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. 

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

FDA commissioner Stephen Hahn at the White House (AP Images)

Un­der fire, FDA to is­sue stricter guid­ance for Covid-19 vac­cine EUA this week — re­port

The FDA has been insisting for months that a Covid-19 vaccine had to be at least 50% effective – a measure of transparency meant to shore public trust in the agency and in a vaccine that had been brought forward at record speed and record political pressure. But now, with concerns of a Trump-driven authorization arriving before the election, the agency may be raising the bar.

The FDA is set to release new guidance that would raise safety and efficacy requirements for a vaccine EUA above earlier guidance and above the criteria used for convalescent plasma or hydroxychloroquine, The Washington Post reported. Experts say this significantly lowers the odds of an approval before the election on November 3, which Trump has promised despite vocal concerns from public health officials.

#ES­MO20: Push­ing in­to front­line, Mer­ck and Bris­tol My­ers duke it out with new slate of GI can­cer da­ta

Having worked in parallel for years to move their respective PD-1 inhibitors up to the first-line treatment of gastrointestinal cancers, Merck and Bristol Myers Squibb finally have the data at ESMO for a showdown.

Comparing KEYNOTE-590 and CheckMate-649, of course, comes with the usual caveats. But a side-by-side look at the overall survival numbers also offer some perspective on a new frontier for the reigning checkpoint rivals, both of whom are claiming to have achieved a first.

Samit Hirawat (Bristol Myers Squibb)

Af­ter bruis­ing re­jec­tion, blue­bird and Bris­tol My­ers Squibb land ide-cel pri­or­i­ty re­view. But will it mat­ter for the CVR?

With the clock all but up, the FDA accepted and handed priority review to Bristol Myers Squibb and bluebird bio’s BCMA CAR-T, keeping a narrow window open for Celgene investors to still cash in on the $9 CVR from the $63 billion Celgene merger.

The acceptance comes five months after the two companies weres slammed with a surprise refuse-to-file that threatened to foreclose the CVR entirely. Today’s acceptance sets the FDA decision date for March 27, 2021 – or precisely 4 days before the CVR deadline of March 31. Given the breakthrough designation and strong pivotal data — 81.5% response rate, 35.2% complete response rate — priority review was largely expected.

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Blueprint CEO Jeff Albers (file photo)

Blue­print plots re­turn to FDA with new Ay­vak­it da­ta in rare con­di­tion — and the an­a­lysts cheer

Over a decade after launch, Blueprint Medicines nabbed the first approval for their first drug earlier this year. Now, as they move forward with a Roche-partnered global launch, they’re touting data that could push them into more patients.

The Jeff Albers-led Cambridge biotech released their full pivotal data for Ayvakit in patients with advanced systemic mastocytosis. In one 53-person study, they showed that 76% of patients responded to the drug, 36% had complete responses and that on average their responses lasted for just over 3 years. A smaller, 32-patient study had a 75% response rate and most were still responding after 10.4 months, the last follow-up.

President Donald Trump (via AP Images)

Signs of an 'Oc­to­ber Vac­cine Sur­prise' alarm ca­reer sci­en­tists. HHS con­tin­ues to claim Azar “will de­fer com­plete­ly to the FDA"

President Donald Trump, who seems intent on announcing a Covid-19 vaccine before Election Day, could legally authorize a vaccine over the objections of experts, officials at the FDA and even vaccine manufacturers, who have pledged not to release any vaccine unless it’s proved safe and effective.

In podcasts, public forums, social media and medical journals, a growing number of prominent health leaders say they fear that Trump — who has repeatedly signaled his desire for the swift approval of a vaccine and his displeasure with perceived delays at the FDA — will take matters into his own hands, running roughshod over the usual regulatory process.

#ES­MO20: Bris­tol My­ers marks Op­di­vo's sec­ond ad­ju­vant win — eye­ing a stan­dard of care gap

Moving into earlier and earlier treatment lines, Bristol Myers Squibb is reporting that adjuvant treatment with Opdivo has doubled the time that esophageal or gastroesophageal junction cancer patients stay free of disease.

With the CheckMate-577 data at ESMO, CMO Samit Hirawat said, the company believes it can change the treatment paradigm.

While a quarter to 30% of patients typically achieve a complete response following chemoradiation therapy and surgery, the rest do not, said Ronan Kelly of Baylor University Medical Center. The recurrence rate is also high within the first year, Hirawat added.

UP­DAT­ED: Two wild weeks for Grail end in $8B Il­lu­mi­na buy­out

Grail’s whirlwind two weeks have ended in the wealthy arms of its former founder and benefactors.

Illumina has shelled out $8 billion to reacquire the closely-watched liquid biopsy startup they spun out just 5 years ago and sold off much of its shares just 3 years ago. The deal comes nearly two weeks after the well-heeled startup filed for a potentially massive IPO — one that was disrupted just a week later when Bloomberg reported that Illumina was in talks to buy their former spinout for up to $8 billion.

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