The 2nd Malaysia Conference on Rare Disorders with the theme “Developing Strategies for a National Rare Disease Plan” was a significant event for rare disease patients in Malaysia. For the first time, three rare disease patient support groups in Malaysia namely the Malaysian Rare Disorders Society (MRDS), the Malaysia Lysosomal Diseases Association (MLDA) and the Malaysia Metabolic Society (MMS) collaborated to organise a national rare disease conference and for the first time stakeholders involved with rare disease patients sat down together to discuss strategies for developing a national rare disease plan.
More than fifty invited participants from various ministries, universities, the pharmaceutical industry, rare disease patient support organisations and other NGOs attended the Conference. The Deputy Director-General of Health (Medical), Datuk Dr S. Jeyaindran Tan Sri Sinnadurai delivered the keynote address on behalf of the Director-General of Health, Datuk Dr Noor Hisham Bin Abdullah. In his speech, Dr Jeyaindran said that the Ministry of Health is in full support of the Conference and to address issues of rare diseases by networking and involving stakeholders.
The Conference programme consisted of four forums with the following themes:
• Developing strategies for a national rare disease plan
• Coordinated national activity
• Multidiscipline clinical care and systematic approach for rare disease patients
• On time treatment
The forums had international and local speakers speaking on the themes and their talks served as reference to two roundtable discussions that followed. Professor Hugh Dawkin from the Health Department of Western Australia began the session by giving an overview of the global and the Australian scenarios in developing a national rare disease plan. This was followed by Dr Keng Wee Teik from Hospital Kuala Lumpur (HKL) who presented an overview of the rare disease situation in Malaysia. Dr Shuan-Pei Lin from the Taiwan Foundation of Rare Disorders gave an account of the implementation of Taiwan’s Rare Disease and Orphan Drug Act.
Dr Rowani Mohd Rawi from Universiti Sains Malaysia (USM) provided a rare insight to the orphan drugs situation in Malaysia. The final presentations were case studies by Professor Thong Meow Keong and Ms Juliana Lee (University Malaya Medical Centre) on the “Impact of Genetic Counselling in Rare Genetic Disorders”, Dr Chew Hui Bein (HKL) on “Challenges in the Diagnosis and Treatment of Rare Diseases in Malaysia”, and Dr Rowani Mohd Rawi on “Challenges Faced by Outskirts University Hospitals (outside Klang Valley)”.
There was a general consensus by the participants that rare disease patients have rights founded on the Universal Declaration of Human Rights and other international conventions; that ‘rare disease’ should be given formal recognition; that rare disease patient support groups join together to form an alliance to speak with one voice; that there was an all-round support for the development of a national rare disease plan; and that a multi-stakeholder committee be formed to further explore and discuss matters raised during the Conference.