The practice of pre-marketing safety assessment in drug development

Journal of Biopharmaceutical Statistics

Volumn 23, Issue 1, 2013, Pages 3-25

  Chuang Stein, Christy ^a   Xia, H Amy ^b  

^a PFIZER INC   (United States)

^b AMGEN INC   (United States)

Author keywords

Designated medical events; Graphical display of safety data; Multiplicity adjustment; Program safety analysis plan; Targeted medical event; Three tier approach

Indexed keywords

ACCURACY; ALGORITHM; BAYES THEOREM; BIOSTATISTICS; CLINICAL PRACTICE; CONFIDENCE INTERVAL; COOPERATION; DRUG DEVELOPMENT; DRUG INDUSTRY; DRUG LABELING; DRUG MARKETING; DRUG SAFETY; HUMAN; INFERENTIAL STATISTICS; INFORMATION PROCESSING; KNOWLEDGE; META ANALYSIS (TOPIC); ORGANIZATIONAL STRUCTURE; PATIENT SAFETY; PATTERN RECOGNITION; PHASE 3 CLINICAL TRIAL (TOPIC); POLICY; POST HOC ANALYSIS; PRIORITY JOURNAL; PROBABILITY; RECOGNITION; REGULATORY MECHANISM; REVIEW; RISK ASSESSMENT; RISK MANAGEMENT; SIMULATION; STATISTICAL ANALYSIS; STATISTICAL SIGNIFICANCE;

CLINICAL TRIALS AS TOPIC; DRUG DISCOVERY; DRUG INDUSTRY; HUMANS; MARKETING; PATIENT SAFETY; PHARMACEUTICAL PREPARATIONS;

EID: 84873297083     PISSN: 10543406     EISSN: 15205711     Source Type: Journal    
DOI: 10.1080/10543406.2013.736805     Document Type: Review

References (31)

