Trump’s 2021 bud­get re­quest seeks mod­est fund­ing in­crease for FDA

The Trump ad­min­is­tra­tion on Mon­day un­veiled its FY 2021 bud­get pro­pos­al, seek­ing a 10% re­duc­tion in over­all fund­ing for the De­part­ment of Health and Hu­man Ser­vices (HHS), but a slight in­crease in fund­ing for the FDA.

For the FDA, the bud­get re­quests $6.2 bil­lion over­all, in­clud­ing $3.3 bil­lion in dis­cre­tionary bud­get au­thor­i­ty (BA) and $2.9 bil­lion in user fees.

Steven Gross­man, deputy ex­ec­u­tive di­rec­tor of the Al­liance for a Stronger FDA, told Fo­cus that his group’s pre­lim­i­nary analy­sis is that the pro­pos­al in­cludes a $46 mil­lion in­crease in the BA (tax­pay­er fund­ed) ap­pro­pri­a­tions, which in­cludes $21 mil­lion in ad­di­tion­al funds for de­vices, $2 mil­lion in ad­di­tion­al funds for pro­grams in the FDA com­mis­sion­er’s of­fice and $24 mil­lion in to­tal rent items. Off­set­ting this are: $1 mil­lion in de­creas­es for an­i­mal drugs and the Na­tion­al Cen­ter for Tox­i­co­log­i­cal Re­search, the al­liance said.

“In rel­a­tive terms, we are pleased that the Ad­min­is­tra­tion’s com­mit­ment re­mains strong, even while many oth­er worth­while agen­cies are pro­posed for cuts. In ab­solute terms, fur­ther in­creas­es are go­ing to be need­ed be­cause the mis­sion and re­spon­si­bil­i­ties of the FDA are still grow­ing and the sci­ence is be­com­ing more com­plex,” a spokesper­son for the Al­liance told Fo­cus.

The FDA said the BA in­crease is $25.4 mil­lion more than the FY 2020 en­act­ed lev­el and the $2.9 bil­lion for user fees is an in­crease of $240 mil­lion com­pared to 2020.

Wish List

On the de­vice side, the HHS bud­get-in-brief in­cludes an in­crease of $18 mil­lion to con­tin­ue build­ing a knowl­edge man­age­ment sys­tem and por­tal for med­ical de­vices, fund­ing for which was al­so in­clud­ed in last year’s pro­pos­al. This in­creased fund­ing is im­por­tant as the FDA au­tho­rizes, on av­er­age, 12 new or mod­i­fied de­vices every busi­ness day. In 2019, the FDA al­so gave the “break­through” des­ig­na­tion to more than 100 de­vices, dou­bling the num­ber of des­ig­na­tions in 2018.

“This in­vest­ment would al­low FDA to trans­form the agency’s pre­mar­ket re­view and post­mar­ket sur­veil­lance pro­grams, and short­en re­view cy­cles with­out com­pro­mis­ing pa­tient safe­ty, quick­ly iden­ti­fy and ad­dress safe­ty sig­nals and cy­ber vul­ner­a­bil­i­ties, and spur the de­vel­op­ment of in­no­v­a­tive, safer, more ef­fec­tive de­vices,” HHS said.

The bud­get al­so pro­vides $45 mil­lion to in­crease the en­force­ment of opi­oids at in­ter­na­tion­al mail fa­cil­i­ties. HHS said this in­vest­ment will en­able the FDA to in­spect 100,000 pack­ages per year.

In ad­di­tion, the plan seeks a leg­isla­tive pro­pos­al to mod­ern­ize the over-the-counter drug mono­graph sys­tem and es­tab­lish a user fee for an es­ti­mat­ed $28 mil­lion in FY 2021. The Sen­ate passed an OTC mono­graph bill in De­cem­ber 2019.

An­oth­er more than $8 mil­lion in­crease is ex­pect­ed to sup­port an FDA-wide AI strat­e­gy to in­vest in work­force, al­go­rith­mic ca­pa­bil­i­ties, da­ta and tech­ni­cal in­fra­struc­ture. This in­vest­ment will “ad­vance and pro­mote con­sumer-friend­ly AI and dig­i­tal health med­ical de­vices,” HHS said.

