School of Business Administration, Shenyang Pharmaceutical University, Shenyang, China.
International Research Center for Medical Administration, Peking University, Beijing, China.
BMJ Open. 2023 Oct 18;13(10):e064811. doi: 10.1136/bmjopen-2022-064811.
To evaluate the impacts of the 2017 adjustment of National Reimbursement Drug List (NRDL) on orphan drugs hospital procurement volumes and spending in China.
We used an interrupted time series design covering the period from 2016 to 2018 to analyse changes in hospital procurement volumes and spending of orphan drugs for which were included in the 2017 NRDL.
The study was conducted in China. Orphan drug procurement data of 789 public hospitals (594 tertiary hospitals and 195 secondary hospitals) were derived from the Chinese Medical Economic Information (CMEI).
Monthly orphan drugs hospital procurement volumes and spending.
Nine orphan drugs were included in the 2017 NRDL (seven were directly included, and two were included after price negotiation). Comparing to orphan drugs not included in the NRDL, hospital procurement volumes ([Formula: see text] =43 312, p<0.001) and spending ([Formula: see text] =6 48 927, p<0.001) of the nine included drugs showed significant upward trends after implementation of the 2017 NRDL adjustment.
Our results suggest that the 2017 adjustment of NRDL significantly changed the usage and spending on certain orphan drugs. The increase in orphan drug hospital procurement volumes should improve rare disease patients' access to these orphan drugs.
评估 2017 年国家医保药品目录调整对中国孤儿药医院采购量和支出的影响。
我们采用了一个从 2016 年到 2018 年的中断时间序列设计，分析了被纳入 2017 年国家医保药品目录的孤儿药的医院采购量和支出的变化。
本研究在中国进行。孤儿药采购数据来自中国医疗经济信息（CMEI），涵盖了 789 家公立医院（594 家三级医院和 195 家二级医院）。
每月孤儿药医院采购量和支出。
有 9 种孤儿药被纳入 2017 年国家医保药品目录（7 种是直接纳入的，2 种是经过价格谈判后纳入的）。与未纳入国家医保药品目录的孤儿药相比，这 9 种纳入药物的医院采购量（[公式：见文本] =43312，p<0.001）和支出（[公式：见文本] =648927，p<0.001）在实施 2017 年国家医保药品目录调整后呈现出显著的上升趋势。
我们的结果表明，2017 年国家医保药品目录的调整显著改变了某些孤儿药的使用和支出。孤儿药医院采购量的增加应该会提高罕见病患者对这些孤儿药的可及性。
