Biopharmaceutical Applied Statistics Symposium: Volume 1 Design of Clinical Trials

414 Pages·2018·9.18 MB·English

by  Karl E. Peace

ICSA Book Series in Statistics
Series Editors:
Jiahua Chen · Ding-Geng (Din) Chen
Karl E. Peace
Ding-Geng Chen
Editors
Sandeep Menon
Biopharmaceutical
Applied Statistics
Symposium
Volume 1 Design of Clinical Trials
ICSA Book Series in Statistics
Series editors
JiahuaChen,DepartmentofStatistics,UniversityofBritishColumbia,Vancouver,
Canada
Ding-Geng (Din) Chen, University of North Carolina, Chapel Hill, NC, USA
More information about this series at http://www.springer.com/series/13402
Karl E. Peace Ding-Geng Chen
(cid:129)
Sandeep Menon
Editors
Biopharmaceutical Applied
Statistics Symposium
Volume 1 Design of Clinical Trials
123
Editors
KarlE. Peace Sandeep Menon
Jiann-Ping HsuCollege of Public Health BostonUniversity
Georgia SouthernUniversity Cambridge, MA, USA
Statesboro, GA,USA
Ding-GengChen
SchoolofSocialWork&GillingsSchoolof
GlobalPublic Health
University of NorthCarolina
ChapelHill, NC,USA
and
University of Pretoria
Pretoria, SouthAfrica
ISSN 2199-0980 ISSN 2199-0999 (electronic)
ICSABookSeries inStatistics
ISBN978-981-10-7828-6 ISBN978-981-10-7829-3 (eBook)
https://doi.org/10.1007/978-981-10-7829-3
LibraryofCongressControlNumber:2017964432
©SpringerNatureSingaporePteLtd.2018
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Preface
Currently,therearethreevolumesoftheBASSBookSeries,spanning45chapters.
Chapters in this book are contributed by invited speakers at the annual meetings
oftheBiopharmaceuticalAppliedStatisticsSymposium(BASS).Volume1istitled
Design of Clinical Trials and consists of 15 chapters; Volume 2 is titled
BiostatisticalAnalysisofClinicalTrialsandconsistsof12chapters;andVolume3
istitledPharmaceuticalApplicationsandconsistsof18chapters.Thethreevolumes
include the works of 70 authors or co-authors.
HistoryofBASS:BASSwasfoundedin1994,byDr.KarlE.Peace.Dr.Peace
is the Georgia Cancer Coalition Distinguished Scholar/Scientist, Professor of
Biostatistics,FoundingDirectoroftheCenterforBiostatistics,andSeniorResearch
ScientistintheJiann-PingCollegeofPublicHealthatGeorgiaSouthernUniversity.
Originally, there were three objectives of BASS. Since the first editor founded
the Journal of Biopharmaceutical Statistics (JBS) 3 years before founding BASS,
one of the original objectives was to invite BASS Speakers to create papers from
their BASS presentations and submit to JBS for review and publication. Ergo,
BASS wastobe asource ofpapers submitted toJBStoassistinthegrowth ofthe
new journal JBS. The additional two objectives were:
(cid:129) toprovideaforumforpharmaceuticalandmedicalresearchersandregulatorsto
sharetimelyandpertinentinformationconcerningtheapplicationofbiostatistics
in pharmaceutical environments; and most importantly,
(cid:129) to provide revenues to support graduate fellowships in biostatistics at the
MedicalCollegeofVirginia(MCV)andattheJiann-PingHsuCollegeofPublic
Health at Georgia Southern University (GSU).
After the JBS was on firm footing, the first objective was formally dropped. In
addition, the third objective was expanded to include potentially any graduate
program in biostatistics in the USA.
BASSI(1994)washeldattheHyattRegencyinOrlando,FL;BASSII–IIIwere
held at the Hilton Beach Resort, Inner Harbor, in San Diego, CA; BASS IV–VII
were held at the Hilton Oceanfront Resort Hotel, Palmetto Dunes, in Hilton Head
v
vi Preface
Island, SC; BASS VIII–XII were held at the Desoto Hilton; and BASS XIII–XVI
were held at the Mulberry Inn, both located in the Historic District of Savannah,
GA. BASS XVII was held at the Hilton Resort Hotel at Palmetto Dunes, Hilton
HeadIsland,SC.BASSXVIII–XIXwereheldattheMulberryInninSavannah.To
mark thetwentieth Anniversary BASS meeting, BASSXX was held in Orlando at
the Hilton Downtown Orlando Hotel. BASS XXI was held at the Holiday Inn
Crowne Plaza in Rockville, MD; whereas BASS XXII and XXIII were held at the
Radisson Hotel in Rockville, Maryland.
BASS XXIV (www.bassconference.org) was held at the Hotel Indigo in the
charming historic Georgia city of Savannah. More than 360 tutorials and 57 1-day
or 2-day short courses have been presented at BASS, by the world’s leading
authorities on applications of biostatistical methods attendant to the research,
clinical development, and regulation of biopharmaceutical products. Presenters
represent the biopharmaceutical industry, academia, and government, particularly
the NIH and FDA.
BASS is regarded as one of the premier conferences in the world. It has served
thestatistical,biopharmaceutical,andmedicalresearchcommunitiesforthepast24
yearsbyprovidingaforumfordistinguishedresearchersandscholarsinacademia,
governmentagencies,andindustriestoconductknowledgesharing,ideaexchange,
andcreativediscussionsofthemostup-to-dateinnovativeresearchandapplications
to medical and health care to enhance the health of general public, in addition to
providing support for graduate students in their biostatistics studies. Toward this
latterend,BASShasprovidedfinancialsupportfor75studentsincompletingtheir
master’s or doctorate degree in Biostatistics. In addition, BASS has provided
numerous travel grants to doctorate-seeking students in Biostatistics to attend the