1. Amit, O., Heiberger, R. M., Lane, P. W. (2008). Graphical approaches to the analysis of safety data from clinical trials. Pharmaceutical Statistics 7:20-35. (Pubitemid 351385323)
2. Benjamini, Y., Hochberg, Y. (1995). Controlling the false discovery rate: A practical and powerful approach to multiple testing. Journal of the Royal Statistical Society, Series B 57(1):289-300.
3. Berry, S. M., Berry, D. A. (2004). Accounting for multiplicities in assessing drug safety: a three-level hierarchical mixture model. Biometrics 60:418-426. (Pubitemid 38780201)
4. Berry, S. M., Carlin, B. P., Lee, J. J., Muller, P. (2010). Bayesian Adaptive Methods for Clinical Trials. Boca Raton, FL: Chapman and Hall/CRC Press.
5. Chuang-Stein, C., Le, V., Chen, W. (2001). Recent advancements in the analysis and presentation of safety data. Drug Information Journal 35:377-397. (Pubitemid 32524891)
6. Chuang-Stein, C., Mohberg, N. R. (1993). A unified approach to analyze safety data in clinical trials. In: Gilbert, G., ed. Drug Safety Assessment in Clinical Trials, New York, NY: Marcel Dekker, chap. 9.
7. Chuang-Stein, C. (1994). A new proposal for benefit-less-risk analysis in clinical trials. Controlled Clinical Trials 15:30-43. (Pubitemid 24078603)
8. Chuang-Stein, C., Entsuah, R., Pritchett, Y. (2008). Measures for conducting comparative benefit:risk assessment. Drug Information Journal 42(3):223-233. (Pubitemid 351775941)
9. Cooper, A. J. P., Lettis, S., Chapman, C. L., Evans, S. J., Waller, P. C., Shakir, S., Payvandi, N., Murray, A. B. (2008). Developing tools for the safety specification in risk management plans: Lesson learned from a pilot project. Pharmacoepidemiology and Drug Safety 17:445-454. (Pubitemid 351722030)
10. Council for International Organizations of Medical Sciences (CIOMS) Working Group VI. (2005). Management of Safety Information From Clinical Trials. Geneva: CIOMS.
11. Crowe, B. J., Xia, H. A., Berlin, J. A., Watson, D. J., Shi, H., Lin, S. L., Kuebler. J. Schriver, R. C., Santanello, N. C., Rochester, G., Porter, J. B., Oster, M., Mehrotra, D. V., Li, Z., King, E. C., Harpur, E. S., Hall, D. B. (2010). Recommendations for safety planning, data collection, evaluation and reporting during drug, biologic and vaccine development: A report of the safety planning, evaluation, and reporting team. Clinical Trials 6(5):430-440.
12. Duke, S. (2008). A Case Study for More Effective use of Graphics. Presented at the FDA/Industry Statistics Workshop. Washington, DC, September 17.
13. Gould, A. L. (2002). Drug Safety Evaluation in and After Clinical Trials. Presented at the Deming Conference. Atlanta City, NJ, December 3.
14. Heyse, J., et al. (2008). Evaluation of the safety of rotavirus vaccine: The REST of the story. Clinical Trial 5:131-139. (Pubitemid 351682523)
15. Hughes, S., Hughes, A., Brothers, C., Spreen, W., Thorborn, D., on behalf of the CNA106030 Study Team. (2008). PREDICT-1 (CNA106030): The first powered, prospective trial of pharmacogenetic screening to reduce drug adverse events. Pharmaceutical Statistics 7:121-129. (Pubitemid 351943640)
16. International Conference on Harmonisation (ICH). (1998). ICH Harmonised Tripartite Guideline. Statistical Principles for Clinical Trials E9. Available at http://www.ich.org/products/guidelines/efficacy/article/efficacy-guidelines. html (accessed 30 April 2012).
17. International Conference on Harmonisation (ICH). (2005). Guidance for Industry: E14 Clinical Evaluation of QT/QTc Interval Prolongation and Proarrhythmic Potential for Non Antiarrhythmic Drugs. Available at: http://www.fda.gov/downloads/RegulatoryInformation/Guidances/ucm129357.pdf (accessed 29 September 2011).
18. Lewis, S., Clarke, M. (2001). Forest plots: Trying to see the wood and the trees. British Medical Journal 322:1479-80. (Pubitemid 32575420)
19. Mallal, S., et al. (2008). HLA-B*5701 screening for hypersensitivity to Abacavir. New England Journal of Medicine 358:568-579.
20. Mehrotra, D. V. (2008). Recommendations for Analyses of Adverse Events in Randomized Clinical Trials. Presented at the DIA/FDA/PhRMA Drug Safety Conference; Washington DC, October 14.
21. Mehrotra, D. V., Heyse, J. F. (2004). Use of the false discovery rate for evaluating clinical safety data. Statistical Methods in Medical Research 13:227-38. (Pubitemid 38744605)
22. O'Neill, R. T. (1988). Assessment of safety. In: Peace, K. E., ed. Biopharmaceutical Statistics for Drug Development. New York, NY: Marcel Dekker. pp. 543-604.
23. Pitts, N. E. (1993). Safety surveillance. In: Gilbert, G., ed. Drug Safety Assessment in Clinical Trials. New York, NY: Marcel Dekker, chap. 14.
24. Rochester, C. G. (2009). Lifecycle Planning for Safety Evaluation. Presented at the FDA/Industry Statistics Workshop, Washington, DC, September 25.
25. Temple, R. (2011). A regulator's view of CER. Clinical Trial, published electronically on October 5 at http://ctj.sagepub.com/content/early/2011/10/05/ 1740774511422548.
26. U.S. Food and Drug Administration. (2009). Guidance for industry. Drug-induced Liver Injury: Premarketing Clinical Evaluation. Available at: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/ Guidances/UCM174090.pdf (accessed 29 September 2011).
27. U.S. Food and Drug Administration. (2010). Investigational New Drug Safety Reporting Requirements for Human Drug and Biological Products and Safety Reporting Requirements for Bioavailability and Bioequivalence Studies in Humans (final rule). 21 CFR Parts 312 and 320, published in the Federal Register, Vol. 75, No. 188, September 29, 2010.
28. U.S. Food and Drug Administration. (2010). Draft Guidance for Industry and Investigators. Safety Reporting Requirements for INDs and BA/BE Studies. Available at: http://www.fda.gov/downloads/Drugs/-/Guidances/UCM227351.pdf (accessed 29 November, 2012).
29. Vesikari, T, et al. (2006). Safety and efficacy of a pentavalent human-bovine (WC3) reassortant rotavirus vaccine. New England Journal of Medicine 354:23-22.
30. Xia, H. A., Crowe, B. J., Schriver, R. C., Oster, M., Hall, D. B. (2011a). Planning and core analyses for periodic aggregate safety data reviews. Clinical Trials 8(2):175-82.
31. Xia, H. A., Ma, H., Carlin, B. P. (2011b). Bayesian hierarchical modeling for detecting safety signals in clinical trials. Journal of Biopharmaceutical Statistics 21(5):1006-1029.