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

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

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Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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FDA hands ac­cel­er­at­ed nod to Seagen, Gen­mab's so­lo ADC in cer­vi­cal can­cer, but com­bo stud­ies look even more promis­ing

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David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

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Regenxbio, a small licensor of gene therapy viral vectors spun out from the University of Pennsylvania, is now finding itself in the middle of some major league patent fights.

In addition to a patent suit with Sarepta Therapeutics from last September, Novartis, is now trying to push its smaller partner out of the way. The Swiss biopharma licensed Regenxbio’s AAV9 vector for its $2.1 million spinal muscular atrophy therapy Zolgensma.

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Ex-My­lan em­ploy­ee pleads guilty to in­sid­er trad­ing, il­le­gal­ly deal­ing on FDA ap­provals, earn­ings and Up­john merg­er

A former Mylan IT executive pleaded guilty Friday to an insider trading scheme where he bought and sold stock options on another executive’s advice.

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Volker Wagner (L) and Jeff Legos

As Bay­er, No­var­tis stack up their ra­dio­phar­ma­ceu­ti­cal da­ta at #ES­MO21, a key de­bate takes shape

Ten years ago, a small Norwegian biotech by the name of Algeta showed up at ESMO — then the European Multidisciplinary Cancer Conference 2011 — and declared that its Bayer-partnered targeted radionuclide therapy, radium-223 chloride, boosted the overall survival of castration-resistant prostate cancer patients with symptomatic bone metastases.

In a Phase III study dubbed ALSYMPCA, patients who were treated with radium-223 chloride lived a median of 14 months compared to 11.2 months. The FDA would stamp an approval on it based on those data two years later, after Bayer snapped up Algeta and christened the drug Xofigo.

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Rafaèle Tordjman (Jeito Capital)

Con­ti­nu­ity and di­ver­si­ty: Rafaèle Tord­j­man's women-led VC firm tops out first fund at $630M

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