Got­tlieb lays out re­al world ev­i­dence pri­or­i­ties for 2019 with an eye on dig­i­tal tools

Stress­ing the need for speed and ef­fi­cien­cy, FDA Com­mis­sion­er Scott Got­tlieb on Mon­day of­fered a look at FDA’s plans around re­al-world da­ta (RWD) and re­al-world ev­i­dence (RWE) in 2019.

The speech at the Bi­par­ti­san Pol­i­cy Cen­ter built off a frame­work, re­leased in De­cem­ber, that sought to be­gin the con­ver­sa­tion on how RWD and RWE can sup­port changes to la­bel­ing, in­clud­ing adding or mod­i­fy­ing an in­di­ca­tion, changes in dose, dose reg­i­men or route of ad­min­is­tra­tion; chang­ing or adding new pop­u­la­tions; or the ad­di­tion of com­par­a­tive ef­fec­tive­ness or safe­ty in­for­ma­tion.

“Tra­di­tion­al post­mar­ket stud­ies typ­i­cal­ly re­quire years to de­sign and com­plete and cost mil­lions of dol­lars. By us­ing RWD and RWE, we may be able to pro­vide pa­tients and providers with im­por­tant an­swers much soon­er – iden­ti­fy­ing a broad­er range of safe­ty sig­nals more quick­ly, and fol­low­ing up on them much more ef­fec­tive­ly,” Got­tlieb said, not­ing this is es­pe­cial­ly im­por­tant for treat­ments for un­com­mon con­di­tions, such as rare tu­mor types.

Got­tlieb al­so laid out four ad­di­tion­al ac­tiv­i­ties the FDA is tak­ing in con­nec­tion to RWE and RWD this year.

First, to sup­port the in­te­gra­tion of dig­i­tal tech­nolo­gies in clin­i­cal tri­als, the FDA is plan­ning to con­vene a stake­hold­er meet­ing to help de­vel­op a frame­work on how dig­i­tal sys­tems can be used to en­hance the over­sight of clin­i­cal tri­als.

“To take one ex­am­ple, re­mote- and risk-based mon­i­tor­ing can pro­vide bet­ter reg­u­la­to­ry over­sight,” he said. “These ap­proach­es may low­er de­vel­op­ment costs, and en­able more tri­al sites to an­swer im­por­tant sci­en­tif­ic and clin­i­cal ques­tions as a way to im­prove pa­tient care.”

He al­so said the FDA will use dig­i­tal tech­nolo­gies to bring clin­i­cal tri­als to the pa­tient, rather than al­ways re­quir­ing the pa­tient to trav­el to the in­ves­ti­ga­tor.

“To sup­port the de­vel­op­ment and adop­tion of de­cen­tral­ized tri­als, the FDA es­tab­lished a for­mal work­ing group on de­cen­tral­ized tri­als, and we’re work­ing to­wards writ­ing a guid­ance doc­u­ment to out­line our ap­proach. We’ll
have much more to say about this in the next month,” he said.

And third, the FDA is ex­plor­ing how re­view­ers can gain more in­sight in­to how la­bel­ing changes in­form provider pre­scrib­ing de­ci­sions and pa­tient out­comes. The FDA’s In­for­ma­tion Ex­change and Da­ta Trans­for­ma­tion (IN­FORMED) is us­ing RWD to ex­am­ine the im­pact of a re­cent FDA la­bel­ing change for two ap­proved prod­ucts from weight-based dos­ing to flat-dos­ing of im­mune check­point in­hibitors.

In 2019, IN­FORMED is go­ing to be work­ing with FDA’s med­ical prod­uct cen­ters to de­vel­op an FDA cur­ricu­lum on ma­chine learn­ing and ar­ti­fi­cial in­tel­li­gence in part­ner­ship with ex­ter­nal aca­d­e­m­ic part­ners.

