Pelosi drug pric­ing bill promis­es sav­ings, but could gag R&D — CBO analy­sis

The De­moc­rats’ drug pric­ing bill — un­veiled by Speak­er Nan­cy Pelosi last month — could save Medicare spend­ing by $345 bil­lion over a sev­en-year pe­ri­od, a new analy­sis sug­gests. But the ven­omous cli­mate of im­peach­ment pro­ceed­ings and the in­ten­si­fy­ing dis­cord be­tween the De­mo­c­rat-con­trolled House and Re­pub­li­can-ma­jor­i­ty Sen­ate por­tends the bill will un­like­ly ever be­come law.

Tech­ni­cal­ly, both sides of the aisle agree drug prices in the Unit­ed States need some low­er­ing. The De­moc­rats’ bill, H.R.3 – Low­er Drug Costs Now Act of 2019, is en­gi­neered to em­pow­er the HHS to ne­go­ti­ate prices for the 125 most ex­pen­sive pre­scrip­tion drugs with­out at least two com­peti­tors — the Trump ad­min­is­tra­tion has al­ready backed such a mea­sure for the Vet­er­ans As­so­ci­a­tion. Un­der the bill, prices for this cat­e­go­ry of med­i­cines are not in­tend­ed to ex­ceed 120% of the av­er­age price in cer­tain oth­er coun­tries (Aus­tralia, Cana­da, France, Ger­many and the Unit­ed King­dom), akin to a pro­pos­al float­ed by Trump ear­li­er this year, which sug­gest­ed prices be pegged against what oth­er na­tions were pay­ing as part of an “in­ter­na­tion­al pric­ing in­dex”.

Apart from un­der­stand­ably elic­it­ing the ire of the bio­phar­ma in­dus­try — which holds the crown for the least fa­vored sec­tor by Amer­i­cans, falling be­hind the fed­er­al gov­ern­ment it­self — the bill has al­so met with crit­i­cism from mem­bers of the GOP. The dis­sent — shared by both fac­tions — stems from the be­lief that the ini­tia­tive would in­evitably chill in­vest­ment in phar­ma­ceu­ti­cal re­search and sti­fle in­no­va­tion.

In the fresh, but pre­lim­i­nary, analy­sis pub­lished on Fri­day by the Con­gres­sion­al Bud­get Of­fice (CBO), the bill would not on­ly cut fed­er­al di­rect spend­ing for Medicare by $345 bil­lion over the 2023-2029 pe­ri­od but would change the com­plex­ion of the drug mar­ket by chang­ing in­cen­tives that man­u­fac­tur­ers cur­rent­ly en­joy.

“A man­u­fac­tur­er that was dis­sat­is­fied with a ne­go­ti­a­tion could pull a drug out of the U.S. mar­ket en­tire­ly, though CBO ex­pects that would be un­like­ly for drugs al­ready be­ing sold in the Unit­ed States. Man­u­fac­tur­ers would ini­tial­ly set list prices of some new drugs in the U.S. high­er than un­der ex­ist­ing law, al­though the net prices paid by con­sumers over time could be low­er in many such cas­es,” the re­port said.

The CBO al­so cau­tioned that the bill could al­so mo­ti­vate man­u­fac­tur­ers to spike drug prices in coun­tries out­side the Unit­ed States to make up for lost rev­enue, or not sell cer­tain med­i­cines at all out­side the Unit­ed States to elim­i­nate ref­er­ence prices for US ne­go­ti­a­tions.

PhRMA, a pow­er­ful phar­ma lob­by group, took is­sue with this claim. “This re­flects a lack of un­der­stand­ing of for­eign pric­ing sys­tems, which have laws and reg­u­la­tions that pro­hib­it man­u­fac­tur­ers from uni­lat­er­al­ly set­ting prod­uct prices and/or in­creas­ing the prices of prod­ucts,” a spokesper­son not­ed in an email to End­points News.

The CBO’s analy­sis al­so mir­rored some of the con­cerns ex­pressed by Re­pub­li­cans. Al­though in the short term low­er prices will en­hance ac­cess to med­i­cines and im­prove health — over the longer term,  di­min­ished spend­ing on re­search and de­vel­op­ment will cul­mi­nate in the in­tro­duc­tion of few­er new drugs, the re­port pre­dict­ed.

