Oh Cana­da! There is no San­ta Claus to the north giv­ing out cheap drugs to US pa­tients

Bioreg­num Opin­ion Col­umn by John Car­roll

This morn­ing HHS Sec­re­tary Alex Azar took an­oth­er step to­ward open­ing the door to drugs im­port­ed from Cana­da. The still-de­vel­op­ing pol­i­cy ini­tia­tives pave the way to get­ting states to set up pro­grams for im­port­ing less ex­pen­sive drugs, as well as set­ting up a path for drug­mak­ers to bring in ther­a­pies sold abroad.

With less than a year to go to the elec­tion, you could say Pres­i­dent Don­ald Trump has a vest­ed in­ter­est in cuff­ing the bio­phar­ma in­dus­try — as he promised from the start — be­fore he goes back to vot­ers sell­ing 4 more years in the White House.

But let’s get re­al here. Any­one who thinks you can out­source the drug pric­ing is­sue to Cana­da, and get them to act as a proxy for the Amer­i­can mar­ket, has got to be dream­ing.

I’m not go­ing in­to the safe­ty is­sue, the way lob­by­ists want to when it comes to drug im­por­ta­tion. If you stick with rep­utable dis­trib­u­tors in Cana­da, there won’t be a safe­ty is­sue. This ar­gu­ment is about mon­ey, and that’s where we need to fo­cus.

I’ve had se­ri­ous con­ver­sa­tions with se­nior phar­ma ex­ecs who ap­pear per­fect­ly will­ing to sac­ri­fice Eu­ro­pean rev­enue if nec­es­sary in or­der to safe­guard their all-im­por­tant US mar­kets. What do you think these phar­ma com­pa­nies would do with their small Cana­di­an mar­ket­ing ops if they threat­ened US prof­it mar­gins?

If a Cana­di­an ne­go­tia­tor is stand­ing in as a sub­sti­tute to rep­re­sent state mar­kets in the US, their drug prices are go­ing to go sky high or their sup­ply will be cur­tailed. And Cana­da is not go­ing to al­low that. Once the sup­ply of a sin­gle drug is crimped, the hue and cry in that coun­try would be im­mense. They want to pre­serve low­er prices and ac­cess for Cana­di­ans, not go on some cru­sade to ex­tend that to the US. And if glob­al phar­ma play­ers can’t rein in a rogue op­er­a­tion, what hap­pens to the con­tracts they re­ly on to do busi­ness?

Yeah.

In­ter­est­ing­ly, the New York Times re­port on the pol­i­cy de­vel­op­ment to­day says a 2003 law won’t even al­low for im­por­ta­tion of high-priced bi­o­log­ics like Hu­mi­ra — the thorn in the side of law­mak­ers. Ab­b­Vie jacked up that price year af­ter year to cre­ate a cash cow it re­lied on to get them to the next chap­ter — which turned out to be the Al­ler­gan buy­out. And Cana­da can’t help.

This is a non-starter. It al­ways has been for the 20 years it’s been de­bat­ed. Yet it re­mains a spot­light is­sue, un­der­scor­ing the pauci­ty of new ideas when it comes to the much-need­ed task of re­form­ing the pric­ing sys­tem in the US. Drug af­ford­abil­i­ty and ac­cess is a huge is­sue in the US, where large groups of pa­tients can’t af­ford their end of the bill. Let’s not triv­i­al­ize the cri­sis by promis­ing a so­lu­tion from Cana­da that will nev­er come.

So­cial im­age: Shut­ter­stock

2019 Trin­i­ty Drug In­dex Eval­u­ates Ac­tu­al Com­mer­cial Per­for­mance of Nov­el Drugs Ap­proved in 2016

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

UP­DAT­ED: FDA’s golodirsen CRL: Sarep­ta’s Duchenne drugs are dan­ger­ous to pa­tients, of­fer­ing on­ly a small ben­e­fit. And where's that con­fir­ma­to­ry tri­al?

Back last summer, Sarepta CEO Doug Ingram told Duchenne MD families and investors that the FDA’s shock rejection of their second Duchenne MD drug golodirsen was due to some concerns regulators raised about the risk of infection and the possibility of kidney toxicity. But when pressed to release the letter for all to see, he declined, according to a report from BioPharmaDive, saying that kind of move “might not look like we’re being as respectful as we’d like to be.”

He went on to assure everyone that he hadn’t misrepresented the CRL.

But Ingram’s public remarks didn’t include everything in the letter, which — following the FDA’s surprise about-face and unexplained approval — has now been posted on the FDA’s website and broadly circulated on Twitter early Wednesday.

