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

The Big Phar­ma dis­card pile; Lay­offs all around while some biotechs bid farewell; New Roche CEO as­sem­bles top team; and more

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With earnings seasons in full swing, we’ve listened in on all the calls so you don’t have to. But news is popping up from all corners, so make sure you check out our other updates, too.

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Rob Davis, Merck CEO

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Merck executives touted nine ongoing trials in its annual earnings call on Thursday, including five studies in Phase III, for Keytruda (pembrolizumab) in combination with other immuno-oncology drugs. The trials include combinations with Merck’s own developments as well as other pharma companies’ candidates, including its melanoma collaboration with Moderna and its mRNA technology plus Keytruda, aimed at creating a personalized vaccine treatment to reduce the risk of cancer recurrence or death.

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Lars Fruergaard Jørgensen, Novo Nordisk CEO (Christopher Goodney/Bloomberg via Getty Images)

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Following in AbbVie’s footsteps, Teva confirmed on Friday that it’s dropping out of the industry trade group Pharmaceutical Research and Manufacturers of America (PhRMA).

Teva didn’t give a reason for its decision to leave, saying only in a statement to Endpoints News that it annually reviews “effectiveness and value of engagements, consultants and memberships to ensure our investments are properly seated.”

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