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Article History

Received: 21 February 2025

Accepted: 28 March 2025

First Online: 27 April 2025

Declarations

:

: Ian R. Woodcock has received honoraria for work performed with Novartis, Biogen, Roche, Avidity and Pfizer. Damian Clark and Didu S. Kariyawasam, have nothing to disclose. Nicholas J.C. Smith has been an invited member of the Novartis Australian SMA advisory Group; he has received consulting fees from Forge Biologics and Actelion Pharmaceuticals and Research support from Abeona Therapeutics, Ultragenyx, Bluebird bio and Cyclo Therapeutics. Maina P. Kava has received an honorarium for participation in a scientific advisory board for Roche. Eppie M. Yiu has received honoraria from Biogen and Roche for participation in scientific advisory boards, honoraria from Biogen paid to their institution for participation in educational activities, and research support from Biogen, Roche, and Novartis. Matthias Bischof and Jane Adams are employees of Novartis and own Novartis stock or other equities. Adrian Peacock is an employee of HTANALYSTS, which undertakes paid health economics consultancy work for industry and Government. Colman Taylor received grants/contracts through the George Institute for Global Health from the Baxter Healthcare Corporation and worked as a consultant providing project oversight for a government review of chemotherapy funding; received grants through the George Institute for Global Health from CSL Bioplasma to conduct the FLUID-TRIPS study; received a grant through the George Institute for Global Health from the National Health and Medical Research Council to fund the PLUS study. He is also co-owner of Health Technology Analysts Pty Ltd (HTANALYSTS), which undertakes consultancy work for pharmaceutical companies, medical device companies, not-for-profit entities, and the Australian Government.

: This article is based on a cost-utility analysis using a combination of decision tree and Markov model structures. No new studies with human participants were included in this analysis, and, therefore, Institutional Review Board approval was not required, patient consent to participate was not necessary, and the Declaration of Helsinki 1964 does not apply.