Al­though the CBO’s analy­sis of the bill is not com­plete, “its pre­lim­i­nary es­ti­mate is that a re­duc­tion in rev­enues of $0.5 tril­lion to $1 tril­lion would lead to a re­duc­tion of ap­prox­i­mate­ly 8 to 15 new drugs com­ing to mar­ket over the next 10 years. The over­all ef­fect on the health of fam­i­lies in the Unit­ed States that would stem from in­creased use of pre­scrip­tion drugs but de­creased avail­abil­i­ty of new drugs is un­clear.”

These cal­cu­la­tions were based on the premise that the FDA ap­proves, on av­er­age, about 30 new drugs an­nu­al­ly. In 2018, the US agency cleared 59 drugs; in 2017, it ap­proved 46; and in 2016 the num­ber was 22. So far this year, the reg­u­la­tor has green-light­ed 30.

“It takes an av­er­age of 10-12 years to de­vel­op a new med­i­cine so the biggest im­pact on in­no­va­tion will oc­cur out­side of the ten-year bud­get win­dow,” the PhRMA spokesper­son added.

Sep­a­rate­ly, an analy­sis by the CMS sug­gest­ed that the bill could cur­tail to­tal US spend­ing on health­care by about $480 bil­lion over a decade — sav­ing Amer­i­can house­holds rough­ly $158 bil­lion in low­er pre­mi­ums and small­er out-of-pock­et costs.

Source: CMS, 2019

Click on the im­age to see the full-sized ver­sion

Ear­li­er this month, Trump claimed that the phar­ma­ceu­ti­cal in­dus­try was be­hind the im­peach­ment in­quiry from House De­moc­rats, with­out of­fer­ing any ev­i­dence to sup­port his as­ser­tion.

“Does Trump’s al­lu­sion to Big Phar­ma’s role as the bankroller of the im­peach­ment “hoax” get con­demned by the oth­er side, there­by de­fend­ing phar­ma? That prob­a­bly won’t hap­pen, but we are be­gin­ning to think the util­i­ty of phar­ma as a po­lit­i­cal punch­ing bag may be start­ing to wear thin giv­en the ab­sur­di­ty of this lat­est al­le­ga­tion,” Baird’s Bri­an Sko­r­ney wrote in a note on Oc­to­ber 4.

So­cial im­age: Nan­cy Pelosi, AP Im­ages

Inside FDA HQ (File photo)

The FDA just ap­proved the third Duchenne MD drug. And reg­u­la­tors still don’t know if any of them work

Last year Sarepta hit center stage with the FDA’s controversial reversal of its CRL for the company’s second Duchenne muscular dystrophy drug — after the biotech was ambushed by agency insiders ready to reject a second pitch based on the same disease biomarker used for the first approval for eteplirsen, without actual data on the efficacy of the drug.

On Wednesday the FDA approved the third Duchenne MD drug, based on the same biomarker. And regulators were ready to act yet again despite the lack of efficacy data.

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FDA ap­proves the third NSOMD drug in 18 months as Roche/Genen­tech beefs up its port­fo­lio of drugs for neu­ro­log­i­cal dis­or­ders

There were no FDA approved treatments for neuromyelitis optica spectrum disorder at the start of 2019. Now, as of Friday, there are three.

The latest entrant to the market is the Roche/Genentech drug satralizumab after US regulators gave it the thumbs up late Friday. An IL-6 inhibitor, the drug joins Alexion’s Soliris and AstraZeneca spinout Viela Bio’s Uplizna. The annual cost of satralizumab — which will hit the market as Enspryng — will be $190,000 for 13 doses, a Genentech spokesperson said, though the first year of treatment requires 15 doses and cost about $220,000.

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Franz-Werner Haas, CureVac CEO

UP­DAT­ED: On the heels of a snap $1B raise, Cure­Vac out­lines plans to seek emer­gency OK for Covid-19 vac­cine -- shares rock­et up

CureVac is going from being one of the quietest players in the race to develop a new vaccine to fight the worst public health crisis in a century to a challenger for the multibillion-dollar market that awaits the first vaccines to make it over the finish line. Typically low-key at a time of brash comments and incredibly ambitious development timelines from the leaders, CureVac now is jumping straight into the spotlight.

