Comparative policy analysis of national rare disease funding policies in Australia, Singapore, South Korea, the United Kingdom and the United States: a scoping review

Qin Xiang Ng (), Clarence Ong (), Kai En Chan (), Timothy Sheng Khai Ong (), Isabelle Jia Xuan Lim (), Ansel Shao Pin Tang (), Hwei Wuen Chan () and Gerald Choon Huat Koh ()
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Qin Xiang Ng: National University of Singapore and National University Health System
Clarence Ong: National University of Singapore and National University Health System
Kai En Chan: National University
Timothy Sheng Khai Ong: National University
Isabelle Jia Xuan Lim: National University
Ansel Shao Pin Tang: National University
Hwei Wuen Chan: National University
Gerald Choon Huat Koh: National University of Singapore and National University Health System

Health Economics Review, 2024, vol. 14, issue 1, 1-15

Abstract: Abstract Background Rare diseases pose immense challenges for healthcare systems due to their low prevalence, associated disabilities, and attendant treatment costs. Advancements in gene therapy, such as treatments for Spinal Muscular Atrophy (SMA), have introduced novel therapeutic options, but the high costs, exemplified by Zolgensma® at US$2.1 million, present significant financial barriers. This scoping review aimed to compare the funding approaches for rare disease treatments across high-performing health systems in Australia, Singapore, South Korea, the United Kingdom (UK), and the United States (US), aiming to identify best practices and areas for future research. Methods In accordance with the PRISMA-ScR guidelines and the methodological framework by Arksey and O’Malley and ensuing recommendations, a comprehensive search of electronic databases (Medline, EMBASE, and Cochrane) and grey literature from health department websites and leading national organizations dedicated to rare diseases in these countries was conducted. Countries selected for comparison were high-income countries with advanced economies and high-performing health systems: Australia, Singapore, South Korea, the UK, and the US. The inclusion criteria focused on studies detailing drug approval processes, reimbursement decisions and funding mechanisms, and published from 2010 to 2024. Results Based on a thorough review of 18 published papers and grey literature, various strategies are employed by countries to balance budgetary constraints and access to rare disease treatments. Australia utilizes the Life Saving Drugs Program and risk-sharing agreements. Singapore depends on the Rare Disease Fund, which matches public donations. South Korea’s National Health Insurance Service covers specific orphan drugs through risk-sharing agreements. The UK relies on the National Institute for Health and Care Excellence (NICE) to evaluate treatments for cost-effectiveness, supported by the Innovative Medicines Fund. In the US, a combination of federal and state programs, private insurance and non-profit support is used. Conclusion Outcome-based risk-sharing agreements present a practical solution for managing the financial strain of costly treatments. These agreements tie payment to actual treatment efficacy, thereby distributing financial risk and promoting ongoing data collection. Countries should consider adopting and expanding these agreements to balance immediate expenses with long-term benefits, ultimately ensuring equitable access to crucial treatments for patients afflicted by rare diseases.

Keywords: Rare diseases; Orphan drugs; Funding; Health policy; Risk-sharing (search for similar items in EconPapers)
Date: 2024
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DOI: 10.1186/s13561-024-00519-1

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