罕見疾病防治及藥物法施行細則

This set of Rules is formulated in accordance with the regulations of Article 35 of the Rare Diseases and Orphan Drugs Act (hereafter referred to as the Act).

1The disease prevalence mentioned in Paragraph 1, Article 3 of the Act, refers to the annual prevalence rate calculated by the central competent authority with reference to the information reported in accordance with the regulations of Article 7 of the Act and the medical care data from National Health Insurance.

2The central competent authority shall review the above-mentioned annual prevalence rate at least once every three years.

The special circumstances mentioned in Paragraph 1, Article 3 of the Act, refer to the disease prevalence rate that exceeds the criteria announced by the central competent authority or that the prevalence rate is difficult to calculate, and that the methods, medical products, and special dietary foods needed for diagnosis and treatment are indeed difficult to obtain.

The central competent authority shall, in accordance to Paragraph 3, Article 3 of the Act, include the name and the applicable disease in the announcement of the special dietary foods.

The report stipulated in Article 7 of the Act shall be submitted within a month from the day of discovery.

The central competent authority shall include the demographic changes of the rare disease population in the health statistics.

1The central competent authorities shall formulate the plans relating to the organizing and holding of educational activities for the prevention and control of rare diseases annually, in accordance with the regulations of Paragraph 1, Article 11 of the Act.

2In event of mediation stipulated in accordance to the regulations of Paragraph 2, Article 11 of the Act, the central competent authority may provide necessary information to relevant institutions and/or agencies for reference, under informed consent of the patient, or the legal representative, and the legal guardian. For patients lacking disposing capacity, the central competent authority shall obtain informed consent from the respective assistant.

1Similar medical product mentioned in Paragraph 1 and Paragraph 2, Article 17, and Paragraph 1, Article 18 of the Act refers to the following：

21.The active substance(s) and the indication(s) that are the same as the chemical drugs with previously issued permit license upon registration and market approval in accordance with this Act, including structural isomers, complex, salts, esters, chelate or other non-covalent derivatives.

32.The principal molecular structural features and the indication(s) are the same as the biological or macromolecular product with previously issued permit license upon registration and market approval in accordance with this Act.

The right-holders of orphan drugs mentioned in Subparagraph 1, Paragraph 1, Article 18 of this Act, refer to persons who own permit licenses of the orphan medical products.

Applicants for registration and market approval shall submit documents to the central competent authority to prove that the safety or efficacy of the new orphan medical products under application is superior to the orphan medical products already approved in accordance with the regulations of Subparagraph 2, Paragraph 1, Article 18 of the Act.

The failure to meet demands for the drugs mentioned in Subparagraph 3, Paragraph 1, Article 18 of the Act, refers to the failure of the right-holders in supplying in sufficient amount medical products of the rare diseases and those who have been notified by the central competent authority to improve in due time and have not made improvement yet.

When considering the reasonableness of the prices of orphan medical products, the central competent authority shall consult the price analysis information supplied by new applicants and right-holders, The National Health Insurance Pharmaceutical Benefits and Reimbursement Schedule, as well as other related information in accordance with the regulations of Subparagraph 4, Paragraph 1, Article 18 of the Act.

The central competent authority shall commission professional institutions or organizations to conduct the matters stipulated under the regulations of Article 34-1 of the Act.

The documentation format required under the Act and the set of Rules shall be decided by the central competent authority.

The set of Rules shall be implemented on the same day as the day of announcement.