How to use real-world evidence to inform post-marketing drug evaluation: a proposal for conceptual framework_Chinese Journal of Evidence-Based Medicine

Authors：

 SUN Xin ¹ , TAN Jing ¹ , TANG Li ¹ , LI Ling ¹ , WANG Wen ¹ , XIONG Weiyi ² , LIU Jia ² , WU Guizhi ²

1. Chinese Evidence-based Medicine Center and CREAT Group, West China Hospital, Sichuan University, Chengdu, 610041, P.R.China;
2. Center for Drug Re-evaluation and Center for ADR Mouitoring, Chinese State Drug Administration, Beijing, 100000, P.R.China;

Corresponding author：

SUN Xin, Email: sunx79@hotmail.com

Keywords：

Real-world evidence; Post-marketing drug assessment; Policy decision

DOI：

10.7507/1672-2531.201803006

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Real-world evidence represents critical evidence to support post-marketing drug monitoring, assessment and policy decisions, and has received extensive attentions. However, an explicit over-arching design and conceptual framework for this specific area is lacking. Divergent opinions on the production of real world evidence are often present among researchers; and understanding about their implications also differ among policy makers and evidence users. In this article, we have proposed, from the regulatory and clinical perspectives, a conceptual framework on the use of real world data for post-marketing drug studies, assessment and policy decisions.

Citation： SUN Xin, TAN Jing, TANG Li, LI Ling, WANG Wen, XIONG Weiyi, LIU Jia, WU Guizhi. How to use real-world evidence to inform post-marketing drug evaluation: a proposal for conceptual framework. Chinese Journal of Evidence-Based Medicine, 2018, 18(4): 277-283. doi: 10.7507/1672-2531.201803006 Copy

