Statistical modeling for Bayesian extrapolation of adult clinical trial information in pediatric drug evaluation

Pharmaceutical Statistics

Volumn 16, Issue 4, 2017, Pages 232-249

  Gamalo Siebers, Margaret ^a   Savic, Jasmina ^b   Basu, Cynthia ^c   Zhao, Xin ^d   Gopalakrishnan, Mathangi ^e   Gao, Aijun ^f   Song, Guochen ^g   Baygani, Simin ^a   Thompson, Laura ^h   Xia, H Amy ⁱ   Price, Karen ^a   Tiwari, Ram ^j   Carlin, Bradley P ^c  

^a ELI LILLY AND COMPANY   (United States)

^b JS Regulatory   (Germany)

^c UNIVERSITY OF MINNESOTA   (United States)

^d JOHNSON AND JOHNSON PHARMACEUTICAL RESEARCH AND DEVELOPMENT   (United States)

^e UNIVERSITY OF MARYLAND   (United States)

^f Chiltern International Ltd   (United States)

^g BIOGEN   (United States)

^h CENTER FOR DEVICES AND RADIOLOGICAL HEALTH   (United States)

ⁱ AMGEN INC   (United States)

^j CENTER FOR DRUG EVALUATION AND RESEARCH   (United States)

more..

Author keywords

commensurate prior; effective sample size; exchangeability; extrapolation; hierarchical model; model fit; power prior

Indexed keywords

ANTICONVULSIVE AGENT; INFLIXIMAB; OXCARBAZEPINE;

ARTICLE; BAYES THEOREM; CHILD; CROHN DISEASE; DATA EXTRACTION; DRUG APPROVAL; DRUG INDICATION; DRUG INFORMATION; DRUG LABELING; DRUG RESEARCH; DRUG SAFETY; EXTRAPOLATION; FOCAL EPILEPSY; HUMAN; MARKOV CHAIN; MINIMUM PLASMA CONCENTRATION; MONTE CARLO METHOD; RHEUMATOID ARTHRITIS; SAMPLE SIZE; SIMULATION; STATISTICAL MODEL; ULCERATIVE COLITIS; ADULT; CLINICAL TRIAL (TOPIC); DRUG SCREENING; METHODOLOGY;

ADULT; BAYES THEOREM; CLINICAL TRIALS AS TOPIC; COLITIS, ULCERATIVE; DRUG EVALUATION; HUMANS; MODELS, STATISTICAL; RESEARCH DESIGN;

EID: 85018379336     PISSN: 15391604     EISSN: 15391612     Source Type: Journal    
DOI: 10.1002/pst.1807     Document Type: Article

References (66)

