A variety of response-adaptive randomization procedures have been proposed in literature
assuming binary outcomes. However, the list is not so long for continuous outcomes though
many real clinical trials deal with continuous treatment responses. In this paper, we
attempt to explore the available procedures together with a comparison of their
performances. Some real-life adaptive trial is also reviewed.
ConnorEMSperlingRSGelberR. Reduction of maternal-infant transmission
of human immunodeficiency virus of type 1 with zidovudine treatment.
N Eng J Med1994; 331:
1173–1180.
YaoQWeiLJ. Play the winner for phase II/III clinical
trials. Stat Med1996; 15:
2413–2423.
4.
WeiLJDurhamS. The randomized play-the-winner rule in medical
trials. J Am Stat Assoc1978; 73:
840–843.
5.
DragalinV. Adaptive designs: terminology and
classifications. Drug Inform J2006; 40:
425–435.
6.
ThompsonWR. On the likelihood that one unknown probability
exceeds another in the view of the evidence of the two samples.
Biometrika193325: 275–294.
7.
RobbinsH. Some aspects of the sequential design of
experiments. Bull Am Math Soc1952; 58:
527–535.
8.
AnscombeFJ. Sequential medical trials.
J Am Stat Assoc1963; 58:
365–384.
9.
ColtonT. A model for selecting one of two medical
treatments. J Am Stat Assoc1963; 58:
388–400.
10.
RosenbergerWFLachinJM. The use of response-adaptive designs in clinical
trials. Control Clin Trials1993; 14:
471–484.
11.
RosenbergerWF. New directions in adaptive
designs. Stat Sci1996; 11:
137–149.
12.
RosenbergerWFLachinJL. Randomisation in clinical trials: theory and
practice, New York:
Wiley, 2002.
13.
ChowS-CChangM. Adaptive design methods in clinical trials,
Boca Raton, FL: Chapman and
Hall/CRC, 2006.
14.
HuFRosenbergerWF. The theory of response adaptive randomization in
clinical trials, Hoboken, NJ:
Wiley Interscience, 2006.
15.
ZelenM. Play the winner rule and the controlled clinical
trial. J Am Stat Assoc1969; 64:
131–146.
16.
DurhamSDFlournoyNLiW. Sequential designs for maximizing the probability
of a favorable response. Can J Stat1998; 3:
479–495.
17.
IvanovaA. A play-the-winner type urn model with reduced
variability. Metrika2003; 58:
1–13.
18.
ZhangLXChanWSCheungSH. A generalized drop-the-loser urn for
clinical trials with delayed responses. Stat Sin2007; 17:
387–409.
19.
BiswasA. Adaptive designs in phase III clinical trials:
controversies and progress. Stat Meth Med Res2001; 10:
353–364.
20.
WilsonTWLacourcièreYBarnesCC. The antihypertensive efficacy of losartan and
amlodipine assessed with office and ambulatory blood pressure
monitoring. Can Med Assoc J1998; 8:
469–476.
21.
LooCMitchellPSachdevP. Double-blind controlled investigation of
transcranial magnetic stimulation for the treatment of resistant major
depression. Am J Psychiatry1999; 156:
946–948.
22.
TamuraRNFariesDEAndersenJS. A case study of an adaptive clinical trial
in the treatment of out-patients with depressive disorder. J Am
Stat Assoc1994; 89:
768–776.
23.
DworkinRHCorbinAEYoungJP. Pregabalin for the treatment of posther
petic neuralgia. A randomized, placebo-controlled trial.
Neurology2003; 60:
1274–1283.
24.
ZhangLXRosenbergerWF. Response-adaptive randomization for clinical
trials with continuous outcomes. Biometrics2006; 62:
562–569.
25.
BandyopadhyayUBiswasABhattacharyaR. Kernel based response-adaptive design for
continuous responses. Commun Stat: Theor Meth2008; 38:
2691–2705.
26.
BandyopadhyayUBiswasABhattacharyaR. A new response-adaptive design for continuous
treatment responses for phase III clinical trials. J Stat Plann
Infer2011; 141:
2256–2265.
27.
FuXShenZChenY. Randomised placebo-controlled trial of use
of topical recombinant bovine basic fibroblast growth factor for second-degree
burns. Lancet1998; 352:
1661–1664.
28.
BandyopadhyayUBiswasA. Delayed response in randomized play-the-winner
rule: a decision theoretic outlook. Calcutta Stat Assoc
Bull1996; 46:
69–88.
29.
RosenbergerWF. Asymptotic inference with response-adaptive
treatment allocation designs. Ann Stat1993; 21:
2098–2107.
JennisonCTurnbullBW. Group sequential methods with applications to clinical
trials, Boca Raton, FL: Chapman
and Hall/CRC, 2000.
43.
HardwickJStoutQFFlexible algorithms for creating and analyzing adaptive sampling
procedures. In: FlournoyNRosenbergerWFWongWK (eds). New developments and applications in experimental
design, Hayward: Institute of
Mathematical Statistics, 1998, pp.
91–105.
44.
BiswasABhattacharyaRZhangL. Optimal response-adaptive designs for continuous
responses in phase III trials. Biometrical J2007; 49:
928–940.
45.
BiswasAMandalSOptimal adaptive designs in phase III clinical trials for continuous
responses with covariates. In: Di BucchianicoALauterHWynnHP (eds). moDa 7 – advances in model-oriented design and
analysis, Heidelberg:
Physica-Verlag, 2004, pp.
51–58.
46.
BandyopadhyayUBhattacharyaR. Response adaptive procedures with dual
optimality. Stat Neerl2009; 63:
353–367.
47.
