Joe Biden, AP Images

Biden 2022 bud­get wish list calls for Medicare ne­go­ti­a­tions on drug prices, new $6.5B NIH agency

Pres­i­dent Joe Biden late last week re­leased his FY 2022 bud­get plan, call­ing for Medicare to ne­go­ti­ate pay­ment for cer­tain high-cost drugs, in ad­di­tion to the cre­ation of a new NIH agency to de­vel­op next-gen bio­med­ical break­throughs, and a slight in­crease in FDA fund­ing.

“The Pres­i­dent sup­ports re­forms that would bring down drug prices by let­ting Medicare ne­go­ti­ate pay­ment for cer­tain high-cost drugs and re­quir­ing man­u­fac­tur­ers to pay re­bates when drug prices rise faster than in­fla­tion,” the bud­get says, of­fer­ing few de­tails on how to ac­com­plish these re­forms but not­ing that they could “yield over half a tril­lion in Fed­er­al sav­ings over 10 years.”

House En­er­gy & Com­merce Com­mit­tee chair Frank Pal­lone (D-NJ) praised the an­nounce­ment on rein­ing in drug prices, adding, “The days of Amer­i­cans pay­ing three to four times more than peo­ple in oth­er coun­tries for the same pre­scrip­tion drugs are num­bered.”

While the over­all plan of­fers most­ly a high-lev­el wish list for Con­gress to mull over as it de­cides how to fund the gov­ern­ment next year, both FDA and NIH pro­vid­ed de­tails on what they’re look­ing in­to.

Chief among the new ad­di­tions is a re­quest for $6.5 bil­lion to cre­ate a new, NIH-based Ad­vanced Re­search Pro­jects Agency for Health (ARPA-H), tasked with de­vel­op­ing new ways to help pre­vent, de­tect, and treat dis­eases like can­cer, di­a­betes, and Alzheimer’s. While there may be some crossover be­tween this agency and oth­er NIH re­search at the NCI and else­where, the new agency will use term-lim­it­ed pro­gram man­agers and a fed­er­al ad­vi­so­ry pan­el for in­ter­a­gency co­or­di­na­tion and idea gen­er­a­tion.

“Po­ten­tial ar­eas of trans­for­ma­tive re­search dri­ven by ARPA-H in­clude an ‘in­no­va­tion fun­nel’ for ac­cu­rate, wear­able, am­bu­la­to­ry blood pres­sure tech­nol­o­gy, prepa­ra­tion of mR­NA vac­cines against com­mon forms of can­cer, or ac­cel­er­at­ing de­vel­op­ment of ef­fi­cient gene/drug de­liv­ery sys­tems to tar­get any or­gan, tis­sue, or cell type – a zip code for the hu­man body,” NIH said.

Mod­eled af­ter the De­fense Ad­vanced Re­search Pro­jects Agency (DARPA), the new agency could be a boon for the bio­phar­ma in­dus­try, as NIH ex­plains most of the fund­ing from ARPA-H will go to in­dus­try, uni­ver­si­ties, and non­prof­it re­searchers.

As the NIH seeks to add this new $6.5 bil­lion agency, the FDA’s FY 2022 to­tal bud­get re­quest is for $6.5 bil­lion, which would be an in­crease of $343 mil­lion in bud­get au­thor­i­ty (out­side of user fees).

An ad­di­tion­al $75.9 mil­lion will sup­port the FDA’s da­ta mod­ern­iza­tion work, about $39 mil­lion will go to opi­oids-re­lat­ed ef­forts, an in­crease of $18.8 mil­lion will help ad­dress the FDA in­spec­tions de­layed by the pan­dem­ic, $5.6 mil­lion will go for more drug safe­ty sur­veil­lance and over­sight, and $4.7 mil­lion more will go to en­hanc­ing FDA’s abil­i­ty to sup­port and ex­pand health eq­ui­ty and health dis­par­i­ty ef­forts.

In terms of safe­ty sur­veil­lance, the FDA notes that it “cur­rent­ly faces sig­nif­i­cant chal­lenges to its abil­i­ty to main­tain an ef­fi­cient and ef­fec­tive post­mar­ket safe­ty sur­veil­lance pro­gram. Staffing lev­els have not kept pace with the in­creas­ing amount of post­mar­ket work from the grow­ing num­ber and com­plex­i­ties of re­cent ap­provals, and the in­creas­ing amount of da­ta need­ing re­view.”

FDA al­so says it will as­sem­ble a mul­ti­dis­ci­pli­nary team of reg­u­la­to­ry coun­sels, project man­agers, and sci­en­tists to craft a risk-based ap­proach to de­ter­min­ing what types of sci­en­tif­i­cal­ly valid in­for­ma­tion the agency re­quires in­dus­try to sub­mit, and how that in­for­ma­tion will be used.

