现在的位置: 首页时讯速递, 进展交流>正文
[JAMA Netw Open发表论文]:2020年至2024年中国认定突破性疗法肿瘤药物的临床获益、价格与监管批准
2024年12月23日 时讯速递, 进展交流 [JAMA Netw Open发表论文]:2020年至2024年中国认定突破性疗法肿瘤药物的临床获益、价格与监管批准已关闭评论

Original Investigation 

Health Policy

October 16, 2024

Clinical Benefit, Price, and Regulatory Approval of Cancer Drugs Granted Breakthrough Therapy Designation in China, 2020-2024

Xingxian Luo, Xin Du, Xufeng Lv, et al

JAMA Netw Open. 2024;7(10):e2439080. doi:10.1001/jamanetworkopen.2024.39080

Key Points

Question  What are the differences in clinical benefit, clinical development time, novelty, and price of cancer drugs for breakthrough therapy designation (BTD) vs non-BTD cancer drugs in China?

Findings  In a cross-sectional analysis of 18 BTD drugs and 32 non-BTD drugs, BTD drugs had significantly shorter clinical development times and higher novelty. There was no significant difference in efficacy, safety, and change in therapeutic prices.

Meaning  These results suggest that the implementation of the BTD policy in China has expedited the availability of medicines for patients with cancer and is conducive to encouraging innovation in drug development with comparable clinical benefits; however, further efforts are still needed to improve the affordability of BTD drugs.

Abstract

Importance  The China National Drug Administration (NMPA) established the breakthrough therapy designation (BTD) in 2020 to encourage the accelerated development of drugs for the prevention and treatment of diseases that are life-threatening. However, the differences between BTD and non-BTD cancer drugs regarding clinical benefit, regulatory approval, and price are unclear.

Objectives  To compare BTD and non-BTD cancer drugs in clinical benefit (defined as efficacy and safety), novelty, time to approval, and average monthly treatment price.

Design, Setting, and Participants  This cross-sectional study analyzes the original indication of BTD and non-BTD novel cancer drugs approved by the NMPA between July 8, 2020, and July 8, 2024. Data on efficacy, safety, regulatory approval, and price of cancer drugs were extracted from pivotal clinical trials based on review reports published by the NMPA, peer-reviewed articles or meeting reports, and winning bid prices for cancer drugs in the Chinese provincial-level centralized procurement process.

Main Outcomes and Measures  The main outcome was the efficacy and safety associated with BTD vs non-BTD cancer drugs, including progression-free survival (PFS), response rate (RR), duration of response, serious adverse events, grade 3 or higher adverse events, and treatment-related deaths. In addition, the time to approval, novelty, and initial and latest average monthly treatment prices were evaluated, as well as the average annual reduction rate (AARR; the sum of the reduction rates divided by the number of years for the monthly treatment price) for these cancer drugs.

Results  Between July 2020 and July 2024, 18 BTD (36%) and 32 non-BTD (64%) cancer drugs were approved by the NMPA. The median (IQR) clinical development time for BTD drugs was significantly shorter than for non-BTD drugs (5.6 [95% CI, 4.3-7.3] vs 6.6 [95% CI, 6.0-8.5] years; P = .02). No significant differences were observed in PFS (HR, 0.44 [95% CI, 0.38-0.52] vs HR, 0.51 [95% CI, 0.40-0.65]; P = .20), PFS gained (median [IQR], 5.4 [3.9-7.0] vs 2.7 [2.6-5.9] months; P = .77), RR (58% [95% CI, 45%-74%] vs 59% [95% CI, 51%-69%]; P = .85), and duration of response (median [IQR], 18.0 [15.0-21.6] vs 11.1 [7.4-17.4] months; P = .09) between BTD and non-BTD drugs. The rates of serious adverse events (37% [95% CI, 26%-52%] vs 32% [95% CI, 27%-36%]; P = .45), adverse events grade 3 or higher (64% [95% CI, 53%-77%] vs 55% [95% CI, 45%-68%]; P = .31), and treatment-related deaths (2% [95% CI, 1%-4%] vs 1% [95% CI, 1%-2%]; P = .10) were similar between BTD and non-BTD drugs. BTD drugs are more likely to be first-in-class drugs (5 of 18 [28%] vs 1 of 32 [3%]; P = .02). Differences in the median (IQR) initial ($5665 [$3542-$9321] vs $3361 [$2604-$5474]; P = .06) and latest ($5665 [$1553-$9321] vs $2145 [$1318-$4276]; P = .18) average monthly treatment prices for BTD drugs and non-BTD drugs were not significant. The median (IQR) AARRs for BTD drugs and non-BTD drugs were 15.2% (0%-46.9%) and 19.8% (1.0%-42.9%), respectively.

Conclusions and Relevance  The findings of this cross-sectional study suggest that BTD has facilitated faster time to market for cancer drugs and improved novelty, but the price of treatment is relatively higher. There was no significant difference on comparative efficacy and safety.

抱歉!评论已关闭.

×
腾讯微博