Economic evaluation methods for anticancer-drugs with basket trial design_Chinese Journal of Evidence-Based Medicine

Authors：

MA Yue ^1,2 , WU Yao ^1,2 , MA Aixia ^1,2 ,  LI Hongchao ^1,2

1. College of International Pharmaceutical Business, China Pharmaceutical University, Nanjing 211198, P. R. China;
2. Center for Pharmacoeconomics and Outcomes Research, China Pharmaceutical University, Nanjing 211198, P. R. China;

Corresponding author：

LI Hongchao, Email: lihongchao@cpu.edu.cn

Keywords：

Basket trial; Anticancer drug; Pharmacoeconomics; Healthcare decision making

DOI：

10.7507/1672-2531.202201113

Video：

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Objective To introduce economic evaluation methods for anticancer-drugs with basket trial design, and to provide references for related research and decision-making. Methods A case analysis was conducted on economic evaluation methods for anticancer-drugs with basket trial design, which was issued by Canadian Agency for Drugs and Technologies in Health (CADTH) in the Economic Guidance Report. Moreover, both the advantages and disadvantages of the methods were analyzed in accordance with the characteristics of basket trials. Results Pooled analysis and tumor-specific analysis were two methods frequently employed in the case analysis. However, great uncertainties were available in both of them. The uncertainty of the former was mainly reflected in the heterogeneity of the targeted population, while the uncertainty of the latter was mainly shown in the insufficient sample size of the subgroup. Conclusion Currently, economic evaluation methods for anticancer-drugs with basket trial design are immature. Thus, researchers are required to explore the methods of innovation evaluation with lower uncertainty; reimbursement decision-makers should fully consider the uncertainty of evaluation results and enterprises should collect the real-world data for the demands of evaluation to promote the reasonable allocation of healthcare resources in China.

Citation： MA Yue, WU Yao, MA Aixia, LI Hongchao. Economic evaluation methods for anticancer-drugs with basket trial design. Chinese Journal of Evidence-Based Medicine, 2022, 22(7): 862-868. doi: 10.7507/1672-2531.202201113 Copy

1.	IARC. Latest global cancer data: Cancer burden rises to 19.3 million new cases and 10.0 million cancer deaths in 2020 questions and answers (Q&A). Available at: https://www.iarc.who.int/faq/latest-global-cancer-data-2020-qa/.
2.	崔积钰, 韩优莉. 基于中美日新药上市现状对比的抗肿瘤新药发展策略研究. 中国卫生政策研究, 2021, 14(8): 52-59.
3.	Degtiar I. A review of international coverage and pricing strategies for personalized medicine and orphan drugs. Health Policy, 2017, 121(12): 1240-1248.
4.	Park JJH, Hsu G, Siden EG, et al. An overview of precision oncology basket and umbrella trials for clinicians. CA Cancer J Clin, 2020, 70(2): 125-137.
5.	Simon R. New designs for basket clinical trials in oncology. J Biopharm Stat, 2018, 28(2): 245-255.
6.	The U.S. National Library of Medicine. Clinical Trials.gov. Available at: https://www.clinicaltrials.gov/ct2/results?cond=&term=basket+trial&cntry=&state=&city=&dist=.
7.	Nosrati M, Nikfar S. Conducting economic evaluation based on basket clinical trial in the area of precision medicine. Expert Rev Pharmacoecon Outcomes Res, 2021, 21(2): 169-171.
8.	Redig AJ, Jänne PA. Basket trials and the evolution of clinical trial design in an era of genomic medicine. J Clin Oncol, 2015, 33(9): 975-977.
9.	Janiaud P, Serghiou S, Ioannidis JPA. New clinical trial designs in the era of precision medicine: an overview of definitions, strengths, weaknesses, and current use in oncology. Cancer Treat Rev, 2019, 73: 20-30.
10.	The U. S. FDA. Master protocols: efficient clinical trial design strategies to expedite development of oncology drugs and biologics guidance for industry. Available at: https://www.fda.gov/.
11.	Renfro LA, Sargent DJ. Statistical controversies in clinical research: basket trials, umbrella trials, and other master protocols: a review and examples. Ann Oncol, 2017, 28(1): 34-43.
12.	Canadian Agency for Drugs and Technologies in Health. Larotrectinib for neurotrophic tyrosine receptor kinase (NTRK) locally advanced or metastatic solid tumors – details. Available at: https://www.cadth.ca/larotrectinib-neurotrophic-tyrosine-receptor-kinase-ntrk-locally-advanced-or-metastatic-solid.
13.	Canadian Agency for Drugs and Technologies in Health. Larotrectinib. Available at: https://www.cadth.ca/larotrectinib.
14.	国家医疗保障局, 国家人力资源社会保障部. 关于印发《国家基本医疗保险, 工伤保险和生育保险药品目录（2021年）》的通知. Available at: http://www.nhsa.gov.cn/art/2021/12/3/art_37_7429.html.
15.	国家医疗保障局. 基本医疗保险用药管理暂行办法. Available at: http://www.nhsa.gov.cn/art/2020/7/31/art_37_3387.html.
16.	Goodman C, Villarivera C, Gregor K, et al. Regulatory, policy, and operational considerations for outcomes-based risk-sharing agreements in the U. S. market: opportunities for reform. J Manag Care Spec Pharm, 2019, 25(11): 1174-1181.
17.	李伟, 施慧, 丁锦希, 等. 英国癌症药物基金的疗效风险分担协议分析. 中国新药杂志, 2021, 30(17): 1559-1564.
18.	Berger ML, Sox H, Willke RJ, et al. Good practices for real-world data studies of treatment and/or comparative effectiveness: Recommendations from the joint ISPOR-ISPE Special Task Force on real-world evidence in health care decision making. Pharmacoepidemiol Drug Saf, 2017, 26(9): 1033-1039.

