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

The top 10 block­buster drugs in the late-stage pipeline — Eval­u­ate adds 6 new ther­a­pies to heavy-hit­ter list

Vertex comes in for a substantial amount of criticism for its no-holds-barred tactical approach toward wresting the price it wants for its commercial drugs in Europe. But the flip side of that coin is a highly admired R&D and commercial operation that regularly wins kudos from analysts for their ability to engineer greater cash flow from the breakthrough drugs they create.

Both aspects needed for success in this business are on display in the program backing Vertex’s triple for cystic fibrosis. VX-659/VX-445 + Tezacaftor + Ivacaftor — it’s been whittled down to 445 now — was singled out by Evaluate Pharma as the late-stage therapy most likely to win the crown for drug sales in 5 years, with a projected peak revenue forecast of $4.3 billion.

The latest annual list, which you can see here in their latest world preview, includes a roster of some of the most closely watched development programs in biopharma. And Evaluate has added 6 must-watch experimental drugs to the top 10 as drugs fail or go on to a first approval. With apologies to the list maker, I revamped this to rank the top 10 by projected 2024 sales, instead of Evaluate's net present value rankings.

It's how we roll at Endpoints News.

Here is a quick summary of the rest of the top 10:

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How small- to mid-sized biotechs can adopt pa­tient cen­tric­i­ty in their on­col­o­gy tri­als

By Lucy Clos­sick Thom­son, Se­nior Di­rec­tor of On­col­o­gy Pro­ject Man­age­ment, Icon

Clin­i­cal tri­als in on­col­o­gy can be cost­ly and chal­leng­ing to man­age. One fac­tor that could re­duce costs and re­duce bar­ri­ers is har­ness­ing the pa­tient voice in tri­al de­sign to help ac­cel­er­ate pa­tient en­roll­ment. Now is the time to adopt pa­tient-cen­tric strate­gies that not on­ly fo­cus on pa­tient needs, but al­so can main­tain cost ef­fi­cien­cy.

John Reed at JPM 2019. Jeff Rumans for Endpoints News

Sanofi's John Reed con­tin­ues to re­or­ga­nize R&D, cut­ting 466 jobs while boost­ing can­cer, gene ther­a­py re­search

The R&D reorganization inside Sanofi is continuing, more than a year after the pharma giant brought in John Reed to head the research arm of the Paris-based company.
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UP­DAT­ED: Chica­go biotech ar­gues blue­bird, Third Rock 'killed' its ri­val, pi­o­neer­ing tha­lassemia gene ther­a­py in law­suit

Blue­bird bio $BLUE chief Nick Leschly court­ed con­tro­ver­sy last week when he re­vealed the com­pa­ny’s be­ta tha­lassemia treat­ment will car­ry a jaw-drop­ping $1.8 mil­lion price tag over a 5-year pe­ri­od in Eu­rope — mak­ing it the plan­et’s sec­ond most ex­pen­sive ther­a­py be­hind No­var­tis’ $NVS fresh­ly ap­proved spinal mus­cu­lar at­ro­phy ther­a­py, Zol­gens­ma, at $2.1 mil­lion. A Chica­go biotech, mean­while, has been fum­ing at the side­lines. In a law­suit filed ear­li­er this month, Er­rant Gene Ther­a­peu­tics al­leged that blue­bird and ven­ture cap­i­tal group Third Rock un­law­ful­ly prised a vi­ral vec­tor, de­vel­oped in part­ner­ship with the Memo­r­i­al Sloan Ket­ter­ing Can­cer Cen­ter (MSK), from its grasp, and thwart­ed the de­vel­op­ment of its sem­i­nal gene ther­a­py.

A new num­ber 1 drug? Keytru­da tapped to top the 10 biggest block­busters on the world stage by 2024

Analysts may be fretting about Keytruda’s longterm prospects as a host of rival therapies elbow their way to the market. But the folks at Evaluate Pharma are confident that last year’s $7 billion earner is headed for glory, tapping it to beat out the current #1 therapy Humira as AbbVie watches that franchise swoon over the next 5 years.

