Adaptive seamless designs have been considered as one possible way to shorten the time and patient exposure necessary to discover, develop, and demonstrate the benefits of a new drug. We introduce the concept of adaptive designs and describe the current statistical methodologies that relate to adaptive seamless designs. We also describe the decision process involved with seamless designs and present some illustrative examples.
DragalinV.Adaptive designs: terminology and classification. Drug Inf J.2006; 40: 425–436.
2.
QuinlanJ.A., KramsM.Implementing adaptive designs: logistical and operational considerations. Drug Inf J.2006; 40: 437–444.
3.
BechhoferR.E., KieferJ., SobelM.Sequential Identification and Ranking Problems.Chicago: University of Chicago Press; 1968.
4.
PaulsonE.A selection procedure for selecting the population with the largest mean from k normal populations. Ann Math Stat.1964; 35: 174–180.
5.
ThallP.F., SimonR., EllenbergS.S.A two-stage design for choosing among several experimental treatments and a control in clinical trials. Biometrics.1989; 45: 537–547.
6.
SchaidD.J., WieandS., TherneauT.M.Optimal two stage screening designs for survival comparisons. Biometrika.1990; 77: 659–663.
7.
StallardN., ToddS.Sequential designs for phase III clinical trials incorporating treatment selection. Stat Med.2003; 22: 689–703.
HellmichM.Monitoring clinical trials with multiple arms. Biometrics.2001; 57: 892–898.
10.
BischoffW., MillerF.Adaptive two-stage test procedures to find the best treatment in clinical trials. Biometrika.2005; 92: 197–212.
11.
ToddS., StallardN.A new clinical trial design combining phases II and III: sequential designs with treatment selection and a change of end-point. Drug Inf J.2005; 39: 109–118.
12.
BauerP., KöhneK.Evaluation of experiments with adaptive interim analyses. Biometrics.1994; 50: 1029–1041.
13.
BauerP., KieserM.Combining different phases in the development of medical treatments within a single trial. Stat Med.1999; 18: 1833–1848.
14.
BrannathW., PoschM., BauerP.Recursive combination tests. J Am Stat Assoc.2002; 97: 236–244.
15.
MüllerH.H., SchäferH.Adaptive group sequential designs for clinical trials: combining the advantages of adaptive and classical group sequential approaches. Biometrics.2001; 57: 886–819.
16.
LiuQ., PledgerG.W.Phase II and III combination designs to accelerate drug development. J Am Stat Assoc.2005; 100: 493–502.
17.
PoschM., KoenigF., BrannathW., Dunger-BaldaufC., BauerP.Testing and estimation in flexible group sequential designs with adaptive treatment selection. Stat Med.2005; 24: 3697–3714.
18.
BauerP., EinfaltJ.Application of adaptive designs-a review. Biometrical J.2006, 48: 14.
19.
KellyP.J., StallardN., ToddS.An adaptive group sequential design for phase II/III clinical trials that select a single treatment from several. J Biopharm Stat.2005; 15: 641–658.
20.
InoueL.Y.T., ThallP.F., BerryD.A.Seamlessly expanding a randomized phase II trial to phase III. Biometrics.2002; 58: 823–831.
21.
BerryD.A., MüllerP., GrieveA.P.Adaptive Bayesian designs for dose-ranging drug trials. In Case Studies in Bayesian Statistics V. Lecture Notes in Statistics.New York: Springer; 2002: 162: 99–181.
22.
BretzF., PinheiroJ.C., BransonM.Combining multiple comparisons and modeling techniques in dose response studies. Biometrics.2005; 61: 738–748.
23.
CoburgerS., WassmerG.Sample size reassessment in adaptive clinical trials using a bias corrected estimate. Biometrical J.2003; 45: 812–825.
24.
BrannathW., KönigF., BauerP.Improved repeated confidence bounds in trials with a maximal goal. Biomedical J.2003; 45: 311–324.
25.
SampsonA.R., SillM.W.Drop-the-losers design: normal case. Biometrical J.2005; 47: 257–281.
26.
StallardN., ToddS.Point estimates and confidence regions for sequential trials involving selection. J. Statist Plan Inference.2005; 135: 402–419.
27.
US Food and Drug Administration.Guidance for Clinical Trial Sponsors. Establishment and Operation of Clinical Trial Data Monitoring Committees.Rockville MD: FDA; 2006. Available at: http://www.fda.gov/cber/gdlns/clindatmon.htm. Accessed August 15, 2006.