P240: Taiwan's Pediatric or Rare Severe Disease Priority Review Voucher Program: Enhancing Novel Drug Accessibility
Poster Presenter
Cheng Chi Chen
Clinical Reviewer
Center For Drug Evaluation Taiwan
Objectives
The goal is to examine the impact of Pediatric or Rare Severe Disease (PRD) Priority Review Voucher Program on New Drug Applications (NDAs) and its potential to accelerate the availability of innovative drugs for special populations in Taiwan.
Method
We analyzed PRD designation applications from 2020 to 2023, including annual case numbers, utilization in pediatric or rare diseases, treatment domains, granting rate, types of deficiencies, approval rate, and actual review time of PRD Priority Review Voucher Program against standard review for NDA.
Results
In Taiwan, according to “Rare Disease and Orphan Drug Act,” the term “orphan drugs” must be utilized in genetic rare diseases. Therefore, TFDA introduced a new pathway known as PRD Priority Review Voucher Program on November 18, 2019. This aims to expedite the assessment of novel drugs addressing unmet medical needs, particularly for serious pediatric conditions or critical diseases with a prevalence of fewer than five cases per ten thousand individuals over past three years in Taiwan.
Benefits include obtaining a TFDA decision within 240 days, a one-third reduction compared to the standard 360-day review for NDAs, and an exemption from submitting BSE applications in advance, with ethnic differences evaluated during the NDA review. Priority review vouchers are awarded for novel drugs targeting pediatric diseases with confirmed clinical trials in Taiwan.
From 2020 to 2023, 49 drugs sought PRD designation (18, 9, 11, and 11 cases in respective years). Analysis reveals:
(1) 90% focused on rare diseases, 6% on pediatric diseases, and 4% on both, with only 10% specifically for pediatric diseases.
(2) Treatment domains for rare diseases included oncology, hemophilia, resistant myasthenia gravis, pustular psoriasis, multidrug-resistant HIV, and limited representation for neuroblastoma and neurofibromatosis in pediatric diseases.
(3) PRD designation granting rate was 76%, with 77% for rare diseases and 60% for pediatric diseases.
(4) Common deficiencies included presenting incidence instead of prevalence, failure to demonstrate domestic prevalence over past three years, and insufficient clinical advancements.
(5) NDAs with PRD designation reached a 100% approval rate, whereas an 85% overall approval rate for standard review. More importantly, NDAs with the PRD designation had an average review time of 218 days compared to 325 days for standard review, emphasizing earlier patient access. Lastly, TFDA has not issued any priority review vouchers to date.
Conclusion
Authorities worldwide are dedicated to motivating the availability of new medications, and this becomes particularly challenging in regions with limited market size such as Taiwan. To tackle such difficulties, TFDA launched the PRD Priority Review Voucher Program in late 2019 to optimize the drug evaluation process, aiming to hasten innovative drug approvals for special populations, particularly in pediatric or rare disease domains. Our analysis revealed that a majority of the novel drugs under this program were targeted toward rare diseases, especially in oncology. Furthermore, these designated NDAs underwent a significantly shorter review time and achieved a higher approval rate compared to the standard review. Despite the program’s limited focus on pediatric conditions, its capacity to expedite reviews and enhance patient accessibility highlights its promising role in addressing unmet medical needs within Taiwan's healthcare landscape. Adopting additional incentives in the future to attract novel pediatric drugs to enter Taiwan may be crucial.
