Page 52 - 2018食藥署年報(英文版)

Section 5 Policy on Orphan Drug Management

Origin of Policy

The “Rare Disease and Orphan Drug Act” was finalized on January 14, 2000, and became
effective from August 9 the same year. Since then, Taiwan has become the 5th region which had

passed rare disorder-related acts in the world (after the US (1983), Japan (1993), Australia (1997)
and EU (2000)), indicating that Taiwan keeps in step with global trends regarding the fulfilment
of patient needs.
Although the US is the first country in the world stipulating regulatory systems regarding
rare disorder and orphan drugs, whereby promulgated the “Orphan Drug Acts” long ago in 1983,
Taiwan displays a more inclusive attitude toward such topic by combining the “Rare Disorder
Act” and “Orphan Drug Act”. The “Rare Disease and Orphan Drug Act” covers topics such as

preventive eugenic health, health education, patient welfare, international collaboration and
medical team staffing. The comprehensive scope of the Act suggesting that the management
of rare diseases in Taiwan is not limit to orphan drug regulation, but has been referred to
multidisciplinary teamwork (including disease prevention, medical assistance public health, etc.).

Implementation Measures

To encourage pharmaceutical companies to manufacture or importing orphan drugs, “Rare
Disease and Orphan Drug Act” specifies that the valid duration of orphan drug licenses has
been extended up to 10 years. During the 10-year period, no other drug registration of similar
classifications will be approved to protect the interest of the thereof pharmaceutical company.

In addition, under the premises of quality, efficacy and safety of medicinal products, the
required application documents for orphan drug registration could be simplified (e.g. cancelling
the requirement of certification of approval issued by one of the top 10 advanced countries,
exemptions from sample testing, lower registration fee, etc.) to facilitate the manufacturs and
importation of orphan drugs, therefore extend the lifespan of patients with rare diseases.

Outcomes and Benefits

As of the end of December 2017, a total of 99 drugs are launched to be subject to “Rare
Disease and Orphan Drug Act” and a total of 85 orphan drug licenses have been issued.

According to Article 21 of the “Rare Disease and Orphan Drug Act”, the central competence
authorities are required to publish a list of approved orphan drugs on the annual report, indicating
information such as the number of uses, number of indicated patients, adverse events and other
relevant reports. Starting from the 1st issue of the “Annual Report on Drugs for Rare Disease”

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