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2 min read
Introduction
Access to treatment for rare diseases remains a growing priority within Malaysia’s healthcare system. To address this need, the Malaysian Ministry of Health (MOH) has created a dedicated approval pathway for rare disease orphan drugs, also known as
orphan medicines.
This process ensures that patients with rare conditions can receive essential treatments that may not yet be registered in the country. Here's a step-by-step overview of how the approval process works for orphan drugs for rare diseases.
1. Application Submission
Healthcare providers or licensed companies (holding a government contract) must submit an application to the Pharmaceutical Services Programme (PSP) under the MOH.
- MOH Institutions: Use the “Permohonan Memperoleh Dan Menggunakan Ubat Yang Memerlukan Kelulusan Khas” form (Appendix C).
- Other Institutions: Use the “Application to Import/Manufacture Unregistered Products for the Treatment of Life-Threatening Illnesses” form (Appendix D).
2. Supporting Documents
Applicants must submit at least three of the following supporting documents to validate the quality and safety of the drugs for rare diseases:
- Good Manufacturing Practice (GMP) Certificate
- Certificate of Analysis (CoA) for the finished product
- Certificate of Pharmaceutical Product (CPP)
- Product label artwork
- Product package insert
- Clinical evidence (if available)
These documents are crucial in ensuring that orphan disease drugs meet regulatory and therapeutic standards.
3. Review Process
Once the application and documents are submitted, the PSP secretariat will screen them for completeness. If complete, the application undergoes an internal review and assessment to determine eligibility and compliance with the guidelines for orphan drugs.
4. Approval Issuance
Upon approval, an official authorization is issued to the applicant, allowing the use or import of the medicine for orphan drugs for rare diseases in the designated institution.
5. Orphan Medicine Designation
For medicines specifically indicated for rare diseases, companies can apply for Orphan Product status with the National Pharmaceutical Regulatory Agency (NPRA). When required, the NPRA may consult rare disease experts or patient organizations for input on the application.
This designation can offer additional Regulatory support for introducing orphan disease drugs into the market.
6. Post-Approval Compliance
Once approved, healthcare institutions must comply with distribution and usage procedures for treating patients with rare diseases. This ensures responsible handling and tracking of the medicine's use.
Conclusion
Malaysia’s structured approach to rare disease orphan drug approvals demonstrates a commitment to patient access and safety, even for small patient populations. However, the process requires precision in documentation and compliance with Regulatory protocols.
At Freyr, we support pharmaceutical companies in navigating Malaysia’s orphan medicine pathway—from application preparation to regulatory liaison and post-approval compliance. With our in-depth understanding of local guidelines and global best practices, we help ensure timely access to life-saving therapies for rare disease patients.
Need guidance through Malaysia’s rare medicinal product approval process? Contact Freyr to learn how we can help.
