Feasibility assessment of using the MiToS staging system for conducting economic evaluation in amyotrophic lateral sclerosis

ABSTRACT

Objectives

This study assessed the feasibility of using the Milano-Torino staging (MiToS) system for conducting economic evaluation to measure health outcomes in amyotrophic lateral sclerosis (ALS).

Methods

A Markov model was developed using the MiToS system and evaluated with a hypothetical treatment versus standard of care. Health utilities and transition probabilities were derived from the literature. Four-time horizons (1, 5, 10, and 20 years) were examined. Treatment effects of 20–35% relative risk reduction (RRR) of progressing to the next MiToS stage were assessed. Three patient distribution scenarios were tested: (1) all patients began in stage 0; (2) patient distribution based on real-world TONiC study; (3) distribution based on the PRO-ACT database. Health outcomes (quality-adjusted life-years [QALYs], life-years [LYs]) were reported with a 3% discount rate.

Results

A time horizon of 10 years fully captured treatment benefits: incremental QALYs were 0.28–0.60, 0.21–0.45, and 0.26–0.55 for scenarios 1–3, respectively; incremental LYs were 0.56–1.17, 0.46–0.97, and 0.53–1.11, respectively.

Conclusion

MiToS-based staging can be used for conducting economic analyses in ALS. Estimated incremental QALY and LY gains were meaningful within the context of ALS, for hypothetical treatments with RRR of 20–35%.

KEYWORDS:

• Amyotrophic lateral sclerosis
• economic evaluation
• Markov model
• Milano-Torino staging
• health outcomes

Article highlights

• Our aim was to assess the feasibility of using the Milano-Torino staging (MiToS) system for conducting economic evaluation measuring health outcomes in amyotrophic lateral sclerosis (ALS).

• We developed a Markov model using the MiToS system and evaluated it with a hypothetical treatment versus standard of care using health utilities and transition probabilities from the literature.

• We found that a 10-year time horizon entirely captured treatment benefits.

• For hypothetical treatments with RRR of 20–35%, estimated incremental quality-adjusted life-years and life-years gains were meaningful within the context of ALS.

• We therefore conclude that a MiToS-based staging can be used for conducting economic analyses in ALS.

Declaration of interest

P Gebrehiwet is an employee of and owns stock in Cytokinetics, Incorporated. S Aggarwal and O Topaloglu are employees at Novel Health Strategies and were financially compensated for their work by Cytokinetics, Incorporated. A Chiò serves on the advisory board for Amylyx, Biogen, Cytokinetics, Incorporated, Denali Pharma, and Mitsubishi Tanabe.

The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Author contributions

P Gebrehiwet contributed to the study design and concept and originally implemented the model proposal in excel and drafted the manuscript. A Chiò gave expert opinion support. All authors contributed to the critical review of the manuscript and approval of the final version.

Acknowledgments

Editorial support for this manuscript was provided by Geraldine Thompson, PhD, and Andrea Schauenburg, PhD, on behalf of Engage Scientific Solutions, Horsham, UK, and was funded by Cytokinetics, Incorporated.

Ethics approval

This analysis was based on published data. As this was not an interventional study, approval from an ethics committee or institutional review board was not required.

Data availability statement

All data generated or analyzed during this study are included in this published article and its supplementary information files.

Reviewer disclosures

A reviewer on this manuscript has disclosed being an author of a manuscript on a similar topic that was submitted to this journal. Peer reviewers on this manuscript have no other relevant financial relationships or otherwise to disclose.

Supplemental data

Supplemental data for this article can be accessed online at https://doi.org/10.1080/14737167.2024.2306819

Additional information

Funding

This study was funded by Cytokinetics, Incorporated.