Statistics: Analysis and Presentation of Safety Data

Stephens' Detection and Evaluation of Adverse Drug Reactions: Principles and Practice, Sixth Edition

Volumn , Issue , 2012, Pages 349-388

  Evans, Stephen J W ^a   Nitsch, Dorothea ^a  

^a LONDON SCHOOL OF HYGIENE AND TROPICAL MEDICINE   (United Kingdom)

Author keywords

Bayesian; Disproportionality; Epidemiology; Harms; Proportional reporting ratios; Safety; Sequential probability ratio tests; Statistics; Survival analysis

Indexed keywords

EID: 84886165109     PISSN: None     EISSN: None     Source Type: Book    
DOI: 10.1002/9780470975053.ch6     Document Type: Chapter

References (82)

1. Senn S. Statistical Issues in Drug Development. 2nd edition. Chichester: John Wiley & Sons, Ltd, 2007.
2. Aronson JK, Ferner RE. Clarification of terminology in drug safety. Drug Saf 2005; 28(10): 851-70.
3. US Food and Drug Administration. Guidance for the Use of Bayesian Statistics in Medical Device Clinical Trials. http://www.fda.gov/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/ucm071072.htm.
4. Glasziou P, Chalmers I, RawlinsM, McCulloch P. When are randomised trials unnecessary? Picking signal from noise. BMJ 2007; 334: 349-51.
5. Aronson JK, Hauben M. Anecdotes that provide definitive evidence. BMJ 2006; 332: 1267-9.
6. Howick J, Glasziou P, Aronson JK. The evolution of evidence hierarchies: what can Bradford Hill's "guidelines for causation" contribute? J R Soc Med 2009; 102: 186-94.
7. Ioannidis J, Contopoulos-IoannidisD. Reporting of safety data from randomised trials. Lancet 1998; 352: 1752-3.
8. Ioannidis J, Lau J Completeness of safety reporting in randomized trials: an evaluation of 7 medical areas. JAMA 2001; 285: 437-43.
9. Ioannidis J, Evans S, Gøtzsche PC, O'Neill RT, Altman DG, Schulz K, Moher D; CONSORT Group. Better reporting of harms in randomized trials: an extension of the CONSORT statement. Ann InternMed 2004; 141(10): 781-8.
10. Ludbrook J. Multiple comparison procedures updated. Clin Exp Pharmacol Physiol 1998; 25: 1032-7.
11. Chuang-Stein C, Le V, Chen W. Recent advancements in the analysis and presentation of safety data. Drug Inform J 2001; 35: 377-97.
12. Rossouw JE, Anderson GL, Prentice RL, LaCroix AZ, Kooperberg C, Stefanick ML, Jackson RD, Beresford SA, Howard BV, Johnson KC, Kotchen JM, Ockene J;Writing Group for theWomen's Health Initiative Investigators. Risks and benefits of estrogen plus progestin in healthy postmenopausal women. JAMA 2002; 288(3): 321-33.
13. Altman D. Practical Statistics for Medical Research. London: Chapman and Hall, 1991.
14. Rothman KJ, Greenland S, Lash TL. Modern Epidemiology. 3rd edition. Philadelphia: Lippincott Williams & Wilkins, 2008.
15. Stricker B, Tijessen J. Serum sickness-like reactions to ceflacor. J Clin Epidemiol 1992; 45: 1177-84.
16. O'Neill R. Statistical analyses of AE data from clinical trials. special emphasis on serious events. Drug Inform J 1987; 21: 9-20.
17. Peto R, Pike MC, Armitage P, Breslow NE, Cox DR, Howard SV, Mantel N, McPherson K, Peto J, Smith PG. Design and analysis of randomized clinical trials requiring prolonged observation of each patient. II. Analysis and examples. Br J Cancer 1977; 35(1): 1-39.
18. Collett D. Modelling Survival Data in Medical Research. 2nd edition. London: Chapman & Hall, 2003.
19. Machin D, Cheung YB, Parmar M. Survival Analysis: a Practical Approach. 2nd edition. Chichester: JohnWiley & Sons Ltd, 2006.
