Fol­low­ing up on Trump's or­ders, the FDA wants to know: which reg­u­la­tions and pa­per­work re­quire­ments need to go?

As part of work un­der two ex­ec­u­tive or­ders, FDA cen­ters on Thurs­day sought com­ments on which ex­ist­ing reg­u­la­tions and re­lat­ed pa­per­work re­quire­ments could be mod­i­fied, re­pealed or re­placed, to re­duce the bur­den on in­dus­try while al­low­ing the reg­u­la­tor to con­tin­ue its pub­lic health mis­sion while ful­fill­ing statu­to­ry oblig­a­tions.

Back in Feb­ru­ary, when Pres­i­dent Don­ald Trump first an­nounced his or­der to re­duce reg­u­la­to­ry bur­dens, sev­er­al ex­perts point­ed Fo­cus to some out­dat­ed reg­u­la­tions that could be re­moved with lit­tle im­pact on pub­lic health and the agency’s mis­sion, though there are still ques­tions as to what types of reg­u­la­tions will be tar­get­ed.

And as An­na Abram, FDA’s deputy com­mis­sion­er for pol­i­cy, plan­ning, leg­is­la­tion and analy­sis, not­ed in a blog post on Thurs­day, there’s “a lot of ground to cov­er” as FDA’s reg­u­la­tions com­prise more than 4,000 pages in the Code of Fed­er­al Reg­u­la­tions.

In gen­er­al, though, the agency says it’s look­ing for com­ment and sup­port­ing tech­ni­cal, sci­en­tif­ic, eco­nom­ic or oth­er da­ta from those sig­nif­i­cant­ly af­fect­ed by FDA reg­u­la­tions, in­clud­ing con­sumers, pa­tients and care­givers, re­searchers, health­care in­sti­tu­tions, the reg­u­lat­ed in­dus­try, trade as­so­ci­a­tions, pub­lic in­ter­est or­ga­ni­za­tions, acad­e­mia, and state, lo­cal and trib­al gov­ern­ments.

“Some reg­u­la­tions may not ad­e­quate­ly re­flect ad­vances in sci­ence, tech­nol­o­gy or changes in in­dus­try prac­tice. Oth­ers may be geared to­ward prod­ucts and prac­tices that have large­ly ceased to ex­ist,” Abram said, of­fer­ing the ex­am­ple of how the ma­nip­u­la­tion of FDA’s rules on Risk Eval­u­a­tion and Mit­i­ga­tion Strate­gies (REMS) can cre­ate ob­sta­cles to the time­ly en­try of gener­ic com­pe­ti­tion.

Fea­tured in Thurs­day’s Fed­er­al Reg­is­ter no­tices are ques­tions FDA says it is us­ing to guide its ini­tial re­view, in­clud­ing:

  • Is the reg­u­la­tion still cur­rent, or is it out­dat­ed or un­nec­es­sary in some way?
  • Have reg­u­lat­ed en­ti­ties had dif­fi­cul­ties com­ply­ing with the reg­u­la­tion?
  • Does the reg­u­la­tion im­pose re­quire­ments that are al­so pro­vid­ed for in vol­un­tary or con­sen­sus stan­dards or guid­ance by third par­ty or­ga­ni­za­tions (e.g., In­ter­na­tion­al Coun­cil for Har­mon­i­sa­tion, In­ter­na­tion­al Or­ga­ni­za­tion for Stan­dard­iza­tion, Codex Al­i­men­ta­r­ius)?
  • Does the reg­u­la­tion con­tain re­dun­dant, out­dat­ed, or un­nec­es­sary col­lec­tions of in­for­ma­tion or re­ten­tion of records, e.g., re­port­ing, record­keep­ing, or la­bel­ing re­quire­ments?
  • Could the goal of the reg­u­la­tion be achieved by less cost­ly means that would pro­vide the same lev­el of pub­lic health pro­tec­tion?
  • What fac­tors should FDA con­sid­er in se­lect­ing and pri­or­i­tiz­ing reg­u­la­tions and re­port­ing re­quire­ments for re­form?


Re­view of Ex­ist­ing Gen­er­al Reg­u­la­to­ry and In­for­ma­tion Col­lec­tion Re­quire­ments of the Food and Drug Ad­min­is­tra­tion

Re­view of Ex­ist­ing Cen­ter for Drug Eval­u­a­tion and Re­search Reg­u­la­to­ry and In­for­ma­tion Col­lec­tion Re­quire­ments

Re­view of Ex­ist­ing Cen­ter for Bi­o­log­ics Eval­u­a­tion and Re­search Reg­u­la­to­ry and In­for­ma­tion Col­lec­tion Re­quire­ments

Re­view of Ex­ist­ing Cen­ter for De­vices and Ra­di­o­log­i­cal Health Reg­u­la­to­ry and In­for­ma­tion Col­lec­tion Re­quire­ments

Im­age: Get­ty, End­points News, The White House

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 for more in­for­ma­tion.


Zachary Brennan

managing editor, RAPS

Deborah Dunsire. Lundbeck

UP­DAT­ED: Deb­o­rah Dun­sire is pay­ing $2B for a chance to leap di­rect­ly in­to a block­buster show­down with a few of the world's biggest phar­ma gi­ants

A year after taking the reins as CEO of Lundbeck, Deborah Dunsire is making a bold bid to beef up the Danish biotech’s portfolio of drugs in what will likely be a direct leap into an intense rivalry with a group of giants now carving up a growing market for new migraine drugs.

