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P118: Facilitating the Accessibility of Orphan Drugs: Analysis of Orphan Drug Cases from 2018 to 2023 in Taiwan

Poster Presenter

Yueh-Wen Lan

Senior Project Manager
Center for Drug Evaluation
Taiwan

Objectives

This study aims to analyze the current status of orphan drugs in Taiwan and attempt to investigate the effect of regulatory measures facilitating the accessibility.

Method

1. Retrieved and analyzed the orphan drug data from TFDA Database and CDE Database for the period 2018 to 2023. 2. Drugs with the same active ingredient(s) were regarded as the same drug despite differences in unit doses (strengths) and/or dosage forms.

Results

In Taiwan, rare diseases are defined as prevalence rate lower than 1 per 10,000, which are life-threatening, mostly genetically related, and designated by the government. To facilitate the accessibility of orphan drugs, the government enacted The Rare Disease and Orphan Drug Act in 2000 and established a special fund for rare diseases in 2005. Generally, before obtaining marketing authorization, orphan drug in Taiwan must complete both designation and NDA. However, drugs can be imported or manufactured through specific permission once the designation has been granted. The agency also provides several incentives for orphan drug, including reducing application fees, waiving the CPP requirement, and 10-year marketing exclusivity. To understand the impact, we analyzed the licensing and review status in Taiwan. The results are summarized as follows: 1) Until 2023, there are 110 designated orphan drugs, 72 of them have been authorized. In terms of the therapeutic area, brain or nervous system disorders and lysosomal storage disorders play the first and second largest proportions of authorized orphan drugs, which accounted for 32% and 18%, respectively. 2) The annual number of orphan drug designations was 9, 6, 13, 8, 13, and 6 respectively from 2018 to 2023. The review period ranged from 93 to 153 days with an average of 121 days. Brain or nervous system disorders and lysosomal storage disorders play the first and second largest proportions of these designations, which accounted for 49% and 9%, respectively. 3) The annual completed number of orphan drug NDA was 3, 5, 6, 10, 10, and 7, respectively from 2018 to 2023. The review time decreased from 322 to 276 with an average of 297 days, which is below the target of 330 days announced by TFDA. The six-year average approval rate was 89%. Among these cases, 39% were biological products. Brain or nervous system disorders plays the major role (37%) whereas hematologic and immune disorders is the second (6%).

Conclusion

The analysis revealed that the review time demonstrates a downward trend. Moreover, the number of orphan drug NDA shows an increasing trend. There are 65% of designated drugs have obtained licenses. Recently, the authorities have further proposed 2 programs to facilitate orphan drugs accessibility and NDA: 1) Parallel review of designation and NDA: In November 2023, an expedited review program was announced for drugs approved by the USFDA, EMA, or MHLW. Under this program, applicants can apply for orphan drug designation and NDA simultaneously. The authority predicts that this parallel review process will reduce the review time by 75 days. 2) Parallel assessment of NDA and Health Technology Assessment (HTA): Since 2024, applicants can request a parallel assessment of National Health Insurance reimbursement pricing evaluation during the NDA review period if the application meets the specific criteria. It is estimated that the pricing evaluation can be completed within 6 months after obtaining the license. The benefits in orphan drugs accessibility after implementing these measures are foreseeable in the future. As an ICH member, we are committed to establishing a more efficient, comprehensive and high-quality regulatory environment. The government aims to encourage stakeholders to enter the market, thereby advocating for the rights of patients.