Sequential Statistical Methods for Prospective Postmarketing Safety Surveillance

Pharmacoepidemiology, Fifth Edition

Volumn , Issue , 2012, Pages 852-867

  Kulldorff, Martin ^a  

^a HARVARD MEDICAL SCHOOL   (United States)

Author keywords

Drug safety surveillance; Group sequential method; Pharmacovigilance; Sequential analysis; Sequential probability ratio test; Vaccine safety

Indexed keywords

EID: 84888735923     PISSN: None     EISSN: None     Source Type: Book    
DOI: 10.1002/9781119959946.ch46     Document Type: Chapter

References (53)

1. Varricchio F, Iskander J, Destefano F, Ball R, Pless R, Braun MM, et al. Understanding vaccine safety information from the Vaccine Adverse Event Reporting System. Pediatr Infect Dis J 2004; 23: 287-94.
2. Chen RT, DeStefano F, Davis RL, Jackson LA, Thompson RS, Mullooly JP, et al. The Vaccine Safety Datalink: Immunization research in health maintenance organizations in the USA. Bull World Health Organ 2000; 78: 186-94.
3. Platt R, Wilson M, Chan KA, Benner JS, Marchibroda J, McClellan M. The New Sentinel Network - improving the evidence of medical -product safety. N Engl J Med 2009; 361: 645-7.
4. Food and Drug Administration. The Future of Drug Safety - Promoting and Protecting the Health of the Public. FDA ' s Response to the Institute of Medicine ' s 2006 Report, Jan 2007. http://www.fda.gov/downloads/Drugs/ DrugSafety/PostmarketDrugSafetyInformationfor PatientsandProviders/UCM171627.pdf(accessed July 2011).
5. 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: 483-6.
6. Moore N, Kreft-Jais C, Haramburu F, Noblet C, Andrejak M, Ollagnier M, et al. 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: 513-18.
7. Bate A, Lindquist M, Edwards IR, Olsson S, Orre R, Lansner A, et al. A Bayesian neural network method for adverse drug reaction signal generation. Am J Clin Pharmacol 1998; 54: 315-21.
8. DuMouchel W. Bayesian data mining in large frequency tables with an application to the FDA spontaneous reporting system. Am Stat 1993; 53: 177-90.
9. Szarfman A, Machado SG, O'Neill RT. Use of screening algorithms and computer systems to effi ciently signal higher -than-expected combinations of drugs and events in the US FDA 's spontaneous reports database. Drug Saf 2002; 25: 381-92.
10. Davis RL, Kolczak M, Lewis E, Nordin J, Goodman M, Shay DK, et al. Active surveillance of vaccine safety: a system to detect early signs of adverse events. Epidemiology 2005; 16: 336-41.
11. Lieu TA, Kulldorff M, Davis RL, Lewis EM,Weintraub E, Yih K, et al. Real-time vaccine safety surveillance for the early detection of adverse events. Med Care 2007; 45: 89-95.
12. Wald A. Sequential tests of statistical hypotheses. Ann Math Stat 1945; 16: 117-86.
13. Wald A. Sequential Analysis. New York: Wiley, 1947.
14. Page ES. Continuous Inspection Schemes. Biometrika 1954; 41: 100-15.
15. Grigg OA, Farewell VT, Spiegelhalter DJ. Use of risk -adjusted CUSUM and RSPRT charts for monitoring in medical contexts. Stat Methods Med Res 2003; 12: 147-70.
16. Abt K. Poisson sequential sampling modifi ed towards maximal safety in adverse event monitoring. Biometrical J 1998; 40: 21-41.
17. Kulldorff M, Davis R, Kolczak M, Lewis E, Lieu T, Platt R. A maximized sequential probability ratio test for drug and vaccine safety surveillance. Seq Anal 2011; 30: 58-78.
18. Lorden G. Open-ended tests for Koopman -Darmois Families. Ann Statistics 1973; 1: 633-43.
19. Lehmann EL. Testing Statistical Hypotheses, 2nd edn. New York: Springer -Verlag, 1986.
20. Li L, Kulldorff M. A conditional maximized sequential probability ratio test for pharmacovigilance. Stat Med 2010; 29: 284-95.
21. Jennison C, Turnbull BW. Repeated confi dence intervals for group sequential clinical trials. Control Clin Trials 1984; 5: 33-45.
22. Jennison C, Turnbull BW. Group Sequential Methods with Applications to Clinical Trials. Boca Raton, FL: CRC Press, 2000.
23. Whitehead J. The Design and Analysis of Sequential Clinical Trials. New York: Wiley, 1997.
24. Armitage P. Sequential methods in clinical trials. Am J Pub Health 1958; 48: 1395-402.
25. Emerson S, Kittelson J, Gillen D. Tutorial in biostatistics: Frequentist evaluation of group sequential clinical trial designs. Stat Med 2007; 26: 5047-80.
26. Emerson S, Kittelson J, Gillen D. Bayesian evaluation of group sequential clinical trial designs. Stat Med 2007; 26: 1431-49.
27. Hocine MN, Musonda P, Andrews NJ, Farrington CP. Sequential case series analysis for pharmacovigilance. J Royal Statistical Society A, 2009; 172: 213-36.
28. Li L. A conditional sequential sampling procedure for drug safety surveillance. Stat Med 2009; 28: 3124-38.
29. Shih MC, Lai TL, Heyse JF, Chen J. Sequential generalized likelihood ratio tests for vaccine safety evaluation. Stat Med 2010; 29: 2698-708.
