FDA's Janet Wood­cock: the clin­i­cal tri­als sys­tem is 'bro­ken'

The clin­i­cal tri­als sys­tem is “bro­ken” and there needs to be new ways to col­lect and uti­lize pa­tient da­ta, Janet Wood­cock, di­rec­tor of FDA’s Cen­ter for Drug Eval­u­a­tion and Re­search, told a work­shop at the Na­tion­al Acad­e­mies of Sci­ences, En­gi­neer­ing, and Med­i­cine to­day.

The com­ment came at the end of Wood­cock’s talk in which she al­so not­ed that use of mas­ter pro­to­cols (pro­to­cols for tri­als that look at mul­ti­ple ther­a­pies in a sin­gle dis­ease or a sin­gle treat­ment in mul­ti­ple dis­eases) and the de­vel­op­ment of new clin­i­cal tri­al net­works “need to be the fu­ture.”

Both the 21st Cen­tu­ry Cures Act and the new user fee laws will ex­pand FDA’s use of so-called Re­al World Ev­i­dence in cer­tain cir­cum­stances, as FDA com­mis­sion­er Scott Got­tlieb out­lined Tues­day, though Wood­cock not­ed there has been “very lit­tle his­tor­i­cal use of re­al world ex­pe­ri­ence in drug reg­u­la­to­ry de­ci­sions about ef­fec­tive­ness.”

The use of RWE in de­ter­min­ing ef­fec­tive­ness is “ob­vi­ous­ly most im­por­tant for in­cen­tives” for in­dus­try, she said, while cau­tion­ing RWE works when there’s a “big ef­fect” but it’s a lot more dif­fi­cult in un­cov­er­ing small­er ef­fects be­cause “so many bi­as­es are in­tro­duced.”

As far as sit­u­a­tions in which drug de­vel­op­ers might be able to use RWE, Wood­cock not­ed sim­i­lar­i­ties with the med­ical de­vice in­dus­try, and sin­gled out the de­vel­op­ment of bio­mark­ers, ex­pand­ed in­di­ca­tions (she of­fered the ex­am­ple of Ver­tex’s cys­tic fi­bro­sis drug Ka­ly­de­co) and pos­si­bly eval­u­at­ing an in­ves­ti­ga­tion­al drug in a “hy­brid mod­el” that can use some RWE with ran­dom­iza­tion.

“Let’s make gen­er­at­ing this ev­i­dence a lot eas­i­er and ran­dom­iz­ing with­in the care sys­tem as much as we can,” Wood­cock said in the Q&A por­tion of the work­shop.

Draft guid­ance on RWE and a new frame­work on its use are both ex­pect­ed to be re­leased by FDA be­fore 2021, though Wood­cock said she could not spec­u­late on when ex­act­ly the guid­ance would be ready, not­ing there’s a di­rect cor­re­la­tion be­tween de­lays in a guid­ance’s re­lease and in­ter­est in a guid­ance.

Ro­ry Collins, head of the Nuffield De­part­ment of Pop­u­la­tion Health at the Uni­ver­si­ty of Ox­ford, al­so told par­tic­i­pants that just re­form­ing the ICH guid­ance on good clin­i­cal prac­tices might not be enough, as the doc­u­ment might need to be re-writ­ten com­plete­ly or just aban­doned.

How­ev­er, Wood­cock coun­tered that at its heart, reg­u­la­tion is of­ten just aimed at the bot­tom 1% of com­pa­nies, which is a prob­lem, though the ICH GCP guid­ance is nec­es­sary.

“You wouldn’t be­lieve what some will do – there are what I call bot­tom feed­ers out there that will as­ton­ish any­one. We have to have some kind of struc­ture to guard against that,” she added.


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.

Im­age: Janet Wood­cock ap­pears be­fore a House com­mit­tee in­ves­ti­gat­ing drug prices last year. CQ Roll Call, AP Im­ages

In­side Track: Be­hind the Scenes of a Ma­jor Biotech SPAC

Dr. David Hung and Michelle Doig are no strangers to the SPAC phenomenon. As Founder and CEO of Nuvation Bio, a biotech company tackling some of the greatest unmet needs in oncology, Dr. Hung recently took the company public in one of this year’s biggest SPAC related deals. And as Partner at Omega Funds, Doig not only led and syndicated Nuvation Bio’s Series A, but is now also President of the newly formed, Omega-sponsored, Omega Alpha SPAC (Nasdaq: OMEG; oversubscribed $138m IPO priced January 6, 2021).

Aduhelm OK 'bit­ter­sweet' for ALS ad­vo­cates; Con­trast­ing Covid-19 vac­cine read­outs; GSK joins TIG­IT bat­tle; 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.

