SQZ's out­side-the-box man­u­fac­tur­ing method slash­es pro­duc­tion time in ear­ly in study

At ASCO 2021 in June of last year, SQZ Biotech showcased a glimpse of its unorthodox cell therapy manufacturing tech. And on Wednesday, the Watertown, MA, company announced that its first-generation system showed comparable or better performance than a conventional clean-room-based manufacturing process.

The study was non-clinical. Clinical trials are expected by the first half of 2023.

SQZ’s device opens up a temporary window by cell-squeezing to deliver cargoes into cells. Its average processing time was less than six hours per batch, which is more than half the time than conventional methods. The company is planning to use the technology in its first red blood cell derived program for celiac disease. That IND is set to be submitted in the first half of 2023, the company said.

Bobby Sheng, Bora Pharmaceuticals CEO

With new ac­qui­si­tion, Bo­ra to ven­ture in­to bi­o­log­ics

Last week, Taiwan-based CDMO Bora Pharmaceuticals announced that it acquired Eden Biologics. Now, it says that purchase has helped established Bora Biologics, expanding into the biopharmaceutical market.

The acquisition of the company’s assets, which are located in the Hsinchu Biomedical Science Park in Taiwan, is helping Bora build its presence in the biopharma world by expanding production capacity of cell lines for the production of protein drugs. It will also improve the quality control and inspection specifications, as well as cell bank generation. The facility has four 500-liter bioreactors that have been approved by European and Taiwanese regulators.

Paul Chaplin, Bavarian Nordic president and CEO

With mon­key­pox cas­es ris­ing, one Eu­ro­pean coun­try is lock­ing down a small­pox vac­cine con­tract

As the global number of confirmed and suspected monkeypox cases continues to slowly climb, one country is trying to get a head start on potential vaccine stocking.

Bavarian Nordic signed a contract with an undisclosed European nation to supply its smallpox vaccine in response to new cases this month, the company announced Thursday morning. The continent saw its first monkeypox case confirmed about two weeks ago, with both the UK and Portugal seeing cases, according to the Washington Post.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 142,600+ biopharma pros reading Endpoints daily — and it's free.