Drugs on Trial PDF Download

Author: Andreas-Holger Maehle
Publisher: BRILL
ISBN: 9004333290
Category : Medical
Languages : en
Pages : 362

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Book Description
This book describes the main issues of eighteenth-century pharmacology and therapeutics and provides detailed case studies of three key areas: lithontriptics (remedies against urinary stones), opium, and Peruvian bark (quinine).

Author: National Research Council
Publisher: National Academies Press
ISBN: 030918651X
Category : Medical
Languages : en
Pages : 163

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Book Description
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.

Author: Tom Brody
Publisher: Academic Press
ISBN: 0128042583
Category : Medical
Languages : en
Pages : 897

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Book Description
Clinical Trials, Second Edition, offers those engaged in clinical trial design a valuable and practical guide. This book takes an integrated approach to incorporate biomedical science, laboratory data of human study, endpoint specification, legal and regulatory aspects and much more with the fundamentals of clinical trial design. It provides an overview of the design options along with the specific details of trial design and offers guidance on how to make appropriate choices. Full of numerous examples and now containing actual decisions from FDA reviewers to better inform trial design, the 2nd edition of Clinical Trials is a must-have resource for early and mid-career researchers and clinicians who design and conduct clinical trials. Contains new and fully revised material on key topics such as biostatistics, biomarkers, orphan drugs, biosimilars, drug regulations in Europe, drug safety, regulatory approval and more Extensively covers the "study schema" and related features of study design Incorporates laboratory data from studies on human patients to provide a concrete tool for understanding the concepts in the design and conduct of clinical trials Includes decisions made by FDA reviewers when granting approval of a drug as real world learning examples for readers

Author: Institute of Medicine
Publisher: National Academies Press
ISBN: 0309316324
Category : Medical
Languages : en
Pages : 304

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Book Description
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.

Author: National Academies of Sciences, Engineering, and Medicine
Publisher: National Academies Press
ISBN: 0309442583
Category : Medical
Languages : en
Pages : 111

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Book Description
On March 3-4, 2016, the National Academies of Sciences, Engineering, and Medicine's Forum on Neuroscience and Nervous System Disorders held a workshop in Washington, DC, bringing together key stakeholders to discuss opportunities for improving the integrity, efficiency, and validity of clinical trials for nervous system disorders. Participants in the workshop represented a range of diverse perspectives, including individuals not normally associated with traditional clinical trials. The purpose of this workshop was to generate discussion about not only what is feasible now, but what may be possible with the implementation of cutting-edge technologies in the future.

Author: Institute of Medicine
Publisher: National Academies Press
ISBN: 0309068886
Category : Medical
Languages : en
Pages : 86

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Book Description
Increasingly over the past five years, uncertainty about reimbursement for routine patient care has been suspected as contributing to problems enrolling people in clinical trials. Clinical trial investigators cannot guarantee that Medicare will pay for the care required, and they must disclose this uncertainty to potential participants during the informed consent process. Since Medicare does not routinely "preauthorize" care (as do many commercial insurers) the uncertainty cannot be dispelled in advance. Thus, patients considering whether to enter trials must assume that they may have to pay bills that Medicare rejects simply because they have enrolled in the trial. This report recommends an explicit policy for reimbursement of routine patient care costs in clinical trials. It further recommends that HCFA provide additional support for selected clinical trials, and that the government support the establishment of a national clinical trials registry. These policies (1) should assure that beneficiaries would not be denied coverage merely because they have volunteered to participate in a clinical trial; and (2) would not impose excessive administrative burdens on HCFA, its fiscal intermediaries and carriers, or investigators, providers, or participants in clinical trials. Explicit rules would have the added benefit of increasing the uniformity of reimbursement decisions made by Medicare fiscal intermediaries and carriers in different parts of the country. Greater uniformity would, in turn, decrease the uncertainty about reimbursement when providers and patients embark on a clinical trial.

Author: Institute of Medicine
Publisher: National Academies Press
ISBN: 9780309171144
Category : Medical
Languages : en
Pages : 222

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Book Description
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.