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John Chiminski, Catalent CEO - File Photo

'It's a growth play': Catal­ent ac­quires Bris­tol-My­er­s' Eu­ro­pean launch pad, ex­pand­ing glob­al CD­MO ops

Catalent is staying on the growth track.

Just two months after committing $1.2 billion to pick up Paragon and take a deep dive into the sizzling hot gene therapy manufacturing sector, the CDMO is bouncing right back with a deal to buy out Bristol-Myers’ central launchpad for new therapies in Europe, acquiring a complex in Anagni, Italy, southwest of Rome, that will significantly expand its capacity on the continent.

There are no terms being offered, but this is no small deal. The Anagni campus employs some 700 staffers, and Catalent is planning to go right in — once the deal closes late this year — with a blueprint to build up the operations further as they expand on oral solid, biologics, and sterile product manufacturing and packaging.

This is an uncommon deal, Catalent CEO John Chiminski tells me. But it offers a shortcut for rapid growth that cuts years out of developing a green fields project. That’s time Catalent doesn’t have as the industry undergoes unprecedented expansion around the world.

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Arc­turus ex­pands col­lab­o­ra­tion, adding $30M cash; Ku­ra shoots for $100M raise

→  Rare dis­ease play­er Ul­tragenyx $RARE is ex­pand­ing its al­liance with Arc­turus $ARCT, pay­ing $24 mil­lion for eq­ui­ty and an­oth­er $6 mil­lion in an up­front as the two part­ners ex­pand their col­lab­o­ra­tion to in­clude up to 12 tar­gets. “This ex­pand­ed col­lab­o­ra­tion fur­ther so­lid­i­fies our mR­NA plat­form by adding ad­di­tion­al tar­gets and ex­pand­ing our abil­i­ty to po­ten­tial­ly treat more dis­eases,” said Emil Kakkis, the CEO at Ul­tragenyx. “We are pleased with the progress of our on­go­ing col­lab­o­ra­tion. Our most ad­vanced mR­NA pro­gram, UX053 for the treat­ment of Glyco­gen Stor­age Dis­ease Type III, is ex­pect­ed to move in­to the clin­ic next year, and we look for­ward to fur­ther build­ing up­on the ini­tial suc­cess of this part­ner­ship.”

Neil Woodford. Woodford Investment Management via YouTube

Wood­ford braces po­lit­i­cal storm as UK fi­nan­cial reg­u­la­tors scru­ti­nize fund sus­pen­sion

The shock of Neil Wood­ford’s de­ci­sion to block with­drawals for his flag­ship fund is still rip­pling through the rest of his port­fo­lio — and be­yond. Un­der po­lit­i­cal pres­sure, UK fi­nan­cial reg­u­la­tors are now tak­ing a hard look while in­vestors con­tin­ue to flee.

In a re­sponse let­ter to an MP, the Fi­nan­cial Con­duct Au­thor­i­ty re­vealed that it’s opened an in­ves­ti­ga­tion in­to the sus­pen­sion fol­low­ing months of en­gage­ment with Link Fund So­lu­tions, which tech­ni­cal­ly del­e­gat­ed Wood­ford’s firm to man­age its funds.

Gilead baits new al­liance with $45M up­front, div­ing in­to the busy pro­tein degra­da­tion field

Gilead is jump­ing on board the pro­tein degra­da­tion band­wag­on. And they’re turn­ing to a low-pro­file Third Rock start­up for the ex­per­tise. But if you were look­ing for a trans­for­ma­tion­al deal to kick up fresh en­thu­si­asm for Gilead, you’ll have to re­main pa­tient.

This one will have a long way to go be­fore they get in­to the clin­ic.

The big biotech said Wednes­day morn­ing that it is pay­ing $45 mil­lion up­front and re­serv­ing a whop­ping $2.3 bil­lion in biotech bucks if San Fran­cis­co-based Nurix can point the way to new can­cer ther­a­pies, as well as drugs for oth­er, un­spec­i­fied dis­eases.