{"product_id":"clinical-trials-in-the-era-of-real-world-evidence-paperback-softback-9781394369652","title":"Clinical Trials in the Era of Real-World Evidence (Paperback \/ softback) 9781394369652","description":"\u003cfont face=\"Georgia\"\u003e\r\n\u003cp\u003e\u003cfont size=\"6\"\u003eClinical Trials in the Era of Real-World Evidence\u003c\/font\u003e\u003cbr\u003e\r\n\r\n\r\n\r\n\r\n\r\n\u003c\/p\u003e\n\u003cp\u003e\u003cfont size=\"4\"\u003eJie Chen (Author), Naitee Ting (Author), Feinan Lu (Author)\u003c\/font\u003e\u003c\/p\u003e\r\n\r\n\u003cp\u003e\u003cfont size=\"3\"\u003e9781394369652, Wiley\u003c\/font\u003e\u003c\/p\u003e\r\n\r\n\u003cp\u003e\u003cfont size=\"3\"\u003ePaperback \/ softback, published 6 May 2026\u003c\/font\u003e\u003c\/p\u003e\r\n\r\n\u003cp\u003e\u003cfont size=\"3\"\u003e464 pages\u003cbr\u003e24.6 x 20.3 x 3 cm, 0.885 kg\u003c\/font\u003e\u003c\/p\u003e\r\n\r\n\r\n\r\n\r\n\r\n\u003cp align=\"justify\"\u003e\u003cstrong\u003e\u003cfont size=\"3\"\u003e\u003cp\u003e\u003cb\u003eIntegrate RCTs and real-world evidence for stronger regulatory submissions\u003c\/b\u003e\u003c\/p\u003e \u003cp\u003eSince the passage of the 21st Century Cures Act, the landscape of clinical research has transformed dramatically. Today, the integration of real-world data (RWD) and real-world evidence (RWE) with traditional randomized controlled trials (RCTs) is not just innovative - it is essential for modern drug development and regulatory decision-making.\u003c\/p\u003e \u003cp\u003eThis comprehensive volume bridges the methodologies of conventional RCTs and emerging RWE studies, offering systematic approaches for leveraging diverse data sources throughout the clinical development lifecycle. Grounded in regulatory statistics yet forward-looking in scope, the book explores Stein's continuum of study designs - from traditional RCTs to pragmatic trials and observational studies - and demonstrates how RWD can enhance trial feasibility, inform outcome selection, provide external controls for single-arm trials, and strengthen post-approval safety monitoring.\u003c\/p\u003e \u003cp\u003eReaders will find detailed explorations of:\u003c\/p\u003e \u003cul\u003e \u003cli\u003eA history of clinical trials, evidence-based medicine, and the regulatory frameworks governing drug approval\u003c\/li\u003e \u003cli\u003eTreatment switching and estimands in RWE studies\u003c\/li\u003e \u003cli\u003eAdvanced analytical considerations including precision medicine, vaccine effectiveness, safety evaluation, and sensitivity analysis\u003c\/li\u003e \u003cli\u003ePractical roadmaps for designing robust RWE studies\u003c\/li\u003e \u003cli\u003eArtificial intelligence and machine learning in clinical research\u003c\/li\u003e \u003c\/ul\u003e \u003cp\u003eDesigned as both a reference and a graduate-level textbook, this book serves clinical researchers such as clinicians and biostatisticians, regulatory professionals, public health scientists, and industry practitioners seeking to harness the power of RWD and RWE to generate credible, actionable evidence for modern medicine.