Author: Institute of Medicine
Publisher: National Academies Press
ISBN: 0309163358
Category : Medical
Languages : en
Pages : 151

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Book Description
An ideal health care system relies on efficiently generating timely, accurate evidence to deliver on its promise of diminishing the divide between clinical practice and research. There are growing indications, however, that the current health care system and the clinical research that guides medical decisions in the United States falls far short of this vision. The process of generating medical evidence through clinical trials in the United States is expensive and lengthy, includes a number of regulatory hurdles, and is based on a limited infrastructure. The link between clinical research and medical progress is also frequently misunderstood or unsupported by both patients and providers. The focus of clinical research changes as diseases emerge and new treatments create cures for old conditions. As diseases evolve, the ultimate goal remains to speed new and improved medical treatments to patients throughout the world. To keep pace with rapidly changing health care demands, clinical research resources need to be organized and on hand to address the numerous health care questions that continually emerge. Improving the overall capacity of the clinical research enterprise will depend on ensuring that there is an adequate infrastructure in place to support the investigators who conduct research, the patients with real diseases who volunteer to participate in experimental research, and the institutions that organize and carry out the trials. To address these issues and better understand the current state of clinical research in the United States, the Institute of Medicine's (IOM) Forum on Drug Discovery, Development, and Translation held a 2-day workshop entitled Transforming Clinical Research in the United States. The workshop, summarized in this volume, laid the foundation for a broader initiative of the Forum addressing different aspects of clinical research. Future Forum plans include further examining regulatory, administrative, and structural barriers to the effective conduct of clinical research; developing a vision for a stable, continuously funded clinical research infrastructure in the United States; and considering strategies and collaborative activities to facilitate more robust public engagement in the clinical research enterprise.

Author: National Research Council
Publisher: National Academies Press
ISBN: 0309166659
Category : Medical
Languages : en
Pages : 320

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Book Description
New and improved therapies to treat and protect against drug dependence and abuse are urgently needed. In the United States alone about 50 million people regularly smoke tobacco and another 5 million are addicted to other drugs. In a given year, millions of these individuals attemptâ€"with or without medical assistanceâ€"to quit using drugs, though relapse remains the norm. Furthermore, each year several million teenagers start smoking and nearly as many take illicit drugs for the first time. Research is advancing on promising new means of treating drug addiction using immunotherapies and sustained-release (depot) medications. The aim of this research is to develop medications that can block or significantly attenuate the psychoactive effects of such drugs as cocaine, nicotine, heroin, phencyclidine, and methamphetamine for weeks or months at a time. This represents a fundamentally new therapeutic approach that shows promise for treating drug addiction problems that were difficult to treat in the past. Despite their potential benefits, however, several characteristics of these new methods pose distinct behavioral, ethical, legal, and social challenges that require careful scrutiny. Such issues can be considered unique aspects of safety and efficacy that are fundamentally related to the distinct nature and properties of these new types of medications.

Author: Shivaani Kummar
Publisher: Academic Press
ISBN: 0128125705
Category : Medical
Languages : en
Pages : 234

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Book Description
Novel Designs of Early Phase Trials for Cancer Therapeutics provides a comprehensive review by leaders in the field of the process of drug development, the integration of molecular profiling, the changes in early phase trial designs, and endpoints to optimally develop a new generation of cancer therapeutics. The book discusses topics such as statistical perspectives on cohort expansions, the role and application of molecular profiling and how to integrate biomarkers in early phase trials. Additionally, it discusses how to incorporate patient reported outcomes in phase one trials. This book is a valuable resource for medical oncologists, basic and translational biomedical scientists, and trainees in oncology and pharmacology who are interested in learning how to improve their research by using early phase trials. Brings a comprehensive review and recommendations for new clinical trial designs for modern cancer therapeutics Provides the reader with a better understanding on how to design and implement early phase oncology trials Presents a better and updated understanding of the process of developing new treatments for cancer, the exciting scientific advances and how they are informing drug development