PHARMACEUTICAL STATISTICS

Pharmaceut. Statist. 2007; 6: 67–68

Published online in Wiley InterScience (www.interscience.wiley.com). DOI: 10.1002/pst.259

Literature Review October–December 2006

Scott D. Patterson3, Kevin Carroll2 and Steven Julious1,*,y

1Pharmaceutical Statistics Medical Statistics Group, University of Sheffield, Regent

Court, 30 Regent Street, Sheffield S1 4DA, UK2AstraZeneca Pharmaceuticals, Global Clinical Information Science, Alderley Park,

Macclesfield, UK3GlaxoSmithKline Pharmaceuticals, 2301 Renaissance Boulevard, King of Prussia, PA

19406, USA

INTRODUCTION

This review covers the following journals received during the

period from October to December 2006:

* Applied Statistics, volume 55, parts 4 and 5.* Biometrical Journal, volume 48, issue 5.* Biometrics, volume 62, parts 2 and 3.* Biometrika, volume 93, issue 3.* Biostatistics, volume 7, part 4, and volume 8, part 1.* Computational Statistics & Data Analysis, volume 51, parts

1 to 5.* Clinical Trials, volume 3, parts 4 and 5.* Drug Information Journal, volume 40, part 4.* Journal of Biopharmaceutical Statistics, volume 16, parts 5

and 6.* Journal of the Royal Statistical Society, Series A, volume

169 part 4.* Statistics in Medicine, volume 25, Parts 20 to 24.* Statistical Methods in Medical Research, volume 15, parts 4

and 5.

SELECTED HIGHLIGHTS FROM THE

LITERATURE

A guideline on undertaking simulations

* Burton A, Altman DG, Royston P, Holder RL. The design

of simulation studies in medical statistics. Statistics in

Medicine 2006; 25(24): 4279–4292.

Phase I

The area of determining the maximum tolerated doses

particularly in the oncology field has a growing literature with

the methodologies beginning to cross-over into other therapeu-

tic areas. The following papers may be worth looking at:

* Zohar S, O’Quigley J. Optimal designs for estimating the

most successful dose. Statistics in Medicine 2006; 25(24):

4311–4320.* Ivanaova A. Escalation, group and A+B designs for dose-

finding trials. Statistics in Medicine 25(21): 3668–3678.* Yin G, Li Y, Ji Y. Bayesian dose-finding in phase I/II

clinical trials using toxicity and efficacy odds ratios.

Biometrics 62: 777–787.

Phase II

The Journal of Biopharmaceutical Statistics (Issue 5 by guest

editors N. Ting and A. Grieve) is devoted to the statistics of

dose–response studies and covers a variety of topics from

designs used in Phase I to multiple comparison issues.

Sample size calculations

There are a number of interesting articles this month on sample

size calculation including the following article on the real

situation where you have a very bounded outcome

* Tsonaka R, Rizopoulos D, Lesaffre E. Power and sample

size calculations for discrete bounded outcome scores.

Statistics in Medicine 2006; 25(24): 4241–4251.

While the following paper discusses calculations for survival

trials with time varying relationships.

* Li B, Grambsch P. Sample size calculation in survival trials

accounting for time-varying relationship between noncom-

Copyright # 2007 John Wiley & Sons, Ltd.Received \60\re /teci

yE-mail: S.A.Julious@Sheffield.ac.uk

*Correspondence to: Steven Julious, Medical StatisticsGroup, University of Sheffield, Regent Court, 30 RegentStreet, Sheffield S1 4DA, UK.

pliance and risk of outcome event. Clinical Trials 2006; 3(4):

349–359.

Sample size re-estimation in Phase III and IV may need to be

employed in situations where design assumptions are uncertain.

This is the topic of a review in:

* Chuang-Stein C, Anderson K, Gallo P, Collins S. Sample

size re-estimation: a review and recommendations. Drug

Information Journal 2006; 40(4): 475–484.

Interim analyses, flexible designs and Data

Monitoring Committees

The Drug Information Journal (Issue 4) contains a large number

of papers on the topic of adaptive designs

A non-technical paper on the topic of adaptive designs with

separate commentaries by Jennison and Turnbull; Proschan;

Bauer and Frisen that may be of interest is

* Burman CF, Sonesson C. Are flexible designs sound?

Biometrics 2006; 62: 664–683.

Data Analysis Issues

Survival analysis is always an interesting field of research and

the following paper, through real world examples, discusses the

situation of monitoring trials where there are two opposing

survival endpoints for example time to relapse and time to cure

* Salter A, Raab G, Day S. Analysing sequential events in

clinical trials. Clinical Trials 2006; 3(5): 421–430.

Use of Bayesian statistics in medical research is a hot topic

for the moment, and an historical review in the recent past may

be found in:

* Ashby D. Bayesian statistics in medicine: a 25 year review.

Statistics in Medicine 2006; 25: 3589–3631.

Miscellaneous

Stephen Senn has written a couple of articles in Statistics in

Medicine which may be worth a read. The first is an article

under the special series to mark 25 years of Statistics in

Medicine

* Senn S. Cross-over trials in Statistics in Medicine: the first

‘25’ years. Statistics in Medicine 2006; 25(20): 3430–3442.

While the second discusses the issues of ANCOVA over

change from baseline.

* Senn S. Change from baseline and analysis of covariance

revisited. Statistics in Medicine 2006; 25(24): 4334–4344.

Copyright # 2007 John Wiley & Sons, Ltd. Pharmaceut. Statist. 2007; 6: 67–68

Literature Review68