Written by Durrah Sharifah Ahmad Azlan, senior research executive at the Institute for Democracy and Economic Affairs (IDEAS)

Treatment options for rare disease patients are advancing at remarkable speed. Nearly 50 gene therapies have been approved globally, with thousands more in development. Yet, for many families living with rare diseases, these treatments remain out of reach. These innovations have arrived, but access has not. Gene therapies are costly and require specialised clinical capacity, posing significant challenges for health systems that are still developing their regulatory and specialist ecosystems.

So far, Malaysia has approved only one gene therapy and there is no established procurement pathway to make it accessible. Beyond the price of the medicines themselves, costs extend to specialist training, infrastructure and long-term care for complex genetic conditions. There is, however, a strategic opportunity for Malaysia to respond differently by attracting more clinical trials for emerging therapies.

Clinical trial gap

Clinical trials carry inherent risks but they also provide early, and often cost-free, access to breakthrough treatments. In a recently published article by the Hong Kong Journal of Paediatrics, Malaysian parents in Perlis and Kedah whose children participated in clinical trials expressed their appreciation for receiving free treatment, particularly with the rising costs of living.

Despite these benefits, participation remains uneven globally. In 2024, the Access to Medicine Foundation reported low- and middle-income countries are frequently bypassed for clinical trials despite making up 80% of the world population. As a result, data on how diverse populations respond to new therapies is limited. For many Malaysian families, exclusion from trials often means prolonged waiting, financial strain and the emotional burden of fundraising for treatment abroad.

Angelman syndrome as a policy pilot

For some conditions, the window for opportunities remains open. One such example is Angelman syndrome (AS), a rare genetic disorder affecting approximately one in 12,000 to 20,000 live births or an estimated five to eight Malaysian infants annually. Children with AS experience severe developmental delay, absent or minimal speech, movement and balance disorders, seizures and lifelong care needs.

Encouragingly, AS is caused by only a single genetic error and is not degenerative. This creates genuine therapeutic potential. Several approaches are already in clinical trials globally, with researchers racing to develop the first approved gene therapy for the condition.

In Malaysia, a small but growing community is taking shape, joining the expanding rare diseases community. Families and advocates convened the country’s first national AS meeting last August, marking a significant milestone in awareness and solidarity.

What is holding Malaysia back?

Malaysia has potential in strengthening the overall medical talent pool and hospital infrastructure. The extra challenge lies in the depth and coordination of its rare disease ecosystem, which remains fragmented and underdeveloped, particularly for conditions like AS. Global research sponsors prioritise coordinated clinical infrastructure, predictable regulatory pathways and meaningful patient engagement. Many emerging rare disease therapies also require specialised administration and monitoring, demanding sustained investment in training and facilities. These are areas in which Malaysia can improve.

Malaysia could explore partnerships with more established organisations overseas or similar collaborative frameworks to build local capacity. For example, patient-led organisations such as the Foundation for Angelman Syndrome Therapeutics (FAST) working to cure AS have funded dedicated training centres in the US, ensuring doctors are prepared to run trials and deliver therapies safely.

Clinical trials need robust systems for identifying eligible participants and medical experts who can provide ongoing support. Where these systems are weak or absent, countries are more likely to be overlooked. This ultimately deepens inequity, as treatments become available only when they are fully commercialised and financially out of reach.

Building a trial-friendly environment as an investment

Public spending on rare diseases is often stereotyped as a high cost for a small population. Yet, it can also be understood as a strategic investment.

The economics case for clinical research is increasingly recognised. Prime Minister Datuk Seri Anwar Ibrahim asserted last year that clinical research generates employment, technology transfer and economic growth. Since 2012, Malaysia’s clinical research ecosystem has contributed more than RM1.6 billion to gross national income and created thousands of high-skilled jobs. Globally, the clinical trial market is projected to reach US$120 billion (RM473.2 billion) by 2035.

Time to act

The window to bring clinical trials to Malaysia is open, but it will not remain so indefinitely. AS may affect only a small number of children each year, yet it offers a practical policy pilot. By building capacity around it, Malaysia can develop the regulatory, clinical and community foundations needed to support a broader rare disease population while being a regional leader.

Rather than remaining a passive recipient of innovations developed elsewhere, Malaysia can position itself as an active contributor. Doing so would ensure that families are no longer left waiting on the margins of medical progress.

This article was featured in The Edge Malaysia Weekly on March 9, 2026 – March 15, 2026

The views expressed in this article are solely those of the authors and do not necessarily represent the views or positions of IDEAS Malaysia. All opinions are the author’s own.