Appraising National Policy and Access to Orphan Drugs for Rare Disease Treatment in Malaysia
as
by Azuwana Supian
Appraising National Policy and Access to Orphan Drugs for Rare Disease Treatment in Malaysia – by Azwa
RECAP: RARE DISEASE FACTS
PATIENT JOURNEY
RARE DISEASE: CURRENT POLICY LANDSCAPE
MALAYSIAN ORPHAN MEDICINES GUIDELINE
MALAYSIA: ACCESS TO MEDICINES
MALAYSIAN REGISTERED ORPHAN MEDICINES
CHALLENGES IN ACCESSING ORPHAN MEDICINES
IMPACT OF PATIENTS & HEALTHCARE SYSTEM
KEY RECOMMENDATIONS FOR POLICY ENHANCEMENT
FOSTERING COLLABORATION & SUPPORT NETWORKS
CONCLUSION: TOWARDS A BRIGHTER FUTURE
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ORPHAN MEDICINES �DESIGNATION & REGISTRATION
TABLE OF CONTENT
Appraising National Policy and Access to Orphan Drugs for Rare Disease Treatment in Malaysia – by Azwa
�
80% are of genetic origin
30% of these childhood rare die before 5 year old
75% affect children
A life-threatening or chronically debilitating rare condition affecting a small percentage of the general population.
RECAP: RARE DISEASE FACTS
Appraising National Policy and Access to Orphan Drugs for Rare Disease Treatment in Malaysia – by Azwa
PATIENT JOURNEY
Appraising National Policy and Access to Orphan Drugs for Rare Disease Treatment in Malaysia – by Azwa
MALAYSIA: ACCESS TO MEDICINES
AIM: To improve the health of the nation through strengthening equitable access and promoting the rational use of medicines to achieve better health outcomes for Malaysians
.
quality
effective
safety
affordable
Appraising National Policy and Access to Orphan Drugs for Rare Disease Treatment in Malaysia – by Azwa
CURRENT POLICY LANDSCAPE
National Rare Disease Policy
Healthcare Infrastructure
Funding for Orphan Drugs
Appraising National Policy and Access to Orphan Drugs for Rare Disease Treatment in Malaysia – by Azwa
National Rare Disease Policy
Malaysia has made strides in recognizing rare diseases through a national policy framework, aiming to provide guidance for diagnosis, management, and support.
However, its implementation and comprehensive scope are still evolving, particularly regarding consistent funding and access mechanisms.
CURRENT POLICY LANDSCAPE
will publish
soon… ☺
Appraising National Policy and Access to Orphan Drugs for Rare Disease Treatment in Malaysia – by Azwa
TOR :
NATIONAL RARE DISEASE COMMITTEE
CHAIRPERSON
Deputy Director General of Health (Medical)
Technical Committee on Clinical Management & Support Services
Technical Committee on Orphan Drugs
Technical Committee on Advocacy & Health Education
SECRITARIAT
O&G & Peadiatric Services Unit, Medical Development Division
6. To provides policies and strategies for prevention of rare diseases through advocacy and public health education including prenatal diagnosis and newborn screening.
Appraising National Policy and Access to Orphan Drugs for Rare Disease Treatment in Malaysia – by Azwa
RARE DISEASE DEFINITION IN MALAYSIA
492 type of
rare disease
Definition: Rare disease is defined as a life-threatening and/or chronically debilitating rare condition as listed in the Malaysian Rare Disease List.
The criteria for inclusion a disease in the Malaysian Rare Disease List are :-
Appraising National Policy and Access to Orphan Drugs for Rare Disease Treatment in Malaysia – by Azwa
CURRENT POLICY LANDSCAPE
Specialised centres and expert networks are being established to cater patients with rare diseases.
While urban areas may have better access, significant disparities persist in rural regions, affecting timely diagnosis and continuous
care for many.
Healthcare Infrastructure
Appraising National Policy and Access to Orphan Drugs for Rare Disease Treatment in Malaysia – by Azwa
Genetic Clinic
Visiting Clinic
Funding for Orphan Drugs
Funding for orphan drugs primarily comes from government allocations and patient-specific programs.
The current system faces challenges in affordability, sustainability and equitable access due to the high cost of many orphan medicines and limited budget allocations.