1. R. English, Y. Lebovitz, R. Griffin, Institute of Medicine (US) Forum on Drug Discovery Development and Translation. Transforming clinical research in the United States. Challenges and opportunities: Workshop summary, 2010.
2. US Congress, Best pharmaceuticals for children act, Available from: http://www.fda.gov/opacom/laws/pharmkids/pharmkids.html, 2002 (accessed: August 1, 2016).
3. US Congress, Pediatric research equity act, Available from: http://frwebgate.access.gpo.gov/cgi-bin/getdoc.cgi?dbname=108_cong_public_laws&docid=f:publ155, 2003 (accessed: August 1, 2016).
4. S. G. Selevan, C. A. Kimmel, P. Mendola, Environmental Health Perspectives 2000, 108(Suppl 3), 451.
5. M. D. Miller, M. A. Marty, A. Arcus, J. Brown, D. Morry, M. Sandy, International Journal of Toxicology 2002, 21(5), 403.
6. M. Roth-Cline, J. Gerson, P. Bright, C. S. Lee, R. M. Nelson, in Pediatric Clinical Pharmacology, Springer, Berlin Heidelberg 2011, 219.
7. P. H. Y. Caldwell, S. B. Murphy, P. N. Butow, J. C. Craig, The Lancet 2004, 364(9436), 803.
8. European Medicines Agency, Concept paper on extrapolation of efficacy and safety in medicine development, Available from: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2013/04/WC500142358.pdf, 2013 (accessed: August 1, 2016).
9. European MA, Reflection paper on extrapolation of efficacy and safety in pediatric medicine development, Available from: http://www.ema.europa.eu/docs/en_GB/document_library/Regulatory_and_procedural_guideline/2016/04/WC500204187.pdf, 2016 (accessed: August 1, 2016).
10. Y. Mulugeta, J. S. Barrett, R. Nelson, A. T. Eshete, A. Mushtaq, L. Yao, N. Glasgow, A. E. Mulberg, D. Gonzalez, D. Green, J. Florian, The Journal of Clinical Pharmacology 2016, 56(11), 1326.
11. J. Dunne, W. J. Rodriguez, M. D. Murphy, B. N. Beasley, G. J. Burckart, J. D. Filie, L. L. Lewis, H. C. Sachs, P. H. Sheridan, P. Starke, L. P. Yao, Pediatrics 2011, 128(5), e1242.
12. H. Sachs, Pediatric extrapolation, Available from: http://www.fda.gov/downloads/NewsEvents/MeetingsConferencesWorkshops/UCM415474.pdf, 2014 (accessed: August 1, 2016).
13. B. P. Carlin, T. A. Louis, Bayesian Methods for Data Analysis, 3rd ed., CRC Press, Boca Raton, FL 2009.
14. S. M. Berry, B. P. Carlin, J. J. Lee, P. Muller, Bayesian Adaptive Methods for Clinical Trials, CRC Press, Boca Raton, FL 2010.
15. S. N. Goodman, J. T. Sladky, Clinical Trials 2005, 2(4), 305.
16. D. A. Schoenfeld, H. Zheng, D. M. Finkelstein, Clinical Trials 2009, 6(4), 297–304.
17. M. Gastonguay, Application of population pharmacokinetic and pharmacodynamic modeling and simulation n, Available from: http://www2.cde.org.tw/action/uploadfile/2012/0327/GastonguayTaiwanNatlUExamples.pdf, 2009 (accessed: August 1, 2016).
18. I. Wadsworth, L. V. Hampson, T. F. Jaki, Statistical Methods in Medical Research 2016, 0962280216631359.
19. J. T. Mondick, L. Gibiansky, M. R. Gastonguay, J. M. Skolnik, M. Cole, G. J. Veal, A. V. Boddy, A. V. Boddy, P. C. Adamson, J. C. Barrett, The Journal of Clinical Pharmacology 2008, 48(1), 35.
20. U.S. Department of Health and Human Services, Guidance for the use of Bayesian statistics in medical device clinical trials, Available from: www.fda.gov/medicaldevices/deviceregulationandguidance/guidancedocuments/ucm071072.htm, 2010 (accessed: August 1, 2016).
21. U.S. Department of Health and Human Services, Leveraging existing clinical data for extrapolation to pediatric uses of medical devices, Available from: http://www.fda.gov/ucm/groups/fdagov-public/@fdagov-meddev-gen/documents/document/ucm444591.pdf, 2015 (accessed: August 1, 2016).
22. L. Thompson, G. Pennello, Technical Report, Food and Drug Administration, Center for Devices and Radiological Health 2015.
23. CMPH, Guideline on clinical trials in small populations, 2006.
24. T. Bayes, Philosophical Transactions of the Royal Society of London 1765, 54, 296.
25. A. Birnbaum, Journal of the American Statistical Association 1962, 57(298), 269.
26. D. J. Spiegelhalter, N. G. Best, B. P. Carlin, A. Van Der Linde, Journal of the Royal Statistical Society: Series B (Statistical Methodology) 2002, 64(4), 583.
27. D. Lunn, C. Jackson, N. Best, A. Thomas, D. J. Spiegelhalter, The Bugs Book: A Practical Introduction to BAyesian Analysis, CRC Press, Boca Raton, FL 2012.
28. J. M. Bernardo, Far East Journal of Mathematical Sciences 1996, 4, 111–122.
29. S. Muthukumarana, R. C. Tiwari, Statistical Methods in Medical Research 2012, 25(1), 352.
30. H. Schmidli, S. Gsteiger, S. Roychoudhury, A. O'Hagan, D. Spiegelhalter, B. Neuenschwander, Biometrics 2014, 70(4), 1023.
31. M. West, Journal of the Royal Statistical Society, Ser. B 1993, 55, 409.
32. J. M. Bernardo, A. F. M. Smith, Bayesian Theory, Wiley, New York 1994.
33. T. S. Ferguson, The Annals of Statistics 1974, 2(4), 615.
