The com­pas­sion­ate use poll: A lop­sided vote fa­vors post­ing poli­cies, with plen­ty of fret­ting

We ran a snap poll yes­ter­day on com­pas­sion­ate use poli­cies, and there’s clear­ly plen­ty of pos­i­tive sen­ti­ment among our read­ers back­ing some sort of eas­i­ly ac­ces­si­ble, on­line state­ment ex­plain­ing each biotech’s poli­cies on ex­pand­ed ac­cess to ex­per­i­men­tal drugs.

The vote land­ed 70% for, 30% against post­ing a com­pas­sion­ate use pol­i­cy on­line among the 149 votes we col­lect­ed in the last 24 hours. But maybe that has a lot to do with the fact that a very sim­i­lar sized group al­so said that such pol­i­cy state­ments don’t ap­ply to their com­pa­nies. Among the 49 who said it did ap­ply, 33 said their com­pa­nies didn’t post a pol­i­cy, against 16 who said they did.

That two-to-one ra­tio against, though, is still a far cry from the neg­li­gi­ble 4% of small biotechs tracked by Avalere Health which post­ed such poli­cies.

No, said one poll re­spon­dent: “The de­ci­sion to try an ex­per­i­men­tal med­i­cine is very com­plex (sci­en­tif­i­cal­ly, eth­i­cal­ly and reg­u­la­to­ri­ly) and mak­ing it ‘easy’ is go­ing to open a Pan­do­ra’s box that we’re not ready for.”

The ‘no’ camp al­so ex­pressed con­cerns that a sin­gle ad­verse event from com­pas­sion­ate use could de­rail a pro­gram, as well as wor­ries about the cost of pro­vid­ing drugs for small com­pa­nies years away from mak­ing any mon­ey.

A lot of the com­ments for the “yes” vote echoed this sen­ti­ment:

“Post­ing com­pas­sion­ate use poli­cies on your web­site does not promise that your case will be ac­cept­ed, but at least it pro­vides trans­paren­cy on their pol­i­cy and of­fers the po­ten­tial to be treat­ed, ” said one.

Yes, but. “Com­pas­sion­ate Use should on­ly be of­fered for drugs suc­cess­ful­ly shown to be both ef­fi­ca­cious and safe in Phase IIb/III tri­als. Pri­or to that, it is not ad­vis­able. Re­mem­ber “first do no harm,” says an­oth­er.

In­deed, one read­er al­so steered me to Bio­Marin’s pol­i­cy, say­ing that it’s com­mon for such state­ments to re­strict any com­pas­sion­ate use to drugs which have fin­ished Phase III, when you have sol­id ef­fi­ca­cy and safe­ty da­ta, but be­fore the FDA has act­ed.

Art Ca­plan

That’s a nar­row win­dow. NYU’s not­ed bioethi­cist Art Ca­plan tells me he’s seen com­pas­sion­ate use re­quests for drugs in every­thing from an­i­mal-stage test­ing on up. Some small com­pa­nies will start en­ter­tain­ing re­quests as ear­ly as Phase I. And no biotech should ex­pect the pleas to be re­strict­ed for use in the des­ig­nat­ed dis­eases be­ing stud­ied. It could be for a dif­fer­ent dose as well as a sep­a­rate in­di­ca­tion, which is what caught Chimerix in the mid­dle of an on­line mob as the par­ents of one young boy sought ac­cess for its lead ther­a­py through a pub­lic lob­by­ing ef­fort that sud­den­ly went vi­ral.

Iron­i­cal­ly, I checked out Chimerix’s web site and couldn’t find any men­tion of a com­pas­sion­ate use pol­i­cy. The biotech, which has ex­pe­ri­enced a cou­ple of set­backs with the pro­gram, de­clined com­ment.

Any time you get press cov­er­age of a new drug, Ca­plan tells me, you raise the chances of com­pas­sion­ate use re­quests. And Ca­plan doesn’t sign on with the post-Phase III on­ly camp. The im­por­tant thing is mak­ing sure the drug has a well-es­tab­lished safe­ty pro­file and at least a hint of ef­fi­ca­cy. For Ca­plan, Phase IIb is where you hit the sweet spot for con­sid­er­ing these re­quests.

And he adds that he wasn’t in the least bit sur­prised to hear that few biotechs have clear­ly enun­ci­at­ed poli­cies spelled out on­line.

“If you have a com­pa­ny with one CEO and 20 peo­ple chained to a bench, web­site de­vel­op­ment is not a top pri­or­i­ty,” says Ca­plan. But that doesn’t mean they shouldn’t do it.

Alice Shaw, Lung Cancer Foundation of America

Top ALK ex­pert and can­cer drug re­searcher Al­ice Shaw bids adieu to acad­e­mia, hel­lo to No­var­tis

Jay Bradner has recruited a marquee oncology drug researcher into the ranks of the Novartis Institutes for BioMedical Research. Alice Shaw is jumping from prestigious posts intertwined through Mass General, Harvard and Dana-Farber to take the lead of NIBR’s translational clinical oncology group.