“This ap­proach—which will be dy­nam­ic—may fac­tor in dif­fer­en­tial risks in­clud­ing those as­so­ci­at­ed with ac­tive phar­ma­ceu­ti­cal in­gre­di­ents, ex­cip­i­ents, or de­liv­ery mech­a­nisms. FDA’s goal with these ef­forts is to de­ter­mine which set of in­for­ma­tion we can both re­quire of in­dus­try and uti­lize in­ter­nal­ly to max­i­mize the val­ue of da­ta that we re­ceive, while con­sid­er­ing the le­gal, fi­nan­cial, and eco­nom­ic bur­dens im­posed on all stake­hold­ers. This team will evolve in­to a per­ma­nent, cross-dis­ci­pline func­tion ded­i­cat­ed to post-mar­ket safe­ty pol­i­cy,” the FDA said.

In terms of leg­isla­tive pro­pos­als, the agency seeks to fur­ther clar­i­fy the FDA’s au­thor­i­ty to re­quire in­for­ma­tion that would im­prove its abil­i­ty to as­sess phar­ma­ceu­ti­cal crit­i­cal in­fra­struc­ture as well as man­u­fac­tur­ing qual­i­ty and ca­pac­i­ty.

“For ex­am­ple, FDA is seek­ing to re­quire de­tailed drug list­ings on a quar­ter­ly ba­sis for fin­ished drug prod­uct or in-process ma­te­r­i­al, re­gard­less of whether they were di­rect­ly or in­di­rect­ly im­port­ed in­to the U.S.,” the agency says.

The FDA al­so notes that it cur­rent­ly has lim­it­ed au­thor­i­ty to re­quest records and oth­er in­for­ma­tion in ad­vance of or in lieu of drug in­spec­tions, and it seeks to ex­pand FDA’s au­thor­i­ty in this ca­pac­i­ty. The agency al­so seeks to amend the FD&C Act, to al­low for the dis­clo­sure of cer­tain non-pub­lic in­for­ma­tion to state, lo­cal, and US ter­ri­to­r­i­al gov­ern­ment agen­cies.

Mean­while, as gene and cell-based ther­a­pies con­tin­ue to be sub­mit­ted to the agency, the FDA’s Cen­ter for Bi­o­log­ics Eval­u­a­tion and Re­search is re­quest­ing $900,000 to ac­cel­er­ate ef­forts to mod­ern­ize and stream­line the re­view of these com­plex bi­o­log­ics. CBER al­so says it’s de­vel­op­ing a reg­u­la­to­ry pro­gram for in­di­vid­u­al­ized ther­a­pies and fos­ter­ing glob­al reg­u­la­to­ry con­ver­gence for cell and gene ther­a­pies.

To help that cause, NIH notes that it’s al­so work­ing to re­solve a pro­duc­tion bot­tle­neck for gene-based ther­a­pies as wait times to pro­duce vec­tor ther­a­pies that meet the man­u­fac­tur­ing stan­dards nec­es­sary for clin­i­cal tri­als are long, of­ten one to two years.

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

David Livingston (Credit: Michael Sazel for CeMM)

Renowned Dana-Far­ber sci­en­tist, men­tor and bio­phar­ma ad­vi­sor David Liv­ingston has died

David Livingston, the Dana-Farber/Harvard Med scientist who helped shine a light on some of the key molecular drivers of breast and ovarian cancer, died unexpectedly last Sunday.

One of the senior leaders at Dana-Farber during his nearly half century of work there, Livingston was credited with shedding light on the genes that regulate cell growth, with insights into inherited BRCA1 and BRCA2 mutations that helped lay the scientific foundation for targeted therapies and earlier detection that have transformed the field.

With hun­dreds of mil­lions spent on failed ac­cel­er­at­ed ap­provals, re­searchers call for faster FDA with­drawals

Between 2017 and 2019, Medicare spent more than $220 million on cancer drugs for which the indications were either voluntarily pulled by their applicants or FDA’s oncology adcomm had recommended their withdrawal.

That kind of massive spending on cancer drugs lacking overall survival benefit is wasteful and risks harming people’s health, a research letter published in JAMA Internal Medicine on Monday said. The researchers from Harvard and the London School of Economics called on the FDA to move faster in both requiring timely postmarketing trials and accelerating the speed in pulling these dangling approvals when the confirmatory studies fail.

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No­vo CEO Lars Fruer­gaard Jør­gensen on R&D risk, the deal strat­e­gy and tar­gets for gen­der di­ver­si­ty


I kicked off our European R&D summit last week with a conversation involving Novo Nordisk CEO Lars Fruergaard Jørgensen. Novo is aiming to launch a new era of obesity management with a new approval for semaglutide. And Jørgensen had a lot to say about what comes next in R&D, how they manage risk and gender diversity targets at the trendsetting European pharma giant.