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2.	孙鑫, 谭婧, 唐立, 等. 重新认识真实世界研究. 中国循证医学杂志, 2017, 17(2): 126-130.
3.	王雯, 刘艳梅, 谭婧, 等. 回顾性数据库研究的概念、策划与研究数据库构建. 中国循证医学杂志, 2018, 18(2): 230-237.
4.	谭婧, 程亮亮, 王雯, 等. 患者登记研究的策划与患者登记数据库构建: 基于观察性设计的真实世界研究. 中国循证医学杂志, 2017, 17(12): 1365-1372.
5.	U.S. Food and Drug Administration. FDA's Sentinel Initiative 2016. Available at: https://www.fda.gov/Safety/ FDAsSentinelInitiative/default.htm.
6.	Oyinlola JO, Campbell J, Kousoulis AA. Is real world evidence influencing practice? A systematic review of CPRD research in NICE guidances. BMC Health Serv Res, 2016, 16: 299.
7.	Ko CY, Hall BL, Hart AJ, et al. The American college of surgeons national surgical quality improvement program: achieving better and safer surgery. Jt Comm J Qual Patient Saf, 2015, 41(5): 199-204.
8.	Garrison LP, Jr., Neumann PJ, Erickson P, et al. Using real-world data for coverage and payment decisions: the ISPOR real-world data task force report. Value Health, 2007, 10(5): 326-335.
9.	Zhao Y, Pinnow E, Major JM. A systematic assessment of key design and performance characteristics of drug exposure registries requested by the U.S. Food and Drug Administration. Pharmacoepidemiol Drug Saf, 2018, 27(3): 299-306.
10.	Brand M, Muller A, Alsop J, et al. Results from a 9-year Intensive Safety Surveillance Scheme (IS (3)) in miglustat (Zavesca ((R)))-treated patients. Pharmacoepidemiol Drug Saf, 2015, 24(3): 329-333.
11.	Shin SM, Kim CS, Karkada N, et al. Post-marketing safety surveillance conducted in Korea (2008-2013) following the introduction of the rotavirus vaccine, RIX4414 (Rotarix). Hum Vaccin Immunother, 2016, 12(10): 2590-2594.
12.	Kyriacou DN, Lewis RJ. Confounding by Indication in Clinical Research. JAMA, 2016, 316(17): 1818-1819.
13.	Cortese S, Panei P, Arcieri R, et al. Safety of methylphenidate and atomoxetine in children with attention-deficit/hyperactivity disorder (ADHD): data from the Italian National ADHD Registry. CNS Drugs, 2015, 29(10): 865-877.
14.	Eapen ZJ, Lauer MS, Temple RJ. The imperative of overcoming barriers to the conduct of large, simple trials. JAMA, 2014,311(14): 1397-1398.
15.	Wechsler ME, Yawn BP, Fuhlbrigge AL, et al. Anticholinergic vs long-acting beta-agonist in combination with inhaled corticosteroids in black adults with asthma: the BELT randomized clinical trial. JAMA, 2015, 314(16): 1720-1730.
16.	Li L, Shen J, Bala MM, et al. Incretin treatment and risk of pancreatitis in patients with type 2 diabetes mellitus: systematic review and meta-analysis of randomised and non-randomised studies. BMJ, 2014, 348: g2366.
17.	Li L, Li S, Deng K, et al. Dipeptidyl peptidase-4 inhibitors and risk of heart failure in type 2 diabetes: systematic review and meta-analysis of randomised and observational studies. BMJ, 2016, 352: i610.
18.	Liu J, Li L, Deng K, et al. Incretin based treatments and mortality in patients with type 2 diabetes: systematic review and meta-analysis. BMJ, 2017, 357: j2499.
19.	Guyatt GH, Oxman AD, Vist GE, et al. GRADE: an emerging consensus on rating quality of evidence and strength of recommendations. BMJ, 2008, 336(7650): 924-926.
20.	Andrews J, Guyatt G, Oxman AD, et al. GRADE guidelines: 14. Going from evidence to recommendations: the significance and presentation of recommendations. J Clin Epidemiol, 2013, 66(7): 719-725.
21.	Andrews JC, Schunemann HJ, Oxman AD, et al. GRADE guidelines: 15. Going from evidence to recommendation-determinants of a recommendation's direction and strength. J Clin Epidemiol, 2013, 66(7): 726-735.
22.	Siemieniuk RA, Foroutan F, Mirza R, et al. Antiretroviral therapy for pregnant women living with HIV or hepatitis B: a systematic review and meta-analysis. BMJ Open, 2017, 7(9): e019022.
23.	Siemieniuk RAC, Lytvyn L, Mah MJ, et al. Antiretroviral therapy in pregnant women living with HIV: a clinical practice guideline. BMJ, 2017, 358: j3961.
24.	U.S. Food and Drug Administration. Exenatide (marketed as BYETTA): Acute Pancreatitis. Available at: http://www.fda.gov/Drugs/DrugSafety/DrugSafetyNewsletter/ucm119034.htm#exenatide.
25.	Cox E, Martin BC, Van Staa T, et al. Good research practices for comparative effectiveness research: Approaches to mitigate bias and confounding in the design of nonrandomized studies of treatment effects using secondary data sources: The international society for pharmacoeconomics and outcomes research good research practices for retrospective database analysis task force report -Part II. Value Health, 2009, 12(8):1053-1061.
26.	Lenzer J. Consumer group calls for antidiabetes drug to be withdrawn. BMJ, 2012, 344: e3259.
27.	Cohen D. Has pancreatic damage from glucagon suppressing diabetes drugs been underplayed? BMJ, 2013, 346: f3680.
28.	Kmietowicz Z. Potential harms of type 2 diabetes drugs have been ignored, finds BMJ investigation. BMJ, 2013,346: f3782.
29.	Scirica BM, Bhatt DL, Braunwald E, et al. Saxagliptin and cardiovascular outcomes in patients with type 2 diabetes mellitus. N Engl J Med, 2013, 369(14): 1317-1326.
30.	Yanoff L. Saxagliptin assessment of vascular outcomes recorded in patients with diabetes mellitus (SAVOR). Available at: http://www.fda.gov/downloads/advisorycommittees/committeesmeetingmaterials/drugs/endocrinologicandmetabolicdrugsadvisorycommittee/ucm444144.pdf.
31.	Zannad F, Cannon CP, Cushman WC, et al. Heart failure and mortality outcomes in patients with type 2 diabetes taking alogliptin versus placebo in EXAMINE: a multicentre, randomised, double-blind trial. Lancet, 2015, 385(9982): 2067-2076.
32.	Green JB, Bethel MA, Armstrong PW, et al. Effect of sitagliptin on cardiovascular outcomes in Type 2 diabetes. N Engl J Med, 2015, 373(3): 232-242.
33.	Li L, Li S, Liu J, et al. Glucagon-like peptide-1 receptor agonists and heart failure in type 2 diabetes: systematic review and meta-analysis of randomized and observational studies. BMC Cardiovasc Disord, 2016, 16(1): 91.
34.	Sutton AJ, Cooper NJ, Lambert PC, et al. Meta-analysis of rare and adverse event data. Expert Rev Pharmacoecon Outcomes Res, 2002, 2(4): 367-379.
35.	Nissen SE, Wolski K. Effect of rosiglitazone on the risk of myocardial infarction and death from cardiovascular causes. N Engl J Med, 2007, 356(24): 2457-2471.
36.	Grieff M, Bushinsky DA. Diuretics and disorders of calcium homeostasis. Semin Nephrol, 2011, 31(6): 535-541.
37.	Tsvetov G, Hirsch D, Shimon I, et al. Thiazide treatment in primary hyperparathyroidism-a new indication for an old medication? J Clin Endocrinol Metab, 2017, 102(4): 1270-1276.
38.	Berger ML, Dreyer N, Anderson F, et al. Prospective observational studies to assess comparative effectiveness: the ISPOR good research practices task force report. Value in Health, 2012, 15(2): 217-230.
39.	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. Value Health, 2017, 20(8): 1003-1008.

