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Insights into Support Systems for Orphan Drug Development: A Comparative Study
Ye Leng1, Yu Tang2, Weijie Yu2
1School of Basic Medicine and Clinical Pharmacy, China Pharmaceutical University, Nanjing, 211198, China.
Therapeutic Innovation & Regulatory Science
|July 14, 2025
Summary
Developing orphan drugs for rare diseases (RDs) requires harmonized support systems. This study proposes a framework and finds the US, EU, and China have basic conditions, with China needing improvements in incentives and infrastructure.
Area of Science:
- Health Policy and Orphan Drug Development
- Rare Diseases Research and Regulatory Science
Background:
- Orphan drug development addresses significant unmet medical needs for rare diseases (RDs).
- A harmonized support system is crucial for advancing orphan drug development globally.
Purpose of the Study:
- To propose a framework and dimensions of support systems influencing orphan drug development.
- To assess the current state of orphan drug development conditions in the US, EU, and China.
- To suggest action plans for strengthening global orphan drug development initiatives.
Main Methods:
- Development of a support system framework encompassing legal, technical, motivational, and logistical factors.
- Comparative analysis of orphan drug development conditions across the US, EU, and China based on the framework.
- Formulation of strategic action plans for improving orphan drug development.
Main Results:
- The US, EU, and China have established foundational conditions for orphan drug development.
- China demonstrates deficiencies in economic incentives and fundamental infrastructure compared to the US and EU.
- Specific areas for improvement in China include pricing, reimbursement, and real-world data (RWD) integration.
Conclusions:
- A comprehensive framework is essential for evaluating and enhancing orphan drug development support systems.
- Targeted improvements, particularly in China, are needed to meet the needs of rare disease patients.
- The study provides a reference for national and global strategies to advance orphan drug development worldwide.
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