Scott Got­tlieb to bio­phar­ma: You want­ed us to help mod­ern­ize R&D, now go out and do it

FDA com­mis­sion­er Scott Got­tlieb is us­ing the fi­nal days of his brief term to scold the drug R&D world for fail­ing to keep up. Wide­ly praised for his work aimed at mod­ern­iz­ing the re­search tech and strate­gies used to ad­vance new drugs, Got­tlieb says that drug de­vel­op­ers aren’t tak­ing ad­van­tage of the new, more ef­fi­cient ap­proach­es to clin­i­cal work — and that needs to change.

It’s not just the drug mak­ers, says Got­tlieb. CROs, who do much of the heavy lift­ing in the clin­ic, share a con­sid­er­able por­tion of the blame.

“Un­for­tu­nate­ly, we’ve seen a con­tin­ued re­luc­tance to adopt in­no­v­a­tive ap­proach­es among spon­sors and clin­i­cal re­search or­ga­ni­za­tions,” not­ed Got­tlieb in a state­ment. “In some cas­es, the busi­ness mod­el adopt­ed by the clin­i­cal tri­al es­tab­lish­ment just isn’t com­pat­i­ble with the kind of pos­i­tive but dis­rup­tive changes that cer­tain in­no­va­tions can en­able. We ap­pre­ci­ate that sci­en­tif­ic and tech­ni­cal com­plex­i­ty is a re­al and on­go­ing chal­lenge, but in­dus­try and acad­e­mia al­so need to in­vest in and lever­age these ap­proach­es and de­vel­op new in­cen­tives that re­ward col­lab­o­ra­tion and da­ta shar­ing across the clin­i­cal re­search en­ter­prise.”

He goes on in a jar­gon-laden style:

New re­search par­a­digms are need­ed to break down bar­ri­ers be­tween re­al world da­ta and clin­i­cal re­search, so that ev­i­dence can be shared rapid­ly to im­prove both do­mains across a learn­ing health care sys­tem. For in­stance, more tri­als can in­cor­po­rate da­ta from elec­tron­ic health records, and adopt elec­tron­ic in­formed con­sent, to en­roll more pa­tients in clin­i­cal tri­als clos­er to where they live and work. This can re­duce bar­ri­ers to clin­i­cal tri­al par­tic­i­pa­tion and ac­cel­er­ate re­searchers’ abil­i­ty to ask and an­swer im­por­tant ques­tions.

Got­tlieb goes on with the lat­est in a se­ries of guid­ances aimed at stream­lin­ing R&D. The list in­cludes the use of com­put­er­ized sys­tems for over­sight, ad­vice on adding pa­tients to tri­als with more “chal­leng­ing con­di­tions,” to make tri­als bet­ter re­flect re­al world con­di­tions and more about avoid­ing an­i­mals in drug de­vel­op­ment work.

The in­dus­try has been cheer­ing Got­tlieb and the FDA on for this kind of work. Now Got­tlieb, who winds up his tenure at the agency at the end of this month, wants the bio­phar­ma world to put it to prac­tice.

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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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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De­spite a slow start to the year for deals, PwC pre­dicts a flur­ry of ac­tiv­i­ty com­ing up

Despite whispers of a busy year for M&A, deal activity in the pharma space is actually down 30% on a semi-annualized basis, according to PwC’s latest report on deal activity. But don’t rule out larger deals in the second half of the year, the consultants said.

PwC pharmaceutical and life sciences consulting solutions leader Glenn Hunzinger expects to see Big Pharma companies picking up earlier stage companies to try and fill pipeline gaps ahead of a slew of big patent cliffs. Though a bear market continues to maul the biotech sector, Hunzinger said recent deals indicate that pharma companies are still paying above current trading prices.

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.

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.”

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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Joe Papa (Ryan Remiorz/The Canadian Press via AP, File)

Joe Pa­pa re­signs as chair of Bausch Health as bil­lion­aire John Paul­son takes over

Joe Papa, chair of Bausch Health, officially resigned on Thursday and the board appointed billionaire hedge fund manager John Paulson as the new chair, effective immediately.

The specialty pharma company sought to make clear that Papa’s abrupt departure “was not due to any dispute or disagreement with the Company, its management or the Board on any matter relating to the Company’s operations, policies or practices.”

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