The FDA al­so is plan­ning new work to com­bat drug short­ages, in­clud­ing a pro­pos­al that would ex­pand the FDA’s au­thor­i­ty to re­quire, when like­ly to help pre­vent or mit­i­gate a short­age, “that an ap­pli­cant eval­u­ate, sub­mit stud­ies to FDA, and la­bel a prod­uct with the longest pos­si­ble ex­pi­ra­tion date that FDA agrees is sci­en­tif­i­cal­ly jus­ti­fied.”

In ad­di­tion, the FDA’s jus­ti­fi­ca­tion in­cludes a pro­pos­al that would en­sure the FDA “has time­ly and ac­cu­rate in­for­ma­tion about like­ly or con­firmed na­tion­al short­ages of es­sen­tial de­vices to en­able FDA to take steps to pro­mote the con­tin­ued avail­abil­i­ty of de­vices of pub­lic health im­por­tance. Specif­i­cal­ly, FDA is seek­ing au­thor­i­ty to: re­quire firms to no­ti­fy FDA of an an­tic­i­pat­ed sig­nif­i­cant in­ter­rup­tion in the sup­ply of an es­sen­tial de­vice; re­quire all man­u­fac­tur­ers of de­vices de­ter­mined to be es­sen­tial to pe­ri­od­i­cal­ly pro­vide FDA with in­for­ma­tion about the man­u­fac­tur­ing ca­pac­i­ty of the es­sen­tial de­vice(s) they man­u­fac­ture; and au­tho­rize the tem­po­rary im­por­ta­tion of de­vices whose risks pre­sent­ed when pa­tients and health­care providers lack ac­cess to crit­i­cal­ly im­por­tant med­ical de­vices out­weigh com­pli­ance with U.S. reg­u­la­to­ry stan­dards.”

On the top­ic of pri­or­i­ty re­view vouch­ers (PRVs), the FDA is seek­ing clar­i­fi­ca­tion of the nov­el­ty stan­dard for a bi­o­log­i­cal prod­uct, based on its con­tain­ing key struc­tur­al fea­tures not con­tained in a pre­vi­ous­ly li­censed bi­o­log­i­cal prod­uct, which it says “would help pro­vide a more pre­dictable stan­dard for spon­sors seek­ing PRVs.”

And the FDA is seek­ing to al­low for ex­pe­dit­ed ac­cess to de­vices that would oth­er­wise be re­viewed un­der the pre­mar­ket ap­proval or de no­vo clas­si­fi­ca­tions path­ways if they are in­tend­ed to treat or di­ag­nose a life-threat­en­ing or ir­re­versibly de­bil­i­tat­ing dis­ease or con­di­tion and ad­dress an un­met med­ical need us­ing a two-step ap­proval. “These de­vices would be el­i­gi­ble for pro­vi­sion­al ap­proval based on a demon­stra­tion of safe­ty and per­for­mance plus ad­di­tion­al risk mit­i­ga­tions and could re­main on the mar­ket af­ter an es­tab­lished time pe­ri­od on­ly af­ter a demon­stra­tion of rea­son­able as­sur­ance of safe­ty and ef­fec­tive­ness,” the jus­ti­fi­ca­tion adds.

Com­ments

Al­though the num­bers or the pro­pos­als are not fi­nal, and some may stress that the bud­get plan is noth­ing more than a po­lit­i­cal ma­neu­ver, the Al­liance for a Stronger FDA not­ed: “The re­al­i­ty: whether Con­gress ac­cepts or re­jects the Pres­i­dent’s pro­pos­als or adds more mon­ey, his num­bers will be used as a base­line when the sub­com­mit­tees mark-up. That mat­ters.”

The bud­get al­so pro­pos­es to move the Cen­ter for To­bac­co Prod­ucts out of the FDA to cre­ate a new agency with­in HHS to fo­cus on to­bac­co reg­u­la­tion.