“The agency will al­so pi­lot a com­pet­i­tive fel­low­ship pro­gram un­der IN­FORMED in ar­ti­fi­cial in­tel­li­gence and ma­chine learn­ing that al­lows post-doc­tor­al fel­lows from lead­ing aca­d­e­m­ic cen­ters to join the FDA for two-year fel­low­ships to de­vel­op high-im­pact AI-based reg­u­la­to­ry sci­ence tools by work­ing close­ly with men­tors from the agency’s med­ical prod­uct cen­ters,” Got­tlieb said.

He al­so not­ed that in on­col­o­gy, the FDA cur­rent­ly has new drug ap­pli­ca­tions un­der re­view where RWD and RWE are help­ing to in­form on­go­ing eval­u­a­tions.

The FDA’s On­col­o­gy Cen­ter of Ex­cel­lence (OCE) is work­ing with Friends of Can­cer Re­search, the Na­tion­al Can­cer In­sti­tute and oth­er stake­hold­ers to har­mo­nize ref­er­ence stan­dards for as­sess­ing tu­mor mu­ta­tion­al bur­den to help iden­ti­fy can­cer pa­tients who are more like­ly to re­spond to im­munother­a­py.

“OCE is al­so work­ing on a project ex­plor­ing whether it’s pos­si­ble to use re­al world end­points, like time to treat­ment dis­con­tin­u­a­tion (TTD) from in lega­cy datasets as a po­ten­tial re­al-world end­point for prag­mat­ic ran­dom­ized clin­i­cal tri­als for FDA-ap­proved ther­a­pies in the off-la­bel set­ting, and whether syn­thet­ic con­trol arms based on pri­or clin­i­cal tri­als can be used for tu­mor types where the stan­dard of care has not changed in decades, and the prog­no­sis is es­pe­cial­ly poor,” he added.


Scott Got­tlieb at a 2016 event. BI­PAR­TI­SAN POL­I­CY CEN­TER

First pub­lished here. Reg­u­la­to­ry Fo­cus is the flag­ship on­line pub­li­ca­tion of the Reg­u­la­to­ry Af­fairs Pro­fes­sion­als So­ci­ety (RAPS), the largest glob­al or­ga­ni­za­tion of and for those in­volved with the reg­u­la­tion of health­care and re­lat­ed prod­ucts, in­clud­ing med­ical de­vices, phar­ma­ceu­ti­cals, bi­o­log­ics and nu­tri­tion­al prod­ucts. Email news@raps.org for more in­for­ma­tion.

How pre­pared is bio­phar­ma for the cy­ber dooms­day?

One of the largest cyberattacks in history happened on a Friday, Eric Perakslis distinctly remembers.

Perakslis, who was head of Takeda’s R&D Data Sciences Institute and visiting faculty at Harvard Medical School at the time, had spent that morning completing a review on cybersecurity for the British Medical Journal. Moments after he turned it in, he heard back from the editor: “Have you heard what’s going on right now?”

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Scoop: Boehringer qui­et­ly shut­ters a PhII for one of its top drugs — now un­der re­view

Boehringer Ingelheim has quietly shut down a small Phase II study for one of its lead drugs.

The private pharma player confirmed to Endpoints News that it had shuttered a study testing spesolimab as a therapy for Crohn’s patients suffering from bowel obstructions.

A spokesperson for the company tells Endpoints:

Taking into consideration the current therapeutic landscape and ongoing clinical development programs, Boehringer Ingelheim decided to discontinue our program in Crohn’s disease. It is important to note that this decision is not based on any safety findings in the clinical trials.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Pearl Huang, Dunad Therapeutics CEO (Ken Richardson, PR Newswire)

Long­time biotech leader Pearl Huang takes the reins as CEO of No­var­tis-backed up­start

It has only been a few months since Pearl Huang exited the top seat at Cygnal Therapeutics, but now she’s back at the helm of another biotech.

