Evolution of drug regulations and regulatory innovation for anticancer drugs in China

Liu, Yang; Zhang, Ning; Xie, Cuicui; Jiang, Yale; Qin, Yunhe; Zhou, Liyun; Yi, Fan; Ren, Lianjie; Chen, Yin; Yang, Huan; Xie, W.; Zhai, Qixiao; Li, Guanqiao; Chen, Hongzhuan; Chen, Xiaoyuan

doi:10.1016/j.apsb.2022.08.004

Cited by 13 publications

(7 citation statements)

References 32 publications

(47 reference statements)

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“…Additionally, there are some emerging institutions in China that lack sufficient experience and sound quality management systems. Regulations and measures have been taken by both Chinese health authority and emerging institutions to improve their quality systems [ 9 ].…”

Section: Figurementioning

confidence: 99%

Unveiling quality of clinical trial in China: from concern to confirmation

Huang,

Hou,

Fang

et al. 2024

Cancer Communications

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Section: Figurementioning

confidence: 99%

Unveiling quality of clinical trial in China: from concern to confirmation

Huang,

Hou,

Fang

et al. 2024

Cancer Communications

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“…Driven by continued innovation, worldwide spending on anticancer drugs reached $185 billion in 2021 and is projected to exceed $300 billion by 2026 [3]. China, being the largest developing country, has optimized its regulatory policies for innovative drugs, leading to the launch of numerous anticancer drugs in recent years [4][5][6]. Consequently, expenditure on anticancer drugs in China increased from $4.8 billion in 2016 to $13 billion in 2021, marking an increase of $8.2 billion [3].…”

Section: Introductionmentioning

confidence: 99%

Price negotiation and pricing of anticancer drugs in China: An observational study

Zhou,

Lan,

et al. 2024

PLoS Med

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Background 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. Methods and findings 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. Conclusions 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.

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“…Most importantly, domestic standalone clinical trials in China are also no longer a prerequisite when applying for new drug market authorization by NMPA: they can be replaced by MRTs enrolling Chinese patients, or be exempted for drugs treating rare diseases ( Bajaj et al, 2019 ). After China joined The International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) in 2017, the acceptance of overseas clinical data in drug approval decisions has been further improved ( Liu et al, 2022 ). The existing literature has measured the absolute and relative drug lag in China ( Zhou et al, 2019 ; Liu et al, 2022 ; Luo et al, 2023 ), but only focuses on oncology drugs, leaving other diseases understudied.…”

Section: Introductionmentioning

confidence: 99%

“…After China joined The International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) in 2017, the acceptance of overseas clinical data in drug approval decisions has been further improved ( Liu et al, 2022 ). The existing literature has measured the absolute and relative drug lag in China ( Zhou et al, 2019 ; Liu et al, 2022 ; Luo et al, 2023 ), but only focuses on oncology drugs, leaving other diseases understudied. Moreover, whether the recent reform efforts, particularly the relaxation of clinical research requirements, influence the drug lag in China likewise awaits answers.…”

Section: Introductionmentioning

confidence: 99%

Bridging the new drug access gap between China and the United States and its related policies

Zhu,

Chen

2024

Front. Pharmacol.

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Introduction: The access gap for novel pharmaceuticals between China and the developed countries is a major public health issue in China. It is crucial to understand the determinants of this gap to ensure timely access to new drugs and enhance patient health.Methods: We included all new drugs approved by the US Food and Drug Administration (FDA) between 2012 and 2019, and collected their approval timings in China. Major factors of interest comprised orphan designation and expedited review pathways granted by the FDA, along with the proportion of Asian subjects in the pivotal trial supporting the FDA approval and whether the trial included study sites in China. The elapsed time from the FDA approval to the market authorization in China constituted the time-to-event outcome, and Cox proportional-hazards regression was used for multivariate analysis.Results: A total of 327 new drugs were approved by the FDA between 2012 and 2019, among which 41.3% were found to be authorized in China as of 1 November 2023. The median lag time for the mutually approved drugs was 3.5 years. The Cox model found that orphan drugs had lower likelihood of being approved in China (HR = 0.59, 95% CI 0.39–0.89; p = 0.011), while the FDA’s Breakthrough-Therapy drugs (HR = 2.33, 95% CI 1.39–3.89; p = 0.001) and Fast-Track drugs (HR = 1.58, 95% CI 1.05–2.38; p = 0.028) had shorter lag times. In the pivotal trials that supported the FDA approvals, a higher proportion of Asian subjects was associated with faster drug entry into the Chinese market (HR = 1.02, 95% CI 1.01–1.03; p < 0.001), and the inclusion of study sites in China mainland was likewise conducive to reducing the drug lag (HR = 5.30, 95% CI 3.20–8.77; p < 0.001). After the trials with China-based sites supported the FDA approvals, 77.8% of the trials also supported the subsequent approvals in China.Discussion: China’s involvement in global drug co-development can streamline clinical development, by reducing repeated trials solely in the Chinese population. This is primarily due to the openness of the Chinese drug agency towards overseas clinical data and is a positive sign that encourages global drug developers to include Chinese patients in their development plans as early as possible.

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Evolution of drug regulations and regulatory innovation for anticancer drugs in China

Cited by 13 publications

References 32 publications

Unveiling quality of clinical trial in China: from concern to confirmation

Unveiling quality of clinical trial in China: from concern to confirmation

Price negotiation and pricing of anticancer drugs in China: An observational study

Bridging the new drug access gap between China and the United States and its related policies

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