Measures for conducting comparative benefit:risk assessment

Drug Information Journal

Volumn 42, Issue 3, 2008, Pages 223-233

  Chuang Stein, Christy ^a,d   Entsuah, Richard ^b   Pritchett, Yili ^c  

^a PFIZER INC   (United States)

^b MERCK RESEARCH LABORATORIES   (United States)

^c ABBOTT LABORATORIES   (United States)

^d NONE   (United States)

Author keywords

Benefit less risk measure; Benefit:risk index; Benefit:risk ratio; Global benefit:risk score; Number needed to treat; Q TWiST; TWiST

Indexed keywords

COMPARATIVE STUDY; DRUG EFFICACY; DRUG SAFETY; PRIORITY JOURNAL; QUANTITATIVE STUDY; REVIEW; RISK ASSESSMENT; RISK BENEFIT ANALYSIS;

EID: 44649167607     PISSN: 00928615     EISSN: None     Source Type: Journal    
DOI: 10.1177/009286150804200304     Document Type: Review

References (38)

1. Federal Food, Drug, and Cosmetic Act. Available at: http://en.wikipedia. org/wiki/Food%2C_Drug%2C_and_Cosmetic_Act.
2. Principles of international law recognized in the charter of the Nüremberg Tribunal and in the judgment of the tribunal (1950). Available at: http://www.icrc.org/ihl.nsf/FULL/390?OpenDocument.
3. Al-Khatib SM. Placebo-controls in short-term clinical trials of hypertension. Presentation to the FDA Cardiovascular and Renal Drugs Advisory Committee, April 25, 2006.
4. History of Declaration of Helsinki. Available at: http://cme.cancer.gov/ c01/a02_02.htm.
5. The Belmont Report (1979). US Department of Health and Human Services website. Available at: http://www.hhs.gov/ohrp/humansubjects/guidance/belmont. htm.
6. Krantz JC Jr. New drugs and the Kefauver-Harris Amendment. J New Drugs. 1966;6:77-79.
7. Thalidomide, approval history and FDA alert. Food and Drug Administration website. Available at: http://www.fda.gov/cder/drug/infopage/thalidomide/ default.htm.
8. Thalidomide. Available at: http://en.wikipedia.org/wiki/Thalidomide.
9. Accutane and the iPLEDGE Risk Management Plan. Food and Drug Administration website. Available at: http://www.fda.gov/cder/drug/advisory/ isotretinoin2005.htm.
10. Lotronex, package insert and additional information. Food and Drug Administration website. Available at: http://www.fda.gov/cder/drug/infopage/ lotronex/lotronex.htm.
11. Korting HC, Schäfer-Korting M, eds. The Benefit/Risk Ratio: A Handbook for the Rational Use of Potentially Hazardous Drugs. Boca Raton, FL: CRC Press; 1999.
12. Psaty BM, Potter JD. Editorial: risks and benefits of celecoxib to prevent recurrent adenomas. N Engl J Med. 2006;355:950-952.
13. Gelber RD, Gelman RS, Goldhirsch A. A quality-of-life oriented endpoint for comparing treatments. Biometrics. 1989;45:781-795.
14. Glasziou PP, Simes RJ, Gelber RD. Quality adjusted survival analysis. Stat Med. 1990;9:1259-1276.
15. Irish W, Sherrill B, Cole B, Gard C, Gledenning GA, Mouridsen H. Quality-adjusted survival in a crossover trial of letrozole versus tamoxifen in postmenopausal women with advanced breast cancer. Ann Oncol. 2005;10:1458-1462.
16. Payne JT, Loken MK. A survey of the benefits and risks in the practice of radiology. CRC Crit Rev Clin Radiol Nucl Med. 1975;6:425-439.
17. Chang RW, Fineberg HV. Risk-benefit consideration in the management of polymyalgia rheumatica. Med Decis Making. 1983;3:459-475.
18. Chuang-Stein C, Mohberg NR, Sinkula MS. Three measures for simultaneously evaluating benefits and risks using categorical data from clinical trials. Stat Med. 1991;10:1349-1359.
19. Entsuah R, Gorman JM. Global benefit-risk assessment of antidepressants: venlafaxine XR and fluoxetine. J Psychiatric Res. 2002;36:111-118.
20. Entsuah R, Gao B. Global benefit-risk evaluation of antidepressant action: comparison of pooled data for venlafaxine, SSRIs and placebo. CNS Spectrums. 2002;7:882-888.
21. Pritchett Y, Tamura R. The application of global benefit-risk assessment in clinical trial design and some statistical considerations. Pharm Stat. May 30, 2007 [Epub ahead of print].
22. Hutton JL. Number needed to treat: properties and problems. J R Stat Soc. 2000;163:403-419.
23. Lesaffre E, Pledger G. A note on the number needed to treat. Control Clin Trials. 1999;20:439-447.
24. Walter SD. Number needed to treat (NNT) estimation of a measure of clinical benefit. Stat Med. 2001;20:3947-3962.
25. Grieve A. The number needed to treat: a useful clinical measure or a case of the Emperor's new clothes? Pharm Stat. 2003;2:87-102.
26. Duncan BW, Olkin I. Bias of estimate of number needed to treat. Stat Med. 2005;24:1837-1848.
27. Chuang-Stein C. A new proposal for benefit-less-risk analysis in clinical trials. Control Clin Trials. 1994;15:30-43.
28. Chuang-Stein C, Mohberg NR, Musselman DM. Organization and analysis of safety data using a multivariate approach. Stat Med. 1992;11:1075-1089.
29. Women's Health Initiative. Available at: http://www.nhlbi.nih.gov/whi/.
30. Manson JE, et al. Estrogen plus progestin and the risk of coronary heart disease. N Engl J Med. 2003;349:523-534.
31. Freedman L, Anderson G, Kipnis V, et al. Approaches to monitor the results of long-term disease prevention trials: examples from the Women's Health Initiative. Control Clin Trials. 1996;17:509-525.
32. Bross IDJ. How to use RIDIT analysis? Biometrics. 1958;14:18-38.
33. O'Brien PC. Procedures for comparing samples with multiple endpoints. Biometrics. 1984;40:1079-1087.
34. Mussen F, Salek S, Walker S. A quantitative approach to benefit-risk assessment of medicines - part 1: the development of a new model using multi-criteria decision analysis. Pharmacoepidemiol Drug Safety. 2007;16(Suppl 1):S2-S15.
35. Mussen F, Salek S, Walker S. A quantitative approach to benefit-risk assessment of medicines - part 2: the practical application of a new model. Pharmacoepidemiol Drug Safety. 2007;16 (Suppl 1):S16-S41.
36. Tysabri. Approval history and important information. Available at: http://www.accessdata.fda.gov/scripts/cder/drugsatfda/index.cfm?fuseaction= Search.DrugDetails.
37. Temple R. Quantitative decision analysis: a work in progress. Clin Pharmacol Ther. 2007;82:127-130.
38. Hughes DA, Bayoumi AM, Pirmohamed M. Current assessment of risk-benefit by regulators: is it time to introduce decision analyses? Clini Pharmacol Ther. 2007;82:123-127.