從那他珠單抗不良反應處理看生物制劑風險監測——基于嚴重不良反應報告與相關管理文件的系統評價_《中國循證醫學雜志》

作者：

李媛媛 ,  李幼平 , 孫鑫 , 王莉

四川大學華西醫院循證醫學與臨床流行病學教研室（成都 610041）;

關鍵詞：

那他珠單抗不良反應處理生物制劑風險監測系統評價

DOI：

10.7507/1672-2531.20090214

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目的　基于導致那他珠單抗撤市的不良反應報告及其審評途徑，深入分析其撤市原因，為更安全有效完善類似生物制劑的報告方法提供決策參考。
方法　計算機檢索MEDLINE、EMbase、和美國FDA 網站，納入病例報道及審評途徑、藥品上市撤市審評相關情況的信息。
結果　最終納入4 篇病例報道及14 篇官方文獻。那他珠單抗第一次上市后的臨床試驗中有3 例發生進行性多灶性白質腦病（PML），其中2 例通過血透糾正。重新上市兩年后，32 000 例受試者中共發現3 例新發PML（0.06‰），但未見死亡報道。鑒于那他珠單抗其治療多發性硬化（MS）、克羅恩病（CD）的不可替代性，FDA 決定在嚴密監控下病人仍可用此藥。
結論　 ① 那他珠單抗治療MS 和CD 的最嚴重不良反應均為PML，目前可防可治。上市期間其療效及不良反應監測結果均提示現有審批制度相對完善可行，可供其他高風險藥物如中藥注射劑的快速審評或標準審評上市提供借鑒。② 那他珠單抗重新上市對其他藥物研發公司不僅是重要的風險管理先例，也為一些有可疑副作用的藥物重返市場或重新開展臨床試驗的可能性帶來了希望。③ 由于那他珠單抗是經快速通道審評，在研Good Clinical Practice（GCP）研究與上市后評價是交疊進行，因此必須不斷進行追蹤，提供新證據。

引用本文： 李媛媛,李幼平,孫鑫,王莉. 從那他珠單抗不良反應處理看生物制劑風險監測——基于嚴重不良反應報告與相關管理文件的系統評價. 中國循證醫學雜志, 2009, 09(11): 1185-1192. doi: 10.7507/1672-2531.20090214 復制

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20.	Shankar G, Pendley C, Stein KE. A risk—based bioanalytical strategy for the assessment of antibody immune responses against biological drugs. Nat Biotechnol, 2007, 25(5):555-561.
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26.	Yousry TA, Habil M, Major EO, et al. Evaluation of Patients Treated with Natalizumab for Progressive Multifocal Leukoencephalopathy. N Engl J Med, 2006, 354: 924-933.
27.	Information on Natalizumab (marketed as Tysabri). [cited 2009, Aug, 15]. Available from: URL: http://www.fda.gov/Drugs/ DrugSafety/PostmarketDrugSafetyInformationforPatientsandProvid ers/ucm107198.htm.
28.	Tysabri Relaunch Solid, As Efficacy Trumps Risk. [cited 2009, Aug, 15]. Available from: URL: (http://blogs.wsj.com/health/2008/02/29/ tysabri-relaunch-solid-as-efficacy-trumps-risk/).
29.	Walsh G. Biopharmaceutical benchmarks 2006. Nat Biotechnol, 2006, 24(7): 769-776.
30.	Aggarwal S. What’s fueling the biotech engine? Nat Biotechnol, 2007, 25(10): 1097-2011.

