KUALA LUMPUR (Feb 23): Malaysia is still exploring the introduction of gene therapy as a potential curative treatment for thalassaemia patients in the country, but its high cost remains a major obstacle, the Dewan Rakyat was told on Monday.

Health Minister Datuk Seri Dr Dzulkefly Ahmad, while describing the treatment as highly effective, highlighted that the price tag — estimated at up to RM13 million per patient — is currently beyond the government’s immediate financial capacity.  

“The cost today is extremely high and we are not in a position to make upfront payments. However, technological developments — particularly in countries like China — are expected to drive prices down. We hope access will improve in the near future,” he said during the oral question-and-answer session.

For context, gene therapy is a form of precision medicine involving the extraction of a patient’s own stem cells, which are genetically modified in a laboratory before being reintroduced into the body to correct the underlying defect.

Dzulkefly noted that more than 90% of patients who receive these therapies no longer require lifelong blood transfusions. Several such treatments have been approved by international regulators, including the US Food and Drug Administration and the European Medicines Agency.

He added that local institutions, including the Institute for Medical Research (IMR) and other research bodies, have begun building expertise in gene-based therapies, including gene addition and other advanced modalities.

However, he cautioned that despite its curative potential, widespread implementation remains limited due to the need for specialised gene therapy facilities, multidisciplinary expertise, high-standard laboratory infrastructure, robust referral systems and sustainable healthcare financing mechanisms.

Dzulkefly stressed the importance of international collaboration to reduce costs and democratise access, particularly for low- and middle-income countries (LMICs).

Latest data from the Ministry of Health showed that as of 2023, Malaysia had 9,554 registered thalassaemia patients. Thalassaemia is a hereditary blood disorder caused by impaired haemoglobin production, resulting in anaemia of varying severity. 

The lifetime cost of care for a transfusion-dependent thalassaemia (TDT) patient — from childhood until age 40 — is estimated at US$606,665 (nearly RM3 million) per patient, covering both medical and related out-of-pocket expenses borne by patients and their families, according to the MOH. 

For now, the Clinical Practice Guidelines (CPG) Management of Thalassaemia (Second Edition 2024) identify haematopoietic stem cell transplantation (HSCT) as the only established curative option for transfusion-dependent thalassaemia in Malaysia.

Since 2007, a total of 212 patients have been cured through HSCT. The cost of the procedure ranges between RM100,000 and RM120,000 per patient — significantly lower than gene therapy.

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