Download or read book Regulatory Effectiveness Methodology written by S. Cohen and published by . This book was released on 1979 with total page 550 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Download or read book Regulatory Effectiveness Methodology Phase II Research written by and published by . This book was released on 1979 with total page 556 pages. Available in PDF, EPUB and Kindle. Book excerpt: An integrated package of methods is presented for evaluating the benefits and costs of USCG recreational boating safety (and similar) programs and regulations. A general procedure for developing specific accident model data bases is presented along with a selection guide of model forms. An Accident Recovery Model is described in depth and used to develop benefit estimations. Guidelines for and examples of the multistate benefit analysis technique are presented. The Box-Jenkins approach to time series analysis is described and used to generate forecasts of future boating fatalities. Equations are developed for predicting the potential benefits of contemplated regulations. Analytic techniques, including intervention analysis and Benefit Assessment Diagram methods, are presented for assessing the past benefit effects of USCG regulations. Benefit evaluation examples include the level flotation, safe powering, safe loading, and basic flotation regulations. Some basic interrelationship models are presented to illustrate the nature of the marine market and the cost transfers required for tracking cost elements.
Download or read book The Prevention and Treatment of Missing Data in Clinical Trials written by National Research Council and published by National Academies Press. This book was released on 2010-12-21 with total page 163 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.
Download or read book Evaluation of Strategies for the Phase II to Phase III Progression in Treatment Discovery written by Brittany J. Sanchez and published by . This book was released on 2014 with total page 112 pages. Available in PDF, EPUB and Kindle. Book excerpt: The goal of clinical research is to improve the health of the population through the prevention, diagnosis, and treatment of disease. Clinical trials are essential for reliably evaluating a proposed treatment to determine whether it should be adopted into clinical practice. Current standards involve the evaluation of a new treatment through several phases of investigation. After preliminary evaluations of the safety and ethics of further study, promising treatments are studied in preliminary screening trials and then ultimately large, confirmatory trials. Although well developed, the "treatment discovery process" is lengthy, expensive, and has low success rates for treatments even at confirmatory phases of the investigation. Improvements to trial design and implementation are necessary for better achieving the goals of clinical research. In this research, we consider the progression of studies for investigating a new treatment, and discuss strategies in a framework that encompasses the period from the start of preliminary Phase II studies to the completion of the confirmatory Phase III studies. Using a general notational framework for evaluating new treatments, we examine optimality criteria for a strategy that best addresses the often competing goals of science, ethics, and efficiency. These optimality criteria include not only the standard frequentest operating characteristics of type I error and power and the standard Bayesian criteria of positive and negative predictive values, but also the efficiency considerations of the number of new treatments identified in a setting with limited resources. We parameterize the Phase II and Phase III designs using frequentest type I error and power in such a way as to attain high Bayesian positive predictive value (PPV). We then explore the impact specific choices of those design parameters have on the number of effective and ineffective treatments identified with constrained resources. We illustrate how allowing for early trial termination for efficacy or futility with a group sequential design (GSD) within Phase II and/or Phase III improves efficiency in terms of the number of subjects used on average for identifying effective therapies. Other methods for improving efficiency by eliminating the time spent between Phase II and Phase III have been proposed. A "seamless" Phase II/III trial design is one that combines the Phase II screening stage with the Phase III confirmatory stage. We consider how a single sequential design differs from the optimal approach of independent stages. We then explore how the traditional approach of adapting hypotheses at the end of Phase II fits in with the newer adaptive methods. We discuss how powering of Phase III based on Phase II results mimics adaptive sample size re-estimation / re-powering of study and does not offer improvement beyond that of GSDs. Bias in the estimate of the treatment effect is a result of the lack of precision of small samples inherent in Phase II studies and at early interim analyses. We investigate how such bias can be addressed with adjustment methods. We then examine differences between conducting subgroup analyses when there exist homogeneous versus heterogeneous effects and how inflation of the type I error can be controlled in this setting and in the setting of considering multiple summary measures. In our research, we thus demonstrate that the optimal Phase II to Phase III progression defined by an acceptable PPV and a maximal number of effective treatments can be identified for an anticipated prevalence and hypothesized resources by a parameterization of type I error and power at Phase II. We recognize that several approaches lead to the same optimality criteria, and that the chosen strategy will depend on individual objectives of clinical researchers, trial sponsors, regulatory agencies, patients on study, and those who might benefit from new knowledge about treatments being studied.
