What does biotech think about ‘right-to-try'? It might sur­prise you

“Right-to-try” has made its way back to the head­lines, fill­ing our Twit­ter feeds with opin­ions on whether the House should re­ject or em­brace a bill that would al­low pa­tients ac­cess to un­ap­proved ther­a­pies. We know what politi­cians think, and what the pub­lic thinks. What does biotech think?

We asked our­selves that ques­tion here at End­points News, so we craft­ed a brief sur­vey pos­ing this ques­tion to our read­ers: Do you ap­prove or dis­ap­prove of “right-to-try”?

We had ex­pec­ta­tions of what you might say. We were wrong.

The sur­vey was en­light­en­ing on sev­er­al ac­counts. The da­ta were com­pelling, but — un­sur­pris­ing­ly — the jew­els were in your com­ments. They were a far cry more thought­ful and nu­anced than the com­ment feeds of many news sites. For that, we were proud. We were sur­prised, how­ev­er, by how much the top­ic po­lar­ized you. Giv­en an au­di­ence that of­ten stands unit­ed on top­ics like these, we were in­stead in­trigued to find “right-to-try” di­vid­ed our read­ers.

Near­ly 58% of our read­ers dis­ap­prove of the cur­rent leg­is­la­tion, while 42% ap­prove of “right-to-try.”

The top­ic in­spired a del­uge of com­ments among those who re­spond­ed. With 1,194 votes on the top­ic, near­ly half — 544 —took the time to write out their thoughts on the is­sue. I read through hun­dreds of com­ments to find out what was be­hind your votes.

But first, the oblig­a­tory ex­plain­er for those of you who missed the de­bate.

In our sur­vey, those who ap­proved of the leg­is­la­tion shared over­whelm­ing­ly con­sis­tent rea­son­ing. Many had a vari­a­tion of the fol­low­ing re­sponse from an anony­mous com­menter:

Ter­mi­nal­ly ill pa­tients who have no oth­er treat­ment op­tions should be al­lowed to try a treat­ment or ther­a­py if they think it will ex­tend their life. Why should we leg­is­late treat­ments? Shouldn’t some­one be able to make their own de­ci­sion about their health?

Those who dis­ap­proved of the leg­is­la­tion, how­ev­er, had sev­er­al rea­sons why. Most com­mon was con­cern for pa­tient safe­ty. This com­menter put it most suc­cinct­ly:

It may ex­pose peo­ple with­out choice to use­less and dan­ger­ous med­i­cines lead­ing to more suf­fer­ing. There is an un­found­ed pre­sump­tion that ex­per­i­men­tal drugs work. They don’t in most of the cas­es. Ex­per­i­men­tal drugs should al­ways be test­ed un­der con­trolled con­di­tions first.

That con­cern for pa­tient safe­ty was close­ly fol­lowed by wor­ries that “right-to-try” would pose a risk to busi­ness. The fear is that phar­ma would some­how be held ac­count­able for dis­as­trous out­comes, ei­ther through lit­i­ga­tion or the bad press fol­low­ing an ex­per­i­men­tal treat­ment gone wrong.

This may be suit­able for some pa­tients with ter­mi­nal ill­ness, how­ev­er, the risks of ex­pand­ing this pol­i­cy are very dan­ger­ous and risk mak­ing a cau­tious in­dus­try even less con­fi­dent (be­cause of the risk of lit­i­ga­tion), which will sti­fle fu­ture in­no­va­tion and thus harm pa­tients fur­ther in the long run.

Or more plain­ly:

We can­not claim any good anec­do­tal da­ta but must live with all tox­i­c­i­ty da­ta.

Then there was the over­whelm­ing agree­ment among dis­senters that the bill was com­plete­ly un­nec­es­sary, as the FDA’s Ex­pand­ed Ac­cess pol­i­cy (of­ten called “com­pas­sion­ate use”) al­ready gets ex­per­i­men­tal drugs to peo­ple who are out of oth­er op­tions. The FDA al­ready ap­proves 99% of these re­quests, but some­times makes small safe­ty-re­lat­ed changes such as dos­ing or fre­quen­cy with which the pa­tient takes the drug.

