Policies for use of real-world data in drug reimbursement decision_Chinese Journal of Evidence-Based Medicine

Authors：

HUANG Tao ¹ , LI Can ¹ , HUANG Xianqin ¹ , LI Huangqianyu ² , WU Jing ³ , MAO Zongfu ^4,5 , SHI Luwen ^1,2 ,  GUAN Xiaodong ^1,2

1. Pharmacy Administration and Clinical Pharmacy, Peking University, Beijing 100191, P. R. China;
2. International Research Center for Medicinal Administration, Peking University, Beijing 100191, P. R. China;
3. School of Pharmaceutical Science and Technology, Tianjin University, Tianjin 300072, P. R. China;
4. School of Public Health, Wuhan University, Wuhan 430071, P. R. China;
5. Global Health Institute, Wuhan University, Wuhan 430071, P. R. China;

Corresponding author：

GUAN Xiaodong, Email: guanxiaodong@pku.edu.cn

Keywords：

Real-world; Drug reimbursement; Policy; Process; Reimbursement mode

DOI：

10.7507/1672-2531.202401104

Video：

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Objective To analyze the limitations and challenges for the use of real-world data in the decision making of drug reimbursement through literature review and provide standard process and guideline for the real-world study supporting drug reimbursement. Methods By summarizing the relevant policies, regulations, and guiding principles of major drug regulatory agencies worldwide, the study analyzed the applicable conditions, framework, and reimbursement mode for using real-world evidence in the decision making of drug reimbursement. Results The study found that the health technology assessment departments of major developed countries and Asian countries have used real -world evidence to evaluate the drug efficacy and safety. The application scope of real-world data for reimbursement decision included describing the treatment process of the disease, assessing economic burden, verifying economic models, and evaluating the efficacy and safety of drugs. Some developed countries including the United Kingdom and the United States had released guidelines or frameworks of the real-world study for reimbursement decision. The process and framework of using real-world data in reimbursement decision could be divided into three models: coverage with evidence development, outcome-based contract, and re-assessment. Conclusion Real-world data has been widely used in the process of health technology assessment. To adapt to the development of the pharmaceutical industry and to meet the needs of clinical patients, it is urgent to standardize the process of collecting real-world data and formulate the scope and process of using real-world data in the reimbursement process.

Citation： HUANG Tao, LI Can, HUANG Xianqin, LI Huangqianyu, WU Jing, MAO Zongfu, SHI Luwen, GUAN Xiaodong. Policies for use of real-world data in drug reimbursement decision. Chinese Journal of Evidence-Based Medicine, 2024, 24(5): 510-515. doi: 10.7507/1672-2531.202401104 Copy