BiswasABhattacharyaR. An optimal response-adaptive design with dual
constraints. Stat Probab Lett2010; 80:
177–185.
48.
AntogniniBAGiovagnoliA. On the large sample optimality of sequential
designs for comparing two or more treatments. Sequential
Anal2005; 24:
205–217.
49.
SilveySD. Optimum design,
London: Chapman and
Hall, 1980.
50.
ZhangLRosenbergerWF. Response-adaptive randomization for survival
trials: the parametric approach. J R Stat Soc Ser C2007; 34:
562–569.
51.
TymofyeyevYRosenbergerWFHuF. Implementing optimal allocation in sequential
binary response experiments. J Am Stat Assoc2007; 102:
224–234.
52.
ZhuHHuF. Implementing optimal allocation in sequential
continuous response experiments. J Stat Plann Inf2009; 139:
2420–2430.
53.
BiswasAMandalSBhattacharyaR. Multi-treatment optimal response-adaptive designs
for phase III clinical trials. J Korean Stat Soc2010; 40:
33–44.
54.
SverdlovOTymofyeyevYWongWK. Optimal response-adaptive randomizes designs for
multi-armed survival trials. Stat Med2011; 30:
2800–2810.
55.
BeggCBIglewiczB. A treatment allocation procedure for sequential
clinical trials. Biometrics1980; 36:
81–90.
56.
AtkinsonAC. Optimum biased coin designs for sequential
clinical trials with prognostic factors. Biometrika1982; 69:
61–67.
57.
AtkinsonAC. Optimum biased-coin designs for sequential
treatment allocation with covariate information. Stat
Med1999; 18:
1741–1752.
58.
AtkinsonAC. Bayesian and other biased-coin designs for
sequential clinical trials. Tatra Mountains Math Pub1999; 17:
133–139.
59.
AtkinsonACBiswasA. Bayesian adaptive biased-coin designs for
clinical trials with normal responses. Biometrics2005; 61:
118–125.
60.
WongWKZhuW. Optimum treatment allocation rules under a
variance heterogeneity model. Stat Med2008; 27:
4581–4595.
61.
BandyopadhyayUBiswasA. Adaptive designs for normal responses with
prognostic factors. Biometrika2001; 88:
409–419.
62.
BiswasACoadDS. A general multi-treatment adaptive design for
multivariate responses. Sequential Anal2005; 24:
139–158.
63.
BiswasAHuangHHHuangWT. Covariate-adjusted adaptive designs for
continuous responses in a phase III clinical trial: recommendation for
practice. J Biopharm Stat2006; 16:
227–239.
64.
ZhangLXHuFCheungSH. Asymptotic properties of
covariate-adjusted response-adaptive designs. Ann Stat2007; 35:
1166–1182.
65.
ZhangLXHuF. A new family of covariate-adjusted response
adaptive designs and their properties. Appl Math A J Chin Uni
Ser B2009; 24:
1–13.
66.
AntogniniABZgoraiouMCovariate adjusted designs for combining efficiency, ethics and randomness
in normal response trials. In: GiovagnoliAAtkinsonACTorsneyB (eds). moDa 9 – advances in model-oriented design and
analysis, Berlin, Heidelberg:
Springer-Verlag, 2010, pp.
17–24.
67.
FriedmanLMFurbergCDDeMetsDL. Fundamentals of clinical trials, 3rd
ed. New York, NY:
Springer-Verlag, 1998.
68.
BandyopadhyayUBhattacharyaR. An urn based covariate adjusted response adaptive
allocation design. Stat Meth Med Res2012. doi: 10.1177/0962280212437479.
69.
BiswasAAngersJF. A Bayesian adaptive design in clinical trials for
continuous responses. Stat Neerl2002; 56:
400–414.
70.
HuFRosenbergerWFZhangL-X. Asymptotically best response-adaptive
randomization procedures. J Stat Plann Inf2006; 136:
1911–1922.
71.
HuFZhangL-XHeX. Efficient randomized-adaptive
designs. Ann Stat2009; 37:
2543–2560.
72.
EfronB. Forcing a sequential experiment to be
balanced. Biometrika1971; 58:
403–417.
73.
WeiLJ. Exact two-sample permutation tests based on the
randomized play-the-winner rule in medical trials.
Biometrika1988; 75:
603–606.
74.
IvanovaARosenbergerWF. A comparison of urn designs for randomized
clinical trials of K > 2 treatments. J
Biopharm Stat2000; 10:
93–107.
75.
BaiZDHuFRosenbergerWF. Asymptotic properties of adaptive designs for
clinical trials with delayed response. Ann Stat2002; 30:
122–129.
76.
BiswasA. Generalized delayed response in randomized
play-the-winner rule. Commun Stat Simul Comput2003; 32:
259–274.
77.
HuFZhangLXCheungSH. Doubly adaptive biased coin designs with
delayed responses. Can J Stat2008; 36:
541–559.
78.
RoutCCRockeDALevinJ. A reevaluation of the role of crystalloid
preload in the prevention of hypotension associated with spinal anesthesia for elective
cesarean section. Anesthesiology1993; 79:
262–269.
79.
BiswasADewanjiA. A Randomized longitudinal play-the-winner design
for repeated binary data. Aus N Z J Stat2004; 46:
675–684.
80.
BiswasADewanjiA. Inference for a RPW-type clinical trial with
repeated monitoring for the treatment of rheumatoid arthritis.
Biometrical J2004; 46:
769–779.
81.
BerryDA. Adaptive clinical trials: the promise and the
caution. J Clin Oncol2011; 29:
606–609.
82.
KornELFreidlinB. Outcome-adaptive randomization: is it
useful?J Clin Oncol201129: 771–776.