\u003c\/p\u003e\u003c\/font\u003e\u003c\/strong\u003e\u003c\/p\u003e\r\n\r\n\u003cp\u003e\u003cfont size=\"3\"\u003e\u003cp\u003ePreface xvii\u003c\/p\u003e \u003cp\u003eAcknowledgments xxi\u003c\/p\u003e \u003cp\u003eAcronyms xxiii\u003c\/p\u003e \u003cp\u003e\u003cb\u003eChapter 1 Introduction 1\u003c\/b\u003e\u003c\/p\u003e \u003cp\u003e1.1 Medical Product Development Pathway 1\u003c\/p\u003e \u003cp\u003e1.2 Development of Evidence-Based Medicine 2\u003c\/p\u003e \u003cp\u003e1.3 The 21st Century Cures Act 4\u003c\/p\u003e \u003cp\u003e1.4 Regulatory Guidance and Related Documents 4\u003c\/p\u003e \u003cp\u003e1.5 Discussion and Summary 8\u003c\/p\u003e \u003cp\u003e1.6 Supplements 9\u003c\/p\u003e \u003cp\u003e\u003cb\u003eChapter 2 A Brief History and Critical Components of Clinical Trials 10\u003c\/b\u003e\u003c\/p\u003e \u003cp\u003e2.1 Lady Tasting Tea 10\u003c\/p\u003e \u003cp\u003e2.2 Alpha 12\u003c\/p\u003e \u003cp\u003e2.3 Permutation Test 13\u003c\/p\u003e \u003cp\u003e2.4 Selection of Control 16\u003c\/p\u003e \u003cp\u003e2.5 Parallel Versus Crossover Trials 19\u003c\/p\u003e \u003cp\u003e2.6 Blinding 20\u003c\/p\u003e \u003cp\u003e2.7 Process of a Clinical Trial 21\u003c\/p\u003e \u003cp\u003e2.8 Supplements 26\u003c\/p\u003e \u003cp\u003e\u003cb\u003eChapter 3 Clinical Development Process of a New Drug 27\u003c\/b\u003e\u003c\/p\u003e \u003cp\u003e3.1 Clinical Development Plan 27\u003c\/p\u003e \u003cp\u003e3.2 Phase I Clinical Trials 31\u003c\/p\u003e \u003cp\u003e3.3 Phase II Clinical Trials 36\u003c\/p\u003e \u003cp\u003e3.4 Phase III Trials 44\u003c\/p\u003e \u003cp\u003e3.5 New Drug Application (NDA) 49\u003c\/p\u003e \u003cp\u003e3.6 Phase IV Studies 50\u003c\/p\u003e \u003cp\u003e3.7 Supplements 51\u003c\/p\u003e \u003cp\u003e\u003cb\u003eChapter 4 Design Considerations for Phase III Confirmatory Trials 52\u003c\/b\u003e\u003c\/p\u003e \u003cp\u003e4.1 Drug Label 52\u003c\/p\u003e \u003cp\u003e4.2 Selection of Primary Indication 53\u003c\/p\u003e \u003cp\u003e4.3 Multi-Regional Clinical Trials (MRCT) 54\u003c\/p\u003e \u003cp\u003e4.4 Selection of Endpoint(s) 56\u003c\/p\u003e \u003cp\u003e4.5 Selection of Control 57\u003c\/p\u003e \u003cp\u003e4.6 Selection of Dose(s) 61\u003c\/p\u003e \u003cp\u003e4.7 Additional Considerations 63\u003c\/p\u003e \u003cp\u003e4.8 Supplements 67\u003c\/p\u003e \u003cp\u003e\u003cb\u003eChapter 5 Regulatory Submission and Approval 69\u003c\/b\u003e\u003c\/p\u003e \u003cp\u003e5.1 International Council of Harmonisation (ICH) 69\u003c\/p\u003e \u003cp\u003e5.2 Prescription Drug User Fee Act (PDUFA) 71\u003c\/p\u003e \u003cp\u003e5.3 Pre-submission Meetings 72\u003c\/p\u003e \u003cp\u003e5.4 Common Technical Documents and Submission 73\u003c\/p\u003e \u003cp\u003e5.5 Advisory