1. 唐立, 康德英, 喻佳洁, 等. 实效性随机对照试验: 真实世界研究的重要设计. 中国循证医学杂志, 2017, 17(9): 999-1004.
2. 孙鑫, 谭婧, 唐立, 等. 重新认识真实世界研究. 中国循证医学杂志, 2017, 17(2): 126-130.
3. 王雯, 刘艳梅, 谭婧, 等. 回顾性数据库研究的概念、策划与研究数据库构建. 中国循证医学杂志, 2018, 18(2): 230-237.
4. 谭婧, 程亮亮, 王雯, 等. 患者登记研究的策划与患者登记数据库构建: 基于观察性设计的真实世界研究. 中国循证医学杂志, 2017, 17(12): 1365-1372.
5. U.S. Food and Drug Administration. FDA's Sentinel Initiative 2016. Available at: https://www.fda.gov/Safety/ FDAsSentinelInitiative/default.htm.
6. Oyinlola JO, Campbell J, Kousoulis AA. Is real world evidence influencing practice? A systematic review of CPRD research in NICE guidances. BMC Health Serv Res, 2016, 16: 299.
7. Ko CY, Hall BL, Hart AJ, et al. The American college of surgeons national surgical quality improvement program: achieving better and safer surgery. Jt Comm J Qual Patient Saf, 2015, 41(5): 199-204.
8. Garrison LP, Jr., Neumann PJ, Erickson P, et al. Using real-world data for coverage and payment decisions: the ISPOR real-world data task force report. Value Health, 2007, 10(5): 326-335.
9. Zhao Y, Pinnow E, Major JM. A systematic assessment of key design and performance characteristics of drug exposure registries requested by the U.S. Food and Drug Administration. Pharmacoepidemiol Drug Saf, 2018, 27(3): 299-306.
10. Brand M, Muller A, Alsop J, et al. Results from a 9-year Intensive Safety Surveillance Scheme (IS (3)) in miglustat (Zavesca ((R)))-treated patients. Pharmacoepidemiol Drug Saf, 2015, 24(3): 329-333.
11. Shin SM, Kim CS, Karkada N, et al. Post-marketing safety surveillance conducted in Korea (2008-2013) following the introduction of the rotavirus vaccine, RIX4414 (Rotarix). Hum Vaccin Immunother, 2016, 12(10): 2590-2594.
12. Kyriacou DN, Lewis RJ. Confounding by Indication in Clinical Research. JAMA, 2016, 316(17): 1818-1819.
13. Cortese S, Panei P, Arcieri R, et al. Safety of methylphenidate and atomoxetine in children with attention-deficit/hyperactivity disorder (ADHD): data from the Italian National ADHD Registry. CNS Drugs, 2015, 29(10): 865-877.
14. Eapen ZJ, Lauer MS, Temple RJ. The imperative of overcoming barriers to the conduct of large, simple trials. JAMA, 2014,311(14): 1397-1398.
15. Wechsler ME, Yawn BP, Fuhlbrigge AL, et al. Anticholinergic vs long-acting beta-agonist in combination with inhaled corticosteroids in black adults with asthma: the BELT randomized clinical trial. JAMA, 2015, 314(16): 1720-1730.
16. Li L, Shen J, Bala MM, et al. Incretin treatment and risk of pancreatitis in patients with type 2 diabetes mellitus: systematic review and meta-analysis of randomised and non-randomised studies. BMJ, 2014, 348: g2366.
17. Li L, Li S, Deng K, et al. Dipeptidyl peptidase-4 inhibitors and risk of heart failure in type 2 diabetes: systematic review and meta-analysis of randomised and observational studies. BMJ, 2016, 352: i610.
18. Liu J, Li L, Deng K, et al. Incretin based treatments and mortality in patients with type 2 diabetes: systematic review and meta-analysis. BMJ, 2017, 357: j2499.
19. Guyatt GH, Oxman AD, Vist GE, et al. GRADE: an emerging consensus on rating quality of evidence and strength of recommendations. BMJ, 2008, 336(7650): 924-926.
20. Andrews J, Guyatt G, Oxman AD, et al. GRADE guidelines: 14. Going from evidence to recommendations: the significance and presentation of recommendations. J Clin Epidemiol, 2013, 66(7): 719-725.
21. Andrews JC, Schunemann HJ, Oxman AD, et al. GRADE guidelines: 15. Going from evidence to recommendation-determinants of a recommendation's direction and strength. J Clin Epidemiol, 2013, 66(7): 726-735.
