Cum­mings trig­gers probe in­to pric­ing prac­tices of dozen ma­jor drug­mak­ers

An in­ves­ti­ga­tion in­to drug price goug­ing by the pro­lif­ic chair­man of the Com­mit­tee on Over­sight and Re­form Eli­jah Cum­mings kicked off on Mon­day with in­quiries sent to a dozen ma­jor drug­mak­ers seek­ing in­for­ma­tion on pric­ing prac­tices, days af­ter a leg­isla­tive pack­age aimed at curb­ing sky­rock­et­ing pre­scrip­tion drug prices was un­veiled.

Rep. Eli­jah Cum­mings

The probe is de­signed to un­pack the ra­tio­nale be­hind dra­mat­ic drug price hikes, how the pro­ceeds from drug sales are be­ing used by man­u­fac­tur­ers and what can be done to cut prices.

“Re­search and de­vel­op­ment ef­forts on ground­break­ing med­ica­tions have made im­mea­sur­able con­tri­bu­tions to the health of Amer­i­cans… But the on­go­ing es­ca­la­tion of prices by drug com­pa­nies is un­sus­tain­able,” Cum­mings said in a state­ment.

Let­ters were sent to 12 drug­mak­ers — Ab­b­Vie $AB­BV, Am­gen $AMGN, As­traZeneca $AZN, Cel­gene $CELG, Lil­ly $LLY, J&J $JNJ, Mallinck­rodt $MNK, No­var­tis $NVS, No­vo Nordisk $NVO, Pfiz­er $PFE, Sanofi $SNY and Te­va $TE­VA re­gard­ing var­i­ous drugs they sell — seek­ing in­for­ma­tion on price hikes, in­vest­ments in R&D, and strate­gies em­ployed by the man­u­fac­tur­ers to pre­serve mar­ket share. The let­ters are fo­cused on drugs that are among the costli­est to Medicare Part D, among the most ex­pen­sive per ben­e­fi­cia­ry, or had the largest price in­creas­es over a five-year pe­ri­od. Ac­cord­ing to a re­port by the non-prof­it Amer­i­can As­so­ci­a­tion of Re­tired Per­sons (AARP), which rep­re­sents 40 mil­lion Amer­i­cans, be­tween 2006 and 2017 the prices of 267 com­mon­ly used pre­scrip­tion drugs rose by an av­er­age 8.4% each year, far out­strip­ping the gen­er­al in­fla­tion rate of 2.1% over the pe­ri­od.

The first pub­lic hear­ing on the sub­ject is sched­uled for Jan­u­ary 29.

Da­ta show that de­spite the high­er cost of health­care borne by the Unit­ed States, the re­gion ac­tu­al­ly per­forms worse across var­i­ous health mea­sures ver­sus a num­ber of oth­er high-in­come na­tions. Ac­cord­ing to a re­cent Politi­co/Har­vard poll, an over­whelm­ing ma­jor­i­ty of Amer­i­cans ranked ad­dress­ing the cost of med­i­cines as a top pri­or­i­ty for the new Con­gress.

The Trump ad­min­is­tra­tion has pro­posed steps to curb prices, and var­i­ous law­mak­ers have al­so made it a cor­ner­stone  is­sue, in­clud­ing Sen­a­tor Chuck Grass­ley, the new chair of the Sen­ate Fi­nance Com­mit­tee, and pres­i­den­tial hope­ful Sen­a­tor Eliz­a­beth War­ren.

 

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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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).

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?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
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.

Stephen Hahn, AP

The FDA has de­val­ued the gold stan­dard on R&D. And that threat­ens every­one in drug de­vel­op­ment

Bioregnum Opinion Column by John Carroll

A few weeks ago, when Stephen Hahn was being lightly queried by Senators in his confirmation hearing as the new commissioner of the FDA, he made the usual vow to maintain the gold standard in drug development.

Neatly summarized, that standard requires the agency to sign off on clinical data — usually from two, well-controlled human studies — that prove a drug’s benefit outweighs any risks.

Over the last few years, biopharma has enjoyed an unprecedented loosening over just what it takes to clear that bar. Regulators are more willing to drop the second trial requirement ahead of an accelerated approval — particularly if they have an unmet medical need where patients are clamoring for a therapy.

That confirmatory trial the FDA demands can wait a few years. And most everyone in biopharma would tell you that’s the right thing for patients. They know its a tonic for everyone in the industry faced with pushing a drug through clinical development. And it’s helped inspire a global biotech boom.

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Eli Lil­ly’s $1.6B can­cer drug failed to spark even the slight­est pos­i­tive gain for pa­tients in its 1st PhI­II

Eli Lilly had high hopes for its pegylated IL-10 drug pegilodecakin when it bought Armo last year for $1.6 billion in cash. But after reporting a few months ago that it had failed a Phase III in pancreatic cancer, without the data, its likely value has plunged. And now we’re getting some exact data that underscore just how little positive effect it had.

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Am­gen aug­ments Asia foothold by tak­ing over Astel­las joint ven­ture in Japan

California-based Amgen, which does the bulk of its business in the United States, made its ambition to reinvigorate its growth prospects by expanding its presence in Asia clear at the sidelines of the JP Morgan healthcare conference in San Francisco earlier this month.

The Thousand Oaks-based company on Thursday executed its plan to dissolve the joint venture with Astellas — created in 2013 — to operate the unit independently in Japan. With its rapidly aging population, the region represents an appealing market for Amgen’s osteoporosis treatments Prolia and Evenity as well as a cholesterol-lowering injection Repatha.

Daphne Zohar (PureTech)

PureTech bags $200M from sale of Karuna shares — still siz­zling from promis­ing schiz­o­phre­nia da­ta

Cashing in on the exuberance around Karuna Therapeutics and its potential blockbuster CNS drug, PureTech has sold a chunk of the biotech’s shares to Goldman Sachs for $200 million.

Boston-based PureTech had helped Eli Lilly vet Steve Paul launch Karuna and invent its lead program, which combines two old drugs that both act on the muscarinic receptor and balances each other out. Xanomeline, a discard from Lilly, stimulates the M1 and M4 receptors; trospium is an muscarinic receptor antagonist approved to treat overactive bladders.

UP­DAT­ED: New play­ers are jump­ing in­to the scram­ble to de­vel­op a vac­cine as pan­dem­ic pan­ic spreads fast

When the CNN news crew in Wuhan caught wind of the Chinese government’s plan to quarantine the city of 11 million people, they made a run for one of the last trains out — their Atlanta colleagues urging them on. On the way to the train station, they were forced to skirt the local seafood market, where the coronavirus at the heart of a brewing outbreak may have taken root.

And they breathlessly reported every moment of the early morning dash.

In shuttering the city, triggering an exodus of masked residents who caught wind of the quarantine ahead of time, China signaled that they were prepared to take extreme actions to stop the spread of a virus that has claimed 17 lives, sickened many more and panicked people around the globe.

CNN helped illustrate how hard all that can be.

The early reaction in the biotech industry has been classic, with small-cap companies scrambling to headline efforts to step in fast. But there are also new players in the field with new tech that has been introduced since the last of a series of pandemic panics that could change the usual storylines. And they’re volunteering for a crash course in speeding up vaccine development — a field where overnight solutions have been impossible to prove.

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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.