In October 2019, Abhijit Banerjee, Esther Duflo, and Michael Kremer jointly won the 51st Sveriges Riksbank Prize in Economic Sciences in Memory of Alfred Nobel for their experimental approach to alleviating global poverty. But what is the exact scope of their experimental method, known as randomized control trials (RCTs)? Which sorts of questions are RCTs able to address and which do they fail to answer? The first of its kind, Randomized Control Trials in the Field of Development: A Critical Perspective provides answers to these questions, explaining how RCTs work, what they can achieve, why they sometimes fail, how they can be improved and why other methods are both useful and necessary. Bringing together leading specialists in the field from a range of backgrounds and disciplines (economics, econometrics, mathematics, statistics, political economy, socioeconomics, anthropology, philosophy, global health, epidemiology, and medicine), it presents a full and coherent picture of the main strengths and weaknesses of RCTs in the field of development. Looking beyond the epistemological, political, and ethical differences underlying many of the disagreements surrounding RCTs, it explores the implementation of RCTs on the ground, outside of their ideal theoretical conditions and reveals some unsuspected uses and effects, their disruptive potential, but also their political uses. The contributions uncover the implicit worldview that many RCTs draw on and disseminate, and probe the gap between the method's narrow scope and its success, while also proposing improvements and alternatives. Without disputing the contribution of RCTs to scientific knowledge, Randomized Control Trials in the Field of Development warns against the potential dangers of their excessive use, arguing that the best use for RCTs is not necessarily that which immediately springs to mind. Written in plain language, this book offers experts and laypeople alike a unique opportunity to come to an informed and reasoned judgement on RCTs and what they can bring to development.

Clinical trials are used to elucidate the most appropriate preventive, diagnostic, or treatment options for individuals with a given medical condition. Perhaps the most essential feature of a clinical trial is that it aims to use results based on a limited sample of research participants to see if the intervention is safe and effective or if it is comparable to a comparison treatment. Sample size is a crucial component of any clinical trial. A trial with a small number of research participants is more prone to variability and carries a considerable risk of failing to demonstrate the effectiveness of a given intervention when one really is present. This may occur in phase I (safety and pharmacologic profiles), II (pilot efficacy evaluation), and III (extensive assessment of safety and efficacy) trials. Although phase I and II studies may have smaller sample sizes, they usually have adequate statistical power, which is the committee's definition of a "large" trial. Sometimes a trial with eight participants may have adequate statistical power, statistical power being the probability of rejecting the null hypothesis when the hypothesis is false. Small Clinical Trials assesses the current methodologies and the appropriate situations for the conduct of clinical trials with small sample sizes. This report assesses the published literature on various strategies such as (1) meta-analysis to combine disparate information from several studies including Bayesian techniques as in the confidence profile method and (2) other alternatives such as assessing therapeutic results in a single treated population (e.g., astronauts) by sequentially measuring whether the intervention is falling above or below a preestablished probability outcome range and meeting predesigned specifications as opposed to incremental improvement.

Randomized controlled trials are one of the most powerful and revolutionary tools of research. This book is a convenient and accessible description of the underlying principles and practice of randomized controlled trials and their role in clinical decision-making. Structured in a jargon-free question-and-answer format, each chapter provides concise and understandable information on a different aspect of randomized controlled trials, from the basics of trial design and terminology to the interpretation of results and their use in driving evidence-based medicine. The authors end each chapter with their musings, going beyond the evidence or citations, and sometimes even beyond orthodox correctness to share their thoughts and concerns about different aspects of randomized controlled trials, and their role within the health system. Updated to include insights from the last decade, this second edition challenges over-reliance on randomized controlled trials by debating their strengths and limitations and discussing their optimal use in modern healthcare. It also includes a new and increasingly relevant chapter on the ethics of randomized trials. World renowned writers and thinkers Drs Jadad and Enkin bring you this invaluable book for busy health professionals who wish to understand the theory of randomized controlled trials and their influence on clinical, research or policy decisions.

The Textbook of Pharmacoepidemiology provides a streamlined text for evaluating the safety and effectiveness of medicines. It includes a brief introduction to pharmacoepidemiology as well as sections on data sources, methodology and applications. Each chapter includes key points, case studies and essential references. One-step resource to gain understanding of the subject of pharmacoepidemiology at an affordable price Gives a perspective on the subject from academia, pharmaceutical industry and regulatory agencies Designed for students with basic knowledge of epidemiology and public health Includes many case studies to illustrate pharmacoepidemiology in real clinical setting