“This new agency would be led by a Sen­ate-con­firmed Di­rec­tor in or­der to in­crease di­rect ac­count­abil­i­ty and more ef­fec­tive­ly re­spond to this crit­i­cal area of pub­lic health con­cern,” the pro­pos­al says, not­ing that it al­so would al­low the FDA com­mis­sion­er to fo­cus on its tra­di­tion­al mis­sion of en­sur­ing the safe­ty of the na­tion’s food and med­ical prod­ucts sup­ply.

House Ap­pro­pri­a­tions Com­mit­tee Chair­woman Ni­ta Lowey (D-NY) said in a state­ment: “The Pres­i­dent’s lat­est bud­get is a dis­as­trous re­peat of the mis­placed pri­or­i­ties and cal­lous cuts he has pur­sued un­suc­cess­ful­ly in past re­quests. This reck­less pro­pos­al back­tracks on fund­ing lev­els agreed to in the bi­par­ti­san bud­get agree­ment and would gut in­vest­ments in Amer­i­ca’s work­ing fam­i­lies with deep cuts to crit­i­cal pro­grams like med­ical re­search, ed­u­ca­tion, and cli­mate sci­ence.”

Sen. Lamar Alexan­der (R-TN) added in a state­ment: “I ap­pre­ci­ate the pres­i­dent’s bud­get sug­ges­tions and will care­ful­ly con­sid­er his rec­om­men­da­tions as Con­gress be­gins the process to fund the fed­er­al gov­ern­ment for the next fis­cal year. Un­der the Con­sti­tu­tion, it is Con­gress’ job to set spend­ing pri­or­i­ties and pass ap­pro­pri­a­tions bills, and as a mem­ber of the Sen­ate Ap­pro­pri­a­tions Com­mit­tee, my pri­or­i­ties will con­tin­ue to be mak­ing sure our na­tion­al de­fense, na­tion­al lab­o­ra­to­ries, the Na­tion­al In­sti­tutes of Health and na­tion­al parks have the re­sources they need.”


RAPS: First pub­lished in Reg­u­la­to­ry Fo­cus™ by the Reg­u­la­to­ry Af­fairs Pro­fes­sion­als So­ci­ety, the largest glob­al or­ga­ni­za­tion of and for those in­volved with the reg­u­la­tion of health­care prod­ucts. Click here for more in­for­ma­tion.

Author

Zachary Brennan

managing editor, RAPS

Lessons for biotech and phar­ma from a doc­tor who chased his own cure

After being struck by a rare disease as a healthy third year medical student, David Fajgenbaum began an arduous journey chasing his own cure. Amidst the hustle of this year’s JP Morgan conference, the digital trials platform Medable partnered with Endpoints Studio to share Dr. Fajgenbaum’s story with the drug development industry.

What follows is an edited transcript of the conversation between Medable CEO Dr. Michelle Longmire and Dr. Fajgenbaum, and it is full of lessons for biotech executives charged with bringing the next generation of medicines to patients.

Kathy High (file photo)

Gene ther­a­py pi­o­neer Kathy High has left Spark af­ter com­plet­ing $4.3B union with Roche

Kathy High dedicated the past seven years of her life shepherding experimental gene therapies she’s developed at Children’s Hospital of Philadelphia toward the market as president and head of R&D at Spark Therapeutics. Now that the biotech startup is fully absorbed into Roche — with an FDA approval, a $4.3 billion buyout and a promising hemophilia program to boast — she’s ready to move on.

Roche confirmed her departure with Endpoints News and noted “she will take some well-deserved time off and then will begin a new chapter in a sabbatical at a university.”

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Tim Mayleben (file photo)

Es­pe­ri­on's goldilocks cho­les­terol fight­er wins FDA ap­proval — will its 'tra­di­tion­al' pric­ing ap­proach spur adop­tion?

It’s more effective than decades-old statins but not as good as the injectable PCSK9 — the goldilocks treatment for cholesterol-lowering, bempedoic acid, has secured FDA approval.

Its maker, Esperion Therapeutics, is betting that their pricing strategy — a planned list price of between $10 to $11 a day — will help it skirt the pushback the PCSK9 cholesterol fighters, Repatha and Praluent, got from payers for their high sticker prices.

