Del 0 - Statistics in Practice

Practical Guide to Designing Phase II Trials in Oncology

Inbunden, Engelska, 2014

AvSarah R. Brown,Walter M. Gregory,Christopher J. Twelves,Julia M. Brown

989 kr

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How to identify optimal phase II trial designsProviding a practical guide containing the information needed to make crucial decisions regarding phase II trial designs, A Practical Guide to Designing Phase II Trials in Oncology sets forth specific points for consideration between the statistician and clinician when designing a phase II trial, including issues such as how the treatment works, choice of outcome measure and randomization, and considering both academic and industry perspectives. A comprehensive and systematic library of available phase II trial designs is included, saving time otherwise spent considering multiple manuscripts, and real-life practical examples of using this approach to design phase II trials in cancer are given.A Practical Guide to Designing Phase II Trials in Oncology: Offers a structured and practical approach to phase II trial designConsiders trial design from both an academic and industry perspectiveIncludes a structured library of available phase II trial designsIs relevant to both clinical and statistical researchers at all levelsIncludes real life examples of applying this approachFor those new to trial design, A Practical Guide to Designing Phase II Trials in Oncology will be a unique and practical learning tool, providing an introduction to the concepts behind informed decision making in phase II trials. For more experienced practitioners, the book will offer an overview of new, less familiar approaches to phase II trial design, providing alternative options to those which they may have previously used.

Produktinformation

Utgivningsdatum2014-05-13
Mått160 x 236 x 18 mm
Vikt463 g
FormatInbunden
SpråkEngelska
SerieStatistics in Practice
Antal sidor256
FörlagJohn Wiley & Sons Inc
ISBN9781118570906