20. Clayton D, Hills M. Statistical Models in Epidemiology. Oxford, Oxford University Press, 1993.
21. Egger M, Davey Smith G, Altman DG (editors). Systematic Reviews in Health Care. Meta-analysis in Context. 2nd edition. London: British Medical Association, Blackwell Publishing, 2001.
22. Sutton AJ, Abrams KR, Jones DR, Sheldon TA, Song F. Methods for Meta-Analysis in Medical Research. Chichester: John Wiley & Sons, Ltd, 2000.
23. 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): 2457-71.
24. Bradburn MJ, Deeks JJ, Berlin JA, Russell Localio A. Much ado about nothing: a comparison of the performance of meta-analytical methods with rare events. Stat Med 2007; 26(1): 53-77.
25. Koch GG, Schmid JE, Begun JM, Maier WC. Meta analysis of drug safety data. In: Gilbert GS (editor). Drug Safety Assessment in Clinical Trials. New York: Marcel Dekker, 1993: 279-304.
26. Lee MT, Lazarus R. Meta-analysis of drug safety data with logistic regression. Drug Inform J 1997; 31(4): 1189-93.
27. Davis S, King B, Raine J. Spontaneous reporting-UK. In: Mann R, Andrews E (editors). Pharmacovigilance, 2nd edition. Chichester: Wiley, 2007.
28. Hauben M, Aronson JK. Defining "signal" and its subtypes in pharmacovigilance based on a systematic review of previous definitions. Drug Saf 2009; 32(2): 99-110.
29. HaubenM, Madigan D, Gerrits CM, Walsh L, Van Puijenbroek EP. The role of data mining in pharmacovigilance. Exp Opinion Drug Saf 2005; 4(5): 929-48.
30. Banks D, Woo EJ, Burwen DR, Perucci P, Braun MM, Ball R. Comparing data mining methods in the VAERS database. Pharmacoepidemiol Drug Saf 2005; 14 (9): 601-9.
31. Smith CC, Bennett PM, Pearce HM, Harrison PI, Reynolds DJ, Aronson JK, Grahame-Smith DG. Adverse drug reactions in a hospital general medical unit meriting notification to the Committee on Safety of Medicines. Br J Clin Pharmacol 1996; 42(4): 423-9.
32. van Puijenbroek EP, Bate A, Leufkens HG, Lindquist M, Orre R, Egberts AC. A comparison of measures of disproportionality for signal detection in spontaneous reporting systems for adverse drug reactions. Pharmacoepidemiol Drug Saf 2002; 11(1): 3-10.
33. Evans SJ, Waller PC, Davis S. Use of proportional reporting ratios (PRRs) for signal generation from spontaneous adverse drug reaction reports. Pharmacoepidemiol Drug Saf 2001; 10(6): 483-6.
34. Patwary KW. Report on Statistical Aspects of the Pilot Research Project for International Drug Monitoring. Geneva: WHO, 1969.
35. Egberts AC, Meyboom RH, De Koning FH, Bakker A, Leufkens HG. Non-puerperal lactation associated with antidepressant drug use. Br J Clin Pharmacol 1997; 44(3): 277-81.
36. Moore N, Kreft-Jais C, Haramburu F, Noblet C, Andrejak M, Ollagnier M, Bégaud B. Reports of hypoglycaemia associated with the use of ACE inhibitors and other drugs: a case/non-case study in the French pharmacovigilance system database. Br J Clin Pharmacol 1997; 44(5): 513-8.
37. Hopstadius J, Norén GN, Bate A, Edwards IR. Impact of stratification on adverse drug reaction surveillance. Drug Saf 2008; 31(11): 1035-48.
38. Hopstadius J, Norén GN, Bate A, Edwards IR. Stratification for spontaneous report databases. Drug Saf 2008; 31(12): 1145-7.
39. Evans SJ. Stratification for spontaneous report databases. Drug Saf 2008; 31(11): 1049-52.
40. WHOCollaborating Centre for Drug Statistics Methodology. International language for drug utilization research. http://www.whocc.no.