After taking a few months off — passing an exam in that time to get her captain’s license from the US Coast Guard — she’s been named CEO of Dunad Therapeutics, a biotech focused on developing a small molecule covalent therapies that was founded in 2020. Huang told Endpoints News that two factors attracted her to going back to the c-suite: the company’s technology and its co-founders.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,400+ biopharma pros reading Endpoints daily — and it's free.

Matt Gline, Roivant CEO (John Sciulli/Getty Images for GLG)

Roivant chops sick­le cell gene ther­a­py, der­ma­tol­ogy drugs to fo­cus on 'high­er val­ue pro­ject­s'

Roivant is sweeping a suite of drugs, including a gene therapy for sickle cell disease already in the clinic, out of its pipeline.

Six programs from four of its “vants” are being wound down as part of “a company-wide cost optimization and pipeline reprioritization initiative to reduce our expected operating expenses and prioritize our capital resources.”

When reached by Endpoints News, a spokesperson said, “We don’t anticipate a material reduction in headcount but we will likely reassign some folks to higher value projects as part of winding down specific programs.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,400+ biopharma pros reading Endpoints daily — and it's free.

Laurence Reid, Decibel CEO

Still in pre­clin­i­cal test­ing for ear gene ther­a­pies, Deci­bel touts small snap­shot of chemo-in­duced hear­ing loss drug

Though Decibel Therapeutics has largely pivoted toward gene therapies for the inner ear, its lead clinical candidate simply aims to protect cancer patients from chemotherapy-induced hearing loss. On Tuesday, the biotech presented its first efficacy data for the program, and execs like what they see.

Decibel reported interim results from a Phase Ib study showing the experimental drug, dubbed DB-020, largely protected a small group of patients from losing their hearing. Researchers used a particularly unique study design, administering the compound in one of each patients’ ears before they received cisplatin chemotherapy and placebo in the other.

Alex­ion puts €65M for­ward to strength­en its po­si­tion on the Emer­ald Isle

Ireland has been on a roll in 2022, with several large pharma companies announcing multimillion-euro projects. Now AstraZeneca’s rare disease outfit Alexion is looking to get in on the action.

Alexion on Friday announced a €65 million ($68.8 million) investment in new and enhanced capabilities across two sites in the country, including at College Park in the Dublin suburb of Blanchardstown and the Monksland Industrial Park in the central Irish town of Athlone, according to the Industrial Development Agency of Ireland.

Fed­er­al judge de­nies Bris­tol My­er­s' at­tempt to avoid Cel­gene share­hold­er law­suit

Some Celgene shareholders aren’t happy with how Bristol Myers Squibb’s takeover went down.

On Friday, a New York federal judge ruled that they have a case against the pharma giant, denying a request to dismiss allegations that it purposely slow-rolled Breyanzi’s approval to avoid paying out $6.4 billion in contingent value rights (CVR).

When Bristol Myers put down $74 billion to scoop up Celgene back in 2019, liso-cel — the CAR-T lymphoma treatment now marketed as Breyanzi — was supposedly one of the centerpieces of the deal. After going back and forth on negotiations for about six months, BMS put $6.4 billion into a CVR agreement that required an FDA approval for Zeposia, Breyanzi and Abecma, each by an established date.

Ben Zimmer, Priovant CEO

Roivant un­veils lat­est spin­out as Pfiz­er en­trusts JAK1/TYK2 to Pri­o­vant

In November, Pfizer disclosed it’s spun out the Phase II dual JAK1/TYK2 inhibitor to a startup formed in collaboration with an unnamed, experienced partner.

We now know who the partner is. And as Pfizer and Roivant officially take the wraps off Priovant Therapeutics, the companies reveal that they have started two registrational trials of the drug, brepocitinib, as part of a broader plan to develop a big, first-in-class franchise spanning multiple orphan and specialty autoimmune diseases.

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 144,400+ biopharma pros reading Endpoints daily — and it's free.