Spline-based procedures for dose-finding studies with active control

Statistics in Medicine

Volumn 34, Issue 2, 2015, Pages 232-248

  Helms, Hans Joachim ^a   Benda, Norbert ^b   Zinserling, Jörg ^b   Kneib, Thomas ^c   Friede, Tim ^a  

^a UNIVERSITY MEDICAL CENTER GÖTTINGEN   (Germany)

^b Federal Institute for Drugs and Medical Devices   (Germany)

^c UNIVERSITY OF GÖTTINGEN   (Germany)

Author keywords

Active control; Design considerations; Dose finding study; Spline interpolation

Indexed keywords

ARTICLE; BOOTSTRAPPING; CONTROL GROUP; DOSE CALCULATION; DOSE RESPONSE; PARAMETRIC TEST; PROBABILITY; SIMULATION; SPLINE BASED PROCEDURE; STATISTICAL ANALYSIS; STATISTICAL MODEL; BIOASSAY; COMPUTER SIMULATION; CONFIDENCE INTERVAL; HUMAN; METHODOLOGY; PHASE 2 CLINICAL TRIAL (TOPIC); PROCEDURES; REGRESSION ANALYSIS; STATISTICAL BIAS;

BIAS (EPIDEMIOLOGY); CLINICAL TRIALS, PHASE II AS TOPIC; COMPUTER SIMULATION; CONFIDENCE INTERVALS; DOSE-RESPONSE RELATIONSHIP, DRUG; ENDPOINT DETERMINATION; HUMANS; LIKELIHOOD FUNCTIONS; LOGISTIC MODELS; REGRESSION ANALYSIS; RESEARCH DESIGN;

EID: 84916233588     PISSN: 02776715     EISSN: 10970258     Source Type: Journal    
DOI: 10.1002/sim.6320     Document Type: Article

References (32)

