N Of 1

N-of-1 trials, a type of individualized randomized controlled trial, are relevant to almost every discipline in medicine and psychology. They can tell the clinician with precision whether a treatment works in that individual, which distinguishes from the information available from most other trial designs. They have the potential to revolutionize the way clinical medicine is practiced. Whether you are a busy clinician, a researcher or a student, this book provides everything you need to know about N-of-1 trials. Written and edited by some of the world’s leading experts on N-of-1 trials, the book presents state of the art knowledge about N-of-1 trials, with chapters on ethics, statistics, health economics, design, analysis and reporting, and more. Full of examples and well illustrated, it is a comprehensive compendium of issues surrounding the design, conduct, interpretation and implementation of N-of-1 trials in a health system.

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.

Twenty-five years ago my doctors had no cure for my cancer. So I went on a quest to find my own treatment. This is my story... Glenn Sabin was diagnosed with "incurable" leukemia (CLL). He embarked on a journey researching lifestyle changes, and conducted a personal experiment, chronicled through Harvard, now part of the medical literature.

This book provides a practical guide to the analysis of data from randomized controlled trials (RCT). It gives an answer to the question of how to estimate the intervention effect in an appropriate way. This problem is examined for different RCT designs, such as RCTs with one follow-up measurement, RCTs with more than one follow-up measurement, cluster RCTs, cross-over trials, stepped wedge trials, and N-of-1 trials. The statistical methods are explained in a non-mathematical way and are illustrated by extensive examples. All datasets used in the book are available for download, so readers can reanalyse the examples to gain a better understanding of the methods used. Although most examples are taken from epidemiological and clinical studies, this book is also highly recommended for researchers working in other fields.

Advances in medical, biomedical and health services research have reduced the level of uncertainty in clinical practice. Clinical practice guidelines (CPGs) complement this progress by establishing standards of care backed by strong scientific evidence. CPGs are statements that include recommendations intended to optimize patient care. These statements are informed by a systematic review of evidence and an assessment of the benefits and costs of alternative care options. Clinical Practice Guidelines We Can Trust examines the current state of clinical practice guidelines and how they can be improved to enhance healthcare quality and patient outcomes. Clinical practice guidelines now are ubiquitous in our healthcare system. The Guidelines International Network (GIN) database currently lists more than 3,700 guidelines from 39 countries. Developing guidelines presents a number of challenges including lack of transparent methodological practices, difficulty reconciling conflicting guidelines, and conflicts of interest. Clinical Practice Guidelines We Can Trust explores questions surrounding the quality of CPG development processes and the establishment of standards. It proposes eight standards for developing trustworthy clinical practice guidelines emphasizing transparency; management of conflict of interest ; systematic review-guideline development intersection; establishing evidence foundations for and rating strength of guideline recommendations; articulation of recommendations; external review; and updating. Clinical Practice Guidelines We Can Trust shows how clinical practice guidelines can enhance clinician and patient decision-making by translating complex scientific research findings into recommendations for clinical practice that are relevant to the individual patient encounter, instead of implementing a one size fits all approach to patient care. This book contains information directly related to the work of the Agency for Healthcare Research and Quality (AHRQ), as well as various Congressional staff and policymakers. It is a vital resource for medical specialty societies, disease advocacy groups, health professionals, private and international organizations that develop or use clinical practice guidelines, consumers, clinicians, and payers.

Data sharing can accelerate new discoveries by avoiding duplicative trials, stimulating new ideas for research, and enabling the maximal scientific knowledge and benefits to be gained from the efforts of clinical trial participants and investigators. At the same time, sharing clinical trial data presents risks, burdens, and challenges. These include the need to protect the privacy and honor the consent of clinical trial participants; safeguard the legitimate economic interests of sponsors; and guard against invalid secondary analyses, which could undermine trust in clinical trials or otherwise harm public health. Sharing Clinical Trial Data presents activities and strategies for the responsible sharing of clinical trial data. With the goal of increasing scientific knowledge to lead to better therapies for patients, this book identifies guiding principles and makes recommendations to maximize the benefits and minimize risks. This report offers guidance on the types of clinical trial data available at different points in the process, the points in the process at which each type of data should be shared, methods for sharing data, what groups should have access to data, and future knowledge and infrastructure needs. Responsible sharing of clinical trial data will allow other investigators to replicate published findings and carry out additional analyses, strengthen the evidence base for regulatory and clinical decisions, and increase the scientific knowledge gained from investments by the funders of clinical trials. The recommendations of Sharing Clinical Trial Data will be useful both now and well into the future as improved sharing of data leads to a stronger evidence base for treatment. This book will be of interest to stakeholders across the spectrum of research-from funders, to researchers, to journals, to physicians, and ultimately, to patients.