41. Bate A, Lindquist M, Edwards IR, Olsson S, Orre R, Lansner A, De Freitas RM. A Bayesian neural network method for adverse reaction signal generation. Eur J Clin Pharmacol 1998; 54(4): 315-21.
42. Lindquist M, St°ahl M, Bate A, Edwards IR, Meyboom RH. A retrospective evaluation of a data mining approach to aid finding new adverse drug reaction signals in the WHO international database. Drug Saf 2000; 23(6): 533-42.
43. DuMouchel W. Bayesian data mining in large frequency tables with an application to the FDA spontaneous reporting system. Am Stat 1999; 53: 177-90.
44. Szarfman A, Machado SG, O'Neill RT. Use of screening algorithms and computer systems to efficiently signal higher-than-expected combinations of drugs and events in the US FDA's spontaneous reports database. Drug Saf 2002; 25(6): 381-92.
45. Gould A. Practical pharmacovigilance analysis strategies. Pharmacoepidemiol Drug Saf 2003; 12(7): 559-74.
46. Armitage P. Sequential methods in clinical trials. Am J Public Health 1958; 48: 1395-402.
47. Barnard G. Sequential tests in industrial statistics. J R Stat Soc 1946; Suppl 8: 1-26.
48. Wald A. Sequential Analysis. New York: Wiley, 1947.
49. Whitehead J. The Design and Analysis of Sequential Clinical Trials. Revised 2nd edition. Chichester: Wiley, 1997.
50. Spiegelhalter D, Grigg O, Kinsman R, Treasure T. Risk-adjusted sequential probability ratio tests: applications to Bristol, Shipman, and adult cardiac surgery. Int J Qual Health Care 2003; 15(1): 7-13.
51. Almenoff JS, Pattishall EN, Gibbs TG, DuMouchel W, Evans SJ, Yuen N. Novel statistical tools for monitoring the safety of marketed drugs. Clin Pharmacol Ther 2007; 82(2): 157-66.
52. Bate A, Evans SJ. Quantitative signal detection using spontaneous ADR reporting. Pharmacoepidemiol Drug Saf 2009; 18(6): 427-36.
53. Brown JS, Kulldorff M, Chan KA, Davis RL, Graham D, Pettus PT, Andrade SE, Raebel MA, Herrinton L, Roblin D, Boudreau D, Smith D, Gurwitz JH, Gunter MJ, Platt R. Early detection of adverse drug events within population-based health networks: application of sequential testing methods. Pharmacoepidemiol Drug Saf 2007; 16(12): 1275-84.
54. Brown JS, Kulldorff M, Petronis KR, Reynolds R, Chan KA, Davis RL, Graham D, Andrade SE, Raebel MA, Herrinton L, Roblin D, Boudreau D, Smith D, Gurwitz JH, Gunter MJ, Platt R. Early adverse drug event signal detection within population-based health networks using sequential methods: key methodologic considerations. Pharmacoepidemiol Drug Saf 2009; 18(3): 226-34.
55. Altman D. Adjustment for covariate imbalance. In: Armitage P, Colton T (editors). Encyclopedia of Biostatistics. Chichester: Wiley, 1998.
56. Beardon PH, McGilchrist MM, McKendrick AD, McDevitt DG, MacDonald TM. Primary non-compliance with prescribed medication in primary care. BMJ 1993; 307(6908): 846-8.
57. Greenland S. Randomization, statistics, and causal inference. Epidemiology 1990; 1(6): 421-9.
58. Wolfe F, Flowers N, Burke TA, Arguelles LM, Pettitt D. Increase in lifetime adverse drug reactions, service utilization, and disease severity among patients who will start COX-2 specific inhibitors: quantitative assessment of channeling bias and confounding by indication in 6689 patients with rheumatoid arthritis and osteoarthritis. J Rheumatol 2002; 29(5): 1015-22.
59. Wood L, Coulson R. Revitalizing the General Practice Research Database: plans, challenges, and opportunities. Pharmacoepidemiol Drug Saf 2001; 10(5): 379-83.