1. IARC. Latest global cancer data: Cancer burden rises to 19.3 million new cases and 10.0 million cancer deaths in 2020 questions and answers (Q&A). Available at: https://www.iarc.who.int/faq/latest-global-cancer-data-2020-qa/.
2. 崔积钰, 韩优莉. 基于中美日新药上市现状对比的抗肿瘤新药发展策略研究. 中国卫生政策研究, 2021, 14(8): 52-59.
3. Degtiar I. A review of international coverage and pricing strategies for personalized medicine and orphan drugs. Health Policy, 2017, 121(12): 1240-1248.
4. Park JJH, Hsu G, Siden EG, et al. An overview of precision oncology basket and umbrella trials for clinicians. CA Cancer J Clin, 2020, 70(2): 125-137.
5. Simon R. New designs for basket clinical trials in oncology. J Biopharm Stat, 2018, 28(2): 245-255.
6. The U.S. National Library of Medicine. Clinical Trials.gov. Available at: https://www.clinicaltrials.gov/ct2/results?cond=&term=basket+trial&cntry=&state=&city=&dist=.
7. Nosrati M, Nikfar S. Conducting economic evaluation based on basket clinical trial in the area of precision medicine. Expert Rev Pharmacoecon Outcomes Res, 2021, 21(2): 169-171.
8. Redig AJ, Jänne PA. Basket trials and the evolution of clinical trial design in an era of genomic medicine. J Clin Oncol, 2015, 33(9): 975-977.
9. Janiaud P, Serghiou S, Ioannidis JPA. New clinical trial designs in the era of precision medicine: an overview of definitions, strengths, weaknesses, and current use in oncology. Cancer Treat Rev, 2019, 73: 20-30.
10. The U. S. FDA. Master protocols: efficient clinical trial design strategies to expedite development of oncology drugs and biologics guidance for industry. Available at: https://www.fda.gov/.
11. Renfro LA, Sargent DJ. Statistical controversies in clinical research: basket trials, umbrella trials, and other master protocols: a review and examples. Ann Oncol, 2017, 28(1): 34-43.
12. Canadian Agency for Drugs and Technologies in Health. Larotrectinib for neurotrophic tyrosine receptor kinase (NTRK) locally advanced or metastatic solid tumors – details. Available at: https://www.cadth.ca/larotrectinib-neurotrophic-tyrosine-receptor-kinase-ntrk-locally-advanced-or-metastatic-solid.
13. Canadian Agency for Drugs and Technologies in Health. Larotrectinib. Available at: https://www.cadth.ca/larotrectinib.
14. 国家医疗保障局, 国家人力资源社会保障部. 关于印发《国家基本医疗保险, 工伤保险和生育保险药品目录（2021年）》的通知. Available at: http://www.nhsa.gov.cn/art/2021/12/3/art_37_7429.html.
15. 国家医疗保障局. 基本医疗保险用药管理暂行办法. Available at: http://www.nhsa.gov.cn/art/2020/7/31/art_37_3387.html.
16. Goodman C, Villarivera C, Gregor K, et al. Regulatory, policy, and operational considerations for outcomes-based risk-sharing agreements in the U. S. market: opportunities for reform. J Manag Care Spec Pharm, 2019, 25(11): 1174-1181.
17. 李伟, 施慧, 丁锦希, 等. 英国癌症药物基金的疗效风险分担协议分析. 中国新药杂志, 2021, 30(17): 1559-1564.
18. Berger ML, Sox H, Willke RJ, et al. Good practices for real-world data studies of treatment and/or comparative effectiveness: Recommendations from the joint ISPOR-ISPE Special Task Force on real-world evidence in health care decision making. Pharmacoepidemiol Drug Saf, 2017, 26(9): 1033-1039.

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