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US gov­ern­ment re­port­ed­ly be­gins prepar­ing for Covid-19 chal­lenge tri­als. Are they eth­i­cal?

Controversial human challenge trials for potential Covid-19 vaccines reportedly have a new booster — the US government.

Scientists working for the government have begun manufacturing a strain of the novel coronavirus that could be used in such studies, Reuters reported Friday morning. The trials would enroll healthy volunteers to be vaccinated and then intentionally infected with a weakened coronavirus.

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Phase III read­outs spell dis­as­ter for Genen­tech’s lead IBD drug

Roche had big plans for etrolizumab. Eyeing a hyper-competitive IBD and Crohn’s market where they have not historically been a player, the company rolled out 8 different Phase III trials, testing the antibody for two different uses across a range of different patient groups.

On Monday, Roche released results for 4 of those studies, and they mark a decided setback for both the Swiss pharma and their biotech sub Genentech, potentially spelling an end to a drug they put over half-a-decade and millions of dollars behind.

Stéphane Bancel speaks to President Donald Trump at the White House meeting on March 2 (AP Images)

UP­DAT­ED: Mod­er­na of­fers steep dis­count in US sup­ply deal — but still takes the crown with close to $2.5B in vac­cine con­tracts

The US pre-order for Moderna’s Covid-19 vaccine is in.

Operation Warp Speed is reserving $1.525 billion for 100 million doses of Moderna’s Phase III mRNA candidate, rounding out to about $15 per dose — including $300 million in incentive payments for timely delivery. Given that Moderna has a two-dose regimen, it’s good for vaccinating 50 million people. The US government also has the option to purchase another 400 million doses for a total of $6.6 billion, or $16.5 per dose.

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Trevor Martin (Mammoth)

Eye­ing in-vi­vo edit­ing, Mam­moth li­cens­es Jen­nifer Doud­na’s new CRISPR en­zyme

Last month, Jennifer Doudna revealed in Science a new, “hyper-compact” CRISPR enzyme that was half the size of traditional CRISPR enzymes and could, she suspected, offer a new, more versatile tool for gene editing.

Now, the University of California-Berkeley has licensed that enzyme, known as Casφ, exclusively to a biotech startup she and two former students set up three years ago: Mammoth Biosciences. It’s the second new CRISPR protein Mammoth has licensed from Doudna’s lab, after they licensed Cas14 in 2019.

Sanofi vet Kather­ine Bowdish named CEO of PIC Ther­a­peu­tics; As the world Terns: Liv­er dis­ease biotech makes ex­ec­u­tive changes

PIC Therapeutics hasn’t raised much money, yet. But the fledgling biotech has attracted a high-profile player to the helm.

The Boston-based biotech has handed the reins to Katherine Bowdish as its president and CEO. Bowdish will also join the board of directors of PIC. Bowdish joins from Sanofi where she served as VP and head of R&D strategy, as well as helping launch and lead Sanofi Sunrise, a venture investment and partnering vehicle at Sanofi. Before that, Bowdish held several exec roles at Permeon Biologics, Anaphore, Alexion Pharmaceuticals and Prolifaron (acquired by Alexion).

Martin Shkreli (Shutterstock)

Mar­tin Shkre­li con­tin­ued to or­ches­trate an­ti-com­pet­i­tive schemes for Dara­prim be­hind bars — FTC

Martin Shkreli didn’t just blog, read up on drug development news and run his biotech business with a contraband cell phone in prison. According to the FTC, he was also coordinating the anticompetitive scheme to shield Daraprim — the drug at the center of a price-gouging controversy that earned him the “Pharma Bro” nickname — from generic rivals.

Back in January the FTC, together with New York’s attorney general, launched a federal lawsuit against Shkreli, who’s now serving a 7-year sentence for defrauding investors in his hedge fund, alleging that he effectively created a drug monopoly. While Shkreli’s notorious move to raise the per tablet price of Daraprim from $17.50 to $750 was perfectly legal, the tactics he allegedly deployed to box out competitors weren’t.