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.

FDA


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