According to a story from The Edge Markets, Dr. Lee Boon Chye, Malaysia’s Deputy Minister of Health, has recently announced an initiative to develop a National Framework for Rare Disease, which aims to improve conditions for rare disease patients in Malaysia. Rare diseases are not receiving adequate attention or funding anywhere in the world, but in developing countries, many of the challenges that face rare patients are exacerbated.
Rare Diseases: A Challenge Worldwide
In Malaysia, a substantial portion of patients with rare diseases are never diagnosed or treated, as both doctors and the patients themselves have little knowledge about many rare diseases; this is a problem that rare patients face worldwide. Rare diseases can often be crippling or debilitating, constituting a substantial medical, financial, and psychological burden.
The National Framework for Rare Disease
The Framework aims to achieve a number of different goals. In the short term, the initiative will determine a national definition for a rare disease, which will be the basis for future laws regarding them. Another objective is to strengthen government resources that are oriented towards disease treatment and study. The National Framework for Rare Disease will also foster rare disease advocacy in the public sphere. More long term goals include the creation of rare disease patient registries in Malaysia, which will serve as resources for data gathering and research. The initiative also aims to make it easier for Malaysian rare disease patients to have access to the orphan drugs that they need for treatment.
Ensuring Success
All of these goals from the country’s Ministry of Health constitute necessary and encouraging improvements for the plight of rare disease patients in Malaysia. However, considerations about the daily management and care for these patients are outside of the Ministry’s capabilities. Since patients are often economically vulnerable and uninformed about their disease and the resources that are available, helping rare disease patients will require collaboration across different ministries. Part of the Framework also includes a national governance committee for rare diseases that will include representatives from the Ministry of Women, Family, and Community Development and the Ministry of Education.
These initiatives from Malaysia are undoubtedly steps in the right direction. However, only a holistic approach that encourages collaboration between all relevant government ministries will result in long-term success.
