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The Real World Is Not Enough: Comparability Is Common Obstacle For RWE In US FDA Decisions

 
• By Bridget Silverman

Unsuccessful attempts to use real-world evidence for approvals highlight the need to demonstrate relationship between real-world data and clinical trial patients. The Pink Sheet examines what went wrong for Bluebird/Bristol’s Abecma, Ultragenyx’s Dojolvi, and Karyopharm’s Xpovio.

ARE APPLES COMPARABLE TO ORANGES? • Source: Shutterstock

The importance of ensuring close comparability between treated patients in a single-arm study and a control arm using real-world evidence is made clear by some of the less successful attempts to use RWE of efficacy in US FDA approval decisions.

Bluebird bio/Bristol Myers Squibb Company’s Abecma, Ultragenyx Pharmaceutical Inc.’s Dojolvi, and Karyopharm Therapeutics’s Xpovio were approved...

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