Pa­tient reg­istries may prove valu­able in reg­u­la­to­ry de­ci­sions, EMA study finds

Al­though pa­tient reg­istries can be het­ero­ge­neous in their qual­i­ty and de­sign, a new study in Drug Safe­ty co-au­thored by an EMA of­fi­cial ex­plains how pa­tient reg­istries could pro­vide valu­able da­ta for reg­u­la­to­ry de­ci­sions.

The au­thors ex­plain how such reg­istries can be used when ran­dom­ized con­trolled tri­als are not fea­si­ble, such as with some rare dis­eases, or to meet post­mar­ket­ing com­mit­ments.

“For prod­ucts grant­ed con­di­tion­al mar­ket­ing ap­proval, reg­istry stud­ies may pro­vide post-au­tho­ri­sa­tion da­ta to ful­fil reg­u­la­tor-im­posed spe­cif­ic oblig­a­tions to con­firm safe­ty and/or ef­fec­tive­ness, as is the case with the re­cent­ly au­tho­rised chimeric anti­gen re­cep­tor (CAR) T-cell prod­ucts, tia­gen­le­cleu­cel and axi­cab­ta­gene ciloleu­cel,” the study says.

The eight au­thors, from the EMA, Queens Mary Uni­ver­si­ty of Lon­don, the Uni­ver­si­ty of Gronin­gen in the Nether­lands and the Dutch Med­i­cines Eval­u­a­tion Board, al­so ex­plain how reg­istries are cur­rent­ly un­der­used.

Of 335 prod­ucts rec­om­mend­ed for ap­proval by the EMA’s Com­mit­tee for Med­i­c­i­nal Prod­ucts for Hu­man Use be­tween 2005 and 2013, 31 reg­istries were re­quest­ed to ful­fill a con­di­tion of the mar­ket­ing au­tho­riza­tion, but by De­cem­ber 2017, just ten had been com­plet­ed.

To try to fig­ure out what fac­tors fa­cil­i­tate reg­istry use, the au­thors syn­the­sized ob­ser­va­tions and rec­om­men­da­tions pub­lished in five re­ports aris­ing from a mul­ti-stake­hold­er EMA con­sul­ta­tion and four dis­ease-spe­cif­ic work­shops con­duct­ed as part of the EMA’s Pa­tient Reg­istries Ini­tia­tive.

The study ex­plains how the ab­sence or in­com­plete­ness of 10 dif­fer­ent fac­tors “great­ly im­ped­ed reg­istry use.”
Such fac­tors in­clude the use of com­mon core da­ta sets, com­mon da­ta cod­ing ter­mi­nolo­gies, da­ta ac­cess and shar­ing and da­ta link­age ca­pac­i­ty, among oth­ers.

“From a reg­u­la­to­ry per­spec­tive, the ul­ti­mate re­quire­ment of pa­tient reg­istries is that they per­mit the con­duct of high-qual­i­ty stud­ies that eval­u­ate the safe­ty and ef­fec­tive­ness of med­i­cines. Recog­nis­ing the ben­e­fits and chal­lenges in­her­ent in us­ing ob­ser­va­tion­al da­ta for med­i­cines as­sess­ments, in 2019, EMA will pub­lish method­olog­i­cal and op­er­a­tional ad­vice on han­dling reg­istry da­ta in post-au­tho­ri­sa­tion stud­ies, tak­ing in­to ac­count re­spons­es to its open con­sul­ta­tion on a pre­lim­i­nary dis­cus­sion pa­per,” the study adds.

Pa­tient Reg­istries: An Un­der­used Re­source for Med­i­cines Eval­u­a­tion

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.

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Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

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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.

It’s always a bittersweet moment saying goodbye, but as Josh Sullivan goes off to new adventures we are grateful for the way he’s built up the Endpoints Manufacturing section — which the rest of the team will now carry forward. If you’re not already, this may be a good time to sign up for your weekly dose of drug manufacturing news. Thank you for reading and wish you a restful weekend.