John Carroll: I’m here with Lars Jørgensen, the CEO of Novo Nordisk. Lars, it’s been a really interesting year so far with Novo Nordisk, right? You’ve projected a new era of growing sales. You’ve been able to expand on the GLP-1 franchise that was already well established in diabetes now going into obesity. And I think a tremendous number of people are really interested in how that’s working out. You have forecast a growing amount of sales. We don’t know specifically how that might play out. I know a lot of the analysts have different ideas, how those numbers might play out, but that we are in fact embarking on a new era for Novo Nordisk in terms of what the company’s capable of doing and what it’s able to do and what it wants to do. And I wanted to start off by asking you about obesity in particular. Semaglutide has been approved in the United States for obesity. It’s an area of R&D that’s been very troubled for decades. There have been weight loss drugs that have come along. They’ve attracted a lot of attention, but they haven’t actually ever gained traction in the market. My first question is what’s different this time about obesity? What is different about this drug and why do you expect it to work now whereas previous drugs haven’t?

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Man­u­fac­tur­ing woes for No­vavax’s Covid jab bad­ly dis­rupt plans for roll­out to the poor — re­port

Production problems at a Novavax facility in Maryland have led to delays in the Covax vaccine sharing program. Now, a shortage of 1 billion doses is expected, as the supplier tries to navigate producing a shot up to regulators’ standards, Politico reported Tuesday.

The company has run into trouble with the purity of the vaccine. Novavax has had trouble proving it can produce a shot consistently up to standards, and it has caused significant delays in the rollout to low- and middle-income countries. This follows several delays at Novavax that has put the executive crew on the defensive.

Sur­geons suc­cess­ful­ly at­tach pig kid­ney to a hu­man for the first time, us­ing tech from Unit­ed's Re­vivi­cor

In a first, researchers reportedly successfully transplanted a pig kidney into a human without triggering an immediate immune response this week. And the technology came from the biotech United Therapeutics.

Surgeons spent three days attaching the kidney to the patient’s blood vessels, but when all was said and done, the kidney appeared to be functioning normally in early testing, Reuters and the New York Times were among those to report. The kidney came from a genetically altered pig developed through United’s Revivicor unit.

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Break­ing: Bio­gen sells just $300K worth of Aduhelm in Q3, as ques­tions on long term vi­a­bil­i­ty re­main

Barely anyone is accessing Biogen’s controversial Alzheimer’s treatment, with the company reporting just $0.3 million in Aduhelm sales in the third quarter. Although investors will be looking to the longer term, when CMS may decide to cover the drug and open the floodgates for more coverage, use of the drug is currently stalled.

Since June, when the FDA first signed off on the drug under its accelerated pathway, Biogen said Wednesday that it’s sold a total of $2 million worth of Aduhelm. Biogen said on its earnings call that about 120 sites so far have infused at least one patient with Aduhelm, which is priced at $56,000 annually. Morgan Stanley previously predicted about 14,000 patients will access Aduhelm by the end of 2022.

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Bill Gates at the Global Investment Summit in London, Oct. 19, 2021 (Leon Neal/Pool via AP Images)

Gates Foun­da­tion pledges $120M to ramp up gener­ic sup­ply of Mer­ck­'s Covid-19 pill while ac­tivists blast Pfiz­er's dis­pro­por­tion­ate pow­er

Merck’s molnupiravir may not be officially authorized anywhere in the world yet, but who will get access to it has shaped up to be a huge issue. The Bill & Melinda Gates Foundation is now stepping up to ensure lower-income countries won’t be left behind — and calling on others to follow its lead.

The oral antiviral pill, which was shown to dramatically cut the risk of severe Covid-19 disease and death in a Phase III study, is the latest rallying symbol in the battle against not just the coronavirus but the inequality it’s exposed.

Jon Cole, emergency doc at Minneapolis' Hennepin County Medical Center

FDA warns Min­neso­ta doc for run­ning two ke­t­a­mine tri­als with­out INDs, and with chil­dren and a preg­nant woman

The FDA on Tuesday released a warning letter sent to a Minnesota physician who failed to exclude vulnerable populations from two clinical trials comparing the anesthetic ketamine with haloperidol and midazolam as potential severe agitation treatments.

The letter makes clear that not only did Jon Cole, emergency doc at Minneapolis’ Hennepin County Medical Center, never file INDs for the trials with the FDA, as required by law, he also didn’t write appropriate protocols to ensure that children and pregnant women weren’t enrolled, and didn’t exclude those who were under the influence of intoxicants, in whom the use of ketamine is cautioned.