Stephen Hahn, AP

Hahn stress­es im­por­tance of da­ta, RWE in first all-hands meet­ing

FDA Com­mis­sion­er Stephen Hahn ad­dressed FDA staffers in an all-hands meet­ing for the first time on Thurs­day, ex­plain­ing how he wants to do more with re­al-world ev­i­dence (RWE), which has been a con­tro­ver­sial sub­ject.

While not­ing that the FDA is op­er­at­ing in a time of “un­sur­passed sci­en­tif­ic and tech­no­log­i­cal in­no­va­tion,” Hahn called to un­leash the pow­er of da­ta and “to at­tain more and bet­ter da­ta.” His com­ments build up­on the agency’s tech­nol­o­gy mod­ern­iza­tion plan un­veiled last Sep­tem­ber.

“By har­ness­ing this pow­er, we can im­prove our reg­u­la­to­ry de­ci­sion-mak­ing and more ef­fec­tive­ly con­nect to­day’s ground­break­ing sci­en­tif­ic dis­cov­er­ies with the rapid de­vel­op­ment and ap­proval of new prod­ucts. We can al­so in­crease the knowl­edge pa­tients and con­sumers have to make in­formed de­ci­sions about FDA-reg­u­lat­ed prod­ucts,” he said.

Hahn al­so con­tin­ued to push the agen­da of his pre­de­ces­sor, Scott Got­tlieb, who re­leased an RWE frame­work in De­cem­ber 2018. Al­though the agency has on­ly used RWE in lim­it­ed amounts so far.

“Specif­i­cal­ly, I be­lieve there is great promise in the ef­fec­tive use and in­te­gra­tion of pa­tient-lev­el da­ta or re­al-world ev­i­dence such as elec­tron­ic health records, clin­i­cal tri­als, med­ical stud­ies, and pa­tient reg­istries,” Hahn said.

For­mer FDA Com­mis­sion­ers Robert Califf, Mark Mc­Clel­lan and An­drew von Es­chen­bach al­so of­fered their rec­om­men­da­tions to ex­pand the use of RWE in reg­u­la­to­ry and pay­ment de­ci­sion-mak­ing. But ques­tions on the ap­plic­a­bil­i­ty of RWE linger. A re­cent study in JA­MA Open Net­work found that re­al-world da­ta (RWD) could on­ly be used to fea­si­bly repli­cate 15% of high-pro­file clin­i­cal tri­als con­duct­ed in the US in 2017.

For­mer FDA Com­mis­sion­er Rob Califf al­so said RWE and RWD can in­clude ran­dom­iza­tion and oth­er meth­ods to en­able causal in­fer­ence. “We wor­ried at the time that the terms RWD & RWE would be hi­jacked to fos­ter low qual­i­ty re­search. This must be fought, be­cause well done RWD & RWE with prop­er use of meth­ods for causal in­fer­ence is HIGH­ER qual­i­ty & more gen­er­al­iz­able,” Califf wrote on Twit­ter.

Hahn al­so stressed the need to build up­on the FDA’s Pa­tient-Fo­cused Drug De­vel­op­ment ini­tia­tive so “con­sumers may be even more in­formed and aware about the en­tire life­cy­cle of prod­ucts we reg­u­late in or­der to live more healthy and pro­duc­tive lives.”

And per­haps as a re­sponse to cri­tiques of the agency’s re­cent speedy ap­provals of new drugs, Hahn said the agency needs to “main­tain the vi­tal bal­ance be­tween the speedy ap­proval of new prod­ucts and the in­tegri­ty of the da­ta and the process it­self.”Ed­i­tor’s Note:

Ar­ti­cle up­dat­ed with com­ment from for­mer FDA Com­mis­sion­er Robert Califf.


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.

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.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,300+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,300+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,300+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,300+ biopharma pros reading Endpoints daily — and it's free.

FDA warns Mex­i­can glyc­erin man­u­fac­tur­er for re­fus­ing an in­spec­tion

A drug manufacturing facility in Mexico is drawing the ire of the FDA after it ignored the US regulator’s inspection requests and phone calls.

According to the warning letter issued on June 13, Glicerinas Industriales refused a pre-announced inspection during a phone call with FDA prior to the inspection at the company’s facility in Zapopan, Mexico, a city next to Guadalajara, which was planned for May 16 to May 20.