Rare Disease Prevention and Orphan Drug Act

TFRD had been advocating the adoption of “Rare Disease Prevention and Orphan Drug Act” since the establishment of the foundation. Thanks to the support from medical society, academia, and legislators, the act finally became available in 2000.

How it got started 

In March of 1998, Ms. Serena Wu and Prof. Min-Chieh Tseng, both as parents of rare disease patients, silently worked to promote the systematization and legalization of rare disease issues in Taiwan. On June 16, 1998, the United Daily Evening News reported the issues of rare diseases and orphan drugs, presenting in-depth stories and testimonials of families that suffered from rare diseases. The report highlighted how the medical and social systems overlooked and unfairly treated patients with rare diseases, thus formally unveiling the opportunity for change.

At the beginning, there were 3 versions of laws for Rare Disease Prevention and Orphan Drug Act. The Bureau of Pharmaceutical Affairs (under the administration of Department of Health), the Legislation Yuan and TFRD had different emphasis when drafting the law. The Bureau of Pharmaceutical Affairs emphasized on the location and importance of orphan drugs. However, TFRD knew that rare disease patients wanted more options. Through the continued efforts of the PR, negotiation meetings, and going door-to-door to the legislators. The Bureau of Pharmaceutical Affairs, Legislation Yuan, and TFRD finally consolidated the 3 different laws into one Act, which covered the needs of rare disease patients in respect to: location, designation of orphan drugs, relevant needs, prevention & management of rare diseases, and international medical cooperation.

In January of 2000, after more than forty days and three readings in the Legislative Yuan, the Rare Disease Prevention and Orphan Drug Act was enacted the following month with the announcement from the President. 

After the passage of the Act patients can receive the reimbursements from the government. Patients that have been acknowledged officially as having rare diseases could apply for reimbursement for the medical expenses occurred in local medical center, or regional teaching hospitals. Expenses include diagnosis, treatment, drugs, and special nutritional supplements. The reimbursement cap is 70% of actual expenses but families that qualify for low-income status can receive reimbursements up to 100% for drugs and nutritional supplements for the patient. 

**The official translation by the government is The Rare Disease and Orphan Drug Act. But TFRD wants it to be closer to the Chinese naming to highlight the concept of "prevention ," so we use Rare Disease Prevention and Orphan Drug Act on this website.

Legislative History

Promulgated by Presidential Order Hua Tsung (1) Yi Tzu No. 8900031600 on February 9, 2000.
Deleted Article 12 and Article 37; and amended Article 1, Article 3, Article 5, Article 7 through Article 11, Article 13, Article 15, Article 19, Article 25, Article 32, Article 34 and Article 36, and promulgated by Presidential Order Hua Tsung (1) Yi Tzu No. 09400004921 on January 19, 2005.
Amended Article 6 and Article 33, and promulgated by Presidential Order Hua Tsung (1) Yi Tzu No. 09900331421 on December 8, 2010.
Deleted Article 5; and amended Article 2, Article 3, Article 4, Article 8, Article 10, Article 11, Article 13, Article 15-1, Article 17, Article 22, Article 26, Article 27-1, Article 33 and Article 34-1, and promulgated by Presidential Order Hua Tsung (1) Yi Tzu No. 10400002311 on January 14, 2015.

Article Content

Enforcement Rules Content

Revisions

Rare Disease Prevention and Orphan Drug Act revisions - Follow-up to secure patients’ rights

The 3rd amendment of the Rare Disease Prevention and Orphan Drug Act was publicized on January 14th, 2015. In order to meet the requirements of the amendment, sub-laws were proposed and revised.

The Enforcement Rules of the Rare Disease Prevention and Orphan Drug Act had been revised and publicized by the end of 2015. The Subsidy Regulations of International Medical Cooperation for Rare Disorders, the Reward and Subsidy Regulations of Rare Disease Prevention and Treatment Work, and the Regulations of Rare Diseases and Rare Genetic Diseases Defect Care Services had been publicized in 2016. It is TFRD’s hope that these regulations will bring more supports to patients and researchers of rare disorders.