House pass­es 'right-to-try' — al­low­ing the ter­mi­nal­ly ill to ask com­pa­nies for ex­per­i­men­tal drugs out­side of FDA's purview

The House on Tues­day af­ter­noon passed a Sen­ate-rat­i­fied ver­sion of a bill, known as “right-to-try,” and Pres­i­dent Don­ald Trump has in­di­cat­ed he will sign it — fur­ther proof that the Koch broth­ers-backed Gold­wa­ter In­sti­tute’s politi­ciza­tion of the US Food and Drug Ad­min­is­tra­tion (FDA) is just be­gin­ning.

Rid­ing on the backs of ter­mi­nal­ly ill pa­tients who have run out of op­tions, Re­pub­li­cans pegged the bill as a last-ditch ef­fort to “try any­thing.” At an ear­li­er hear­ing, Rep. Mor­gan Grif­fith (R-VA) even went so far as to say that he would in­ject mon­key urine in­to him­self if “faced with one of these heart-rend­ing sit­u­a­tions.”

The bill’s back­ers, though, have yet to pub­licly dis­close why the pro­gram is specif­i­cal­ly nec­es­sary, or if there are ex­am­ples of ter­mi­nal­ly ill pa­tients who will gain ac­cess to in­ves­ti­ga­tion­al treat­ments be­cause of “right-to-try” (com­pa­nies are still al­lowed to re­ject re­quests) and would not have un­der FDA’s ex­pand­ed ac­cess pro­gram.

The House’s pas­sage of the Sen­ate bill from last Au­gust — af­ter a pre­vi­ous House bill passed but was not tak­en up by the Sen­ate, and af­ter a pre­vi­ous House ver­sion failed to pass — al­so fol­lows the right-to-try move­ment’s spread across state leg­is­la­tures. Pres­i­dent Don­ald Trump has said he would sign the bill and Vice Pres­i­dent Mike Pence said it was a pri­or­i­ty for him.

But Rep. Frank Pal­lone (D-NJ) called the bill “dan­ger­ous,” “not nec­es­sary” and said it “need­less­ly ex­pos­es pa­tients to un­known risks.” This bill is worse than what the House passed pri­or, Pal­lone said, not­ing a loop­hole in it that could ex­pand the num­ber of peo­ple than can use in­ves­ti­ga­tion­al drugs and are not ter­mi­nal­ly ill.

Pub­lic health ex­perts, in­clud­ing FDA Com­mis­sion­er Scott Got­tlieb (though he has since said he backs the bill and that there’s lee­way for im­ple­men­ta­tion), had raised con­cerns on var­i­ous ver­sions of the bill, par­tic­u­lar­ly as FDA al­ready has its own ver­sion of “right-to-try,” known as ex­pand­ed ac­cess, and the agency ap­proves more than 99% of all ex­pand­ed ac­cess re­quests.

Crit­ics of the ex­pand­ed ac­cess pro­gram have said it’s un­nec­es­sar­i­ly bur­den­some, though FDA has made clear that it can re­spond to an emer­gency re­quest with­in 24 hours.

Re­gard­less, with a right-to-try win un­der its belt, Gold­wa­ter now has its sights on eas­ing the off-la­bel pro­mo­tion of med­i­cines and fur­ther ef­forts to un­der­mine the FDA.

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 for more in­for­ma­tion.

Im­age: Shut­ter­stock


Zachary Brennan

managing editor, RAPS

Hal Barron, GSK

Break­ing the death spi­ral: Hal Bar­ron talks about trans­form­ing the mori­bund R&D cul­ture at GSK in a crit­i­cal year for the late-stage pipeline

Just ahead of GlaxoSmithKline’s Q2 update on Wednesday, science chief Hal Barron is making the rounds to talk up the pharma giant’s late-stage strategy as the top execs continue to woo back a deeply skeptical investor group while pushing through a whole new R&D culture.

And that’s not easy, Barron is quick to note. He told the Financial Times:

I think that culture, to some extent, is as hard, in fact even harder, than doing the science.

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Some Big Phar­mas stepped up their game on da­ta trans­paren­cy — but which flunked the test?

The nonprofit Bioethics International has come out with their latest scorecard on data transparency among the big biopharmas in the industry — flagging a few standouts while spotlighting some laggards who are continuing to underperform.

Now in its third year, the nonprofit created a new set of standards with Yale School of Medicine and Stanford Law School to evaluate the track record on trial registration, results reporting, publication and data-sharing practice.

Busy Gilead crew throws strug­gling biotech a life­line, with some cash up­front and hun­dreds of mil­lions in biobucks for HIV deal

Durect $DRRX got a badly needed shot in the arm Monday morning as Gilead’s busy BD team lined up access to its extended-release platform tech for HIV and hepatitis B.

Gilead, a leader in the HIV sector, is paying a modest $25 million in cash for the right to jump on the platform at Durect, which has been using its technology to come up with an extended-release version of bupivacaine. The FDA rejected that in 2014, but Durect has been working on a comeback.

In­tec blitzed by PhI­II flop as lead pro­gram fails to beat Mer­ck­'s stan­dard com­bo for Parkin­son’s

Intec Pharma’s $NTEC lead drug slammed into a brick wall Monday morning. The small-cap Israeli biotech reported that its lead program — coming off a platform designed to produce a safer, more effective oral drug for Parkinson’s — failed the Phase III at the primary endpoint.

Researchers at Intec, which has already seen its share price collapse over the past few months, says that its Accordion Pill-Carbidopa/Levodopa failed to prove superior to Sinemet in reducing daily ‘off’ time. 

Cel­gene racks up third Ote­zla ap­proval, heat­ing up talks about who Bris­tol-My­ers will sell to

Whoever is taking Otezla off Bristol-Myers Squibb’s hands will have one more revenue stream to boast.

The drug — a rising star in Celgene’s pipeline that generated global sales of $1.6 billion last year — is now OK’d to treat oral ulcers associated with Behçet’s disease, a common symptom for a rare inflammatory disorder. This marks the third FDA approval for the PDE4 inhibitor since 2014, when it was greenlighted for plaque psoriasis and psoriatic arthritis.

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Vlad Coric (Biohaven)

In an­oth­er dis­ap­point­ment for in­vestors, FDA slaps down Bio­haven’s re­vised ver­sion of an old ALS drug

Biohaven is at risk of making a habit of disappointing its investors.

Late Friday the biotech $BHVN reported that the FDA had rejected its application for riluzole, an old drug that they had made over into a sublingual formulation that dissolves under the tongue. According to Biohaven, the FDA had a problem with the active ingredient used in a bioequivalence study back in 2017, which they got from the Canadian drugmaker Apotex.

Apotex, though, has been a disaster ground. The manufacturer voluntarily yanked the ANDAs on 31 drugs — in late 2017 — after the FDA came across serious manufacturing deficiencies at their plants in India. A few days ago, the FDA made it official.

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Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

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Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

They kept a massive number of people alive who would otherwise have been facing a death sentence. And they made money.

And throughout, John Pottage has been the chief scientific and chief medical officer.

Until now.

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Chas­ing Roche's ag­ing block­buster fran­chise, Am­gen/Al­ler­gan roll out Avastin, Her­ceptin knock­offs at dis­count

Let the long battle for biosimilars in the cancer space begin.

Amgen has launched its Avastin and Herceptin copycats — licensed from the predecessors of Allergan — almost two years after the FDA had stamped its approval on Mvasi (bevacizumab-awwb) and three months after the Kanjinti OK (trastuzumab-anns). While the biotech had been fielding biosimilars in Europe, this marks their first foray in the US — and the first oncology biosimilars in the country.