Kuala lumpur: The launch of the country’s first-ever National Policy for Rare Diseases in Malaysia, represents a national commitment to ensure that Malaysians with rare diseases receive the attention and care they deserve. Health Minister Datuk Seri Dr Dzulkefly Ahmad said the pre-launch of the National Policy for Rare Diseases in Malaysia, last Aug 27, marked a historic milestone for Malaysia, aimed at addressing long-standing gaps in the diagnosis, treatment and support for individuals affected by rare conditions.

According to BERNAMA News Agency, rare diseases affect fewer than one in 4,000 people, yet the journey to diagnosis and care is often long, uncertain and lonely. Globally, there are up to 8,000 rare conditions but only a small fraction have approved treatments, underscoring the urgency of action in this area.

Structured around five key pillars, Dzulkefly said the policy seeks to reduce the diagnostic odyssey and bring families closer to answers, while ensuring no patients are left behind. “Beyond every statistic is a story of courage, a child, a parent and a family. To them, I say this policy is our promise. You matter and you will not be forgotten,” he emphasized.

The five key pillars outlined in the policy include early detection, fairer access to treatment, stronger research and innovation, development of clinical expertise, and the establishment of a national rare disease database. These initiatives aim to create a healthcare system that is inclusive, equitable, and compassionate, ensuring that no Malaysians are left behind due to the rarity of their condition.

According to the World Health Organisation (WHO), rare diseases are defined as diseases that affect a small portion of the general population and are usually debilitating in nature. These diseases are often chronic, serious, and can be life-threatening. It is estimated that there are between 6,000 and 8,000 rare diseases worldwide, with the majority of them being caused by genetic factors.