Committee Meetings 75\u003c\/p\u003e \u003cp\u003e5.6 Supplements 77\u003c\/p\u003e \u003cp\u003e\u003cb\u003eChapter 6 Overview on Use of RWD and RWE in Regulatory Setting 79\u003c\/b\u003e\u003c\/p\u003e \u003cp\u003e6.1 Categories of RWD and External Data 79\u003c\/p\u003e \u003cp\u003e6.2 Supporting Trial Design and Conduct 80\u003c\/p\u003e \u003cp\u003e6.3 Using RWD and RWE to Support Product Approval 83\u003c\/p\u003e \u003cp\u003e6.4 Fulfilling Post-marketing Requirements and Commitments 88\u003c\/p\u003e \u003cp\u003e6.5 Discussion and Summary 89\u003c\/p\u003e \u003cp\u003e6.6 Supplements 89\u003c\/p\u003e \u003cp\u003e\u003cb\u003eChapter 7 Single-Arm Trials 91\u003c\/b\u003e\u003c\/p\u003e \u003cp\u003e7.1 Necessary Conditions 92\u003c\/p\u003e \u003cp\u003e7.2 Desirable Conditions 92\u003c\/p\u003e \u003cp\u003e7.3 Other Considerations 98\u003c\/p\u003e \u003cp\u003e7.4 Examples 101\u003c\/p\u003e \u003cp\u003e7.5 Conclusion and Summary 104\u003c\/p\u003e \u003cp\u003e7.6 Supplements 105\u003c\/p\u003e \u003cp\u003e\u003cb\u003eChapter 8 Externally Controlled Trials 107\u003c\/b\u003e\u003c\/p\u003e \u003cp\u003e8.1 Types of External Controls 107\u003c\/p\u003e \u003cp\u003e8.2 External Data as a Sole Control Group 111\u003c\/p\u003e \u003cp\u003e8.3 External Data to Augment Concurrent Controls in RCTs 114\u003c\/p\u003e \u003cp\u003e8.4 Assessment of Fit-for-Use External Data 115\u003c\/p\u003e \u003cp\u003e8.5 General Considerations in Using External Controls 117\u003c\/p\u003e \u003cp\u003e8.6 A Targeted-Learning Roadmap for Causal Inference in ECTs 118\u003c\/p\u003e \u003cp\u003e8.7 Discussion and Summary 123\u003c\/p\u003e \u003cp\u003e8.8 Supplements 124\u003c\/p\u003e \u003cp\u003e\u003cb\u003eChapter 9 Master Protocols 126\u003c\/b\u003e\u003c\/p\u003e \u003cp\u003e9.1 Types and Features of Master Protocols 126\u003c\/p\u003e \u003cp\u003e9.2 Estimands in Master Protocols 128\u003c\/p\u003e \u003cp\u003e9.3 Multiplicity 129\u003c\/p\u003e \u003cp\u003e9.4 Master Protocols Using External Controls 130\u003c\/p\u003e \u003cp\u003e9.5 Case Studies for ECTs 134\u003c\/p\u003e \u003cp\u003e9.6 Discussion and Summary 137\u003c\/p\u003e \u003cp\u003e9.7 Supplements 137\u003c\/p\u003e \u003cp\u003e\u003cb\u003eChapter 10 Decentralized Clinical Trials 139\u003c\/b\u003e\u003c\/p\u003e \u003cp\u003e10.1 Elements of DCTs 140\u003c\/p\u003e \u003cp\u003e10.2 Regulatory Guidance and Framework on DCTs 144\u003c\/p\u003e \u003cp\u003e10.3 Statistical Challenges and Considerations 145\u003c\/p\u003e \u003cp\u003e10.4 Examples 154\u003c\/p\u003e \u003cp\u003e10.5 Discussion and Summary 156\u003c\/p\u003e \u003cp\u003e10.6 Supplements 157\u003c\/p\u003e \u003cp\u003e\u003cb\u003eChapter 11 Drug Development for Rare Diseases 159\u003c\/b\u003e\u003c\/p\u003e \u003cp\u003e11.1 Regulatory Guidance for Rare Diseases 160\u003c\/p\u003e \u003cp\u003e11.2 Challenges in Rare Disease Drug Development 