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Med­ical an­i­ma­tion: Mak­ing it eas­i­er for the site and the pa­tient to un­der­stand

Medical animation has in recent years become an increasingly important tool for conveying niche information to a varied audience, particularly to those audiences without expertise in the specialist area. Science programmes today, for example, have moved from the piece-to-camera of the university professor explaining how a complex disease mechanism works, to actually showing the viewer first-hand what it might look like to shrink ourselves down to the size of an ant’s foot, and travel inside the human body to witness these processes in action. Effectively communicating a complex disease pathophysiology, or the novel mechanism of action of a new drug, can be complex. This is especially difficult when the audience domain knowledge is limited or non-existent. Medical animation can help with this communication challenge in several ways.
Improved accessibility to visualisation
Visualisation is a core component of our ability to understand a concept. Ask 10 people to visualise an apple, and each will come up with a slightly different image, some apples smaller than others, some more round, some with bites taken. Acceptable, you say, we can move on to the next part of the story. Now ask 10 people to visualise how HIV’s capsid protein gets arranged into the hexamers and pentamers that form the viral capsid that holds HIV’s genetic material. This request may pose a challenge even to someone with some virology knowledge, and it is that inability to effectively visualise what is going on that holds us back from fully understanding the rest of the story. So how does medical animation help us to overcome this visualisation challenge?

Hal Barron, GSK's president of R&D and CSO, speaks to Endpoints News founder and editor John Carroll in London at Endpoints' #UKBIO19 summit on October 8, 2019

[Video] Cel­e­brat­ing tri­al fail­ures, chang­ing the cul­ture and al­ly­ing with Cal­i­for­nia dream­ers: R&D chief Hal Bar­ron talks about a new era at GSK

Last week I had a chance to sit down with Hal Barron at Endpoints’ #UKBIO19 summit to discuss his views on R&D at GSK, a topic that has been central to his life since he took the top research post close to 2 years ago. During the conversation, Barron talked about changing the culture at GSK, a move that involves several new approaches — one of which involves celebrating their setbacks as they shift resources to the most promising programs in the pipeline. Barron also discussed his new alliances in the Bay Area — including his collaboration pact with Lyell, which we covered here — frankly assesses the pluses and minuses of the UK drug development scene, and talks about his plans for making GSK a much more effective drug developer.

This is one discussion you won’t want to miss. Insider and Enterprise subscribers can log-in to watch the video.

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Flu Virus (Source: CDC)

FDA ex­pands Xofluza ap­proval as Roche strug­gles to catch loom­ing flu mar­ket

As a potentially powerful flu season looms, so does a big test for Roche and its new flu drug, Xofluza. The Swiss giant just got a small boost in advance of that test as the FDA expanded Xofluza’s indication to include patients at high risk of developing flu-related complications.

Xofluza (baloxavir marboxil) was approved last October in the US, the first landmark flu drug approval in 20 years and a much-needed green light for a company that had watched its leading flu drug Tamiflu get eaten alive by generics. Like its predecessor, the pill offered a reduction in flu symptoms but not a cure.

EMA backs sev­en ther­a­pies, in­clud­ing Mer­ck­'s Ebo­la vac­cine

The first-ever Ebola vaccine is on the precipice of approval after the European Medicine’s Agency (EMA) backed the Merck product in this week’s roster of recommendations.

The drugmaker $MRK began developing the vaccine, christened Ervebo, during the West African outbreak that occurred between 2014 and 2016, killing more than 11,000.

The current outbreak in the Democratic Republic of Congo (DRC) has shown case fatality rates of approximately 67%, the agency estimated. Earlier this year, the WHO declared the outbreak — which so far has infected more than 3,000 people — a public health emergency of international concern.

Ronald Herb­st fol­lows Med­Im­mune ex­o­dus to Pyx­is CSO post; Jeff God­dard to suc­ceed CEO of AIT Bio­science

→ The outflow of top execs from MedImmune continues to fill the leadership ranks of smaller biotechs. The latest to take off is Ronald Herbst, the head of oncology research, who’s assuming the CSO post at Pyxis Oncology.  

Herbst was part of the old MedImmune organization AstraZeneca CEO Pascal Soriot restructured earlier this year, reorganizing the company and eliminating the storied subsidiary as a separate organization.

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Mer­ck is tak­ing the ax to its US op­er­a­tions, cut­ting 500 jobs in its lat­est re­or­ga­ni­za­tion

Merck is cutting 500 jobs in its US sales and headquarters commercial teams in its latest effort to find new ways to streamline the operation.

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UP­DAT­ED: J&J's Xarel­to, Amar­in's Vas­cepa are cost-ef­fec­tive, not bud­get friend­ly — ICER

ICER, an increasingly influential cost-effectiveness watchdog in the United States, has concluded in its review of treatments for cardiovascular disease that while the cost of J&J’s Xarelto and Amarin’s Vascepa meet its benchmark for value pricing — the two treatments will not likely treat as many patients as hoped without surpassing the annual budget threshold calculated by ICER for each therapy.

Mi­rati preps its first look at their KRAS G12C con­tender, and they have to clear a high bar for suc­cess

If you’re a big KRAS G12C fan, mark your calendars for October 28 at 4:20 pm EDT.

That’s when Mirati $MRTX will unveil its first peek at the early clinical data available on MRTX849 in presentations at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics in Boston.

Mirati has been experiencing the full effect of a rival’s initial success at targeting the G12C pocket found on KRAS, offering the biotech some support on the concept they’re after — and biotech fans a race to the top. Amgen made a big splash with its first positive snapshot on lung cancer, but deflated sky-high expectations as it proved harder to find similar benefits in other types of cancers.

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