The Orphan Drug Route pathway has been developed by the National Pharmaceutical Regulatory Agency (NPRA) of Malaysia.

This pathway can be used where Orphan drug designation (ODD) is subject to these criteria: (a) A medicine, vaccine, or in vivo diagnostic agent that is primarily intended to treat, prevent, or diagnose a rare disease and, (b) No satisfactory method of diagnosis, prevention or treatment of the condition concerned can be authorized, or if such a method exists, the medicinal product must be of significant benefit to those affected by the condition. Rare disease (RD) refers to the diseases listed in the latest Malaysian RD List. Criteria for inclusion in the Malaysia RD list: (1.) There are or have been confirmed patients locally; (2.) 1 in 4,000; (3.) Disease is a severe condition; and (4.) Inclusion is approved by the national advisory committee. "Significant benefit" refers to the EMA definition that states "a medicine produces a clinically relevant advantage or makes a major contribution to patients care, compared with existing treatment methods". CATEGORIZATION OF ORPHAN MEDICINE - Orphan medicine are categorized according to their use: (1) Emergency Treatment: Immediate intervention for life-saving / avoidance of permanent disability. For accessibility, the medicines shall be available as ready inventory (stock keeping) and shall be managed as emergency item. (2) Lifetime treatment: Lifetime treatment is defined as long term treatment and maintenance therapy for rare disease. The medicines may be in either pharmaceutical dosage forms or non-pharmaceutical dosage forms (e.g. powder and liquid in the form of raw material). The orphan medicine shall be made available accordingly by the facilities that managed the patients. .

Orphan drug regulatory requirements: (1.) Apply for OOD. Orphan drug designation takes 45 working days. After it is granted, proceed to drug registration. (2.) Drug registration: priority review granted automatically with evaluation timelines to be 120 instead of 245 working (full evaluation) days. Certain flexibilities are permitted e.g., phase 2 clinical data may be acceptable for registration (with justification of absence of phase 3 data), zone IVb stability data is not mandatory, etc.