The Taiwan Orphan Drug Pathway pathway has been developed by the Taiwan Food and Drug Administration of Taiwan (Chinese Taipei).

This pathway can be used where This pathway is for orphan drugs and difficult-to-obtain drugs. Taiwan promulgated and enacted the “Rare Disease and Orphan Drug Act” in 2000. According to the Act, the term “rare diseases” refers to diseases with prevalence lower than the standard announced by the central government or with special circumstances, and reviewed by the“Review Committee for Rare Diseases and Orphan Drugs” as well as officially announced by the central government. The prevalence rate of rare diseases in the current public notice is lower than 1 in 10,000. The term orphan drugs as used in the Act therefore refers to drugs with major indications for the prevention, diagnosis and treatment of rare diseases that have been submitted in accordance with the Act for application, recognized through review by the Review Committee specified in Article 4 of the Act, and announced by the central competent authority. According to Article 22 of The Rare Disease and Orphan Drug Act, difficult-to-obtain drugs are those that have difficulties in manufacturing or importing and are also reviewed by the Review Committee and determined to be beneficial for the treatment of specific diseases. The same incentives as those offered for orphan drugs are offered for difficult-to-obtain drugs , including registration, market approval and specific use permission..

This pathway accelerates the regulatory review process.

It is an abridged review (a reliance pathway).

According to the Rare Disease and Orphan Drug Act, the process of orphan drug application has 3 steps: Rare Disease Designation; Orphan Drug Designation; and Orphan Drug Registration. For Orphan Drug Designation, the application should demonstrate that orphan drug criteria have been met (i.e., life-threatening or debilitating nature of condition, especially for genetic disease; medical plausibility; prevalence < 1 in 10,000; and it is unlikely to generate sufficient return on investment). All claims should be substantiated by references. For Orphan Drug Registration, orphan drug designation does not change the standard requirements for NDA. Applicants must prepare and submit data in CTD format. There is also an exemption one Certificate of Pharmaceutical Product (2 CPP for non-orphan drugs) and relaxation of the number of subjects required in clinical trials. The orphan drug designation takes approximately 6-12 months.