National rare diseases governance committee set up applauded

LETTER| The Institute for Democracy and Economic Affairs (IDEAS) applauds the Ministry of Health (MoH) on their efforts in setting up a national governance committee to manage rare diseases in Malaysia. 

Over the past year, IDEAS “Championing for the Rare” project aimed to highlight the voices of patients and advocate for a national policy for rare diseases.

Deputy health minister, Dr Lee Boon Chye, has announced in Parliament that a national framework for rare diseases has been developed to integrate the management of rare diseases in Malaysia through a governance committee.

Many countries worldwide have established the rare diseases and/or orphan drug acts or policies, including Asian countries such as Taiwan, Singapore, China, India and Japan. 

A policy or national framework would provide better access and healthcare services to the rare diseases patients.

Therefore, this initiative by the MoH is very much needed to address the needs of rare diseases patients.

The deputy health minister outlined that the short-term goals of the framework would include deciding on a definition of rare diseases and strengthening the governance of rare diseases services and relevant labs. 

He said in the long term, the committee aims to create policies and better strategies to improve access to orphan products and aspires to set up a registry to collect and analyse patient data.

As noted in IDEAS policy paper “national policy on rare diseases living with dignity: in search of solutions for rare diseases”, deciding on a definition is a vital step forward for the gov't to further effectively support these patients and manage treatment in the country. 

Only 5 percent of rare diseases have available pharmaceutical treatment options and these drugs are often expensive and require gov't support for access.

As highlighted in another IDEAS policy paper “ improving access to orphan drugs”, there is a need to establish a fit-for-purpose framework to evaluate and fund therapies, drawing on other countries’ experiences in decision making. 

IDEAS commends this step to formulate stronger strategies to allow for timely access to appropriate treatment and encourages public awareness on rare diseases for more effective disease management. 

Having a registry in place can feed into research and development in fields related to rare diseases and improve cooperation with insurance companies to cover these diseases in Malaysia.

The committee will also be represented by other ministry stakeholders including Education Ministry, Social Welfare Department and Ministry of Women, Family and Community Development into the rare diseases governance committee. 

IDEAS looks forward to future developments for Rare Disease in Malaysia through this national governance committee and framework.