Cur­rent rules give pa­tients wide ac­cess to ex­per­i­men­tal ther­a­pies. If the ad­min­is­tra­tion is con­cerned about the pro­lif­er­a­tion of reg­u­la­tions, it should avoid en­act­ing un­nec­es­sary ones! — Bernard Munos

A mech­a­nism al­ready ex­ists to make ex­per­i­men­tal drugs avail­able to pa­tients. Right-to-try is a thin­ly veiled at­tempt to re­duce the reg­u­la­to­ry au­thor­i­ty of the gov­ern­ment.

Per­haps most com­pelling were re­spons­es we re­ceived from physi­cians con­cerned that “right-to-try” would per­pet­u­ate snake oil mar­ket­ing tac­tics that prey on vul­ner­a­ble and des­per­ate pa­tients.

I have seen many pa­tients des­per­ate to do any­thing to help their loved ones in the most try­ing of times. I’ve seen a girl whose moth­er was in de­nial she had ter­mi­nal (brain can­cer), pray­ing that the ex­pen­sive moon­shot home­o­path­ic ther­a­py she had found would cure her daugh­ter. I’ve seen pa­tients claim that they had found the cure to can­cer but the rich were hid­ing it to make mon­ey off sick peo­ple. I’ve seen a man plead­ing to do­nate his or­gan to his broth­er think­ing it could save him. These pa­tients are vul­ner­a­ble. They are des­per­ate. They are in de­spair. They are griev­ing. Their worlds have been turned up­side-down. If we al­low ‘right-to-try,’ they WILL be ex­ploit­ed. They will be tak­en ad­van­tage of even by the most well-mean­ing busi­ness in­ter­ests. You need not look fur­ther than the of­ten val­ue-de­struc­tive nu­traceu­ti­cal in­dus­try to rec­og­nize that. This can­not be al­lowed. It does not em­pow­er pa­tients. It shack­les them.

The gen­er­al pub­lic is dra­mat­i­cal­ly mis­in­formed about right to try. It comes to a head dur­ing emo­tion­al times. The me­dia sen­sa­tion­al­ism of the sit­u­a­tion doesn’t help. Re­search or­ga­ni­za­tions use the words ‘life sav­ing clin­i­cal tri­al’ like they all work. This too is a dis­ser­vice. That caus­es peo­ple to feel that they are be­ing de­nied some­thing in­stead of be­ing pro­tect­ed from snake oil.

We put to­geth­er the fol­low­ing word cloud that sums of the com­mon top­ics dis­cussed in com­ments.

Be­cause they were too good to leave out, here are some more com­ments for your pe­rusal:

Pro­vid­ed pa­tients are well in­formed of the risks in­her­ent in us­ing yet-to-be-proven treat­ments (some­thing we should be good at as part of the clin­i­cal tri­als process), and on­ly for life-threat­en­ing con­di­tions and en­sur­ing there is no co­er­cion, I see no rea­son why we should not al­low pa­tients the op­por­tu­ni­ty for a chance to sur­vive. To do oth­er­wise is at least pa­tron­iz­ing, and at worst dis­em­pow­er­ing.

Right-to-try, as it is typ­i­cal­ly framed, is a cyn­i­cal ploy by priv­i­leged Amer­i­cans to sub­vert FDA’s au­thor­i­ty and es­sen­tial­ly buy a waiv­er of the rules de­signed to pro­tect all Amer­i­cans from dan­ger­ous or worth­less drugs. Like in­sur­ance, our en­tire sys­tem of clin­i­cal re­search and reg­u­lat­ed ac­cess to drugs on­ly works if every­one par­tic­i­pates, that in­cludes those who can af­ford ac­cess to ex­per­i­men­tal drugs un­der ‘right-to-try.’ ‘Right-to-try’ framed prop­er­ly would cre­ate in­cen­tives to drug spon­sors and re­quire third-par­ty pay­ers to par­tic­i­pate to en­sure lim­it­ed, reg­u­lat­ed avail­abil­i­ty of ex­per­i­men­tal drugs to Amer­i­cans with se­ri­ous med­ical con­di­tions, re­gard­less of fi­nan­cial means or sta­tus, when the op­tions to treat with ap­proved med­ica­tions or par­tic­i­pate in prop­er­ly vet­ted clin­i­cal re­search stud­ies are not fea­si­ble. The frame­work is in the law al­ready; it just needs to be im­proved up­on.