1. Ruberg SJ. Dose response studies I. Some design considerations. Journal of Biopharmaceutical Statistics 1995; 5(1):1-14.
2. Ruberg SJ. Dose response studies II. Analysis and interpretation. Journal of Biopharmaceutical Statistics 1995; 5(1):15-42.
3. Bornkamp B, Bretz F, Dmitrienko A, Enas G, Gaydos B, Hsu C-H, König F, Krams M, Liu Q, Neuenschwander B, Parke T, Pinheiro J, Roy A, Sax R, Shen F. Innovative approaches for designing and analyzing adaptive dose-ranging trials. Journal of Biopharmaceutical Statistics 2007; 17(6):965-995.
4. Bretz F, Pinheiro JC, Branson M. Combining multiple comparisons and modeling techniques in dose-response studies. Biometrics 2005; 61(3):738-748.
5. Pinheiro J, Bornkamp B, Bretz F. Design and analysis of dose-finding studies combining multiple comparisons and modeling procedures. Journal of Biopharmaceutical Statistics 2006; 16(5):639-656.
6. Bretz F, Hsu J, Pinheiro J, Liu Y. Dose finding - a challenge in statistics. Biometrical Journal 2008; 50(4):480-504.
7. Pinheiro J, Bretz F, Branson M. Analysis of Dose-Response Studies-Modeling Approaches, Statistics for Biology and Health. Springer New York, 2006.
8. Hsu J, Berger R. Stepwise confidence intervals without multiplicity adjustment for dose-response and toxicity studies. Journal of the American Statistical Association 1999; 94(446):468-482.
9. Tamhane AC, Logan B. Multiple test procedures for identifying the minimum effective and maximum safe doses of a drug. Journal of the American Statistical Association 2002; 97(457):293-301.
10. Morales KH, Ibrahim JG, Chen CJ, Ryan LM. Bayesian model averaging with applications to benchmark dose estimation for arsenic in drinking water. Journal of the American Statistical Association 2006; 101(473):9-17.
11. Budtz-Jørgensen E. Estimation of the benchmark dose by structural equation models. Biostatistics 2007; 8(4):675-688.
12. Källén A, Larsson P. Dose response studies: how do we make them conclusive?. Statistics in Medicine 1999; 18(6):629-641.
13. Dette H, Kiss C, Benda N, Bretz F. Optimal designs for dose finding studies with an active control. Journal of the Royal Statistical Society: Series B (Statistical Methodology) 2014; 76(1):265-295.
14. Kirby S, Colman P, Morris M. Adaptive modelling of dose-response relationships using smoothing splines. Pharmaceutical Statistics 2009; 8(4):346-355.
15. Dilleen M, Heimann G, Hirsch I. Non-parametric estimators of a monotonic dose-response curve and bootstrap confidence intervals. Statistics in Medicine 2003; 22:869-882.
16. de Boor C. A Practical Guide to Splines, Applied Mathematical Sciences. Springer, 2001.
17. Efron B. Bootstrap methods: another look at the jackknife. The Annals of Statistics 1979; 7(1):1-26 (English).
18. Ferguson T. A Course in Large Sample Theory. Chapman & Hall, 1996.
19. Krum H, Viskoper RJ, Lacourciere Y, Budde M, Charlon V. The effect of an endothelin-receptor antagonist, bosentan, on blood pressure in patients with essential hypertension. New England Journal of Medicine 1998; 338(12):784-791.
20. Chapple CR, Araño P, Bosch JLHR, DeRidder D, Kramer AEJL, Ridder AM. Solifenacin appears effective and well tolerated in patients with symptomatic idiopathic detrusor overactivity in a placebo- and tolterodine-controlled phase 2 dose-finding study. BJU International 2004; 93(1):71-77.
21. Nauck M, Frid A, Hermansen K, Shah N, Tankova T, Mitha I, Zdravkovic M, Düring M, Matthews D. Efficacy and safety comparison of liraglutide, glimepiride, and placebo, all in combination with metformin, in type 2 diabetes: the lead (liraglutide effect and action in diabetes)-2 study. Diabetes Care 2009; 32:84-90.
22. Pawitan Y. In All Likelihood: Statistical Modelling and Inference Using Likelihood. Oxford University Press, 2001.
23. Dragalin V, Hsuan F, Padmanabhan SK. Adaptive designs for dose-finding studies based on sigmoid e max model. Journal of Biopharmaceutical Statistics 2007; 17(6):1051-1070.
24. Kirby S, Brain P, Jones B. Fitting emax models to clinical trial dose-response data. Pharmaceutical Statistics 2011; 10(2):143-149.
25. Jones B, Layton G, Richardson H, Thomas N. Model-based Bayesian adaptive dose-finding designs for a phase 2 trial. Statistics in Biopharmaceutical Research 2011; 3(2):276-287.
26. Helms HJ, Benda N, Friede T. Point and interval estimators of the target dose in clinical dose-finding studies with active control. Journal of Biopharmaceutical Statistics 2014. [Epub ahead of print].
27. Preacher KJ, Rucker DD, Hayes AF. Addressing moderated mediation hypotheses: theory, methods, and prescriptions. Multivariate Behavioral Research 2007; 42(1):185-227.
28. Friede T, Mitchell C, Müller-Velten G. Blinded sample size reestimation in non-inferiority trials with binary endpoints. Biometrical Journal 2007; 49(6):903-916.
29. Hall CA. On error bounds for spline interpolation. Journal of Approximation Theory 1968; 1:209-218.
30. Hall CA, Meyer WW. Optimal error bounds for cubic spline interpolation. Journal of Approximation Theory 1976; 16:105-122.
31. Bornkamp B, Bretz F, Dette H, Pinheiro J. Response-adaptive dose-finding under model uncertainty. The Annals of Applied Statistics 2011; 5(2B):1611-1631.
32. Dette H, Bretz F, Pepelyshev A, Pinheiro J. Optimal designs for dose-finding studies. Journal of the American Statistical Association 2008; 103(483):1225-1237.