When can observational studies support a drug’s effectiveness? FDA explains in new draft guidance

The FDA on Wednes­day re­leased high­ly-an­tic­i­pat­ed draft guid­ance on when drug spon­sors can use ob­ser­va­tion­al stud­ies to help demon­strate a drug’s ef­fec­tive­ness, for the first time of­fer­ing clar­i­ty on what da­ta and ex­pla­na­tions spon­sors should pro­vide the agency.

A ma­jor fo­cus of the FDA’s new guid­ance is mak­ing sure da­ta from ob­ser­va­tion­al stud­ies are re­li­able and rel­e­vant. Da­ta col­lect­ed in ob­ser­va­tion­al stud­ies have of­ten of­fered hope that a ther­a­py might be ef­fec­tive, on­ly to be lat­er con­tra­dict­ed by ev­i­dence from ran­dom­ized con­trolled tri­als.

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.