annual BASS meeting. This provides a unique opportunity for students to broaden
their education, particularly in the application of biostatistical design and analysis
methods, as well as networking opportunities with biostatisticians from Academia,
the Pharmaceutical Industry, and governmental agencies such as the FDA.
Volume 1 of the BASS Book Series, entitled Design of Clinical Trials,
consists of 15 chapters. Chapter 1 presents statistical approaches to clinical trial
simulations. Chapter 2 presents methods helpful in choosing the best function of
baseline run-in data for use as a covariate in the analysis of treatment data from
Phase III clinical trials in hypertension. Chapter 3 provides methods in designing
adaptive trials in clinical research. Chapter 4 then provides best practices and
recommendations for clinical trial simulations for adaptive designs. Chapter 5
discusses the design and analysis of clinical trials that collect recurrent event data.
Chapter6presentsmethodsforresponse-adaptiveallocationforbinaryoutcomes
in clinical trials from a Bayesian perspective. Chapter 7 addresses the important
topic of high placebo response in neuroscience clinical trials. Chapter 8 presents
methodsfordesigningPhaseIcancerclinicaltrials,forbothsingleandcombination
agents. Chapter 9 discusses the structure for clinical trials that include sequential
data monitoring procedures. Chapter 10 addresses both theory and practice in the
design and data analysis of multiregional clinical trials. Chapter 11 continues
Preface vii
discussionofmultiregionalclinicaltrialswithparticularemphasisonICH-E17and
subpopulations.
Chapter 12 also discusses multiregional clinical trials in the development of
vaccines that are designed as adaptive group-sequential outcome studies. Chapter
13 deals with the development and validation of procedures for collecting
patient-reported outcomes. Chapter 14 presents group-sequential and interim
analysisandconditionalpowermethodsforsurvivaltrialsfromthenonproportional
hazards perspective. Finally, Chap. 15 discusses the design and analysis of dose–
response trials for early clinical development.
We are indebted to all the presenters, program committee, attendees, and vol-
unteers who have contributed to the phenomenal success of BASS over its first 24
years, and to the publisher for expressing interest in and publishing the Series.
Statesboro, USA Karl E. Peace, Ph.D.
Jiann-Ping Hsu College
of Public Health
Georgia Southern Univesity
Chapel Hill, USA/Pretoria, South Africa Ding-Geng Chen, Ph.D.
Professor, University
of North Carolina
Extraordinary Professor
University of Pretoria
Cambridge, USA Sandeep Menon
Vice President and Head
of Early Clinical Development
Biostatistics
Contents
1 A Statistical Approach to Clinical Trial Simulations. . . . . . . . . . . . 1
Stephan Ogenstad
2 Choosing the Function of Baseline Run-in Data for Use as a
Covariate in the Analysis of Treatment Data from Phase III
Clinical Trials in Hypertension . . . . . . . . . . . . . . . . . . . . . . . . . . . . 19
Yi Hao and Karl E. Peace
3 Adaptive Trial Design in Clinical Research . . . . . . . . . . . . . . . . . . 75
Annpey Pong and Shein-Chung Chow
4 Best Practices in Clinical Trial Simulations for Adaptive Study
Designs . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 101
Cristiana Mayer and J. Kyle Wathen
5 Designing and Analyzing Recurrent Event Data Trials. . . . . . . . . . 115
Stephan Ogenstad
6 Response-Adaptive Allocation for Binary Outcomes: Bayesian
Methods from the BASS Conference. . . . . . . . . . . . . . . . . . . . . . . . 149
Roy T. Sabo
7 Addressing High Placebo Response in Neuroscience Clinical
Trials . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 171
Gheorghe Doros, Pilar Lim and Yuyin Liu
8 Phase I Cancer Clinical Trial Design: Single and Combination
Agents. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 205
Ying Yuan, Heng Zhou and Yanhong Zhou
9 Data Monitoring: Structure for Clinical Trials and Sequential
Monitoring Procedures . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 235
David M. Reboussin and Dave L. DeMets
ix
x Contents
10 Design and Data Analysis of Multiregional Clinical Trials
(MRCTs)—Theory and Practice . . . . . . . . . . . . . . . . . . . . . . . . . . . 269
Chi-Tian Chen, Hsiao-Hui Tsou, Jung-Tzu Liu, Chin-Fu Hsiao,
Fei Chen, Gang Li and K. K. G. Lan
11 Multi-Regional Clinical Trials, ICH-E17, and Subpopulations . . . . 287
Yoko Tanaka, Bruce Binkowitz and Bill Wang
12 Adaptive Group-Sequential Multi-regional Outcome Studies in
Vaccines . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 297
Inna Perevozskaya
13 Patient-Reported Outcome Measures: Development and
Psychometric Evaluation. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 317
Lori D. McLeod, Sheri E. Fehnel and Joseph C. Cappelleri
14 Interim Analyses: Design and Analysis Considerations for
Survival Trials When Hazards May Be Nonproportional. . . . . . . . 347
Edward Lakatos
15 On Design and Analysis of Dose-Response Trials for Early
Clinical Development . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 377
Qing Liu
Index .... .... .... .... .... ..... .... .... .... .... .... ..... .... 405