60. Pocock SJ, Smeeth L. Insulin glargine and malignancy: an unwarranted alarm. Lancet 2009; 374(9689): 511-3.
61. Suissa S. Immortal time bias in observational studies of drug effects. Pharmacoepidemiol Drug Saf 2007; 16(3): 241-9.
62. McCandless LC, Douglas IJ, Evans SJ, Smeeth L. Cutting feedback in Bayesian regression adjustment for the propensity score. Int J Biostat 2010; 6(2): Article 16. http://www.bepress.com/ijb/vol6/iss2/16.
63. Hernán MA, Hernández-Díaz S, Werler MM, Mitchell AA. Causal knowledge as a prerequisite for confounding evaluation: an application to birth defects epidemiology. Am J Epidemiol 2002; 155(2): 176-84.
64. Greenland S, Pearl J, Robins JM. Causal diagrams for epidemiologic research. Epidemiology 1999; 10(1): 37-48.
65. Brumback BA, Hernán MA, Haneuse SJ, Robins JM. Sensitivity analyses for unmeasured confounding assuming a marginal structural model for repeated measures. Stat Med 2004; 23(5): 749-67.
66. Greenland S, Morganstern H. Matching and efficiency in cohort studies. Am J Epidemiol 1990; 131: 151-9.
67. Evans S. Commentary: matched cohorts can be useful. BMJ 2003; 326(7385): 357.
68. Farrington CP. Relative incidence estimation from case series for vaccine safety evaluation. Biometrics 1995; 51(1): 228-35.
69. Farrington CP, Whitaker HJ, Hocine MN. Case series analysis for censored, perturbed, or curtailed post-event exposures. Biostatistics 2009; 10(1): 3-16.
70. Whitaker HJ, Hocine MN, Farrington CP. The methodology of self-controlled case series studies. Stat Methods Med Res 2009; 18(1): 7-26.
71. de Vries F, de Vries C, Cooper C, Leufkens B, van Staa TP. Reanalysis of two studies with contrasting results on the association between statin use and fracture risk: the General Practice Research Database. Int J Epidemiol 2006; 35(5): 1301-8.
72. Braitman L, Rosenbaum PR. Rare outcomes, common treatments: analytic strategies using propensity scores. Ann Intern Med 2002; 137(8): 693-5.
73. Wang J, Donnan PT. Propensity score methods in drug safety studies: practice, strengths and limitations. Pharmacoepidemiol Drug Saf. 2001; 10(4): 341-4.
74. McAfee AT, Koro C, Landon J, Ziyadeh N, Walker AM. Coronary heart disease outcomes in patients receiving antidiabetic agents. Pharmacoepidemiol Drug Saf 2007; 16(7): 711-25.
75. Kenward MG, Carpenter J. Multiple imputation: current perspectives. Stat Methods Med Res 2007; 16(3): 199-218.
76. Carpenter JR, Kenward MG, White IR. Sensitivity analysis after multiple imputation under missing at random: a weighting approach. Stat Methods Med Res 2007; 16(3): 259-75.
77. Hennessy S, Berlin JA, Kinman JL, Margolis DJ, Marcus SM, Strom BL. Risk of venous thromboembolism from oral contraceptives containing gestodene and desogestrel versus levonorgestrel: a meta-analysis and formal sensitivity analysis. Contraception 2001; 64(2): 125-33.
78. Kemmeren JM, Algra A, Grobbee DE. Third generation oral contraceptives and risk of venous thrombosis: meta-analysis. BMJ 2001; 323(7305): 131-4.
79. Brewer T, Colditz G. Postmarketing surveillance and adverse drug reactions: current perspectives and future needs. JAMA 1999; 282: 824-9.
80. Temple R. Meta-analysis and epidemiologic studies in drug development and postmarketing surveillance. JAMA 1999; 281: 841-4.
81. Sutton A, Abrams K. Bayesian methods in meta-analysis and evidence synthesis. Stat Methods Med Res 2001; 10(4): 277-303.
82. Rothman, K. No adjustments are needed for multiple comparisons. Epidemiology 1990; 1: 43-6.