HEOA Group, West China School of Public Health and West China Fourth Hospital, Sichuan University, Chengdu, China.
Institute for Healthy Cities and West China Research Center for Rural Health Development, Sichuan University, Chengdu, China.
PLoS Med. 2024 Jan 2;21(1):e1004332. doi: 10.1371/journal.pmed.1004332. eCollection 2024 Jan.
While China has implemented reimbursement-linked drug price negotiation annually since 2017, emphasizing value-based pricing to achieve a value-based strategic purchase of medical insurance, whether drug prices became better aligned with clinical value after price negotiation has not been sufficiently established. This study aimed to assess the changes in prices and their relationship with the clinical value of anticancer drugs after the implementation of price negotiations in China.
In this observational study, anticancer drug indications that were negotiated successfully between 2017 and 2022 were identified through National Reimbursement Drug Lists (NRDL) of China. We excluded extensions of indications for drugs already listed in the NRDL, indications for pediatric use, and indications lacking corresponding clinical trials. We identified pivotal clinical trials for included indications by consulting review reports or drug labels issued by the Center for Drug Evaluation, National Medical Products Administration. We calculated treatment costs as outcome measures based on publicly available prices and collected data on clinical value including safety, survival, quality of life, and overall response rate (ORR) from publications of pivotal clinical trials. The associations between drug costs and clinical value, both before and after negotiation, were analyzed using regression analyses. We also examined whether price negotiation has led to a reduction in the variation of treatment costs for a given value. We included 103 anticancer drug indications, primarily for the treatment of blood cancer, lung cancer, and breast cancer, with 76 supported by randomized controlled trials and 27 supported by single-arm clinical trials. The median treatment costs over the entire sample have been reduced from US$34,460.72 (interquartile range (IQR): 19,990.49 to 55,441.66) to US$13,688.79 (IQR: 7,746.97 to 21,750.97) after price negotiation (P < 0.001). Before price negotiation, each additional month of survival gained was associated with an increase in treatment costs of 3.4% (95% confidence interval (CI) [2.1, 4.8], P < 0.001) for indications supported by randomized controlled trials, and a 10% increase in ORR was associated with a 6.0% (95% CI [1.6, 10.3], P = 0.009) increase in treatment costs for indications supported by single-arm clinical trials. After price negotiation, the associations between costs and clinical value may not have changed significantly, but the variation of drug costs for a given value was reduced. Study limitations include the lack of transparency in official data, missing data on clinical value, and a limited sample size.
In this study, we found that the implementation of price negotiation in China has led to drug pricing better aligned with clinical value for anticancer drugs even after substantial price reductions. The achievements made in China could shed light on the price regulation in other countries, particularly those with limited resources and increasing drug expenditures.
自 2017 年以来，中国每年都进行与报销挂钩的药品价格谈判，强调基于价值的定价，以实现医疗保险的基于价值的战略性购买，但药品价格在谈判后是否与临床价值更加一致尚未得到充分证实。本研究旨在评估中国药品价格谈判实施后，药品价格的变化及其与抗癌药物临床价值的关系。
在这项观察性研究中，我们通过中国国家医保药品目录（NRDL）确定了 2017 年至 2022 年成功谈判的抗癌药物适应证。我们排除了已在 NRDL 中列出的药物适应证扩展、儿科适应证和缺乏相应临床试验的适应证。我们通过查阅药品评估中心、国家药品监督管理局发布的审评报告或药品标签，为纳入的适应证确定了关键性临床试验。我们根据公开价格计算治疗费用作为结果指标，并从关键性临床试验的出版物中收集安全性、生存、生活质量和总缓解率（ORR）等临床价值数据。我们使用回归分析评估了谈判前后药物成本与临床价值之间的关系。我们还研究了价格谈判是否导致特定价值范围内治疗成本的变化减少。我们纳入了 103 种抗癌药物适应证，主要用于治疗血液癌、肺癌和乳腺癌，其中 76 种适应证有随机对照试验支持，27 种适应证有单臂临床试验支持。整个样本的中位治疗成本从谈判前的 34460.72 美元（四分位距（IQR）：19990.49 至 55441.66）降至谈判后的 13688.79 美元（IQR：7746.97 至 21750.97）（P < 0.001）。在价格谈判之前，每增加一个月的生存时间，与随机对照试验支持的适应证的治疗成本增加 3.4%（95%置信区间（CI）[2.1，4.8]，P < 0.001）相关，与单臂临床试验支持的适应证的 ORR 增加 10%相关，治疗成本增加 6.0%（95% CI [1.6，10.3]，P = 0.009）。在价格谈判之后，成本与临床价值之间的关系可能没有显著变化，但特定价值范围内的药物成本变化减少了。研究局限性包括官方数据缺乏透明度、临床价值数据缺失以及样本量有限。
本研究发现，中国实施的价格谈判使抗癌药物的定价与临床价值更加一致，即使在大幅降价后也是如此。中国在这方面取得的成就为其他国家，特别是资源有限和药品支出不断增加的国家的价格监管提供了启示。