On­ly if we have the prop­er con­sent from pa­tients. We can al­so look at nov­el Com­bi­na­tions that do not get ex­e­cut­ed due to IP cor­po­rate is­sues.

This would be a BIG step back­ward to the era pri­or to the 1962 [Ke­fau­ver-Har­ris] Drug Amend­ments to the 1938 Food, Drug, and Cos­met­ic Act. Des­per­ate­ly ill peo­ple should sim­ply NOT have open ac­cess to in­ves­ti­ga­tion­al agents with un­proven ef­fi­ca­cy NOR un­proven safe­ty. Open la­bel ex­ten­sion stud­ies and ear­ly ex­pand­ed ac­cess pro­grams/com­pas­sion­ate use ex­ist, seem to in­crease ac­cess to promis­ing ther­a­pies for which there are some safe­ty and ef­fi­ca­cy da­ta, and maybe should be ex­pand­ed. Fi­nal­ly it is pay­or com­mu­ni­ty (PBM’s and gov­ern­ment bod­ies like NICE in the UK) that are re­spon­si­ble for re­strict­ing ac­cess to ap­proved prod­ucts to a much greater ex­tent than is the case for the agents un­der dis­cus­sion here. If the US Gov­ern­ment is re­al­ly in­ter­est­ed in help­ing more sick peo­ple gain ac­cess to proven much less promis­ing but un­proven ther­a­pies they are look­ing down the wrong road.

We could be back to cof­fee en­e­mas with ‘right-to-try.’ There should be eas­i­er ac­cess to drugs in de­vel­op­ment but blan­ket ‘right-to-try’ isn’t the an­swer.


Il­lus­tra­tion: Shut­ter­stock

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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UP­DAT­ED: Leg­end fetch­es $424 mil­lion, emerges as biggest win­ner yet in pan­dem­ic IPO boom as shares soar

Amid a flurry of splashy pandemic IPOs, a J&J-partnered Chinese biotech has emerged with one of the largest public raises in biotech history.

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As it hap­pened: A bid­ding war for an an­tibi­ot­ic mak­er in a mar­ket that has rav­aged its peers

In a bewildering twist to the long-suffering market for antibiotics — there has actually been a bidding war for an antibiotic company: Tetraphase.

It all started back in March, when the maker of Xerava (an FDA approved therapy for complicated intra-abdominal infections) said it had received an offer from AcelRx for an all-stock deal valued at $14.4 million.

The offer was well-timed. Xerava was approved in 2018, four years after Tetraphase posted its first batch of pivotal trial data, and sales were nowhere near where they needed to be in order for the company to keep its head above water.

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Ruffieux, a member of the pharma leadership team at Roche, spent close to 5 years at the company. But like a small army of manufacturing execs, he gained much of his experience at the other Big Pharma in Basel, remaining at Novartis for 12 years before expanding his horizons.

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AbbVie has announced a collaboration with the Netherlands’ Utrecht University and Erasmus Medical Center and the Chinese-Dutch biotech Harbour Biomed to develop a neutralizing antibody that can treat Covid-19. The antibody, called 47D11, was discovered by AbbVie’s three partners, and AbbVie will support early preclinical work, while preparing for later preclinical and clinical development. Researchers described the antibody in Nature Communications last month.

Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

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Bris­tol My­ers is clean­ing up the post-Cel­gene merg­er pipeline, and they’re sweep­ing out an ex­per­i­men­tal check­point in the process

Back during the lead up to the $74 billion buyout of Celgene, the big biotech’s leadership did a little housecleaning with a major pact it had forged with Jounce. Out went the $2.6 billion deal and a collaboration on ICOS and PD-1.

Celgene, though, also added a $530 million deal — $50 million up front — to get the worldwide rights to JTX-8064, a drug that targets the LILRB2 receptor on macrophages.

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