Tillhör följande kategorier

Onkologi inom Medicin
Medicinsk utrustning och medicinska tekniker inom Medicin

Contributors xvForeword I xviiElizabeth A. EisenhauerForeword II xixRoger A’HernPreface xxi1 Introduction 1Sarah Brown, Julia Brown, Walter Gregory and Chris Twelves1.1 The role of phase II trials in cancer 31.2 The importance of appropriate phase II trial design 51.3 Current use of phase II designs 61.4 Identifying appropriate phase II trial designs 71.5 Potential trial designs 91.6 Using the guidance to design your trial 102 Key Points for Consideration 12Sarah Brown, Julia Brown, Marc Buyse, Walter Gregory, Mahesh Parmar and Chris Twelves2.1 Stage 1 – Trial questions 142.1.1 Therapeutic considerations 142.1.2 Primary intention of trial 162.1.3 Number of experimental treatment arms 172.1.4 Primary outcome of interest 182.2 Stage 2 – Design components 182.2.1 Outcome measure and distribution 182.2.2 Randomisation 212.2.3 Design category 262.3 Stage 3 – Practicalities 332.3.1 Practical considerations 332.4 Summary 353 Designs for Single Experimental Therapies with a Single Arm 36Sarah Brown3.1 One-stage designs 363.1.1 Binary outcome measure 363.1.2 Continuous outcome measure 383.1.3 Multinomial outcome measure 393.1.4 Time-to-event outcome measure 403.1.5 Ratio of times to progression 403.2 Two-stage designs 413.2.1 Binary outcome measure 413.2.2 Continuous outcome measure 503.2.3 Multinomial outcome measure 503.2.4 Time-to-event outcome measure 533.2.5 Ratio of times to progression 543.3 Multi-stage designs 553.3.1 Binary outcome measure 553.3.2 Continuous outcome measure 593.3.3 Multinomial outcome measure 593.3.4 Time-to-event outcome measure 603.3.5 Ratio of times to progression 603.4 Continuous monitoring designs 603.4.1 Binary outcome measure 603.4.2 Continuous outcome measure 633.4.3 Multinomial outcome measure 633.4.4 Time-to-event outcome measure 633.4.5 Ratio of times to progression 643.5 Decision-theoretic designs 643.5.1 Binary outcome measure 643.5.2 Continuous outcome measure 653.5.3 Multinomial outcome measure 653.5.4 Time-to-event outcome measure 653.5.5 Ratio of times to progression 653.6 Three-outcome designs 653.6.1 Binary outcome measure 653.6.2 Continuous outcome measure 663.6.3 Multinomial outcome measure 663.6.4 Time-to-event outcome measure 663.6.5 Ratio of times to progression 673.7 Phase II/III designs 674 Designs for Single Experimental Therapies Including Randomisation 68Sarah Brown4.1 One-stage designs 684.1.1 Binary outcome measure 684.1.2 Continuous outcome measure 704.1.3 Multinomial outcome measure 704.1.4 Time-to-event outcome measure 704.1.5 Ratio of times to progression 724.2 Two-stage designs 724.2.1 Binary outcome measure 724.2.2 Continuous outcome measure 734.2.3 Multinomial outcome measure 744.2.4 Time-to-event outcome measure 754.2.5 Ratio of times to progression 754.3 Multi-stage designs 754.3.1 Binary outcome measure 754.3.2 Continuous outcome measure 754.3.3 Multinomial outcome measure 754.3.4 Time-to-event outcome measure 764.3.5 Ratio of times to progression 764.4 Continuous monitoring designs 764.4.1 Binary outcome measure 764.4.2 Continuous outcome measure 764.4.3 Multinomial outcome measure 764.4.4 Time-to-event outcome measure 764.4.5 Ratio of times to progression 764.5 Three-outcome designs 774.5.1 Binary outcome measure 774.5.2 Continuous outcome measure 774.5.3 Multinomial outcome measure 774.5.4 Time-to-event outcome measure 774.5.5 Ratio of times to progression 774.6 Phase II/III designs 774.6.1 Binary outcome measure 774.6.2 Continuous outcome measure 794.6.3 Multinomial outcome measure 804.6.4 Time-to-event outcome measure 814.6.5 Ratio of times to progression 814.7 Randomised discontinuation designs 824.7.1 Binary outcome measure 824.7.2 Continuous outcome measure 824.7.3 Multinomial outcome measure 824.7.4 Time-to-event outcome measure 824.7.5 Ratio of times to progression 825 Treatment Selection Designs 83Sarah Brown5.1 Including a control arm 845.1.1 One-stage designs 845.1.2 Two-stage designs 845.1.3 Multi-stage designs 885.1.4 Continuous monitoring designs 895.1.5 Decision-theoretic designs 895.1.6 Three-outcome designs 895.1.7 Phase II/III designs – same primary outcome measure at phase II and phase III 895.1.8 Phase II/III designs – different primary outcome measures at phase II and phase III 995.1.9 Randomised discontinuation designs 1025.2 Not including a control arm 1035.2.1 One-stage designs 1035.2.2 Two-stage designs 1065.2.3 Multi-stage designs 1085.2.4 Continuous monitoring designs 1095.2.5 Decision-theoretic designs 1105.2.6 Three-outcome designs 1105.2.7 Phase II/III designs – same primary outcome measure at phase II and phase III 1105.2.8 Randomised discontinuation designs 1116 Designs Incorporating Toxicity as a Primary Outcome 112Sarah Brown6.1 Including a control arm 1126.1.1 One-stage designs 1126.1.2 Two-stage designs 1146.1.3 Multi-stage designs 1156.2 Not including a control arm 1176.2.1 One-stage designs 1176.2.2 Two-stage designs 1186.2.3 Multi-stage designs 1226.2.4 Continuous monitoring designs 1256.3 Toxicity alone 1266.3.1 One stage 1266.3.2 Continuous monitoring 1276.4 Treatment selection based on activity and toxicity 1286.4.1 Two-stage designs 1286.4.2 Multi-stage designs 1296.4.3 Continuous monitoring designs 1297 Designs Evaluating Targeted Subgroups 131Sarah Brown7.1 One-stage designs 1317.1.1 Binary outcome measure 1317.2 Two-stage designs 1327.2.1 Binary outcome measure 1327.3 Multi-stage designs 1357.3.1 Binary outcome measure 1357.3.2 Time-to-event outcome measure 1377.4 Continuous monitoring designs 1387.4.1 Binary outcome measure 1387.4.2 Time-to-event outcome measure 1398 ‘Chemo-radio-sensitisation’ in Head and Neck Cancer 141John Chester and Sarah BrownStage 1 – Trial questions 141Therapeutic considerations 141Primary intention of trial 142Number of experimental treatment arms 142Primary outcome of interest 142Stage 2 – Design components 142Outcome measure and distribution 142Randomisation 143Design category 143Possible designs 144Stage 3 – Practicalities 146Practical considerations for selecting between designs 146Proposed trial design 148Summary 1509 Combination Chemotherapy in Second-line Treatment of Non-small Cell Lung Cancer 151Ornella Belvedere and Sarah BrownStage 1 – Trial questions 152Therapeutic considerations 152Primary intention of trial 152Number of experimental treatment arms 152Primary outcome of interest 152Stage 2 – Design components 153Outcome measure and distribution 153Randomisation 153Design category 153Possible designs 154Stage 3 – Practicalities 155Practical considerations for selecting between designs 155Proposed trial design 158Summary 16210 Selection by Biomarker in Prostate Cancer 163Rick Kaplan and Sarah BrownStage 1 – Trial questions 164Therapeutic considerations 164Primary intention of trial 164Number of experimental treatment arms 164Primary outcome of interest 164Stage 2 – Design components 165Outcome measure and distribution 165Randomisation 165Design category 166Possible designs 167Stage 3 – Practicalities 168Practical considerations for selecting between designs 168Proposed trial design 170Summary 17111 Dose Selection in Advanced Multiple Myeloma 174Sarah Brown and Steve ScheyStage 1 – Trial questions 174Therapeutic considerations 174Primary intention of trial 175Number of experimental arms 175Primary outcome of interest 175Stage 2 – Design components 176Outcome measure and distribution 176Randomisation 176Design category 177Possible designs 177Stage 3 – Practicalities 178Practical considerations for selecting between designs 178Proposed trial design 181Summary 18212 Targeted Therapy for Advanced Colorectal Cancer 185Matthew Seymour and Sarah BrownStage 1 – Trial questions 185Therapeutic considerations 185Primary intention of trial 186Number of experimental treatment arms 186Primary outcome of interest 186Stage 2 – Design components 187Outcome measure and distribution 187Randomisation 187Design category 188Possible designs 189Stage 3 – Practicalities 190Practical considerations for selecting between designs 190Proposed trial design 191Summary 19413 Phase II Oncology Trials: Perspective from Industry 195Anthony Rossini, Steven Green and William Mietlowski13.1 Introduction 19513.2 Commercial challenges, drivers and considerations 19613.3 Selecting designs by strategy 19713.3.1 Basic strategies addressed by phase II studies 19813.3.2 Potential registration 19813.3.3 Exploratory activity 20313.3.4 Regimen selection 20413.3.5 Phase II to Support Predicting Success in Phase IIi 20613.3.6 Phase II safety trials 20813.3.7 Prospective identification of target populations 20913.4 Discussion 210References 213Index 227