Kuala Lumpur, 27 January 2026: The Institute for Democracy and Economic Affairs (IDEAS) today launched its latest policy paper exploring how Malaysia can sustainably finance high-cost therapies for rare diseases amid rapid medical advances and growing fiscal pressure on public health.

The paper, titled ‘Advancing Equitable Access to Spinal Muscular Atrophy (SMA) Gene Therapy: Recommendations for Outcomes-Based Financing and Sustainable Procurement’ is authored by Durrah Sharifah Ahmad Azlan, Senior Research Executive at IDEAS, Nimitraa Youganesparan, Research Executive at IDEAS, Dr Stewart Nixon, Director of Research at IDEAS, and Dr Tae Sok Kun, Head of the Medical Genetics Unit, Universiti Malaya Medical Centre. It examines how financing and procurement approaches can improve access to equitable treatment in Malaysia.

Despite breakthroughs such as gene therapy, many Malaysians with rare diseases continue to struggle with high up-front costs and delayed diagnosis. These challenges are aggravated by limited government health budgets. As the National Policy on Rare Diseases 2025 signals greater intent to improve the lives of persons with rare diseases, this paper highlights how better health financing mechanisms, procurement systems, and clinical depth can support this.

Using Spinal Muscular Atrophy (SMA) as a case study, the paper assesses whether outcomes-based payment models (OBPM) can be adapted within Malaysia’s health system to manage financial risk while expanding access to high-cost therapies. It identifies three priority areas for reform:

• Clinical Readiness and Patient-Centric Outcomes,— Clear and clinically agreed eligibility criteria are needed, prioritising pre-symptomatic and early-diagnosed patients while ensuring pathways for those diagnosed later. Outcomes assessment should go beyond survival to include quality of life, caregiver well being and disease stabilisation. System readiness must also be strengthened through newborn screening and a National Rare Disease Registry.
• Procurement and Financing Innovation — Outcomes-based payment models can help manage uncertainty for high-cost, high-impact therapies. For treatments with more well-established and predictable outcomes, a pure financial payment model may be more appropriate.
• Systemic and Ethical Governance — Sustainable reform requires stronger coordination across health, financial and ethical systems. Key steps include establishing a dedicated process for assessing and publishing the comprehensive financial profile (total costs, patient numbers, long-term burden) of emerging treatments, developing mature risk pooling systems for treatment financing, long-term deepening of insurance markets and coverage, and ethical governance to reduce financial and emotional toll on patients and families.

With the launch of this paper, IDEAS hopes to establish recommendations that demonstrate the fiscal feasibility of sustainable access to transformative, high-cost treatments like SMA gene therapy through strategic procurement and financial innovation.

“There are no silver bullets, but fresh approaches inspired by innovative financing models can bring more emerging treatments to Malaysians. An ambitious medium-term vision to advance patient screening, create deeper specialist ecosystems, fill critical information gaps, and grow financing pools is needed; one befitting the passion of rare disease stakeholders,” said Dr Stewart Nixon, Director of Research at IDEAS and co-author of the paper.

“Patients and families must remain at the centre of any discussion on high-cost therapies. Sustainable access will require greater cross-sector collaboration, ensuring health policy decisions are guided by public interest and protected by strong institutional safeguards,” said Durrah Sharifah Ahmad Azlan, Senior Research Executive at IDEAS and co-author of the paper.

For more information, please download the full paper on our website.

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