Every year, Rare Disease Day is observed on the last day of February to raise awareness about rare diseases and their impact on patients, families, and healthcare systems. In Malaysia, where over 2 million people are estimated to be living with a rare disease, this global initiative is crucial in driving better policies, research, and access to treatment.
What is a Rare Disease?
A rare disease is defined as a condition affecting a small percentage of the population. While individual diseases are uncommon, collectively, rare diseases impact millions worldwide. Many of these conditions are genetic and appear early in life, requiring lifelong medical care and support.
In Malaysia, some of the recognized rare diseases include:
- Duchenne Muscular Dystrophy (DMD) – A progressive muscle-wasting disorder
- Gaucher Disease – A metabolic disorder affecting organs and bones
- Spinal Muscular Atrophy (SMA) – A neuromuscular disease causing muscle weakness
- Pompe Disease – A condition that impairs the body’s ability to break down sugar for energy
Challenges Faced by Rare Disease Patients
Patients with rare diseases often encounter delayed diagnoses, limited treatment options, and high medical costs. Many conditions require specialized medication, but treatment accessibility remains a challenge due to the high costs and limited availability of orphan drugs.
In Malaysia, the government has taken steps to improve rare disease management through the Rare Disease Fund under the Ministry of Health (MOH), which supports selected treatments. However, more collaboration between policymakers, healthcare providers, and pharmaceutical companies is needed to enhance access to diagnosis, treatment, and long-term care.
Program JARANG: Malaysia’s Rare Disease Initiative
To strengthen support for individuals living with rare diseases, Pharm-D Health Sciences has launched Program JARANG—a dedicated initiative to raise awareness, improve patient support, and expand access to critical healthcare resources. The initiative’s slogan, “Jarang Tapi Kita Bersama” (Rare But We Are Together), highlights the spirit of unity and collective action in helping patients and their families.
Program JARANG focuses on three key pillars:
- Awareness & Education: Promoting rare disease knowledge among healthcare professionals, policymakers, and the general public to encourage early diagnosis and better support systems.
- Patient Engagement & Support: Providing resources, community programs, and digital health tools to empower patients and caregivers.
- Policy & Advocacy: Collaborating with stakeholders to enhance healthcare policies, funding, and long-term support for rare disease patients in Malaysia.
The Role of Rare Disease Day
Rare Disease Day plays a vital role in:
- Raising public awareness about the challenges faced by patients
- Encouraging policymakers to enhance support and funding
- Promoting scientific research for better treatment options
- Strengthening patient advocacy groups to give individuals a voice
Advancements in Rare Disease Research
Globally, AI-powered diagnostics and gene therapy innovations are transforming rare disease detection and treatment. Countries like the United States and Singapore have established national rare disease registries to improve early diagnosis and targeted therapy access. Malaysia can benefit from similar advancements through regional partnerships and increased healthcare funding.
How You Can Help
- Support patient organizations like Malaysia Lysosomal Diseases Association (MLDA)
- Advocate for policy changes to improve rare disease funding
- Spread awareness through social media and community events
- Participate in Rare Disease Day activities to show solidarity
Conclusion
Rare diseases may be uncommon, but the challenges faced by patients are significant. By raising awareness, supporting research, and advocating for better policies, we can ensure a brighter future for those living with rare conditions.
With initiatives like Program JARANG paving the way for better healthcare solutions, Malaysia is taking important steps towards inclusive and accessible healthcare for all.
References:
- Malaysia Ministry of Health – Rare Disease Management Initiatives
- Malaysia Lysosomal Diseases Association (MLDA)
- Global Rare Disease Research & Policy Reports
- Pharm-D Health Sciences – Program JARANG (jarang.pharmdhs.com)
