Use of Health Technology Assessment for the Continued Funding of Health Technologies: The Case of Immunoglobulins for the Management of Multifocal Motor Neuropathy

• Published in: Applied health economics and health policy Vol. 22; no. 1; pp. 73 - 84
• Main Authors: Vargas, Constanza, Addo, Rebecca, Lewandowska, Milena, Haywood, Philip, De Abreu Lourenco, Richard, Goodall, Stephen
• Format: Journal Article
• Language: English
• Published: Cham Springer International Publishing 2024; Springer Nature B.V
• Subjects: Case studies; Clinical outcomes; Cost analysis; Disorders; Funding; Health Administration; Health care expenditures; Health Economics; Health services; Health technology assessment; Immunoglobulins; Intervention; Markov chains; Medicine; Medicine & Public Health; Original Research Article; Patients; Pharmacoeconomics and Health Outcomes; Prices; Public Health; Quality of Life Research; Rare diseases; Reimbursement; Sensitivity analysis; Technology assessment; Australia
• ISSN: 1175-5652; 1179-1896
• DOI: 10.1007/s40258-023-00853-1

Introduction
Funding decisions for many health technologies occur without undergoing health technology assessment (HTA), in particular, without assessment of cost effectiveness (CE). Immunoglobulins in Australia are an interesting case study because they have been used for a long time for various rare disorders and their price is publicly available. Undertaking an HTA enables us to assess CE for an intervention for which there is limited clinical and economic evidence. This study presents a post-market review to assess the CE of immunoglobulins for the treatment of multifocal motor neuropathy (MMN) compared with best supportive care.
Methods
A Markov model was used to estimate costs and quality-adjusted life-years (QALYs). Input sources included randomised controlled trials, single-arm studies, the Australian clinical criteria for MMN, clinical guidelines, previous Medical Services Advisory Committee (MSAC) reports and inputs from clinical experts. Sensitivity analyses were conducted to assess the uncertainty and robustness of the CE results.
Results
The cost per patient of treating MMN with immunoglobulin was AU$275,853 versus AU$26,191when no treatment was provided, with accrued QALYs of 6.83 versus 6.04, respectively. The latter translated into a high incremental cost-effectiveness ratio (ICER) of AU$317,552/QALY. The ICER was most sensitive to the utility weights and the price of immunoglobulins. MSAC advised to continue funding of immunoglobulins on the grounds of efficacy, despite the high and uncertain ICER.
Conclusions
Beyond the ICER framework, other factors were acknowledged, including the high clinical need in a patient population for which there are no other active treatments available. This case study highlights the challenges of conducting HTA for already funded interventions, and the efficiency trade-offs required to fund effective high-cost therapies in rare conditions.