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.

M&A: a crit­i­cal dri­ver for sus­tain­able top-line growth in health­care

2021 saw a record $600B in healthcare M&A activity. In 2022, there is an anticipated slowdown in activity, however, M&A prospects remain strong in the medium to long-term. What are future growth drivers for the healthcare sector? Where might we see innovations that drive M&A? RBC’s Andrew Callaway, Global Head, Healthcare Investment Banking discusses with Vito Sperduto, Global Co-Head, M&A.

15 LGBTQ lead­ers in bio­phar­ma; Paul Stof­fels’ Gala­pa­gos re­vamp; As­traZeneca catch­es up in AT­TR; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

A return to in-person conferences also marks a return to on-the-ground reporting. My colleagues Beth Synder Bulik and Nicole DeFeudis were on-site at Cannes Lions, bringing live coverage of pharma’s presence at the ad festival — accompanied by photos from Clara Bui, our virtual producer, that bring you right to the scene. You can find a recap (and links to all the stories) below.

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Abortion-rights protesters regroup and protest following Supreme Court's decision to overturn Roe v. Wade. (AP Photo/Gemunu Amarasinghe)

Fol­low­ing SCO­TUS de­ci­sion to over­turn abor­tion pro­tec­tions, AG Gar­land says states can't ban the abor­tion pill

Following the Supreme Court’s historic decision on Friday to overturn Americans’ constitutional right to an abortion after almost 50 years, Attorney General Merrick Garland sought to somewhat reassure women that states will not be able to ban the prescription drug sometimes used for abortions.

Following the decision, the New England Journal of Medicine also published an editorial strongly condemning the reversal, saying it “serves American families poorly, putting their health, safety, finances, and futures at risk.”

AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

As­traZeneca de­buts first con­sumer cam­paign for its Covid-19 pro­phy­lac­tic Evusheld — and a first for EUA drugs

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

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Joe Wiley, Amryt Pharma CEO

Am­ryt Phar­ma sub­mits a for­mal dis­pute res­o­lu­tion to the FDA over re­ject­ed skin dis­ease drug

The story of Amryt Pharma’s candidate for the genetic skin condition epidermolysis bullosa, or EB, will soon enter another chapter.

After the Irish drugmaker’s candidate, dubbed Oleogel-S10 and marketed as Filsuvez, was handed a CRL earlier this year, the company announced in a press release that it plans to submit a formal dispute resolution request for the company’s NDA for Oleogel-S10.

Sen. Thom Tillis (R-NC) (J. Scott Applewhite/AP Images)

Phar­ma-friend­ly sen­a­tor calls on FDA for a third time to show patent pro­tec­tions should­n't be blamed for high drug prices

North Carolina Republican Sen. Thom Tillis made a name for himself in the 2020 election cycle as the darling of the pharma industry, accepting hundreds of thousands in campaign contributions, even from the likes of Pfizer CEO Albert Bourla.

Those contributions have led Tillis to attempt to re-write patent laws in pharma’s favor, a move which failed to gain steam in 2019, and request for a third time since January that the FDA should help stop “the false narrative that patent protections are to blame for high drug prices.”

EMA signs off on 3 drugs re­cent­ly re­ject­ed by FDA, in­clud­ing Bio­Mar­in's new he­mo­phil­ia gene ther­a­py

The EMA’s human medicines committee on Friday recommended three new drugs for approval or conditional approval, even as their US counterparts have rejected these three for various reasons.

In a major move, CHMP offered a thumbs-up to a conditional marketing authorization for the first gene therapy to treat severe hemophilia A, although the agency cautioned that it’s so far unknown how long the effects of infusion will last.

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Alarmed by side ef­fect, FDA slaps clin­i­cal hold on Sarep­ta's next-gen Duchenne drug

Sarepta Therapeutics’ next-gen Duchenne muscular dystrophy drug has been hit with a clinical hold after investigators flagged a serious case of low magnesium levels in one patient’s blood.

Screening and dosing will be halted in what is known as Part B of the Phase II MOMENTUM study, which has enrolled about half of the planned patients. Sarepta said it will be submitting information on all cases of the condition, known as hypomagnesemia, per the FDA’s request and proposing some changes to the risk mitigation and safety monitoring plan.

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When will the FDA re­scind a break­through des­ig­na­tion? New draft guid­ance spells out the com­pli­cat­ed de­tails

Although it’s exceedingly rare for the FDA to rescind a breakthrough designation once it’s granted, there has been a recent uptick — as in 2020 and 2021 combined, the agency rescinded 17 BTDs, compared to just 18 rescinded from 2015 to 2019 combined.

Protagonist Therapeutics saw this reality up close and personal in April after a clinical hold lifted on its experimental blood cancer drug, as the company revealed that the FDA sought to revoke the BTD. The decision, Protagonist says, stems from “observed malignancies” related to the hold, initially imposed in September 2021.

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