Leading economists address the ongoing challenges to economics in theory and practice in a time of political and economic crises. More than a decade of financial crises, sovereign debt problems, political conflict, and rising xenophobia and protectionism has left the global economy unsettled and the ability of economics as a discipline to account for episodes of volatility uncertain. In this book, leading economists consider the state of their discipline in a world of ongoing economic and political crises. The book begins with three sweeping essays by Nobel laureates Kenneth Arrow (in one of his last published works), Amartya Sen, and Joseph Stiglitz that offer a summary of the theoretical foundations of modern economics—the twin pillars of general equilibrium theory and welfare economics. Contributors then turn to macroeconomic stabilization and growth and, finally, new areas of research that depart from traditional theory, methodology, and concerns: climate change, behavioral economics, and evolutionary game theory. The 2019 Nobel Prize laureates, Abhijit Banerjee, Esther Duflo, and Michael Kremer, contribute a paper on the use of randomized control trials indevelopment economics. Contributors Philippe Aghion, Ingela Alger, Kenneth Arrow, Abhijit Banerjee, Kaushik Basu, Lawrence Blume, Guillermo Calvo, Francesco Caselli, Asli Demirgüç-Kunt, Shantayanan Devarajan, Esther Duflo, Samuel Fankhauser, James Foster, Varun Gauri, Xavier Gine, Gäel Giraud, Gita Gopinath, Robert Hockett, Karla Hoff, Ravi Kanbur, Aart Kraay, Michael Kremer, David McKenzie, Célestin Monga, Maurice Obstfeld, Hamid Rashid, Martin Ravallion, Amartya Sen, Luis Servén, Hyun Song Shin, Nicholas Stern, Joseph Stiglitz, Cass Sunstein, Michael Toman, Jörgen Weibull

Health and human services currently face a series of challenges – such as aging populations, chronic diseases and new endemics – that require highly complex responses, and take place in multiple care environments including acute medicine, chronic care facilities and the community. Accordingly, most modern health care interventions are now seen as ‘complex interventions’ – activities that contain a number of component parts with the potential for interactions between them which, when applied to the intended target population, produce a range of possible and variable outcomes. This in turn requires methodological developments that also take into account changing values and attitudes related to the situation of patients’ receiving health care. The first book to place complex interventions within a coherent system of research enquiry, this work is designed to help researchers understand the research processes involved at each stage of developing, testing, evaluating and implementing complex interventions, and assist them to integrate methodological activities to produce secure, evidence-based health care interventions. It begins with conceptual chapters which set out the complex interventions framework, discuss the interrelation between knowledge development and evidence, and explore how mixed methods research contributes to improved health. Structured around the influential UK Medical Research Council guidance for use of complex interventions, four sections, each comprised of bite-sized chapters written by multidisciplinary experts in the area, focus on: - Developing complex interventions - Assessing the feasibility of complex interventions and piloting them - Evaluating complex interventions - Implementing complex interventions. Accessible to students and researchers grappling with complex interventions, each substantive chapter includes an introduction, bulleted learning objectives, clinical examples, a summary and further reading. The perspectives of various stakeholders, including patients, families and professionals, are discussed throughout as are the economic and ethical implications of methods. A vital companion for health research, this book is suitable for readers from multidisciplinary disciplines such as medical, nursing, public health, health services research, human services and allied healthcare backgrounds.

A comprehensive guide to running randomized impact evaluations of social programs in developing countries This book provides a comprehensive yet accessible guide to running randomized impact evaluations of social programs. Drawing on the experience of researchers at the Abdul Latif Jameel Poverty Action Lab, which has run hundreds of such evaluations in dozens of countries throughout the world, it offers practical insights on how to use this powerful technique, especially in resource-poor environments. This step-by-step guide explains why and when randomized evaluations are useful, in what situations they should be used, and how to prioritize different evaluation opportunities. It shows how to design and analyze studies that answer important questions while respecting the constraints of those working on and benefiting from the program being evaluated. The book gives concrete tips on issues such as improving the quality of a study despite tight budget constraints, and demonstrates how the results of randomized impact evaluations can inform policy. With its self-contained modules, this one-of-a-kind guide is easy to navigate. It also includes invaluable references and a checklist of the common pitfalls to avoid. Provides the most up-to-date guide to running randomized evaluations of social programs, especially in developing countries Offers practical tips on how to complete high-quality studies in even the most challenging environments Self-contained modules allow for easy reference and flexible teaching and learning Comprehensive yet nontechnical

A compendium of cutting-edge statistical approaches to solving problems in clinical oncology, Handbook of Statistics in Clinical Oncology, Second Edition focuses on clinical trials in phases I, II, and III, proteomic and genomic studies, complementary outcomes and exploratory methods. Cancer Forum called the first edition a

Providing a comprehensive foundation for planning, executing, and monitoring public health research of all types, this book goes beyond traditional epidemiologic research designs to cover technology-based approaches emerging in the new public health landscape.

Randomized clinical trials are the primary tool for evaluating new medical interventions. Randomization provides for a fair comparison between treatment and control groups, balancing out, on average, distributions of known and unknown factors among the participants. Unfortunately, these studies often lack a substantial percentage of data. This missing data reduces the benefit provided by the randomization and introduces potential biases in the comparison of the treatment groups. Missing data can arise for a variety of reasons, including the inability or unwillingness of participants to meet appointments for evaluation. And in some studies, some or all of data collection ceases when participants discontinue study treatment. Existing guidelines for the design and conduct of clinical trials, and the analysis of the resulting data, provide only limited advice on how to handle missing data. Thus, approaches to the analysis of data with an appreciable amount of missing values tend to be ad hoc and variable. The Prevention and Treatment of Missing Data in Clinical Trials concludes that a more principled approach to design and analysis in the presence of missing data is both needed and possible. Such an approach needs to focus on two critical elements: (1) careful design and conduct to limit the amount and impact of missing data and (2) analysis that makes full use of information on all randomized participants and is based on careful attention to the assumptions about the nature of the missing data underlying estimates of treatment effects. In addition to the highest priority recommendations, the book offers more detailed recommendations on the conduct of clinical trials and techniques for analysis of trial data.