CURRENT POLICY LANDSCAPE
Appraising National Policy and Access to Orphan Drugs for Rare Disease Treatment in Malaysia – by Azwa
2017
2016
2015
2014
MALAYSIAN ORPHAN MEDICINES�GUIDELINE
Appraising National Policy and Access to Orphan Drugs for Rare Disease Treatment in Malaysia – by Azwa
NO. | ITEM | NORMAL | ORPHAN MEDICINES |
1 | Timeline for evaluation: New chemical entities & biologic | 245 working days | Priority review: 120 working days |
2 | Substantial efficacy and safety evidence | Phase III clinical data is required | Phase II clinical data may be acceptable |
3 | Protocol of analysis, and Certificate of Analysis | 3 batches | at least 1 batch |
4 | Fees for GMP inspection | Foreign Manufacturer:
| Foreign Manufacturer:
|
ORPHAN MEDICINES�DESIGNATION & REGISTRATION
Appraising National Policy and Access to Orphan Drugs for Rare Disease Treatment in Malaysia – by Azwa
https://npra.gov.my/images/2019/12/NCE/List_of_Approved_Orphan_Medicines_updated_March_2025.pdf
10 BIOLOGIC
12 NCE
MALAYSIAN REGISTERED �ORPHAN MEDICINES
Appraising National Policy and Access to Orphan Drugs for Rare Disease Treatment in Malaysia – by Azwa
COST OF ERT
(ENZYME REPLACEMENT THERAPY)
No. | Medicines | Rare diseases | Registered | Formulary | Est. Cost / year MYR (USD) ± |
1 | Idursulfase 6mg Inj (Elaprase) | MPS II | √ | √ | 750,000 (185,000) |
2 | Imiglucerase 400 units Inj (Cerezyme) | Gaucher | √ | √ | 762,000 (187,000) |
3 | Laronidase 2.9mg Inj (Aldurazyme) | MPS I | √ | √ | 500,000 (123,000) |
4 | Alglucosidase 50mg Inj (Myozyme) | Pompe | √ | √ | 500,000 (123,000) |
5 | Elosulfase Alfa 5mg Inj (Vimizim) | MPS IVA | √ | X | 1,000,000 (246,000) |
6 | Galsulfase 5mg Inj (Naglazyme) | MPS VI | √ | X | 1,000,000 (246,000) |
8 | Agalsidase alfa/beta (Fabrazyme) | Fabry | √ | X | 1,000,000 (246,000) |
Appraising National Policy and Access to Orphan Drugs for Rare Disease Treatment in Malaysia – by Azwa
HKL
Supply chain & distribution: HKL- centre for ERT
CHALLENGES IN ACCESSING �ORPHAN MEDICINES
Appraising National Policy and Access to Orphan Drugs for Rare Disease Treatment in Malaysia – by Azwa
CHALLENGES IN ACCESSING �ORPHAN MEDICINES
Appraising National Policy and Access to Orphan Drugs for Rare Disease Treatment in Malaysia – by Azwa
“Funding from the government for rare disease management �is definitely not enough. From the current economic climate, �there is nothing much that we can change”
“Some of our patients received ERT from ICAP, pharmaceutical industries NGO and other local corporate companies.
However, this is just temporary and can end anytime.”
Emotional & Financial Burden
Patients and their families face immense emotional distress and significant financial hardship due to the chronic nature of rare diseases and the high cost of treatment and ongoing care.
Strain on Healthcare Resources
The rarity and complexity of these conditions demand specialized expertise and resources, particularly in terms of training, equipment, and facility capacity.
Delayed Treatment
Delays in diagnosis and access to orphan drugs can lead to irreversible progression of the disease, resulting in poorer health outcomes and reduced quality of life
The challenges in rare disease treatment are impacting not just individual patients but also their families and the broader healthcare infrastructure.
Appraising National Policy and Access to Orphan Drugs for Rare Disease Treatment in Malaysia – by Azwa
IMPACT ON PATIENTS & �HEALTHCARE SYSTEM
Establish a Dedicated Orphan Drug Fund
Create a sustainable, ring-fenced national fund to ensure consistent �and predictable financing for the procurement of approved orphan medicines.
Strengthen Public-Private Partnerships
Foster collaborations between the government, pharmaceutical companies, �and patient advocacy groups to negotiate medicine prices and share the burden �of development and distribution.
Enhance Medical Professional Training
Implement specialized training programs for healthcare providers �to improve early diagnosis, appropriate management, and awareness of available �orphan medicine treatments.
Appraising National Policy and Access to Orphan Drugs for Rare Disease Treatment in Malaysia – by Azwa
KEY RECOMMENDATIONS FOR �POLICY ENHANCEMENT
Patient Advocacy Groups
Empower and support patient advocacy groups to voice patient needs, participate in policy discussions, and provide crucial peer support and information �to families.
International Collaboration
Engage with international rare disease organizations and research initiatives to share best practices, facilitate knowledge exchange, and potentially access broader clinical trials.
Academic & Research Institutions
Encourage universities and research institutions to focus on rare disease research, diagnostics, and novel therapeutic development, contributing to local expertise and innovation.
Pharmaceutical Industry
Work closely with the pharmaceutical industry to explore innovative pricing models, access programs, and partnerships that can make orphan drugs more accessible and affordable in Malaysia.
Appraising National Policy and Access to Orphan Drugs for Rare Disease Treatment in Malaysia – by Azwa
FOSTERING COLLABORATION �& SUPPORT NETWORKS
PATIENT ADVOCACY GROUP
Appraising National Policy and Access to Orphan Drugs for Rare Disease Treatment in Malaysia – by Azwa
It requires a concerted effort from policymakers, �healthcare providers, industry partners, and �patient advocates to build a robust and �compassionate healthcare system �that leaves no one behind.
Appraising National Policy and Access to Orphan Drugs for Rare Disease Treatment in Malaysia – by Azwa
Generally, Malaysia has made significant progress �in managing rare diseases in the last ten years.
�However, giving fair and equitable access to treatment �or orphan medicines are a huge challenge for �rare disease management
CONCLUSION: TOWARDS A BRIGHTER FUTURE
Thank you
Appraising National Policy and Access to Orphan Drugs for Rare Disease Treatment in Malaysia – by Azwa