Barriers and Countermeasures to the Accessibility of Orphan Drugs from a Pharmacoeconomic Perspective

Jian-Ling Tan ( Yichang Central People's Hospital, Yichang, Hubei, 444300, China;Institute of Pharmaceutical Preparations, China Three Gorges University, Yichang, Hubei, 444300, China )

https://doi.org/10.37155/3060-8708-0203-5

Abstract

This paper focuses on the pharmacoeconomic barriers affecting the accessibility of orphan drugs and proposes corresponding countermeasures. It first outlines the fundamental framework and core concepts of pharmacoeconomic evaluation, as well as the unique characteristics involved in assessing orphan drugs. It then analyzes the key economic barriers arising during research and development, production, market access, reimbursement, distribution, and clinical use. Finally, it puts forward strategies such as improving multi-level systems ensuring drug accessibility, innovating payment models, optimizing policies for research, development, and regulatory approval, and strengthening pharmaceutical management and data sharing. The objective is to enhance the accessibility of orphan drugs and provide theoretical support for relevant policy-making.

Keywords

Orphan drugs; Pharmacoeconomics; Accessibility barriers; Countermeasure strategies

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References

[1] Cui, N., Liu, K., Dong, Q., et al. Review and Implementation Effectiveness of Rare Disease Drug Policies in China. Chinese Journal of New Drugs, 2025, 34(19): 2027–2035.
[2] Cao, Y., Liu, X., Wang, S., et al. Current Status and Prospects of Digital Intelligence Technology in Rare Disease Drug Surveillance. Rare Disease Research, 2025, 4(1): 22–29.
[3] Chen, R., Zhang, S. Trends in Orphan Drug R&D. Rare Disease Research, 2024, 3(2): 221–223.
[4] Cui, H., Tang, L., Cui, C., et al. Considerations for Applying Decentralized Clinical Trials in Orphan Drug Clinical Development. Rare Disease Research, 2024, 3(2): 175–180.

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