Demonstrating product value from multiple endpoints in rare disease trials

By Lionel Riou França

Lionel RIOU FRANÇA, Chief Scientific Officer at Aixial Group, has co-authored an article exploring the application of the win ratio method in Pompe disease.

Rare diseases are often multifaceted, and a single endpoint is often not sufficient to fully describe treatment1. Demonstrating product benefits for multiple endpoints can however be challenging in small samples as separate testing can lead to a decrease in power. Many methodologies to combine multiple outcomes have their own limitations. Generalized paired comparisons and win statistics address some of these.

The new publication2 illustrates the use of one such method, the win ratio, to quantify the benefits of avalglucosidase alfa, in late-onset Pompe disease.

The article can be consulted at:

  1. Verbeeck J, Dirani M, Bauer JW, Hilgers R-D, Molenberghs G, Nabbout R. Composite endpoints, including patient reported outcomes, in rare diseases. Orphanet J Rare Dis. 2023;18:262.
  2. Boentert M, Berger KI, Díaz-Manera J, Dimachkie MM, Hamed A, Riou França L, et al. Applying the win ratio method in clinical trials of orphan drugs: an analysis of data from the COMET trial of avalglucosidase alfa in patients with late-onset Pompe disease. Orphanet J Rare Dis. 2024;19:14.


Chief Scientific Officer RWE Business Line | Aixial Group

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