The sky-high expectations for the pair of PCSK9 drugs that were first approved in 2015 quickly simmered — and despite a 60% price cut, coupled with data showing the therapies also significantly cut cardiovascular risk, sales have not really perked up.

Esperion is convinced that by virtue of being a cheaper oral therapy, bempedoic acid will hit that sweet spot in terms of adoption.

“We’re kind of like the old comfortable shoe,” Esperion’s chief commercial officer Mark Glickman remarked in an interview with Endpoints News ahead of the decision date. “It’s an oral product, once-daily and nontitratable — these are things that just resonate so true with patients and physicians and I think we’ve kind of forgotten about that.”

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James Collins, Broad Institute via Youtube

UP­DAT­ED: A space odyssey for new an­tibi­otics: MIT's ma­chine learn­ing ap­proach

Drug development is complex, expensive and comes with lousy odds of success — but in most cases, if you make it across the finish line brandishing a product with an edge (and play your cards right) it can be a lucrative endeavor.

As it stands, the antibiotic market is cursed — it harbors the stink of multiple bankruptcies, a dearth of innovation, and is consequently barely whetting the voracious appetites of big pharma or venture capitalists. Enter artificial intelligence — the biopharma industry’s cure-all for the pesky process of making a therapeutic, including data mining, drug discovery, optimal drug delivery, and addressable patient population.

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Gilead los­es two more patent chal­lenges on HIV pill, set­ting up court­room fight in Delaware

Gilead sustained two more losses in their efforts to rid themselves of an activist-backed patent lawsuit from the US government over a best-selling HIV pill.

Urged on by activists seeking to divert a portion of Gilead’s revenue to clinics and prevention programs, the Department of Health and Human Services made a claim to some of the patents for the best-selling HIV prevention drug, Truvada, also known as PrEP. Gilead responded by arguing in court that HHS’s patents were invalid.

Today, the US Patent and Trademark Office ruled that Gilead was likely to lose the last two of those challenges as well. The USPTO ruled against Gilead on the first two patents earlier this month.

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Tal Zaks (Moderna via YouTube)

For two decades, a new vac­cine tech­nol­o­gy has been slow­ly ap­proach­ing prime time. Now, can it stop a pan­dem­ic?

Two months before the outbreak, Moderna CMO Tal Zaks traveled from Cambridge, MA to Washington DC to meet with Anthony Fauci and the leaders of the National Institutes of Health.

For two years, Moderna had worked closely with NIH researchers to build a new kind of vaccine for MERS, one of the deadliest new viruses to emerge in the 21st century. The program was one test for a new technology designed to be faster, cheaper and more precise than the ways vaccines had been made for over a century. They had gathered evidence the technology could work in principle, and Fauci, the longtime head of the National Institute of Allergy and Infectious Diseases and a longtime advocate for better epidemic preparedness, wanted to see if it, along with a couple of other approaches, could work in a worst-case scenario: A pandemic.

“[We were] trying to find a test case for how to demonstrate if our technology could rapidly prepare,” Zaks told Endpoints News.

Zaks and Fauci, of course, wouldn’t have to wait to develop a new test. By year’s end, an outbreak in China would short circuit the need for one and throw them into 24/7 work on a real-world emergency. They also weren’t the only ones with new technology who saw a chance to help in a crisis.

An ocean away, Lidia Oostvogels was still on vacation and relaxing at her mother’s house in Belgium when her Facebook started changing. It was days after Christmas and on most people’s feeds, the news that China had reported a novel virus to the World Health Organization blurred into the stream of holiday sweaters and fir trees. But on Oostvogels’s feed, full of vaccine researchers and virus experts, speculation boiled: There was a virus in China, something contained to the country, but “exotic,” “weird,” and maybe having to do with animals. Maybe a coronavirus.

Lidia Oostvogels

“I was immediately thinking like, ‘Hey, this is something that if needed, we can play a role,'” Oostvogels told Endpoints.

