Abstract：
BACKGROUND: After the inclusion of more high-cost orphan drugs in China\'s National Reimbursement Drugs List, this study investigated issues relating to patient access to the 7 medicines for 4 rare diseases after listing.
METHODS: This study collected data from a national survey conducted in China. Three aspects associated with the accessibility of medicines, namely, approachability, availability, and affordability, were analyzed using descriptive statistics. In addition, multilevel logistic regression models were used to investigate the associations between patient characteristics and the accessibility of surveyed orphan drugs.
RESULTS: Of the 999 completed responses included in the study, 15% of the patients (n = 150) did not use the medicines because of non-medicine-related issues. Among the 849 patients using the surveyed medications, 64.4% (n = 547) encountered the problem of unavailability, whereas 51.2% (n = 435) reported affordability as an issue, and 49.6% (n = 320) had health expenditure beyond the catastrophic threshold. The data also indicated that Commercial Medical Insurance helped patients to relieve the cost burden on orphan drugs, but the payout of Commercial Medical Insurance failed to influence patients\' decisions to continue the treatments.
CONCLUSIONS: Accessibility of orphan drugs has improved in China after their inclusion in the National Reimbursement Drugs List. Nevertheless, the availability and affordability of medicines remained the barriers for patients to access the desired treatments. It is recommended that further policy refinement in conjunction with the collaboration among healthcare stakeholders is required to deliver better care for patients with rare disease.
METHODS: This study collected data from a national survey conducted in China. Three aspects associated with the accessibility of medicines, namely, approachability, availability, and affordability, were analyzed using descriptive statistics. In addition, multilevel logistic regression models were used to investigate the associations between patient characteristics and the accessibility of surveyed orphan drugs.
RESULTS: Of the 999 completed responses included in the study, 15% of the patients (n = 150) did not use the medicines because of non-medicine-related issues. Among the 849 patients using the surveyed medications, 64.4% (n = 547) encountered the problem of unavailability, whereas 51.2% (n = 435) reported affordability as an issue, and 49.6% (n = 320) had health expenditure beyond the catastrophic threshold. The data also indicated that Commercial Medical Insurance helped patients to relieve the cost burden on orphan drugs, but the payout of Commercial Medical Insurance failed to influence patients\' decisions to continue the treatments.
CONCLUSIONS: Accessibility of orphan drugs has improved in China after their inclusion in the National Reimbursement Drugs List. Nevertheless, the availability and affordability of medicines remained the barriers for patients to access the desired treatments. It is recommended that further policy refinement in conjunction with the collaboration among healthcare stakeholders is required to deliver better care for patients with rare disease.
摘要：
背景：在中国国家报销药品目录中纳入更多的高成本孤儿药之后，这项研究调查了4种罕见疾病上市后患者获得7种药物的相关问题。
方法：本研究收集了在中国进行的一项全国性调查的数据。与药物可获得性相关的三个方面，即,可接近性，可用性,和负担能力，使用描述性统计分析。此外,采用多水平logistic回归模型研究了患者特征与接受调查的孤儿药可及性之间的关系.
结果：在研究中包含的999个已完成的响应中，15％的患者（n=150）因非药物相关问题而未使用药物。在使用接受调查的药物的849名患者中，64.4%(n=547)遇到了不可用的问题，而51.2%(n=435)的人报告说负担能力是一个问题，49.6%(n=320)的医疗支出超过灾难性阈值。数据亦显示,商业医疗保险有助病人减轻孤儿药的费用负担,但商业医疗保险的支付未能影响患者继续治疗的决定。
结论：孤儿药被列入国家报销药品清单后，中国的可及性有所改善。然而，药物的可获得性和可负担性仍然是患者获得所需治疗的障碍。建议进一步完善政策，结合医疗保健利益相关者之间的合作，为罕见疾病患者提供更好的护理。
方法：本研究收集了在中国进行的一项全国性调查的数据。与药物可获得性相关的三个方面，即,可接近性，可用性,和负担能力，使用描述性统计分析。此外,采用多水平logistic回归模型研究了患者特征与接受调查的孤儿药可及性之间的关系.
结果：在研究中包含的999个已完成的响应中，15％的患者（n=150）因非药物相关问题而未使用药物。在使用接受调查的药物的849名患者中，64.4%(n=547)遇到了不可用的问题，而51.2%(n=435)的人报告说负担能力是一个问题，49.6%(n=320)的医疗支出超过灾难性阈值。数据亦显示,商业医疗保险有助病人减轻孤儿药的费用负担,但商业医疗保险的支付未能影响患者继续治疗的决定。
结论：孤儿药被列入国家报销药品清单后，中国的可及性有所改善。然而，药物的可获得性和可负担性仍然是患者获得所需治疗的障碍。建议进一步完善政策，结合医疗保健利益相关者之间的合作，为罕见疾病患者提供更好的护理。
