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.

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,900+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,900+ biopharma pros reading Endpoints daily — and it's free.

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,900+ biopharma pros reading Endpoints daily — and it's free.

We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at with any issues.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Am­gen, Al­ler­gan biosim­i­lar of Roche's block­buster Rit­ux­an clears an­oth­er US piv­otal study 

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.

BeiGene and Mus­tang nail down spe­cial FDA sta­tus for top drugs; Roche bags added cov­er­age for Hem­li­bra

→ BeiGene $BGNE is getting a boost in its drive to field a rival to Imbruvica. The FDA has offered an accelerated review to zanubrutinib, a BTK inhibitor that has posted positive results for mantle cell lymphoma. The PDUFA date lands on February 27, 2020. The drug scored breakthrough status at the beginning of the year.

→ BeiGene isn’t the only biopharma company to gain special regulatory status today. Mustang Bio $MBIO and St. Jude Children’s Research Hospital announced that MB-107, a lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency, also known as bubble boy disease, has been granted Regenerative Medicine Advanced Therapy status.

Trump ad­min­is­tra­tion re­vives bid to get drug list prices on TV ads

The Trump administration is not giving up just yet. On Wednesday, the HHS filed an appeal against a judge’s decision in July to overturn a ruling obligating drug manufacturers to disclose the list price of their therapies in television adverts — hours before it was stipulated to go into effect.

In May, the HHS published a final ruling requiring drugmakers to divulge the wholesale acquisition cost— of a 30-day supply of the drug — in tv ads in a bid to enhance price transparency in the United States. The pharmaceutical industry has vehemently opposed the rule, asserting that list prices are not what a typical patient in the United States pays for treatment — that number is typically determined by the type of (or lack thereof) insurance coverage, deductibles and out-of-pocket costs. Although there is truth to that claim, the move was considered symbolic in the Trump administration’s healthcare agenda to hold drugmakers accountable in a climate where skyrocketing drug prices have incensed Americans on both sides of the aisle.

Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,900+ biopharma pros reading Endpoints daily — and it's free.

Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll