Researchers proposed a patient-preference endpoint for ALS trials
Original source
Composite endpoint for ALS clinical trials based on patient preference: Patient-Ranked Order of Function (PROOF). (opens in a new tab)Compass summarised this from the study's abstract.
Study details
- Studied in
- Human
Researchers developed the Patient-Ranked Order of Function (PROOF), a composite measure that ranks treatment outcomes according to the symptoms patients consider most important. It was based on an online questionnaire completed by 433 people with ALS in the Netherlands and was tested through simulations. The authors said it could help assess treatment benefits and risks, but it has not been shown to improve treatment decisions in a clinical trial.
Why this matters
PROOF could help future ALS trials reflect differences in what patients value, rather than giving every functional domain equal importance. This is a proposed trial-measurement method, not a treatment, so it does not currently change care for people with ALS.
Limitations and context
The work used questionnaire responses from 433 responders in a Dutch population-based registry and simulations rather than a prospective treatment trial. The study established and illustrated the endpoint, but further testing would be needed to show how it performs in actual ALS trials and whether it improves interpretation of treatment effects.