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Christos Kyratsous (via LinkedIn)

He built a MERS treat­ment in 6 months and then the best Ebo­la drug. Now Chris­tos Kyrat­sous turns his sights on Covid-19

TARRYTOWN, NY — In 2015, as the Ebola epidemic raged through swaths of West Africa, Kristen Pascal’s roommates sat her down on their couch and staged an intervention.

“Are you sure this is what you want to be doing with your life?” she recalls them asking her.

Special report

Pascal, a research associate for Regeneron, had been coming home at 2 am and leaving at 6 am. At one point, she didn’t see her roommate for a week. For months, that was life in Christos Kyratsous’ lab as the pair led a company-wide race to develop the first drug that could effectively treat Ebola before the outbreak ended. For Pascal, that was worth it.

“I’m ok, I don’t have Ebola,” Pascal told them. “I see that death toll rising and I can’t not do something about it.”

Last August, Regeneron learned they had succeeded: In a large trial across West Africa, their drug, REGN-EB3, was vastly more effective than the standard treatments. It was surprise news for the company, coming just 10 months into a trial they thought would take several years and a major victory in the global fight against a deadly virus that killed over 2,000 in 2019 and can carry a mortality rate of up to 90%.

For Kyratsous and Pascal, though, it brought only fleeting reprieve. Just four months after the NIH informed them REGN-EB3 worked, Kyratsous was back in his office reading the New York Times for updates on a new outbreak on another continent, and wondering alongside Pascal and senior management whether it was time to pull the trigger again.

In late January, as the death toll swelled and the first confirmed cases outside China broke double digits, they made a decision. Soon they were back on the phone with the multiple government agencies and their coronavirus partners at the University of Maryland’s Level 3 bio lab. The question was simple: Can Kyratsous and his team use a process honed over two previous outbreaks, and create a treatment before the newest epidemic ends? Or worse, if, as world health experts fear, it doesn’t vanish but becomes a recurrent virus like the flu?

“Christos likes things immediately,” Matt Frieman, Regeneron’s coronavirus collaborator at the University of Maryland, told Endpoints. “That’s what makes us good collaborators: We push each other to develop things faster and faster.”

Kristen Pascal (Regeneron)

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The first time Regeneron tried to respond to a global outbreak, it was something of a systems test, Kyratsous explains from his office at Regeneron’s Tarrytown headquarters. Kyratsous, newly promoted, has crammed it with photos of his family, sketches of viral vectors and a shark he drew for his 3-year-old son. He speaks rapidly – an idiosyncrasy his press person says has only been aggravated this afternoon by the contents of his “Regeneron Infectious Diseases”-minted espresso glass – and he gesticulates with similar fluidity, tumbling through antibodies, MERS, the novel coronavirus, Ebola-infected monkeys.

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Bank­rupt an­tibi­otics mak­er Ar­a­digm turns to old part­ner/in­vestor for fi­nal $3M fire sale

Grifols once paid Aradigm $26 million for a stake in its inhaled antibiotics. But with Aradigm now in bankruptcy, the Spanish drugmaker is dishing out a final $3.2 million to buy it all.

The fire sale — which comes one year after Aradigm filed for Chapter 11 following a regulatory trifecta for disaster — will see Grifols obtain assets and IP to Apulmiq (formerly Pulmaquin and Linhaliq in Europe), Lipoquin and free ciprofloxacin. In addition to waiving its claims in the bankruptcy case, Grifols also agreed to milestone payments up to $3 million more upon any regulatory approvals.

DB­V's peanut pre­ven­tion patch ap­proach­es key stage of ap­proval process

Almost a year and a half after DBV Technologies pulled its peanut allergy immunotherapy patch from FDA review, the biotech will get their day in court. The FDA has scheduled an advisory committee hearing for May 15.

In the two-horse race to develop the first immunotherapy for peanut allergy, DBV had the early lead, filing an NDA for their patch in 2018. But on December 20 of that year, the company withdrew their application after, they said, meeting with regulators and determining they had not submitted “sufficient detail regarding data on manufacturing procedures and quality controls.” Aimmune filed their BLA 3 days later and won approval as the first immunotherapy for peanuts this month.