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Biopharmaceutical Applied Statistics Symposium: Volume 1 Design of Clinical Trials PDF

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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 Thisworkissubjecttocopyright.AllrightsarereservedbythePublisher,whetherthewholeorpart of the material is concerned, specifically the rights of translation, reprinting, reuse of illustrations, recitation, broadcasting, reproduction on microfilms or in any other physical way, and transmission orinformationstorageandretrieval,electronicadaptation,computersoftware,orbysimilarordissimilar methodologynowknownorhereafterdeveloped. The use of general descriptive names, registered names, trademarks, service marks, etc. in this publicationdoesnotimply,evenintheabsenceofaspecificstatement,thatsuchnamesareexemptfrom therelevantprotectivelawsandregulationsandthereforefreeforgeneraluse. The publisher, the authors and the editors are safe to assume that the advice and information in this book are believed to be true and accurate at the date of publication. Neither the publisher nor the authorsortheeditorsgiveawarranty,expressorimplied,withrespecttothematerialcontainedhereinor for any errors or omissions that may have been made. The publisher remains neutral with regard to jurisdictionalclaimsinpublishedmapsandinstitutionalaffiliations. ThisSpringerimprintispublishedbytheregisteredcompanySpringerNatureSingaporePteLtd. Theregisteredcompanyaddressis:152BeachRoad,#21-01/04GatewayEast,Singapore189721, Singapore 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

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