P127: Experience Sharing on a Special Legislation Incorporating Rare Diseases, Orphan Drugs, and Patients Safeguarding

Poster Presenter

Ting-Ya Chang

Section Chief
TFDA/Center for Drug Evaluation
Taiwan

Objectives

1. Share Taiwan’s experiences on patient care for rare diseases 2. Describe the regulatory framework for orphan drugs in Taiwan

Method

In Taiwan, designated orphan drugs that have received market approval or special use permits shall submit annual reports describing the amounts used and number of users. We use the annual report database to identify the impact of Rare Disease and Orphan Drug Act on the accessibility of orphan drugs.

Results

Currently, there are 242 diseases recognized as rare diseases in Taiwan. Besides, there are 128 designated orphan drugs. The top three types of these 128 orphan drugs classified by the Anatomical Therapeutic Chemical (ATC) classification include drugs for alimentary tract and metabolism, antineoplastic and immunomodulating agents, and drugs for blood and blood forming organs. Among 242 rare diseases, multiple sclerosis had the highest number of available drugs, followed by idiopathic or heritable pulmonary arterial hypertension (IPAH or HPAH). Nevertheless, rare diseases like myotonic dystrophy, osteogenesis imperfecta and Williams Syndrome, which affect more than 1/100,000 patients in Taiwan, still have no approved treatments currently. Although incentives include market exclusivity, application fees and document exemption, and preferential pricing methods have been rewarded for orphan drug sponsors, other incentives may be required in order to further encourage orphan drug development. We think incentives provided by other regulatory agencies such as tropical disease or rare pediatric disease priority review vouchers may be a helpful approach that we could learn in the future.

Conclusion

There are still many rare diseases without available treatments at present, such as those classified as kidney and urinary system disorders, muscle disorders, connective tissue disorders, congenital malformation syndromes. Although several incentives have been offered to sponsors of orphan drugs, unmet medical needs still remain. Other policies may be needed to support the development of orphan drugs. Hope the findings in our research could benefit future policy making.