Effect of the copayment reduction system on accessibility to orphan drugs in South Korea

ABSTRACT

Objective

This study aims to analyze the effect of the copayment reduction system on accessibility to orphan drugs (ODs) in South Korea.

Methods

Data on approval and reimbursement for drugs designated as ODs for the last 10 years (2012–2021) in South Korea were extracted. Among them, with 136 approved products as of 31 December 2022, the reimbursement rates and lead time to reimbursement between drugs for rare diseases (DRDs) and nondrugs for rare diseases (non-DRDs) were analyzed. The pricing and reimbursement (P&R) pathways between drugs for only rare diseases (DORDs) and drugs for rare and cancerous diseases (DRCDs) were compared.

Results

The reimbursement rates for DRDs and non-DRDs were 54.8% and 33.3%, respectively, and the lead time to reimbursement for DRDs and non-DRDs were 16.1 months and 31.2 months, respectively. The P&R pathways for DORDs and DRCDs were pharmacoeconomic evaluation waivers (21.7% and 52.6%), weighted average price (52.2% and 13.2%), and risk-sharing agreement (30.4% and 81.6%).

Conclusion

The copayment reduction system may act as a driver and also barrier for the reimbursement of ODs. To expand treatment accessibility to ODs, it is necessary to consistently grants benefits in all processes from OD designation to market access.

KEYWORDS:

• accessibility
• copayment reduction system
• drugs for rare diseases
• orphan drugs
• pricing and reimbursement
• rare diseases

Acknowledgement

This research was supported by the Chung-Ang University Research Scholarship Grants in 2023. The authors are grateful to the experts of KRPIA (Korean Research-based Pharma Industry Association) for their support in this study, including (Patient Access Improvement Working Group and Rare Disease Working Group): Yang Hyun Ju, Chae Yu Ri, Oh Eun Young, Lee Hyun Yung, Kim Jin Geol, Kim Eun Suk, Gong Gi Ra, Lee Won Chul, Kim Hyoung Jin, Kim Bo Kyung, Kim Seong Taek, Choi In Hwa, Kim mee gyong, Jeong Jae Hong, Park Eun Ji, Ban Kyung Ah.

Abbreviations

RD: Rare Diseases; R&D: Research & Development; ODs: Orphan Drugs; HTAs: Health Technology Assessments; P&R: Pricing and Reimbursement; DRDs: Drugs for Rare Diseases; PE: Pharmacoeconomic Evaluation; RSA: Risk-Sharing Agreement; ICER: Incremental Cost-Effectiveness Ratio; WAP: Weighted Average Price; DORDs: Drugs for Only Rare Diseases; DRCDs: Drugs for Rare and Cancerous Diseases; DRSDs: Drugs for Rare and Severe Incurable Diseases; CEA: Cost-Effectiveness Analysis; QALY: Quality-Adjusted Life Year.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Declaration of interest

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Author contributions

SE Hwang contributed to the study design, data collection, statistical analysis, manuscript development and review under the guidance of JH Lee. MY Kim, YJ Hong, DY Lee, TH Kim, JH Park and JK Bae participated in data collection, data analysis, manuscript review and revision. JH Lee supervised the entirety of the study right from study designing, data collection and analysis, and manuscript editing to manuscript submission and revision. All the authors have read and approved the final version of the manuscript to be published.

Data availability statement

The datasets analyzed during the current study are available from the corresponding author on reasonable request.

Additional information

Funding

This paper was not funded.