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Real-World Evidence: Amylyx’s Use Of External Controls To Frame Relyvrio’s Survival Benefit Rife With Problems

 
• By Sue Sutter

The post hoc comparison of an exploratory, long-term survival benefit in the CENTAUR trial to historical controls in two ALS patient databases lacked prespecification and a common treatment protocol, and potential differences in prognostic factors may have confounded the results.

Drug Review Profile: Relyvrio
Amylyx attempted to rely on historical controls in showing a survival benefit with Relyvrio in a Phase II trial. • Source: Shutterstock

Amylyx Pharmaceuticals, Inc.’s late-in-review attempt to compare the survival benefit for its amyotrophic lateral sclerosis drug Relyvrio (sodium phenylbutyrate/taurursodiol) in the Phase II CENTAUR trial to historical controls was rife with methodologic problems and may be viewed as a case study in how not to conduct such a comparison using real-world data.

US Food and Drug Administration review documents suggest the primary clinical and statistical reviewers put little faith in the analyses,...

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