Bay­er sounds re­treat from a $670 mil­lion CAR-T pact in the wake of a pa­tient death

Two months after Atara Biotherapeutics hit the hold button on its lead CAR-T 2.0 therapy following a patient death, putting the company under the watchful eye of the FDA, its Big Pharma partners at Bayer are bowing out of a $670 million global alliance. And the move is forcing a revamp of Atara’s pipeline plans, even as research execs vow to continue work on the two drugs allied with Bayer 18 months ago, which delivered a $60 million cash upfront.

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Sanofi and Re­gen­eron clear the fin­ish line in an in­flam­ma­to­ry esoph­a­gus dis­ease, leav­ing Take­da in the dust

With atopic dermatitis rivals breathing down Dupixent’s neck, Sanofi and Regeneron on Friday secured a first win in new territory in what Sanofi’s head of immunology and inflammation Naimish Patel called the fastest approval he’s ever seen.

The FDA approved Dupixent on Friday to treat patients 12 years and older with eosinophilic esophagitis (EoE), an inflammatory condition that causes swelling and scarring of the esophagus. The approval came just a couple months after regulators granted Dupixent priority review, and months ahead of its PDUFA date on Aug. 3.

Try­ing to shake up the Parkin­son's par­a­digm, Ab­b­Vie sub­mits NDA for con­tin­u­ous, 24-hour in­fu­sion ther­a­py

AbbVie is approaching the FDA with a new therapy to potentially treat Parkinson’s disease, using prodrugs of two medications commonly used for the condition.

The Big Pharma submitted its NDA for ABBV-951, a solution of levodopa and carbidopa prodrugs being evaluated in advanced Parkinson’s patients who don’t respond well to oral therapy, AbbVie announced Friday morning. Researchers are hoping a positive Phase III study that reads out in late October will help move things along quickly at the agency.

Paul Hudson, Sanofi CEO (via Getty)

Sanofi's $20B buy­out of Gen­zyme pays off again with Eu­ro­pean OK for first Nie­mann-Pick drug

Sanofi CEO Paul Hudson has made clear his intention to develop new rare disease drugs and broaden his company’s offerings. That effort leaped forward on Friday with the EMA’s signing off on the company’s — and the EU’s — first drug to treat the non-central nervous system manifestations of the rare and debilitating Niemann-Pick disease.

The enzyme replacement therapy, developed to replace patients’ deficient or defective enzyme, known as acid sphingomyelinase, was first developed by Genzyme, which Sanofi acquired for more than $20 billion in 2011. That acquisition has also helped Sanofi pull in sales in the field of MS.

Proac­tive­ly pre­vent­ing short­ages: New FDA guid­ance spells out which drugs re­quire risk man­age­ment plans

As the majority of drug shortages are still associated with manufacturing-related quality issues, the FDA on Thursday published new draft guidance spelling out how to proactively assess risks to manufacturing processes and supply chains, while understanding the market’s vulnerabilities.

While drug shortages peaked in 2011, the FDA says in its new 18-page draft guidance that the number of new drug shortages “has declined significantly since” that peak, reaching a low in 2015 and 2016, thanks in part to a new law’s enactment, known as FDASIA, which helped the agency better prevent or mitigate drug supply disruptions and shortages, and clarified cGMP requirements.

Janet Woodcock (Sipa via AP Images)

FDA com­mis­sion­er an­nounces new role for Janet Wood­cock, down­shift­ing away from drugs

New FDA commissioner Rob Califf told staff in a memo yesterday evening that longtime drug center director Janet Woodcock, who’s now serving as Califf’s principal deputy, would take on more of a role as “a strategic thought partner, with decision-making authorities” at several different offices within FDA. Curiously, the list of offices and centers does not include the Office of New Drugs or the Office of Generic Drugs.

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Frank Pallone (D-NJ), House Energy and Commerce Committee chair (Kevin Dietsch/Pool via AP Images)

House com­mit­tee unan­i­mous­ly ad­vances FDA user fee leg­is­la­tion with ac­cel­er­at­ed ap­proval tweaks

The House Energy and Commerce Committee on Wednesday offered a rare show of bipartisan support for a bill that would provide the FDA with user fees for the next five years.

The committee voted 55-0 to advance the quinquennial user fee bill to the full House floor, which if approved, will allow the FDA to use biopharma funds to hire new reviewers, and hit new marks as outlined in the user fee deals that the FDA and biopharma companies forged over the past several years.