Determinants of Orphan Drug Health Technology Assessment in South Korea: An Empirical Analysis

Se Hee Lee¹Sungkyung Lee²Jong Hyuk Lee^3*Joon Seok Bang^1*

• ¹College of Pharmacy, Sookmyung Women's University, Seoul, Republic of Korea
• ²Korea Institute for Industrial Economics and Trade, Seoul, Republic of Korea
• ³College of Pharmacy, Chung-Ang University, Seoul, Republic of Korea

Background: Orphan drug pricing in South Korea, including its workings and determinants, is a complicated aspect of the country's health care system. Therefore, In this study identified the , we attempt to sort out factors associated with the pricings of orphan drugs in South Korea., through which we hope to render a clear picture on the workings and determinants of orphan drug health technology assessment in South Korea. Methods: The studyWe screened a total of 66 orphan drugs out of the whole set of newly reimbursed drugs during the period offrom 2012 toand 2022. Descriptive The analyses presented the characteristics of the orphan drugs. aAnd we look intoaccounted correlations between treatment cost and other seemingly related variables. , and then we conduct fFormal multivariate regression analyses to pointed out associated factors in multivariate settings. Results: Annual treatment cost per patient displayeds a weakly negative correlation with the number of patients variable. Meanwhile, treatment cost exhibiteds a strongly positive correlation with the average of adjusted prices in seven7 advanced countries, ' prices, reaching 0.99 in an adjusted R-squared value of 0.99. ,Treatment cost also showed and a positive correlation with budget impact. Our findings reveal that the pricing of orphan drugs in South Korea is predominantly influenced by external reference pricing and suggest that drugs targeting smaller patient populations tend to be priced higher. Conclusion: The pricing of orphan drugs in South Korea is predominantly influenced by external reference pricing. Drugs targeting smaller patient populations tend to be priced higher. The study delineates which factors have actually shaped the orphan drug health technology assessment the most, highlighting its interplay with external reference prices, drug characteristics, and Price & Reimbursement pathways. These insights finding can guide policy makers, pharmaceutical companies, and healthcare providers in their efforts to balance patient accessibility with the sustainable development of orphan drug and the financial sustainability of public health insurance. development.