1. Assche GV, Ranst PD, RafSciot PD, et al. Progressive multifocal leukoencephalopathy after natalizumab therapy for Crohn’s disease. N Engl J Med, 2005, 353: 1-7.
2. Demasters BK , Tyler KL. Progressive multifocal leukoencephalopathy complicating treatment with natalizumab and interferon beta-1a for multiple sclerosis. N Engl J Med, 2005, 353: 369-374.
3. Gould A, Atlas S, Green AJ, et al. Pelletier D. Progressive multifocal leukoencephalopathy in a patient treated with natalizumab. N Engl J Med, 2005, 353: 375-381.
4. Hartung HP . New cases of progressive multifocal leukoencephalopathy after treatment with natalizumab. Lancet Neurol, 2009, 8: 28-31.
5. U.S. Department of Health and Human Services. Center for Drug Evaluation and Research 2002 Report to the Nation Improving Public Health Through Human Drugs [cited 2009, Aug, 15]. Available from: URL: http://www.fda.gov/downloads/AboutFDA/ CentersOffices/CDER/WhatWeDo/UCM078985.pdf.
6. U.S. Department of Health and Human Services. Center for Drug Evaluation and Research 2003 Report to the NationReport to the Nation Improving Public Health Through Human Drugs[cited 2009, Aug, 15]. Available from: URL: http://www.fda.gov/downloads/ AboutFDA/CentersOffices/CDER/WhatWeDo/UCM078975.pdf. U.S. Department of Health and Human Services.
7. U.S. Department of Health and Human Services.Center for Drug Evaluation and Research 2004 Report to the NationReport to the Nation Improving Public Health Through Human Drugs[cited 2009, Aug, 15]. Available from: URL: http://www.fda.gov/downloads/ AboutFDA/CentersOffices/CDER/WhatWeDo/UCM078941.pdf..
8. U.S. Department of Health and Human Services.Center for Drug Evaluation and Research 2005 Report to the NationReport to the Nation Improving Public Health Through Human Drugs[cited 2009, Aug, 15]. Available from: URL: http://www.fda.gov/downloads/ AboutFDA/CentersOffices/CDER/WhatWeDo/UCM078935.pdf.
9. U.S. Department of Health and Human Services.Center for Drug Evaluation and Research UPDAE[cited 2009, Aug, 15]. Available from: URL: http://www.fda.gov/downloads/AboutFDA/ CentersOffices/CDER/WhatWeDo/UCM121704.pdf.
10. NME Drug and New Biologic Approvals in 2002[cited 2009, Aug, 15]. Available from: URL: http://www.fda.gov/Drugs/Developmen tApprovalProcess/HowDrugsareDevelopedandApproved/Drugan dBiologicApprovalReports/NMEDrugandNewBiologicApprovals/ ucm081683.htm.
11. NME Drug and New Biologic Approvals in 2003[cited 2009, Aug, 15]. Available from: URL: http://www.fda.gov/Drugs/Developmen tApprovalProcess/HowDrugsareDevelopedandApproved/Drugan dBiologicApprovalReports/NMEDrugandNewBiologicApprovals/ ucm081680.htm.
12. NME Drug and New Biologic Approvals in 2004[cited 2009, Aug, 15]. Available from: URL: http://www.fda.gov/Drugs/Developmen tApprovalProcess/HowDrugsareDevelopedandApproved/Drugan dBiologicApprovalReports/NMEDrugandNewBiologicApprovals/ ucm081677.htm.
13. NME Drug and New Biologic Approvals in 2005[cited 2009, Aug, 15]. Available from: URL: http://www.fda.gov/Drugs/Developmen tApprovalProcess/HowDrugsareDevelopedandApproved/Drugan dBiologicApprovalReports/NMEDrugandNewBiologicApprovals/ ucm081676.htm.
14. NME Drug and New Biologic Approvals in 2006[cited 2009, Aug, 15]. Available from: URL: http://www.fda.gov/Drugs/Developmen tApprovalProcess/HowDrugsareDevelopedandApproved/Drugan dBiologicApprovalReports/NMEDrugandNewBiologicApprovals/ ucm081673.htm.
15. NME Drug and New Biologic Approvals in [cited 2009, Aug, 15]. Available from: URL: http://www.fda.gov/Drugs/DevelopmentA pprovalProcess/HowDrugsareDevelopedandApproved/Drugand BiologicApprovalReports/NMEDrugandNewBiologicApprovals/ ucm081690.htm 2007.
16. CDER New Molecular Entity (NME) Drug and New Biologic Approvals for Calendar Year 2008 Updated through November 30, 2008[cited 2009, Aug, 15]. Available from: URL: http://www.fda. gov/downloads/Drugs/DevelopmentApprovalProcess/HowDrugsare DevelopedandApproved/DrugandBiologicApprovalReports/NMEDr ugandNewBiologicApprovals/UCM081805.pdf.
17. CDER Approval Times for Priority and Standard NMEs and New BLAs CY 1993 – 2008[cited 2009, Aug, 15]. Available from: URL: http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProces s/HowDrugsareDevelopedandApproved/DrugandBiologicApproval Reports/UCM123959.pdf.
18. Fast Track, Accelerated Approval and Priority Review[cited 2009, Aug, 15]. Available from: URL: http://www.fda.gov/ForConsumers/ ByAudience/ForPatientAdvocates/SpeedingAccesstoImportantNew Therapies/ucm128291.htm.
19. 杜蕾. 新藥的快速審批及風險. 上海食品藥品監管情報研究, 2007, (87): 1-4, 47.
20. Shankar G, Pendley C, Stein KE. A risk—based bioanalytical strategy for the assessment of antibody immune responses against biological drugs. Nat Biotechnol, 2007, 25(5):555-561.
21. Baumann A. Early development of therapeutic biologics– pharmacokinetics. Curr DrugMetab, 2006, 7(1): 15-21.
22. Schneeweiss S. Developments in post-marketing comparative effectiveness research. Clin Pharmacol Ther, 2007, 82(2): 143-156.
23. Stricker BH, Psaty BM. Detection, verification, and quantification of adverse drug reactions. BMJ, 2004, 329(7456): 44-47.
24. Giezen TJ, Mantel-Teeuwisse AK, Straus SM, et al. Safety-Related Regulatory Actions for Biologicals Approved in the United States and the European Union. JAMA, 2008, 300(16): 1887-1896.
25. FDA Issues Final Risk Minimization Guiances[EB/OL]. [cited 2009, Aug, 15]. Available from: URL: http://www.fda.gov/bbs/topics/ news/2005/NEW01169.html.
26. Yousry TA, Habil M, Major EO, et al. Evaluation of Patients Treated with Natalizumab for Progressive Multifocal Leukoencephalopathy. N Engl J Med, 2006, 354: 924-933.
27. Information on Natalizumab (marketed as Tysabri). [cited 2009, Aug, 15]. Available from: URL: http://www.fda.gov/Drugs/ DrugSafety/PostmarketDrugSafetyInformationforPatientsandProvid ers/ucm107198.htm.
28. Tysabri Relaunch Solid, As Efficacy Trumps Risk. [cited 2009, Aug, 15]. Available from: URL: (http://blogs.wsj.com/health/2008/02/29/ tysabri-relaunch-solid-as-efficacy-trumps-risk/).
29. Walsh G. Biopharmaceutical benchmarks 2006. Nat Biotechnol, 2006, 24(7): 769-776.
30. Aggarwal S. What’s fueling the biotech engine? Nat Biotechnol, 2007, 25(10): 1097-2011.

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《中國循證醫學雜志》

從那他珠單抗不良反應處理看生物制劑風險監測——基于嚴重不良反應報告與相關管理文件的系統評價

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