34. A. O'Hagan, L. Pericchi, Brazilian Journal of Probability and Statistics 2012, 26(4), 372.
35. J. G. Ibrahim, M. H. Chen, Statistical Science 2000, 15, 46.
36. M. A. Gamalo, R. C. Tiwari, L. M. LaVange, Pharmaceutical Statistics 2014, 13(1), 25–40.
37. Y. Duan, K. Ye, E. P. Smith, Environmetrics 2006, 17(1), 95.
38. H. Zhao, B. P. Hobbs, H. Ma, Q. Jiang, B. P. Carlin, Research report, University of Minnesota Division of Biostatistics 2015.
39. B. P. Hobbs, B. P. Carlin, S. J. Mandrekar, D. J. Sargent, Biometrics 2011, 67(3), 1047.
40. B. P. Hobbs, D. J. Sargent, B. P. Carlin, Bayesian Analysis 2012, 7(3), 639.
41. J. A. Fúquene, J. D. Cook, L. R. Pericchi, Bayesian Analysis 2009, 4(4), 817.
42. T. A. Murray, B. P. Hobbs, T. C. Lystig, B. P. Carlin, Biometrics 2014, 70, 185.
43. T. A. Murray, B. P. Hobbs, B. P. Carlin, Annals of Applied Statistics 2015, 9, 1549.
44. D. J. Spiegelhalter, K. R. Abrams, J. P. Myles, Bayesian Approaches to Clinical Trials and Health-Care Evaluation, Wiley, New York 2004.
45. S. Morita, P. F. Thall, P. Müller, Biometrics 2008, 64(2), 595.
46. D. Malec, Statistics in Medicine 2001, 20(12), 1811.
47. A. Gelman, D. B. Rubin, Statistical Science 1992, 7, 457.
48. A. E. Raftery, S. M. Lewis, Statistical Science 1992, 7, 493.
49. M. Plummer, N. Best, K. Cowles, K. Vines, R News 2006, 6, 7.
50. Stan Development Team, Stan: A C++ library for probability and sampling, version 2.8.0, Available from: http://mc-stan.org/, 2015 (accessed: August 1, 2016).
51. M. Plummer, Jags: Just another Gibbs sampler, Available from: http://mcmc-jags.sourceforge.net/, 2004 (accessed: August 1, 2016).
52. A. E. Gelfand, D. K. Dey, H. Chang, Bayesian Statistics 4 1992, 147.
53. A. E. Gelfand, D. K. Dey, Journal of the Royal Statistical Society, Ser. B 1994, 56, 501.
54. European Parliament and of the Council of 12 December 2006, Regulation (EC) No 1901/2006 of the European Parliament and of the Council of 12 December 2006, Available from: http://ec.europa.eu/health/files/eudralex/vol-1/reg_2006_1901/reg_2006_1901_en.pdff, 2016 (accessed: August 1, 2016).
55. K. Viele, S. Berry, B. Neuenschwander, B. Amzal, F. Chen, N. Enas, B. Hobbs, J. G. Ibrahim, N. Kinnersley, S. Lindborg, S. Micallef, S. Roychoudhury, L. Thompson, Pharmaceutical Statistics 2014, 13(1), 41.
56. C. Basu, M. Ahmed, R. V. Kartha, R. C. Brundage, G. V. Raymond, J. C. Cloyd, B. P. Carlin, Journal of Biopharmaceutical Statistics 2017, 26(6), 1025.
57. J. Hyams, L. Damaraju, M. Blank, J. Johanns, C. Guzzo, H. S. Winter, S. Kugathasan, S. Cohen, J. Markowitz, J. C. Escher, G. Veereman-Wauters, W. Crandall, R. Baldassano, A. Griffiths, T72 Study Group, Clinical Gastroenterology and Hepatology 2012, 10(4), 391.
58. J. S. Hyams, T. Lerer, A. Griffiths, M. Pfefferkorn, M. Stephens, J. Evans, A. Otley, R. Carvalho, D. Mack, A. Bousvaros, J. Rosh, A. Grossman, G. Tomer, M. Kay, W. Crandall, M. Oliva-Hemker, D. Keljo, N. LeLeiko, J. Markowitz, Pediatric Inflammatory Bowel Disease Collaborative Research Group, The American Journal of Gastroenterology 2010, 105(6), 1430.
59. U.S. Department of Health and Human Services, NDA 21014/S003 Pharmacology and Biopharmaceutical Review(s), Available from: http://www.accessdata.fda.gov/drugsatfda_docs/nda/2003/021014_S003_TRILEPTAL%20TABLETS_BIOPHARMR.pdf, 2003 (accessed: August 1, 2016).
60. P. News, Journal of Pediatric Pharmacology and Therapeutics 2016, 21(1), 98.
61. A. L. Gould, T. Jin, L. X. Zhang, W. W. B. Wang, Journal of Biopharmaceutical Statistics 2012, 22(5), 916.
62. U.S. Department of Health and Human Services, General Clinical Pharmacology Considerations for Pediatric Studies for Drugs and Biological Products, Available from: http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm425885.pdf, 2014 (accessed: August 1, 2016).
63. European Medicines Agency, EMA Annual Report 2014, Available from: http://www.ema.europa.eu/docs/en_GB/document_library/Annual_report/2015/04/WC500186306.pdf, 2014 (accessed: August 1, 2016).
64. European Medicines Agency and U.S. Food and Drug Administration, General Principles, EMA-FDA Parallel Scientific Advice, Available from: http://www.ema.europa.eu/docs/en_GB/document_library/Other/2009/11/WC500014868.pdf, 2009 (accessed: August 1, 2016).
65. European Medicines Agency and U.S. Food and Drug Administration, Principles of Interactions: Between EMEA and FDA Pediatric Therapeutics, Available from: http://www.ema.europa.eu/docs/en_GB/document_library/Other/2009/12/WC500017969.pdf, 2007 (accessed: August 1, 2016).
66. European Medicines Agency, Gaucher disease: A strategic collaborative approach from EMA and FDA, Available from: http://www.ema.europa.eu/docs/en_GB/document_library/Regulatory_and_procedural_guideline/2014/05/WC500166587.pdf, 2014 (accessed: August 1, 2016).