Bright and early European time Monday morning the company announced that it will pay up to about $2 billion to buy Alder, a little biotech that is far along the path in developing a quarterly IV formulation of a CGRP drug aimed at cutting back the number of crippling migraines patients experience each month. In a followup call, Dunsire also noted that the company will likely need 200 to 250 reps for this marketing task on both sides of the Atlantic. And analysts were quick to note that the dealmaking at Lundbeck isn’t done, with another $2 billion to $3 billion available for more deals to beef up the pipeline.

Endpoints News

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Tower Bridge in London [Shutterstock]

#UK­BIO19: Join GSK’s Hal Bar­ron and a group of top biotech ex­ecs for our 2nd an­nu­al biotech sum­mit in Lon­don

Over the past 10 years I’ve made a point of getting to know the Golden Triangle and the special role the UK biopharma industry plays there in drug development. The concentration of world class research institutes, some of the most accomplished scientists I’ve ever seen at work and a rising tide of global investment cash leaves an impression that there’s much, much more to come as biotech hubs are birthed and nurtured.

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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Ac­celeron drops a de­vel­op­ment pro­gram as #2 drug fails to spark func­tion­al ben­e­fits in pa­tients with a rare neu­ro­mus­cu­lar ail­ment

Acceleron is scrapping a muscular dystrophy development program underway for its number 2 drug in the pipeline after pouring over some failed mid-stage secondary data.

Gone is the ACE-083 project in patients with facioscapulohumeral muscular dystrophy. Their drug hit the primary endpoint on building muscle but flopped on key secondaries for functional improvements in patients, which execs felt was vital to the drug’s success.

Scott Gottlieb, AP Images

Scott Got­tlieb has a new board po­si­tion to add to the re­sume — and this one is fo­cused on a fa­vorite sub­ject

Scott Gottlieb has another position to add to his lengthy roster of boards and advisory roles in the wake of his departure from the helm of the FDA.

He’ll be joining the advisory board of FasterCures, a think tank which former junk bond king Michael Milken set up to help drive more drugs to the market, looking to accelerate drug R&D. That’s a subject close to the heart of Gottlieb, who blazed a trail at the FDA focused on hustling up the process. That helped endear him to the industry, making him one of the most popular commissioners in FDA history.

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Karyopharm lines up $150 mil­lion cash in­jec­tion to back con­tro­ver­sial drug launch

Karyopharm has entered into a royalty agreement worth up to $150 million to back the launch of their multiple myeloma drug — recently approved by the FDA over the objections of a majority of the agency’s outside experts.

The deal with HealthCare Royalty Partners, worth $75 million now and $75 million once certain regulatory and commercial milestones have been reached, will fund the commercialization of Karyopharm’s oral SINE compound Xpovio (selinexor) for patients with multiple myeloma who have already had at least four prior therapies. The money will help Karyopharm as it markets its newly approved drug and pushes through clinical trials testing the drug on refractory multiple myeloma patients with one to three therapies and patients with treatment-resistant diffuse large B-cell lymphoma. It will give Karyopharm a cushion through mid-2021.

Af­ter a run of CT­LA-4 com­bo fail­ures, sci­en­tists spot­light a way to make it work — in se­lect pa­tients

CTLA-4/PD-(L)1 combinations have been one of the El Dorados of oncology, its promise forever behind that next hill but apparently unattainable after a series of pivotal clinical failures. But researchers at New York’s Memorial Sloan Kettering Cancer Center and the Technical University of Munich think they may know how to fix what’s wrong and boost the drive to next-gen cancer combos.

In a preclinical animal research program, researchers found that within a cell, checkpoints rely on a specific molecule — RNA-sensing molecule RIG-I — to work. If that sounds familiar, it’s because it has already been identified as a target for boosting immune responses and was subject to at least one Phase I/II trial. Pfizer in December allied itself with Kineta with $15 million upfront and $505 million in potential milestones to develop RIG-I immunotherapies, and three years ago Merck purchased German upstart Rigontec for $137 million upfront and over $400 million in potential milestones for the same purpose.

Pur­due Phar­ma files for bank­rupt­cy as first step in $10B opi­oid set­tle­ment

It’s settled. Purdue Pharma has filed for bankruptcy as part of a deal that would see the OxyContin maker hand over $10 billion in cash and other contributions to mitigate the opioid crisis — without acknowledging any wrongdoing in the protracted epidemic that’s resulted in hundreds of thousands of deaths.

The announcement came two weeks after news of a proposed settlement surfaced and largely confirm what’s already been reported.

Lisa M. DeAngelis, MSKCC

MSK picks brain can­cer ex­pert Lisa DeAn­ge­lis as its next CMO — fol­low­ing José Basel­ga’s con­tro­ver­sial ex­it

It’s official. Memorial Sloan Kettering has picked a brain cancer expert as its new physician-in-chief and CMO, replacing José Baselga, who left under a cloud after being singled out by The New York Times and ProPublica for failing to properly air his lucrative industry ties.

His replacement, who now will be in charge of MSK’s cutting-edge research work as well as the cancer care delivered by hundreds of practitioners, is Lisa M. DeAngelis. DeAngelis had been chair of the neurology department and co-founder of MSK’s brain tumor center and was moved in to the acting CMO role in the wake of Baselga’s departure.