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. 

Susan Galbraith, AstraZeneca EVP, oncology R&D, at EUBIO22 (Rachel Kiki for Endpoints News)

Up­dat­ed: As­traZeneca jumps deep­er in­to cell ther­a­py 2.0 space with $320M biotech M&A

Right from the start, the execs at Neogene had some lofty goals in mind when they decided to try their hand at a cell therapy that could tackle solid tumors.

Its founders have helped hone a new approach that would pack in multiple neoantigen targets to create a personalized TCR treatment that would not just make the leap from blood to solid tumors, but do it with durability. And they managed to make their way rapidly to the clinic, unveiling their first Phase I program for advanced tumors just last May.

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Ei­sai’s ex­pand­ed Alzheimer’s da­ta leave open ques­tions about safe­ty and clin­i­cal ben­e­fit

Researchers still have key questions about Eisai’s investigational Alzheimer’s drug lecanemab following the publication of more Phase III data in the New England Journal of Medicine Tuesday night.

In the paper, which was released in conjunction with presentations at an Alzheimer’s conference, trial investigators write that a definition of clinical meaningfulness “has not been established.” And the relative lack of new information, following topline data unveiled in September, left experts asking for more — setting up a potential showdown to precisely define how big a difference the drug makes in patients’ lives.

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Emily Leproust, Twist Bioscience CEO

Twist Bio­science’s 'fac­to­ry of the fu­ture' in Ore­gon could de­liv­er with com­pet­i­tive pric­ing, SVB Se­cu­ri­ties says

The synthetic DNA manufacturer Twist Bioscience has given a peek behind the curtain to several analysts into its “factory of the future” as well as insight into the cost structure, workflow and technology at the site.

The 110,000-square-foot manufacturing site in the city of Wilsonville, OR, just south of Portland, which was announced back in 2020, will double Twist’s production capacity and bring around 400 jobs to the area.

Illustration: Assistant Editor Kathy Wong for Endpoints News

Twit­ter dis­ar­ray con­tin­ues as phar­ma ad­ver­tis­ers ex­tend paus­es and look around for op­tions, but keep tweet­ing

Pharma advertisers on Twitter are done — at least for now. Ad spending among the previous top spenders flattened even further last week, according to the latest data from ad tracker Pathmatics, amid ongoing turmoil after billionaire boss Elon Musk’s takeover now one month ago.

Among 18 top advertisers tracked for Endpoints News, only two are spending: GSK and Bayer. GSK spending for the full week through Sunday was minimal at just under $1,900. Meanwhile, German drugmaker Bayer remains the industry outlier upping its spending to $499,000 last week from $480,000 the previous week. Bayer’s spending also marks a big increase from a month ago and before the Musk takeover, when it spent $16,000 per week.

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Vi­a­tris with­draws ac­cel­er­at­ed ap­proval for top­i­cal an­timi­cro­bial 24 years lat­er

After 24 years without confirming clinical benefit, the FDA announced Tuesday morning that Viatris (formed via Mylan and Pfizer’s Upjohn) has decided to withdraw a topical antimicrobial agent, Sulfamylon (mafenide acetate), after the company said conducting a confirmatory study was not feasible.

Sulfamylon first won FDA’s accelerated nod in 1998 as a topical burn treatment, with the FDA noting that last December, Mylan told the agency that it wasn’t running the trial.

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Paul Hudson, Sanofi CEO (Romuald Meigneux/Sipa via AP Images)

Sanofi and DN­Di aim to elim­i­nate sleep­ing sick­ness in Africa with promis­ing Ph II/III re­sults for new drug

The Drugs for Neglected Diseases initiative (DNDi) and Sanofi today said that their potential sleeping sickness treatment saw success rates of up to 95% from a Phase II/III study investigating the safety and efficacy of single-dose acoziborole.

The potentially transformative treatment for sleeping sickness would mainly be targeted at African countries, according to data published today in The Lancet Infectious Diseases medical journal. The clinical trial was led by DNDi and its partners in the Democratic Republic of the Congo (DRC) and Guinea, with the authors noting:

Digital render of CPI's Medicines Manufacturing Innovation Centre in Glasgow, Scotland (Image: uk-cpi.com)

CPI opens the doors to a new $100M+ man­u­fac­tur­ing fa­cil­i­ty in Scot­land

A manufacturing site that has received interest and investments from large pharma companies and the UK government is opening its doors in Scotland.

The manufacturer CPI (Centre for Process Innovation) has opened a new £88 million ($105 million) “Medicines Manufacturing Innovation Centre” in Glasgow, Scotland, to accelerate the development of manufacturing tech and solve longstanding challenges in medicine development and manufacturing.

Lex­i­con slams FDA over hear­ing de­nial fol­low­ing a CRL for its SGLT2 in­hibitor can­di­date

Lexicon Pharmaceutical is not giving up on its Type I diabetes candidate, despite FDA’s repeated rejections. This week the company laid out is argument again for a hearing on sotagliflozin in response to the FDA’s most recent denial.

The issue goes back to March 2019 when the FDA made very clear to Lexicon and its now departed partner Sanofi that it would not approve their application for a potential Type I diabetes drug because it does not appear to be safe.

Pro­tect­ing its megablock­buster, Janssen chal­lenges Am­gen's Ste­lara biosim­i­lar ahead of planned 2023 launch

Johnson & Johnson unit Janssen on Wednesday sued Amgen over the company’s proposed biosimilar to its megablockbuster Stelara (ustekinumab), after Amgen said it was ready to launch next May or as soon as the FDA signs off on it.

If Amgen carries through with that plan, Janssen told the Delaware district court that the Thousand Oaks, CA-based company will infringe on at least two Janssen patents.