Long way to go: Progress of orphan drug accessibility in China from 2017 to 2022

Liu, Jia; Yu, Yue; Zhong, Ming; Ma, Chao; Shao, Rong

doi:10.3389/fphar.2023.1138996

Front. Pharmacol.

2023

DOI: 10.3389/fphar.2023.1138996

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Long way to go: Progress of orphan drug accessibility in China from 2017 to 2022

Jia Liu

Yue Yu²,

Ming Zhong³

et al.

Abstract: Introduction: Over 400 million patients worldwide suffer from rare diseases. Access to orphan drugs is, therefore, crucial for this population. China has been actively working on improving orphan drug accessibility in the past decades, especially since 2018 when the First National List of Rare Diseases was announced. This study aimed to evaluate the current status of orphan drug accessibility in China regarding availability, daily cost, and affordability.Methods: Market availability of orphan drugs in China wa… Show more

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2024

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Cited by 3 publications

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Measurement properties of the EQ-5D-3L, EQ-5D-5L, and SF-6Dv2 in patients with late-onset Pompe disease

Xu,

Luo,

Dong

2024

Eur J Health Econ

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Objective The objective of this study was to evaluate the psychometric properties of the EQ-5D (3L and 5L) and SF-6Dv2 in a group of Chinese patients with late-on Pompe disease (PD), and compare their performance in this patient group. Methods The data used in this study were obtained from a web-based and cross-sectional survey conducted in China. All participants completed the 3L, 5L, and SF-6Dv2. Information about their sociodemographic status and health conditions was also collected. The measurement properties were assessed by examining ceiling and floor effects, evaluating convergent validity, known-group validity, and test–retest reliability (Intraclass correlation coefficient [ICC] and Gwet’s AC). Results A total of 117 PD patients completed the questionnaire. All dimensions of the 3L showed strong ceiling effects, ranging between 17.1 and 42.7%. All three measures showed good test–retest reliability, with ICC values ranging from 0.85 to 0.87. The Gwet’s AC values showed that four out of five dimensions of the 3L showed very good agreement. All hypothesized correlations between the 3L, 5L, SF-6Dv2, and items of WHODAS were supported, indicating satisfactory convergent validity. The 5L showed stronger correlations (|r|= 0.53–0.84) with WHODAS than the other two measures. The outcomes of ANOVA indicated that the 5L had higher F-statistics than the 3L and SF-6Dv2, indicating a stronger discriminant ability to differentiate most condition groups. Conclusion The 5L demonstrates lower ceiling and floor effects, higher discriminant ability, and better convergent validity than the SF-6Dv2 and 3L in patients with PD. In addition, the 5L may generate a larger utility gain compared to the other two instruments when conducting cost-effectiveness analysis.

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Measurement properties of the EQ-5D-3L, EQ-5D-5L, and SF-6Dv2 in patients with late-onset Pompe disease

Xu,

Luo,

Dong

2024

Eur J Health Econ

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A call to rethink the necessity of and challenges facing academic research organizations in the new era of drug innovation in China

Huang,

Jiang,

Chen

et al. 2024

Drug Discovery Today

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Utilization and affordability of health insurance coverage for rare disease drugs in a first-tier city in Northeast China from 2018 to 2021: a study based on the health insurance claims database

Wang,

Zhou,

et al. 2024

Int J Equity Health

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Objective The accessibility issue of orphan drugs in China is prominent. Based on real-world data from a tier-one city in Northeast China, this study aims to analyze the current usage and affordability of orphan drugs for rare diseases. Methods The data was sourced from the health insurance claims data of a certain city from 2018 to 2021, including a total of 16 orphan drugs. The utilization of orphan drugs is assessed using four indicators: frequency of medical insurance claims, medication cost, defined daily doses (DDDs), and defined daily drug cost (DDDc). Affordability is measured using the concept of catastrophic health expenditure (CHE). Results Between January 2018 and December 2021, there were a total of 2,851 medical insurance claims in the city, with a total medication costs of $3.08 million. Overall, during the study, there was a year-on-year increase in the utilization frequency of individual rare disease drugs in the city, with DDDs rising from 140.22 in 2018 to 3983.63 in 2021. Additionally, the annual medication costs of individual drugs showed a consistent upward trend, increasing from $10,953.53 in 2018 to $120,491.36 in 2021. However, the DDDc of individual drugs decreased from $398.12 in 2018 to $96.65 in 2021.The number of sales and the amount of sales for orphan drugs in community pharmacies have significantly increased. Prior to medical insurance coverage, out of the 16 orphan drugs, 9 drugs had annual treatment costs exceeding CHE for urban residents, and 15 drugs had annual treatment costs exceeding CHE for rural residents. After medical insurance coverage, there were no drugs with out-of-pocket costs exceeding CHE for urban residents, while 8 drugs had out-of-pocket costs exceeding CHE for rural residents. Furthermore, both before and after medical insurance coverage, the four treatment drugs for idiopathic pulmonary arterial hypertension were more affordable compared to the four treatment drugs for multiple sclerosis. Conclusion The usage frequency of orphan drugs in a certain city increased gradually, but the disease burden remained heavy. More policy support should be provided to the priority rare disease populations, and the rare disease medical security and diagnosis and treatment systems should be improved.

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Long way to go: Progress of orphan drug accessibility in China from 2017 to 2022

Cited by 3 publications

References 44 publications

Measurement properties of the EQ-5D-3L, EQ-5D-5L, and SF-6Dv2 in patients with late-onset Pompe disease

Measurement properties of the EQ-5D-3L, EQ-5D-5L, and SF-6Dv2 in patients with late-onset Pompe disease

A call to rethink the necessity of and challenges facing academic research organizations in the new era of drug innovation in China

Utilization and affordability of health insurance coverage for rare disease drugs in a first-tier city in Northeast China from 2018 to 2021: a study based on the health insurance claims database

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