Presentation (CTI)

OBJECTIVES: The Six-Minute Walk Test (6MWT) is a recognized measure of ambulatory function widely used in pivotal trials at the regulatory level for neuromuscular disorders such as Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA), and also applied in non-muscular neurological conditions including amyotrophic lateral sclerosis (ALS), multiple sclerosis (MS), and stroke. However, at the reimbursement level it is not uniformly recognized. The study will analyze HTA submissions (2010-2025) where the 6MWT was used as an endpoint hierarchy and other variables are influencing the decision (favorable, unfavorable or inconclusive) to major agencies (England, France, Germany, USA) reporting 6MWT: (1) outcomes in muscular (DMD, SMA) and non-muscular (ALS, MS, stroke) neurological indications, (2) by reimbursement linkage, (3) role of 6MWT (primary vs. supportive), (4) payer commentary on clinical meaningfulness, and (5) methodological factors influencing acceptance as a surrogate endpoint.
METHODS: A comprehensive list of publicly available HTA dossiers will be identified and analyzed using an AI platform. Inclusion criteria are: (a) a neurological indication (muscular or non-muscular) and (b) acceptance of the 6MWT in contrast to other endpoints . Extracted data will include disease area, endpoint designation, age group, correlation with clinical parameters and/or biomarkers, reported MCID, payer appraisal notes, and reimbursement decisions. Analyses will consist of descriptive statistics of the population under study, context of use, and thematic coding of payer commentary.
RESULTS: Published reviews highlight variability in the relevance of the 6MWT as an endpoint across muscular and non-muscular neurological diseases, necessitating further evaluation of its acceptance in reimbursement decisions. Recommendations will be made to inform trial design and implications for launching innovative therapies.
CONCLUSIONS: This analysis will provide an evidence-based overview of 6MWT acceptance across both muscular and non-muscular neurological HTA submissions and inform best practices for its use as a viable surrogate marker of disease progression in reimbursement decisions.