Snapshot

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

This pathway can be used where orphan medicines .

This pathway accelerates the regulatory review process.

It is an abridged review (a reliance pathway).

Procedure for ORPHAN MEDICINE DESIGNATION Application 1. Submission of Orphan Medicine Designation Application Form (link in References section) to the New Drug Product Section or the Biologics Section, Centre for Product and Cosmetic Evaluation, NPRA. The applicant shall submit the completed application form to the relevant section, depending on the product category. The applicant shall submit a separate application form for designation of same orphan medicine to treat a different rare disease (as same medicinal product may be used to treat different rare disease). 2. Screening of application form The relevant section shall screen the submitted form (i.e., without the dossier). If the form is found incomplete, the application shall be rejected. A complete application shall be further evaluated. 3. Evaluation of application The section may seek advice from relevant specialists or expert panel when deemed necessary. A timeframe of two weeks is allocated for the reply. The section shall prepare an evaluation report to be tabled in the DEC meeting. 4. DEC Meeting The DEC shall make the decision to grant the designation of orphan medicine or otherwise. 5. Issuance of official letter to applicant The decision of the DEC shall be informed to the applicant via official letter. Timeline. - The decision of the DEC to grant the designation of orphan medicine or otherwise will be made within 45 working days upon receipt of application. - The medicinal product that has been granted designation as an orphan medicine will be automatically granted priority review. The timeline for evaluation of the medicinal product (biologics and new chemical entities) is 120 working days. The timeline shall commence after payment has been confirmed by the PRH (i.e., post-screening approval). Cancellation of Orphan Medicine Designation - The NPRA (through input from the DEC) may, at any time and by notice, cancel any orphan medicine designation of an unregistered/registered medicinal product that no longer meets the criteria for such designation. However, the registration status of that medicinal product shall remain valid.