163\u003c\/p\u003e \u003cp\u003e11.3 Strategies to Address the Challenges 168\u003c\/p\u003e \u003cp\u003e11.4 Use of RWD and RWE in Rare Disease Drug Development 174\u003c\/p\u003e \u003cp\u003e11.5 Case Studies 180\u003c\/p\u003e \u003cp\u003e11.6 Discussion and Summary 182\u003c\/p\u003e \u003cp\u003e11.7 Supplements 183\u003c\/p\u003e \u003cp\u003e\u003cb\u003eChapter 12 Time-to-Event Analysis with Treatment Switches 185\u003c\/b\u003e\u003c\/p\u003e \u003cp\u003e12.1 Scenarios of Treatment Switching 185\u003c\/p\u003e \u003cp\u003e12.2 Study Designs Incorporating Treatment Switching 187\u003c\/p\u003e \u003cp\u003e12.3 Strategies to Handle Treatment Switching 189\u003c\/p\u003e \u003cp\u003e12.4 Analytical Methods Handling Treatment Switching 190\u003c\/p\u003e \u003cp\u003e12.5 Considerations for Study Conduct and Data Analysis 195\u003c\/p\u003e \u003cp\u003e12.6 Communication with Regulatory Agencies 196\u003c\/p\u003e \u003cp\u003e12.7 Case Studies 197\u003c\/p\u003e \u003cp\u003e12.8 Discussion and Summary 199\u003c\/p\u003e \u003cp\u003e12.9 Supplements 200\u003c\/p\u003e \u003cp\u003e\u003cb\u003eChapter 13 Precision Medicine 201\u003c\/b\u003e\u003c\/p\u003e \u003cp\u003e13.1 Regulatory Activities and Approvals 202\u003c\/p\u003e \u003cp\u003e13.2 Biomarkers 205\u003c\/p\u003e \u003cp\u003e13.3 Study Designs in Precision Medicine Development 211\u003c\/p\u003e \u003cp\u003e13.4 Analytic Methods and Applications 224\u003c\/p\u003e \u003cp\u003e13.5 Optimal Treatment Regimes 235\u003c\/p\u003e \u003cp\u003e13.6 Discussion and Summary 246\u003c\/p\u003e \u003cp\u003e13.7 Supplements 248\u003c\/p\u003e \u003cp\u003e\u003cb\u003eChapter 14 Vaccine Effectiveness Studies 251\u003c\/b\u003e\u003c\/p\u003e \u003cp\u003e14.1 General Considerations in Vaccine Development 252\u003c\/p\u003e \u003cp\u003e14.2 Immune Response, Immunogenicity, and Early-Phase Clinical Development 254\u003c\/p\u003e \u003cp\u003e14.3 Endpoints, Study Population, and Other Design Considerations in Late-Phase Trials 257\u003c\/p\u003e \u003cp\u003e14.4 Assessing Vaccine Effectiveness Using Real-World Data and Evidence 264\u003c\/p\u003e \u003cp\u003e14.5 Vaccine Safety 265\u003c\/p\u003e \u003cp\u003e14.6 Discussion and Summary 266\u003c\/p\u003e \u003cp\u003e14.7 Supplements 267\u003c\/p\u003e \u003cp\u003e\u003cb\u003eChapter 15 Sensitivity Analyses in Clinical Trials 269\u003c\/b\u003e\u003c\/p\u003e \u003cp\u003e15.1 Primary, Supplementary, Sensitivity, and Exploratory Analyses 269\u003c\/p\u003e \u003cp\u003e15.2 Rationales for Sensitivity Analysis 272\u003c\/p\u003e \u003cp\u003e15.3 Considerations for Sensitivity Analysis 272\u003c\/p\u003e \u003cp\u003e15.4 Methods for Sensitivity Analyses 274\u003c\/p\u003e \u003cp\u003e15.5 Summary and Conclusion 278\u003c\/p\u003e \u003cp\u003e15.6 Supplements 279\u003c\/p\u003e \u003cp\u003e\u003cb\u003eChapter 16 Safety Evaluation 282\u003c\/b\u003e\u003c\/p\u003e \u003cp\u003e16.1 Safety Databases 282\u003c\/p\u003e \u003cp\u003e16.2 Statistical Methods for Analysis of Spontaneous Adverse Event Reports 284\u003c\/p\u003e \u003cp\u003e16.3 A Case Study—Sequential Monitoring in Pragmatic Trials 296\u003c\/p\u003e \u003cp\u003e16.4 Discussion and Summary 299\u003c\/p\u003e \u003cp\u003e16.5 Supplements 300\u003c\/p\u003e \u003cp\u003e\u003cb\u003eChapter 17 Estimands in RWE Studies 302\u003c\/b\u003e\u003c\/p\u003e \u003cp\u003e17.1 Frameworks for Defining Estimands 302\u003c\/p\u003e \u003cp\u003e17.2 Estimands in RWE Studies 307\u003c\/p\u003e \u003cp\u003e17.3 Examples of Estimands in TCTs and RWE Studies 312\u003c\/p\u003e \u003cp\u003e17.4 Discussion and Summary 319\u003c\/p\u003e \u003cp\u003e17.5 Supplements 319\u003c\/p\u003e \u003cp\u003e\u003cb\u003eChapter 18 A Roadmap for Formulating RWE Studies 321\u003c\/b\u003e\u003c\/p\u003e \u003cp\u003e18.1 Who Are the Stakeholders and What Are Their Research Questions? 321\u003c\/p\u003e \u003cp\u003e18.2 What Are the Study Objectives, Designs, and Analytic Methods? 322\u003c\/p\u003e \u003cp\u003e18.3 What Are the Fit-for-Purpose RWD Sources? 322\u003c\/p\u003e \u003cp\u003e18.4 What Are the Treatment Regimes of Interest? 322\u003c\/p\u003e \u003cp\u003e18.5 What Are the Possible Intercurrent Events? 322\u003c\/p\u003e \u003cp\u003e18.6 A Roadmap for Choosing an Appropriate Estimand and RWE Study Design 323\u003c\/p\u003e \u003cp\u003e18.7 Discussion and Summary 323\u003c\/p\u003e \u003cp\u003e18.8 Supplements 325\u003c\/p\u003e \u003cp\u003e\u003cb\u003eChapter 19 Artificial Intelligence and Machine Learning in Clinical Studies 326\u003c\/b\u003e\u003c\/p\u003e \u003cp\u003e19.1 Study Design and Planning 327\u003c\/p\u003e \u003cp\u003e19.2 Study Conduct 330\u003c\/p\u003e \u003cp\u003e19.3 Data Analytics 336\u003c\/p\u003e \u003cp\u003e19.4 Prediction of Clinical Trial Outcomes 346\u003c\/p\u003e \u003cp\u003e19.5 Discussion and Summary 354\u003c\/p\u003e \u003cp\u003e19.6 Supplements 355\u003c\/p\u003e \u003cp\u003eBibliography 361\u003c\/p\u003e \u003cp\u003eIndex 429\u003c\/p\u003e\u003c\/font\u003e\u003c\/p\u003e\r\n\r\n\u003cp\u003e\u003cfont size=\"3\"\u003eSubject Areas: Medicine: general issues [\u003ca title=\"See our other books on Medicine: general issues\" href=\"https:\/\/freshlyprintedbooks.co.uk\/search?q=%22Medicine:%20general%20issues%20%5BMB%5D%22\"\u003eMB\u003c\/a\u003e]\u003c\/font\u003e\u003c\/p\u003e\r\n\r\n\r\n\u003c\/font\u003e","brand":"Wiley","offers":[{"title":"Brand New","offer_id":52433826873624,"sku":"9781394369652","price":82.89,"currency_code":"GBP","in_stock":true}],"thumbnail_url":"\/\/cdn.shopify.com\/s\/files\/1\/0730\/2037\/5320\/files\/9781394369652.jpg?v=1784854463","url":"https:\/\/freshlyprintedbooks.co.uk\/products\/clinical-trials-in-the-era-of-real-world-evidence-paperback-softback-9781394369652","provider":"Freshly Printed Books","version":"1.0","type":"link"}