22. Siemieniuk RA, Foroutan F, Mirza R, et al. Antiretroviral therapy for pregnant women living with HIV or hepatitis B: a systematic review and meta-analysis. BMJ Open, 2017, 7(9): e019022.
23. Siemieniuk RAC, Lytvyn L, Mah MJ, et al. Antiretroviral therapy in pregnant women living with HIV: a clinical practice guideline. BMJ, 2017, 358: j3961.
24. U.S. Food and Drug Administration. Exenatide (marketed as BYETTA): Acute Pancreatitis. Available at: http://www.fda.gov/Drugs/DrugSafety/DrugSafetyNewsletter/ucm119034.htm#exenatide.
25. Cox E, Martin BC, Van Staa T, et al. Good research practices for comparative effectiveness research: Approaches to mitigate bias and confounding in the design of nonrandomized studies of treatment effects using secondary data sources: The international society for pharmacoeconomics and outcomes research good research practices for retrospective database analysis task force report -Part II. Value Health, 2009, 12(8):1053-1061.
26. Lenzer J. Consumer group calls for antidiabetes drug to be withdrawn. BMJ, 2012, 344: e3259.
27. Cohen D. Has pancreatic damage from glucagon suppressing diabetes drugs been underplayed? BMJ, 2013, 346: f3680.
28. Kmietowicz Z. Potential harms of type 2 diabetes drugs have been ignored, finds BMJ investigation. BMJ, 2013,346: f3782.
29. Scirica BM, Bhatt DL, Braunwald E, et al. Saxagliptin and cardiovascular outcomes in patients with type 2 diabetes mellitus. N Engl J Med, 2013, 369(14): 1317-1326.
30. Yanoff L. Saxagliptin assessment of vascular outcomes recorded in patients with diabetes mellitus (SAVOR). Available at: http://www.fda.gov/downloads/advisorycommittees/committeesmeetingmaterials/drugs/endocrinologicandmetabolicdrugsadvisorycommittee/ucm444144.pdf.
31. Zannad F, Cannon CP, Cushman WC, et al. Heart failure and mortality outcomes in patients with type 2 diabetes taking alogliptin versus placebo in EXAMINE: a multicentre, randomised, double-blind trial. Lancet, 2015, 385(9982): 2067-2076.
32. Green JB, Bethel MA, Armstrong PW, et al. Effect of sitagliptin on cardiovascular outcomes in Type 2 diabetes. N Engl J Med, 2015, 373(3): 232-242.
33. Li L, Li S, Liu J, et al. Glucagon-like peptide-1 receptor agonists and heart failure in type 2 diabetes: systematic review and meta-analysis of randomized and observational studies. BMC Cardiovasc Disord, 2016, 16(1): 91.
34. Sutton AJ, Cooper NJ, Lambert PC, et al. Meta-analysis of rare and adverse event data. Expert Rev Pharmacoecon Outcomes Res, 2002, 2(4): 367-379.
35. Nissen SE, Wolski K. Effect of rosiglitazone on the risk of myocardial infarction and death from cardiovascular causes. N Engl J Med, 2007, 356(24): 2457-2471.
36. Grieff M, Bushinsky DA. Diuretics and disorders of calcium homeostasis. Semin Nephrol, 2011, 31(6): 535-541.
37. Tsvetov G, Hirsch D, Shimon I, et al. Thiazide treatment in primary hyperparathyroidism-a new indication for an old medication? J Clin Endocrinol Metab, 2017, 102(4): 1270-1276.
38. Berger ML, Dreyer N, Anderson F, et al. Prospective observational studies to assess comparative effectiveness: the ISPOR good research practices task force report. Value in Health, 2012, 15(2): 217-230.
39. 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. Value Health, 2017, 20(8): 1003-1008.

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How to use real-world evidence to inform post-marketing drug evaluation: a proposal for conceptual framework

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