Download or read book The Social Impact of AIDS in the United States written by National Research Council and published by National Academies Press. This book was released on 1993-02-01 with total page 337 pages. Available in PDF, EPUB and Kindle. Book excerpt: Europe's "Black Death" contributed to the rise of nation states, mercantile economies, and even the Reformation. Will the AIDS epidemic have similar dramatic effects on the social and political landscape of the twenty-first century? This readable volume looks at the impact of AIDS since its emergence and suggests its effects in the next decade, when a million or more Americans will likely die of the disease. The Social Impact of AIDS in the United States addresses some of the most sensitive and controversial issues in the public debate over AIDS. This landmark book explores how AIDS has affected fundamental policies and practices in our major institutions, examining: How America's major religious organizations have dealt with sometimes conflicting values: the imperative of care for the sick versus traditional views of homosexuality and drug use. Hotly debated public health measures, such as HIV antibody testing and screening, tracing of sexual contacts, and quarantine. The potential risk of HIV infection to and from health care workers. How AIDS activists have brought about major change in the way new drugs are brought to the marketplace. The impact of AIDS on community-based organizations, from volunteers caring for individuals to the highly political ACT-UP organization. Coping with HIV infection in prisons. Two case studies shed light on HIV and the family relationship. One reports on some efforts to gain legal recognition for nonmarital relationships, and the other examines foster care programs for newborns with the HIV virus. A case study of New York City details how selected institutions interact to give what may be a picture of AIDS in the future. This clear and comprehensive presentation will be of interest to anyone concerned about AIDS and its impact on the country: health professionals, sociologists, psychologists, advocates for at-risk populations, and interested individuals.
Download or read book Sharing Clinical Trial Data written by Institute of Medicine and published by National Academies Press. This book was released on 2015-04-20 with total page 236 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.
Download or read book Energy Research Abstracts written by and published by . This book was released on 1993 with total page 622 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Download or read book Quantitative Methods for Traditional Chinese Medicine Development written by Shein-Chung Chow and published by CRC Press. This book was released on 2015-10-15 with total page 433 pages. Available in PDF, EPUB and Kindle. Book excerpt: In recent years, many pharmaceutical companies and clinical research organizations have been focusing on the development of traditional Chinese (herbal) medicines (TCMs) as alternatives to treating critical or life-threatening diseases and as pathways to personalized medicine. Quantitative Methods for Traditional Chinese Medicine Development is the first book entirely devoted to the design and analysis of TCM development from a Western perspective, i.e., evidence-based clinical research and development. The book provides not only a comprehensive summary of innovative quantitative methods for developing TCMs but also a useful desk reference for principal investigators involved in personalized medicine. Written by one of the world's most prominent biostatistics researchers, the book connects the pharmaceutical industry, regulatory agencies, and academia. It presents a state-of-the-art examination of the subject for: Scientists and researchers who are engaged in pharmaceutical/clinical research and development of TCMs Those in regulatory agencies who make decisions in the review and approval process of TCM regulatory submissions Biostatisticians who provide statistical support to assess clinical safety and effectiveness of TCMs and related issues regarding quality control and assurance as well as to test for consistency in the manufacturing processes for TCMs This book covers all of the statistical issues encountered at various stages of pharmaceutical/clinical development of a TCM. It explains regulatory requirements; product specifications and standards; and various statistical techniques for evaluation of TCMs, validation of diagnostic procedures, and testing consistency
Download or read book Principles of Pharmacogenetics and Pharmacogenomics written by Russ B. Altman and published by Cambridge University Press. This book was released on 2012-01-23 with total page 283 pages. Available in PDF, EPUB and Kindle. Book excerpt: This textbook presents the latest information on pharmacogenetics and pharmacogenomics for students, professionals and researchers.
Download or read book Journal of Research of the National Bureau of Standards written by and published by . This book was released on 1976 with total page 1270 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Download or read book Publications written by United States. National Bureau of Standards and published by . This book was released on 1977 with total page 766 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Download or read book Catalog of National Bureau of Standards Publications 1966 1976 pt 1 Citations and abstracts v 2 pt 1 Key word index A through L v 2 pt 2 Key word index M through Z written by United States. National Bureau of Standards. Technical Information and Publications Division and published by . This book was released on 1978 with total page 804 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Download or read book Publications of the National Bureau of Standards written by United States. National Bureau of Standards and published by . This book was released on 1976 with total page 744 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Download or read book Publications of the National Bureau of Standards Catalog written by United States. National Bureau of Standards and published by . This book was released on 1977 with total page 748 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Download or read book Publications of the National Institute of Standards and Technology Catalog written by National Institute of Standards and Technology (U.S.) and published by . This book was released on 1977 with total page 746 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Download or read book Small Clinical Trials written by Institute of Medicine and published by National Academies Press. This book was released on 2001-01-01 with total page 221 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.
Download or read book Regulatory Burden written by United States. General Accounting Office and published by . This book was released on 1993 with total page 76 pages. Available in PDF, EPUB and Kindle. Book excerpt:
