Author : J. Venitz
Publisher : Springer Science & Business Media
Page : 224 pages
File Size : 46,27 MB
Release : 2007-03-06
Category : Medical
ISBN : 3540495290
Optimal dose individualization has become more important in improving clinical efficacy and safety. This is due in part to the variability in drug response. Therefore, the role of optimal dose finding in early clinical drug development so as to maximize successful clinical use is emphasized. This book reviews innovative methods, tools and examples of rational drug development strategies, particularly for novel oncological agents.
Author : Institute of Medicine
Publisher : National Academies Press
Page : 221 pages
File Size : 14,84 MB
Release : 2001-01-01
Category : Medical
ISBN : 0309171148
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
Page : 107 pages
File Size : 13,90 MB
Release : 2014-02-06
Category : Medical
ISBN : 0309292492
Improving and Accelerating Therapeutic Development for Nervous System Disorders is the summary of a workshop convened by the IOM Forum on Neuroscience and Nervous System Disorders to examine opportunities to accelerate early phases of drug development for nervous system drug discovery. Workshop participants discussed challenges in neuroscience research for enabling faster entry of potential treatments into first-in-human trials, explored how new and emerging tools and technologies may improve the efficiency of research, and considered mechanisms to facilitate a more effective and efficient development pipeline. There are several challenges to the current drug development pipeline for nervous system disorders. The fundamental etiology and pathophysiology of many nervous system disorders are unknown and the brain is inaccessible to study, making it difficult to develop accurate models. Patient heterogeneity is high, disease pathology can occur years to decades before becoming clinically apparent, and diagnostic and treatment biomarkers are lacking. In addition, the lack of validated targets, limitations related to the predictive validity of animal models - the extent to which the model predicts clinical efficacy - and regulatory barriers can also impede translation and drug development for nervous system disorders. Improving and Accelerating Therapeutic Development for Nervous System Disorders identifies avenues for moving directly from cellular models to human trials, minimizing the need for animal models to test efficacy, and discusses the potential benefits and risks of such an approach. This report is a timely discussion of opportunities to improve early drug development with a focus toward preclinical trials.
Author : Rajesh Krishna
Publisher : CRC Press
Page : 328 pages
File Size : 20,80 MB
Release : 2006-05
Category : Medical
ISBN :
This reference provides a concise overview of the key principles in dose selection and optimization and demonstrates applicability to recent successful new drug applications. Compiling key issues and current research on safety, efficacy, and clinical pharmacology, and PK-PD, this volume critically highlights the multidisciplinary nature of drug development and spans the fields of pharmacokinetics, clinical pharmacology, biostatistics, and experimental medicine.
Author : Divya Vohora
Publisher : Academic Press
Page : 527 pages
File Size : 13,26 MB
Release : 2017-11-14
Category : Medical
ISBN : 0128020989
Pharmaceutical Medicine and Translational Clinical Research covers clinical testing of medicines and the translation of pharmaceutical drug research into new medicines, also focusing on the need to understand the safety profile of medicine and the benefit-risk balance. Pharmacoeconomics and the social impact of healthcare on patients and public health are also featured. It is written in a clear and straightforward manner to enable rapid review and assimilation of complex information and contains reader-friendly features.As a greater understanding of these aspects is critical for students in the areas of pharmaceutical medicine, clinical research, pharmacology and pharmacy, as well as professionals working in the pharmaceutical industry, this book is an ideal resource. - Includes detailed coverage of current trends and key topics in pharmaceutical medicine, including biosimilars, biobetters, super generics, and - Provides a comprehensive look at current and important aspects of the science and regulation of drug and biologics discovery
Author : National Research Council
Publisher : National Academies Press
Page : 163 pages
File Size : 33,20 MB
Release : 2010-12-21
Category : Medical
ISBN : 030918651X
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 : Elizabeth A. Eisenhauer
Publisher : Oxford University Press, USA
Page : 369 pages
File Size : 14,85 MB
Release : 2014-06
Category : Language Arts & Disciplines
ISBN : 0199359016
Phase I trials are a critical first step in the study of novel cancer therapeutic approaches. As this title is the only comprehensive book on this topic, it is a useful resource for oncology trainees or specialists interested in understanding cancer drug development. New to this edition are chapters on Phase 0 Trials and Immunotherapeutics, and updated information on the process, pitfalls, and logistics of Phase I Trials.
Author : National Academies of Sciences, Engineering, and Medicine
Publisher : National Academies Press
Page : 145 pages
File Size : 29,53 MB
Release : 2018-02-12
Category : Medical
ISBN : 0309457971
Advances in cancer research have led to an improved understanding of the molecular mechanisms underpinning the development of cancer and how the immune system responds to cancer. This influx of research has led to an increasing number and variety of therapies in the drug development pipeline, including targeted therapies and associated biomarker tests that can select which patients are most likely to respond, and immunotherapies that harness the body's immune system to destroy cancer cells. Compared with standard chemotherapies, these new cancer therapies may demonstrate evidence of benefit and clearer distinctions between efficacy and toxicity at an earlier stage of development. However, there is a concern that the traditional processes for cancer drug development, evaluation, and regulatory approval could impede or delay the use of these promising cancer treatments in clinical practice. This has led to a number of effortsâ€"by patient advocates, the pharmaceutical industry, and the Food and Drug Administration (FDA)â€"to accelerate the review of promising new cancer therapies, especially for cancers that currently lack effective treatments. However, generating the necessary data to confirm safety and efficacy during expedited drug development programs can present a unique set of challenges and opportunities. To explore this new landscape in cancer drug development, the National Academies of Sciences, Engineering, and Medicine developed a workshop held in December 2016. This workshop convened cancer researchers, patient advocates, and representatives from industry, academia, and government to discuss challenges with traditional approaches to drug development, opportunities to improve the efficiency of drug development, and strategies to enhance the information available about a cancer therapy throughout its life cycle in order to improve its use in clinical practice. This publication summarizes the presentations and discussions from the workshop.
Author : J. Venitz
Publisher : Springer
Page : 216 pages
File Size : 19,51 MB
Release : 2009-09-02
Category : Medical
ISBN : 9783540813262
Optimal dose individualization has become more important in improving clinical efficacy and safety. This is due in part to the variability in drug response. Therefore, the role of optimal dose finding in early clinical drug development so as to maximize successful clinical use is emphasized. This book reviews innovative methods, tools and examples of rational drug development strategies, particularly for novel oncological agents.
Author : Wei Zhao
Publisher : CRC Press
Page : 236 pages
File Size : 47,91 MB
Release : 2014-12-19
Category : Mathematics
ISBN : 1482216752
The growing interest in using combination drugs to treat various complex diseases has spawned the development of many novel statistical methodologies. The theoretical development, coupled with advances in statistical computing, makes it possible to apply these emerging statistical methods in in vitro and in vivo drug combination assessments. Howeve
