FDA fi­nal­izes guid­ance on place­bos and blind­ing for can­cer tri­als

The FDA on Wednes­day fi­nal­ized guid­ance rec­om­mend­ing that drug­mak­ers on­ly use place­bos in can­cer clin­i­cal tri­als in se­lect cir­cum­stances due to eth­i­cal con­cerns that would arise when al­ter­na­tive ther­a­pies are avail­able.

Ac­cord­ing to guid­ance, place­bos should on­ly be con­sid­ered in ran­dom­ized con­trolled tri­als to treat on­co­log­ic dis­ease or hema­to­log­ic ma­lig­nan­cy “when sur­veil­lance is stan­dard of care” or in stud­ies with “cer­tain tri­al de­sign fea­tures (e.g., when the tri­al us­es an add-on de­sign).”

“A place­bo-con­trolled study de­sign may be use­ful or pre­ferred in main­te­nance ther­a­py, in add-on tri­al de­signs, in tri­als of ad­ju­vant ther­a­pies (for which stan­dard of care is sur­veil­lance), and for in­di­ca­tions where no treat­ment is avail­able,” the FDA writes.

The guid­ance pro­vides a set of con­sid­er­a­tions for spon­sors weigh­ing a place­bo-con­trolled tri­al de­sign, not­ing that spon­sors “should pro­vide the ra­tio­nale for the tri­al de­sign” and a de­scrip­tion of their plan for blind­ing and un­blind­ing pa­tients dur­ing the tri­al.

The six-page guid­ance fi­nal­izes a draft ver­sion is­sued in Au­gust 2018 and has been up­dat­ed to clar­i­fy is­sues re­lat­ed to un­blind­ing that were raised dur­ing the pub­lic con­sul­ta­tion.

In a change from the draft guid­ance, the FDA clar­i­fies that when un­blind­ing due to dis­ease re­cur­rence or pro­gres­sion that on­ly the pa­tient and in­ves­ti­ga­tor be un­blind­ed, where­as the draft guid­ance on­ly spec­i­fied that the pa­tient be un­blind­ed.

The FDA al­so main­tains that pa­tients and in­ves­ti­ga­tors should be un­blind­ed when pa­tients ex­pe­ri­ence an ad­verse event sus­pect­ed to be re­lat­ed to the in­ves­ti­ga­tion­al drug when man­ag­ing the ad­verse drug would re­quire treat­ment with “one or more drug prod­ucts with sub­stan­tial tox­i­c­i­ty or in­va­sive pro­ce­dures.”

If the spon­sor plans on main­tain­ing blind­ing when a pa­tient’s dis­ease re­curs or pro­gress­es or af­ter an ad­verse event, the FDA says the spon­sor should pro­vide a jus­ti­fi­ca­tion for main­tain­ing blind­ing and ac­knowl­edge the risks of the ap­proach in the in­formed con­sent doc­u­ments.

The FDA al­so up­dat­ed the guid­ance to note that it does not ad­dress sta­tis­ti­cal con­sid­er­a­tions when un­blind­ing da­ta and sim­pli­fied its ti­tle.


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So­cial im­age: AP Im­ages

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Jason Kelly, Ginkgo Bioworks CEO (Kyle Grillot/Bloomberg via Getty Images)

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Ginkgo Bioworks execs always thought big. But today should redefine just how big an upstart biotech player can dream.

In the largest SPAC deal to clear the hurdles to Nasdaq, the biotech that envisioned everything from remaking synthetic meat to a whole new approach to developing drugs has joined forces with one of the biggest disruptors in biotech to slam the Richter scale on dealmaking.

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FDA un­veils six ICH guide­lines ahead of meet­ing with Health Cana­da

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A clos­er look at the FDA’s more than 700 pan­dem­ic-re­lat­ed record re­quests to re­place on­site in­spec­tions

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Distribution of Moderna's Covid-19 Vaccine (Photo by Paul Sancya - Pool/Getty Images)

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UP­DAT­ED: Feds charge an­oth­er CRO staffer with fak­ing da­ta in a Glax­o­SmithK­line pe­di­atric asth­ma study

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Al Sandrock, Biogen R&D chief (Biogen via YouTube)

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Chris Garabedian (Xontogeny)

Per­cep­tive Ad­vi­sors, Xon­toge­ny bring the band back and then some with a $515M sec­ond fund sniff­ing out lead com­pounds

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