The CRL raises plenty of fresh questions about why the FDA abruptly decided to reverse itself and hand out an OK for a drug a senior regulator at the FDA believed — 5 months ago, when he wrote the letter — is dangerous to patients. It also puts the spotlight back on Sarepta $SRPT, which failed to launch a confirmatory study of eteplirsen, which was only approved after a heated internal controversy at the FDA. Ellis Unger, director of CDER’s Office of Drug Evaluation I, notes that study could have clarified quite a lot about the benefit and risks associated with their drugs — which can cost as much as a million dollars per patient per year, depending on weight.

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How to cap­i­talise on a lean launch

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
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What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

Wuhan virus out­break trig­gers in­evitable small-biotech ral­ly

Every few years, a public health crisis (think Ebola, Zika) spurred by a rogue pathogen triggers a small-biotech rally, as drugmakers emerge from the woodwork with ambitious plans to treat the mounting outbreak. In most cases, that enthusiasm never quite delivers.

Things are no different, as the coronavirus outbreak in Wuhan, China takes hold. There have been close to 300 confirmed human infections in China, and at least four deaths. Coronaviruses are a large family of viruses, which include MERS and SARS. On Tuesday, the CDC reported the virus was detected in a US traveler returning from Wuhan.

Brex­it fears, Wood­ford woes over­shad­owed UK biotech and cut 2019 fi­nanc­ing by al­most half

The venture tide might have subsided, the IPO window may be closing and certain listed biotechs may be having a tough time amid Neil Woodford’s well-publicized demised, but there’s still plenty to celebrate in the UK BioIndustry Association’s eyes.

Overall investment in UK biotech last year fell from the record-breaking £2.2 billion levels of 2018 to £1.3 billion — including £679 million in venture capital, a meager £64 million in IPOs plus £596 million when you add up all public financings, according to a new report from the BIA.

Blue­print Med­i­cines po­ten­tial­ly de­lays Ay­vak­it de­ci­sion; Con­trol beats treat­ment in mesothe­lioma tri­al

→ Blueprint Medicines filed an amendment to its application to get the gastrointestinal stromal tumor (GIST) drug Ayvakit approved in fourth-line GIST, the company disclosed in the prospectus for a new $325 million public offering.  Blueprint got a big accelerated OK on the drug this month in a particular mutation, but because the FDA decided to split their review in two, they didn’t hear on fourth-line GIST. They were supposed to hear before February 14, but this amendment could push that date back by 3 months. Blueprint wrote that the amendment is designed to allow the company to comply with the FDA’s request for data from the Phase III Voyage trial before they give a judgment.

Io­n­is, Akcea boost­ed by a pos­i­tive PhII for their No­var­tis castoff car­dio drug — and they plan to push ahead in­to piv­otals

Late last year Novartis abandoned a cardio drug from Ionis’ spinoff Akcea just after the pharma giant snapped up inclisiran, going the RNAi way in guarding against heart disease in the $9.7 billion Medco buyout.

Now the pharma goliath — which is headed down the PCSK9 road with a drug it believes can be used in a mass population — can get a clearer picture of just what they gave up.

Akcea $AKCA and the mother company $IONS put out a statement early Wednesday saying that their Phase II study of AKCEA-APOCIII-LR delivered solid efficacy data, with the high dose clearly outperforming placebo in significantly reducing triglycerides as a means to cutting the risk of cardiovascular disease. In addition, investigators concluded that the drug slashed apoC-III, very low-density lipoprotein and remnant cholesterol while boosting “good” HDL levels.

Hal Barron and Emma Walmsley, GSK

GSK’s ‘break­through’ BC­MA can­cer drug gets a pri­or­i­ty re­view — and a big win for the on­col­o­gy R&D team

After largely whiffing the past 2 years on the pharma R&D front, GlaxoSmithKline research chief Hal Barron has seized boasting rights to a key win that puts them back in the cancer drug development game.

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Who are the young bio­phar­ma lead­ers shap­ing the in­dus­try? Nom­i­nate them for End­points' spe­cial re­port

Update: Nominations open through end of day, Monday, January 27

Two years ago, when we did our first Endpoints 20-under-40, we profiled a set of up-and-comers who promised to help reshape the industry as we know it. Now we’re back and once again looking for the top 20 biopharma professionals under the age of 40. We’ll be profiling folks who have accomplished a lot at a young age but seem on the verge of accomplishing so much more.