This User’s Guide is a resource for investigators and stakeholders who develop and review observational comparative effectiveness research protocols. It explains how to (1) identify key considerations and best practices for research design; (2) build a protocol based on these standards and best practices; and (3) judge the adequacy and completeness of a protocol. Eleven chapters cover all aspects of research design, including: developing study objectives, defining and refining study questions, addressing the heterogeneity of treatment effect, characterizing exposure, selecting a comparator, defining and measuring outcomes, and identifying optimal data sources. Checklists of guidance and key considerations for protocols are provided at the end of each chapter. The User’s Guide was created by researchers affiliated with AHRQ’s Effective Health Care Program, particularly those who participated in AHRQ’s DEcIDE (Developing Evidence to Inform Decisions About Effectiveness) program. Chapters were subject to multiple internal and external independent reviews. More more information, please consult the Agency website: www.effectivehealthcare.ahrq.gov)

In 1996, the Institute of Medicine (IOM) released its report Telemedicine: A Guide to Assessing Telecommunications for Health Care. In that report, the IOM Committee on Evaluating Clinical Applications of Telemedicine found telemedicine is similar in most respects to other technologies for which better evidence of effectiveness is also being demanded. Telemedicine, however, has some special characteristics-shared with information technologies generally-that warrant particular notice from evaluators and decision makers. Since that time, attention to telehealth has continued to grow in both the public and private sectors. Peer-reviewed journals and professional societies are devoted to telehealth, the federal government provides grant funding to promote the use of telehealth, and the private technology industry continues to develop new applications for telehealth. However, barriers remain to the use of telehealth modalities, including issues related to reimbursement, licensure, workforce, and costs. Also, some areas of telehealth have developed a stronger evidence base than others. The Health Resources and Service Administration (HRSA) sponsored the IOM in holding a workshop in Washington, DC, on August 8-9 2012, to examine how the use of telehealth technology can fit into the U.S. health care system. HRSA asked the IOM to focus on the potential for telehealth to serve geographically isolated individuals and extend the reach of scarce resources while also emphasizing the quality and value in the delivery of health care services. This workshop summary discusses the evolution of telehealth since 1996, including the increasing role of the private sector, policies that have promoted or delayed the use of telehealth, and consumer acceptance of telehealth. The Role of Telehealth in an Evolving Health Care Environment: Workshop Summary discusses the current evidence base for telehealth, including available data and gaps in data; discuss how technological developments, including mobile telehealth, electronic intensive care units, remote monitoring, social networking, and wearable devices, in conjunction with the push for electronic health records, is changing the delivery of health care in rural and urban environments. This report also summarizes actions that the U.S. Department of Health and Human Services (HHS) can undertake to further the use of telehealth to improve health care outcomes while controlling costs in the current health care environment.

This book is a practical resource designed for clinicians, researchers, and advanced students who wish to learn about single-case research designs. It covers the theoretical and methodological underpinnings of single-case designs, as well as their practical application in the clinical and research neurorehabilitation setting. The book briefly traces the history of single-case experimental designs (SCEDs); outlines important considerations in understanding and planning a scientifically rigorous single-case study, including internal and external validity; describes prototypical single-case designs (withdrawal-reversal designs and the medical N-of-1 trial, multiple-baseline designs, alternating-treatments designs, and changing-criterion designs) and required features to meet evidence standards, threats to internal validity, and strategies to address them; addresses data evaluation, covering visual analysis of graphed data, statistical techniques, and clinical significance; and provides a practical ten-step procedure for implementing single-case methods. Each chapter includes detailed illustrative examples from the neurorehabilitation literature. Novel features include: A focus on the neurorehabilitation setting, which is particularly suitable for single-case designs because of the complex and often unique presentation of many patients/clients. A practical approach to the planning, implementation, data analysis, and reporting of single-case designs. An appendix providing a detailed summary of many recently published SCEDs in representative domains in the neurorehabilitation field, covering basic and instrumental activities of daily living, challenging behaviours, disorders of communication and cognition, mood and emotional functions, and motor-sensory disabilities. It is valuable reading for clinicians and researchers in several disciplines working in rehabilitation, including clinical and neuropsychology, education, language and speech pathology, occupational therapy, and physical therapy. It is also an essential resource for advanced students in these fields who need a textbook for specialised courses on research methodology and use of single-case design in applied clinical and research settings.

This book, n=1 is a collaboration of physician health system CEO, venture capital entrepreneur and leading global business advisor and best-selling business author. These differing perspectives provide insights into the forces transforming the global society, business and professions with a focus on US healthcare and its transformation. Healthcare leaders must develop the incisive questions that challenge the orthodoxies hindering organizational transformation. Our experience indicates leaders of successful non-healthcare organizations develop cultures of inquiry that guide their organizations through dramatic market change. Unique individuals are the driving force in the transformation of healthcare. Digitization has democratized information, which feeds the desire of people to act, behave and be treated as unique individuals. Scientific innovation is revealing the importance of our biologic individuality. The financial risk of healthcare is increasingly passed to individuals and providers, fueling changes in financial incentives. An individual with information knows their options, and wants to choose the option most suited to his or her unique healthcare needs and financial means. This profound and fundamental change in the individual’s expectations and behavior is accelerating healthcare transformation. The title of this book, n = 1, is a symbol of the uniqueness of individuals. The n=1 will transform healthcare.