Presented this year at ISPOR US: indirect treatment comparisons and exploratory analyses of a clinical trial


Lionel RIOU FRANÇA, Chief Scientific Officer at Aixial Group, was involved in two analyses involving patients with Late-Onset Pompe Disease (LOPD), a rare disease, presented at ISPOR US 2023.

Lionel RIOU FRANÇA tells us more about these interesting clinical outcomes assessments.

When a new drug enters the market, evidence of its relative effectiveness against the alternatives is useful for health technology assessment and prescribers. However, direct comparisons between all therapeutic alternatives are generally unavailable. They may be unfeasible in the case of multiple already established relevant therapeutic alternatives, or when other treatments were developed while the trial was ongoing. Indirect treatment comparisons are then a valuable approach. The results from one such comparison that I was involved were presented at ISPOR this year:

This was an interesting case in which population-adjusted methods were used, including in a situation involving disconnected networks of evidence.


Chief Scientific Officer RWE Business Line | Aixial Group

We remain available at Aixial Group to advise you in your evidence synthesis and generation strategies as well as to perform the relevant analyses.