30. Yih WK, Nordin JD, Kulldorff M, Lewis E, Lieu TA, Shi P, et al. An assessment of the safety of adolescent and adult tetanus -diphtheria-acellular pertussis (Tdap) vaccine, using active surveillance for adverse events in the Vaccine Safety Datalink. Vaccine 2009; 27: 4257-62.
31. Belongia EA, Irving SA, Shui IM, Kulldorff M, Lewis E, Yin R, et al. Real-time surveillance to assess risk of intussusception and other adverse events after pentavalent, bovine derived rotavirus vaccine. Pediatr Infect Dis J 2010; 29: 1-5.
32. Greene SK, Kulldorff M, Lewis EM, Li R, Yin R, Weintraub ES, et al. Near real -time surveillance for infl uenza vaccine safety: proof -of-concept in the Vaccine Safety Datalink Project. Am J Epidemiol 2010; 171: 177-88.
33. Klein N, Fireman B, Yih WK, Lewis E, Kulldorff M, Ray P, et al. Measles-mumps-rubella-varicella combination vaccine and the risk of febrile seizures. Pediatrics 2010; 126: e1-8.
34. McClure DL, Glanz JM, Xu S, Hambidge SJ, Mullooly JP, Baggs J. Comparison of epidemiologic methods for active surveillance of vaccine safety. Vaccine 2008; 26: 3341-5.
35. Green SK, Kulldorff M, Yin R, Yih WK, Lieu TA, Weintraub ES, et al. Near real -time vaccine safety surveillancewith partially accrued data. Pharmacoepidemiol Drug Saf 2011; 20: 583-90.
36. Lee GM, Greene SK,Weintraub ES, Baggs J, Kulldorff M, Fireman BH, et al. on behalf of the Vaccine Safety Datalink Project. H1N1 and seasonal infl uenza vaccine in the vaccine safety datalink project. Am J Prevent Med 2011; 41: 121-8.
37. Yih WK, Kulldorff M, Fireman BH, Shui IM, Lewis EM, Klein NP, et al. Active Surveillance for adverse events: the experience of the Vaccine Safety Datalink Project. Pediatrics 2011; 127: S54-64.
38. Centers for Disease Control and Prevention (CDC), Advisory Committee on Immunization Practices (ACIP). Update: recommendations from the Advisory Committee on Immunization Practices (ACIP) regarding administration of combination MMRV vaccine. MMWR Morb Mortal Wkly Rep 2008; 57: 258-60.
39. Jacobsen SJ, Ackerson BK, Sy LS, Tran TN, Jones TL, Yao JF, et al. Observational safety study of febrile convulsion following fi rst dose MMRV vaccination in a managed care setting. Vaccine 2009; 27: 4656-61.
40. Burwen D, Sandhu S, MaCurdy T, Kelman J, Gibbs J, Garcia B, et al. Surveillance for Guillain -Barre syndrome after infl uenza vaccination among the Medicare population, 2009 -2010. Pharmacoepidemiol Drug Saf 2010; 19: S15.
41. Qiang Y, Groom A, Redd J, Haberling D, Holman R, Jim C, et al. Safety of the infl uenza A (H1N1) 2009 monovalent vaccine among the Indian health service user population. Pharmacoepidemiol Drug Saf 2010; 19: S329.
42. Enger C, Turnbull B, Gately R, Loughlin J, Chan KA. H1N1 infl uenza vaccine surveillance project. Pharmacoepidemiol Drug Saf 2010; 19: S14-15.
43. Seabroke S, Bryan P, Wise L. The use of observed vs expected analyses in real -time vaccine safety surveillance in the UK during the H1N1 pandemic. Pharmacoepidemiol Drug Saf 2010; 19: S188.
44. Seabroke S. Observed vs expected methodologies in vaccine safety surveillance: practical experience of using observed vs expected analyses for vaccine pharmacovigilance at the UK MHRA. Presentation at the 26th International Conference on Pharmacoepidemiology and Therapeutic Risk Management, Brighton, UK, 2010.
45. Brown JS, Kulldorff M, Chan KA, Davis RL, Graham D, Pettus PT, et al. Early detection of adverse drug events within population -based health networks: application of sequential testing methods. Pharmacoepidemiol Drug Saf 2007; 16: 1275-84.
46. Brown JS, Kulldorff M, Petronis KR, Reynolds R, Chan KA, Davis RL, et al. Early adverse drug event signal detection within population -based health networks using sequential methods: key methodologic considerations. Pharmacoepidemiol Drug Saf 2009; 18: 226-34.
47. Wahl PM, Schneeweiss S, Wasser TE, Eisenberg DF, Rodgers K, Gagne JJ, et al. Development of a claims -based healthcare safety sentinel system featuring signal strengthening during sequential monitoring. Pharmacoepidemiol Drug Saf 2010; 19: S80.
48. Avery TR, Vilk Y, Kulldorff M, Li L, Cheetham TC, Dublin S, et al. Prospective, active surveillance for medication safety in population -based health networks: a pilot study. Pharmacoepidemiol Drug Saf 2010; 19: S304.
49. Chen Y, Strombom I, Marmaduke D, Benichou O, Lipkovich I. The risk of infections among patients treated with TNFi: population -based continuous ADRs monitoring using the maximized sequential probability ratio testing. Pharmacoepidemiol Drug Saf 2010; 19: S5.
50. Pocock S. Group sequential methods in the design and analysis of clinical trials. Biometrika 1977; 64: 191-9.
51. O'Brien P, Fleming T. A multiple testing procedure for clinical trials. Biometrics 1979, 35: 549-56.
52. Kulldorff M. A spatial scan statistic. Commun Stat Theory Methods 1997; 26: 1481-96.
53. Kulldorff M and Information Management Systems Inc. SaTScan: Software for the Spatial and Space - time Scan Statistics, v9.0: 2010. http://www.satscan.org (accessed July 2011).