With the busiest days of June now behind us, we’re starting to think seriously about the second half of the year. In August, we have scheduled a special report where Endpoints will compile a list of the 20 most influential R&D executives in biopharma. Know a luminary who should definitely be included? Nominate them now.

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Who are the lu­mi­nar­ies dri­ving the biggest ad­vances in bio­phar­ma R&D? End­points News is ask­ing for your nom­i­na­tions for a spe­cial re­port

In biopharma, driving a drug to market is the ultimate goal — but none of that happens without a strong research and development program. At the most successful companies, those R&D efforts are spearheaded by true innovators in the field who are always looking for that next novel mechanism of action or breakthrough safety profile.

Now, Endpoints News is asking you to tell us who those guiding lights are.

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Biotech founder placed on leave as $400M Alzheimer's start­up idea comes un­der scruti­ny

Athira Pharma, the Alzheimer’s biotech that emerged out of obscurity last year and raised nearly $400 million for a dark-horse approach to treating neurodegeneration, has found itself in sudden turmoil.

On Tuesday evening, the company released a terse statement announcing that CEO and founder Leen Kawas had been placed on administrative leave while an independent review board investigated “actions stemming” from her doctoral research at Washington State University. Mark Litton, who joined the company as COO two years ago, will take over day-to-day operations, they said.

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Bris­tol My­ers breaks the bank on Ei­sai's fo­late re­cep­tor ADC drug, lay­ing out more than $3B+ for rights

For years, innovation in oncology has been a crapshoot with Big Pharma — the whales at the table — dropping the big bucks for the key to the next generation of tumor fighters. Bristol Myers Squibb hasn’t exactly made a name for being an innovator in the space, but that doesn’t mean it won’t splash in when it sees a potential winner.

Now, with a massive check in hand, the drugmaker is willing to put its intuition to the test.

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Michael Chambers (L) and John Ballantyne

Dana­her strikes deal to buy boom­ing next-gen man­u­fac­tur­er Alde­vron for $9.6B

Life sciences conglomerate Danaher Corp. $DHR has struck a deal to buy the fast-growing Aldevron, one of the world’s top manufacturers of hotly sought-after plasmid DNA, mRNA and recombinant proteins for the burgeoning world of vaccine and drugmakers pushing some game-changing technologies.

Buyout talks set the stage for Danaher to settle on a $9.6 billion cash pact to acquire the private Fargo, ND-based company — a key supplier for a disruptive new Covid vaccine as well as a host of gene and cell therapy and CRISPR gene editing players — founded by Michael Chambers and CSO John Ballantyne as a crew of 2 back in 1998.

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FDA's con­tro­ver­sial Aduhelm de­ci­sion leaves ALS pa­tients feel­ing spurned

The FDA’s controversial approval of Biogen’s Aduhelm drug for Alzheimer’s disease has been met with fierce resistance from all corners of the biopharma industry, but few seem to be as upset with the decision as ALS patients and advocacy groups.

For all that’s already been written and discussed about the agency’s announcement, from the drug’s exorbitantly high price of $56,000 per year to criticism over lowered standards, ALS patients see something more. ALS patients and associations say they largely regarded Aduhelm’s approval as a bittersweet double standard: happy that those with Alzheimer’s have a new drug available, but questioning how the FDA evaluated Biogen’s drug compared to the experimental programs being studied for their own disease.

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As drug­mak­ers spend $6B an­nu­al­ly on DTC ads, sen­a­tors re­vive bill to in­clude list prices in ads

A new GAO report on biopharma companies’ $6 billion annual spending on direct-to-consumer advertising is pushing US Senate Majority Whip Dick Durbin (D-IL) and Sen. Chuck Grassley (R-IA) to reintroduce legislation that would require price disclosures in the ads.

The GAO found that drugmakers spent almost half—$8.2 billion of the $17.8 billion from 2016 to 2018—on DTC ads for drugs in three therapeutic categories, including inflammatory conditions (e.g., arthritis, gout), endocrine and metabolic disorders (e.g., type 2 diabetes, hypothyroidism), and conditions affecting the central nervous system (e.g., depression, multiple sclerosis), according to the new report.

Med­ic­aid com­mis­sion to Con­gress: In­crease re­bates for ac­cel­er­at­ed ap­proval drugs

As the FDA continues to approve more new drugs under its accelerated approval pathway, the non-partisan Medicaid and CHIP Payment and Access Commission (MACPAC) is telling Congress to increase the statutory Medicaid rebates for such drugs until their clinical benefits have been verified.

Higher rebates for drugs with accelerated approvals, a move opposed by the biopharma industry, would mean lower net prices, lessening their financial burden on the health care system while incentivizing the companies to speed the verification of the drugs’ clinical benefits in confirmatory trials. Once those benefits are confirmed, the companies would return to the lower rebates when the accelerated approval is converted into a full approval, MACPAC suggests.