%0 Book %A Institute of Medicine %E Grossmann, Claudia %E Sanders, Julia %E English, Rebecca A. %T Large Simple Trials and Knowledge Generation in a Learning Health System: Workshop Summary %@ 978-0-309-28911-5 %D 2013 %U https://nap.nationalacademies.org/catalog/18400/large-simple-trials-and-knowledge-generation-in-a-learning-health-system %> https://nap.nationalacademies.org/catalog/18400/large-simple-trials-and-knowledge-generation-in-a-learning-health-system %I The National Academies Press %C Washington, DC %G English %K Health and Medicine %P 118 %X Randomized clinical trials (RCTs) are often referred to as the "gold standard" of clinical research. However, in its current state, the U.S. clinical trials enterprise faces substantial challenges to the efficient and effective conduct of research. Streamlined approaches to RCTs, such as large simple trials (LSTs), may provide opportunities for progress on these challenges. Clinical trials support the development of new medical products and the evaluation of existing products by generating knowledge about safety and efficacy in pre- and post-marketing settings and serve to inform medical decision making and medical product development. Although well-designed and -implemented clinical trials can provide robust evidence, a gap exists between the evidence needs of a continuously learning health system, in which all medical decisions are based on the best available evidence, and the reality, in which the generation of timely and practical evidence faces significant barriers. Large Simple Trials and Knowledge Generation in a Learning Health System is the summary of a workshop convened by the Institute of Medicine's Roundtable on Value & Science-Driven Health Care and the Forum on Drug Discovery, Development, and Translation. Experts from a wide range of disciplines--including health information technology, research funding, clinical research methods, statistics, patients, product development, medical product regulation, and clinical outcomes research--met to marshal a better understanding of the issues, options, and approaches to accelerating the use of LSTs. This publication summarizes discussions on the potential of LSTs to improve the speed and practicality of knowledge generation for medical decision making and medical product development, including efficacy and effectiveness assessments, in a continuously learning health system. Large Simple Trials and Knowledge Generation in a Learning Health System explores acceleration of the use of LSTs to improve the speed and practicality of knowledge generation for medical decision making and medical product development; considers the concepts of LST design, examples of successful LSTs, the relative advantages of LSTs, and the infrastructure needed to build LST capacity as a routine function of care; identifies structural, cultural, and regulatory barriers hindering the development of an enhanced LST capacity; discusses needs and strategies in building public demand for and participation in LSTs; and considers near-term strategies for accelerating progress in the uptake of LSTs in the United States. %0 Book %A Institute of Medicine %T Developing a National Registry of Pharmacologic and Biologic Clinical Trials: Workshop Report %@ 978-0-309-10078-6 %D 2006 %U https://nap.nationalacademies.org/catalog/11561/developing-a-national-registry-of-pharmacologic-and-biologic-clinical-trials %> https://nap.nationalacademies.org/catalog/11561/developing-a-national-registry-of-pharmacologic-and-biologic-clinical-trials %I The National Academies Press %C Washington, DC %G English %K Health and Medicine %P 124 %X To improve public confidence in clinical research, a number of public and private groups have called for a publicly accessible, comprehensive, and transparent registry of relevant information on clinical trials for drugs and biologics. The public and various entities within the medical community (health care providers, researchers, medical journal editors, pharmaceutical companies, health insurers, and regulators) have different expectations and perceived needs regarding a public clinical trial registry. The IOM Committee on Clinical Trial Registries hosted a workshop on June 27, 2005, to obtain much-needed input from members of the public, public advocate groups, and the broader community of journal editors, pharmaceutical and biotech leaders, NIH, and the FDA. Participants discussed the data elements that have been at the core of debate and commented on issues of compliance and implementation of a national clinical trial registry. Developing a National Registry of Pharmacologic and Biologic Clinical Trials: Workshop Report inlcudes discussions at the workshop centered on the following five concepts, and are described within this report: 1) Purpose, 2) Which Trials to Include, 3) Delayed Disclosure Mechanism, 4) Reporting Results of Completed Trials, and 5) Compliance. %0 Book %A Institute of Medicine %A National Academies of Sciences, Engineering, and Medicine %T Ovarian Cancers: Evolving Paradigms in Research and Care %@ 978-0-309-38046-1 %D 2016 %U https://nap.nationalacademies.org/catalog/21841/ovarian-cancers-evolving-paradigms-in-research-and-care %> https://nap.nationalacademies.org/catalog/21841/ovarian-cancers-evolving-paradigms-in-research-and-care %I The National Academies Press %C Washington, DC %G English %K Health and Medicine %K Biology and Life Sciences %P 396 %X In an era of promising advances in cancer research, there are considerable and even alarming gaps in the fundamental knowledge and understanding of ovarian cancer. Researchers now know that ovarian cancer is not a single disease—several distinct subtypes exist with different origins, risk factors, genetic mutations, biological behaviors, and prognoses. However, persistent questions have impeded progress toward improving the prevention, early detection, treatment, and management of ovarian cancers. Failure to significantly improve morbidity and mortality during the past several decades is likely due to several factors, including the lack of research being performed by specific disease subtype, lack of definitive knowledge of the cell of origin and disease progression, and incomplete understanding of genetic and non-genetic risk factors. Ovarian Cancers examines the state of the science in ovarian cancer research, identifies key gaps in the evidence base and the challenges to addressing those gaps, considers opportunities for advancing ovarian cancer research, and examines avenues for translation and dissemination of new findings and communication of new information to patients and others. This study makes recommendations for public- and private-sector efforts that could facilitate progress in reducing the incidence of morbidity and mortality from ovarian cancers. %0 Book %A Institute of Medicine %T Sharing Clinical Trial Data: Maximizing Benefits, Minimizing Risk %@ 978-0-309-31629-3 %D 2015 %U https://nap.nationalacademies.org/catalog/18998/sharing-clinical-trial-data-maximizing-benefits-minimizing-risk %> https://nap.nationalacademies.org/catalog/18998/sharing-clinical-trial-data-maximizing-benefits-minimizing-risk %I The National Academies Press %C Washington, DC %G English %K Health and Medicine %P 304 %X 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. %0 Book %A Institute of Medicine %E Nass, Sharyl J. %E Moses, Harold L. %E Mendelsohn, John %T A National Cancer Clinical Trials System for the 21st Century: Reinvigorating the NCI Cooperative Group Program %@ 978-0-309-15186-3 %D 2010 %U https://nap.nationalacademies.org/catalog/12879/a-national-cancer-clinical-trials-system-for-the-21st-century %> https://nap.nationalacademies.org/catalog/12879/a-national-cancer-clinical-trials-system-for-the-21st-century %I The National Academies Press %C Washington, DC %G English %K Health and Medicine %P 316 %X The National Cancer Institute's (NCI) Clinical Trials Cooperative Group Program has played a key role in developing new and improved cancer therapies. However, the program is falling short of its potential, and the IOM recommends changes that aim to transform the Cooperative Group Program into a dynamic system that efficiently responds to emerging scientific knowledge; involves broad cooperation of stakeholders; and leverages evolving technologies to provide high-quality, practice-changing research.