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2.	Corrigan-Curay J, Sacks L, Woodcock J. Real-world evidence and real-world data for evaluating drug safety and effectiveness. JAMA, 2018, 320(9): 867-868.
3.	Makady A, Ham RT, de Boer A, et al. Policies for use of real-world data in health technology assessment (HTA): a comparative study of six HTA agencies. Value Health, 2017, 20(4): 520-532.
4.	Makady A, van Veelen A, Jonsson P, et al. Using real-world data in health technology assessment (HTA) practice: a comparative study of five HTA agencies. Pharmacoeconomics, 2018, 36(3): 359-368.
5.	聂晓璐, 王胜锋, 姚晨, 等. 国内外真实世界数据/证据的相关药械监管政策法规及指导原则比较研究. 药物流行病学杂志, 2022, 31(1): 5-12, 49.
6.	Li G, Liu Y, Xie C, et al. Characteristics of expedited programmes for cancer drug approval in China. Nature Rev Drug Dis, 2021, 20(6): 416-416.
7.	国家医疗保障局, 国家卫生健康委. 关于适应国家医保谈判常态化持续做好谈判药品落地工作的通知. 2021.
8.	Bullement A, Podkonjak T, Robinson MJ, et al. Real-world evidence use in assessments of cancer drugs by NICE. Inter J Tech Assess Health Care, 2020, 36(4): 388-394.
9.	Lou J, Kc S, Toh KY, et al. Real-world data for health technology assessment for reimbursement decisions in Asia: current landscape and a way forward. Inter J Tech Assess Health Care, 2020, 36(5): 474-480.
10.	Oyebode O, Garrett Z, George E, et al. Evidence requirements for reimbursement of pharmaceuticals across europe. Inter J Tech Assess Health Care, 2015, 31(1-2): 59-67.
11.	Use of real-world evidence in single-drug assessments. 2018.
12.	Procedure and guidelines for CADTH CDR. 2020.
13.	Therapeutic Goods Administration. Real world evidence and patient reported outcomes in the regulatory context. 2021.
14.	NICE. NICE real-world evidence framework. 2022.
15.	CADTH. Developing a Canadian real-world evidence action plan across the drug life cycle. 2020.
16.	IQWIG. Development of scientific concepts for the generation of routine practice data and their analysis for the benefit assessment of drugs according to 35a social code book V – rapid report. 2019.
17.	HAS. Real-world studies for the assessment of medicinal products and medical devices. 2021.
18.	Hampson G, Towse A, Dreitlein WB, et al. Real-world evidence for coverage decisions: opportunities and challenges. J Compara Effect Res, 2018, 7(12): 1133-1143.
19.	Dreyer NA. Advancing a framework for regulatory use of real-world evidence: when real is reliable. Therap Innov Reg Sci, 2018, 52(3): 362-368.
20.	FDA. Use of Real-world evidence to support regulatory decision-making for medical devices. 2017.
21.	FDA. Use of electronic health record data in clinical investigations guidance for industry. 2018.
22.	FDA. Framework for FDA’s real-world evidence program. 2018.
23.	FDA. Submitting documents using real-world data and real-world evidence to FDA for drug and biological products. 2022.
24.	Hines PA, Janssens R, Gonzalez-Quevedo R, et al. A future for regulatory science in the European Union: the European Medicines Agency’s strategy. Nat Rev Drug Discov, 2020, 19(5): 293-294.
25.	EMA. Guideline on registry-based studies. 2021.
26.	PMDA. Basic principles on utilization of registry for applications. 2021.
27.	PMDA. Points to consider for ensuring the reliability in utilization of registry data for applications. 2021.
28.	国家药监局. 关于发布真实世界证据支持药物研发与审评的指导原则(试行)的通告. 2020.
29.	国家药监局. 关于发布真实世界数据用于医疗器械临床评价技术指导原则(试行)的通告. 2020.
30.	ISPOR. Optimizing managed access: lessons from the cancer drugs fund. 2023.
31.	Jade-Xoan P. Authorisation for early access to medicinal products: HAS assessment doctrine. 2021.
32.	Zeitler E, Gilstrap L, Coylewright M, et al. Coverage with evidence development: where are we now? 2022.
33.	Cathy K. US outcomes-based contracts: big uptick in interest, but not execution. 2016.
34.	Chan K, Nam S, Evans B, et al. Developing a framework to incorporate real-world evidence in cancer drug funding decisions: the Canadian real-world evidence for value of cancer drugs (CanREValue) collaboration. BMJ open, 2020, 10(1): e032884.
35.	Curtis LH, Sola-Morales O, Heidt J, et al. Regulatory and HTA considerations for development of real-world data derived external controls. Clin Pharm Therap, 2023, 114(2): 303-315.
36.	Wang W, Tan J, Wu J, et al. Use of real world data to improve drug coverage decisions in China. BMJ, 2023, 381: e068911.
37.	U. S. FDA. Real-world data: assessing electronic health records and medical claims data to support regulatory decision-making for drug and biological products. 2021.

1. Sherman RE, Anderson SA, Dal Pan GJ, et al. Real-world evidence - What is it and what can it tell us. N Engl J Med, 2016, 375(23): 2293-2297.
2. Corrigan-Curay J, Sacks L, Woodcock J. Real-world evidence and real-world data for evaluating drug safety and effectiveness. JAMA, 2018, 320(9): 867-868.
3. Makady A, Ham RT, de Boer A, et al. Policies for use of real-world data in health technology assessment (HTA): a comparative study of six HTA agencies. Value Health, 2017, 20(4): 520-532.
4. Makady A, van Veelen A, Jonsson P, et al. Using real-world data in health technology assessment (HTA) practice: a comparative study of five HTA agencies. Pharmacoeconomics, 2018, 36(3): 359-368.
5. 聂晓璐, 王胜锋, 姚晨, 等. 国内外真实世界数据/证据的相关药械监管政策法规及指导原则比较研究. 药物流行病学杂志, 2022, 31(1): 5-12, 49.
6. Li G, Liu Y, Xie C, et al. Characteristics of expedited programmes for cancer drug approval in China. Nature Rev Drug Dis, 2021, 20(6): 416-416.
7. 国家医疗保障局, 国家卫生健康委. 关于适应国家医保谈判常态化持续做好谈判药品落地工作的通知. 2021.
8. Bullement A, Podkonjak T, Robinson MJ, et al. Real-world evidence use in assessments of cancer drugs by NICE. Inter J Tech Assess Health Care, 2020, 36(4): 388-394.
9. Lou J, Kc S, Toh KY, et al. Real-world data for health technology assessment for reimbursement decisions in Asia: current landscape and a way forward. Inter J Tech Assess Health Care, 2020, 36(5): 474-480.
10. Oyebode O, Garrett Z, George E, et al. Evidence requirements for reimbursement of pharmaceuticals across europe. Inter J Tech Assess Health Care, 2015, 31(1-2): 59-67.
11. Use of real-world evidence in single-drug assessments. 2018.
12. Procedure and guidelines for CADTH CDR. 2020.
13. Therapeutic Goods Administration. Real world evidence and patient reported outcomes in the regulatory context. 2021.
14. NICE. NICE real-world evidence framework. 2022.
15. CADTH. Developing a Canadian real-world evidence action plan across the drug life cycle. 2020.
16. IQWIG. Development of scientific concepts for the generation of routine practice data and their analysis for the benefit assessment of drugs according to 35a social code book V – rapid report. 2019.
17. HAS. Real-world studies for the assessment of medicinal products and medical devices. 2021.
18. Hampson G, Towse A, Dreitlein WB, et al. Real-world evidence for coverage decisions: opportunities and challenges. J Compara Effect Res, 2018, 7(12): 1133-1143.
19. Dreyer NA. Advancing a framework for regulatory use of real-world evidence: when real is reliable. Therap Innov Reg Sci, 2018, 52(3): 362-368.
20. FDA. Use of Real-world evidence to support regulatory decision-making for medical devices. 2017.
21. FDA. Use of electronic health record data in clinical investigations guidance for industry. 2018.
22. FDA. Framework for FDA’s real-world evidence program. 2018.
23. FDA. Submitting documents using real-world data and real-world evidence to FDA for drug and biological products. 2022.
24. Hines PA, Janssens R, Gonzalez-Quevedo R, et al. A future for regulatory science in the European Union: the European Medicines Agency’s strategy. Nat Rev Drug Discov, 2020, 19(5): 293-294.
25. EMA. Guideline on registry-based studies. 2021.
26. PMDA. Basic principles on utilization of registry for applications. 2021.
27. PMDA. Points to consider for ensuring the reliability in utilization of registry data for applications. 2021.
28. 国家药监局. 关于发布真实世界证据支持药物研发与审评的指导原则(试行)的通告. 2020.
29. 国家药监局. 关于发布真实世界数据用于医疗器械临床评价技术指导原则(试行)的通告. 2020.
30. ISPOR. Optimizing managed access: lessons from the cancer drugs fund. 2023.
31. Jade-Xoan P. Authorisation for early access to medicinal products: HAS assessment doctrine. 2021.
32. Zeitler E, Gilstrap L, Coylewright M, et al. Coverage with evidence development: where are we now? 2022.
33. Cathy K. US outcomes-based contracts: big uptick in interest, but not execution. 2016.
34. Chan K, Nam S, Evans B, et al. Developing a framework to incorporate real-world evidence in cancer drug funding decisions: the Canadian real-world evidence for value of cancer drugs (CanREValue) collaboration. BMJ open, 2020, 10(1): e032884.
35. Curtis LH, Sola-Morales O, Heidt J, et al. Regulatory and HTA considerations for development of real-world data derived external controls. Clin Pharm Therap, 2023, 114(2): 303-315.
36. Wang W, Tan J, Wu J, et al. Use of real world data to improve drug coverage decisions in China. BMJ, 2023, 381: e068911.
37. U. S. FDA. Real-world data: assessing electronic health records and medical claims data to support regulatory decision-making for drug and biological products. 2021.

Chinese Journal of Evidence-Based Medicine

Policies for use of real-world data in drug reimbursement decision

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