Appropriate Dose Selection How To Optimize Clinical Drug Development
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Appropriate Dose Selection - How to Optimize Clinical Drug Development by J. Venitz,W. Sittner Pdf
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.
Dose Optimization in Drug Development by Rajesh Krishna Pdf
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.
Dose Finding in Drug Development by Naitee Ting Pdf
If you have ever wondered when visiting the pharmacy how the dosage of your prescription is determined this book will answer your questions. Dosing information on drug labels is based on discussion between the pharmaceutical manufacturer and the drug regulatory agency, and the label is a summary of results obtained from many scientific experiments. The book introduces the drug development process, the design and the analysis of clinical trials. Many of the discussions are based on applications of statistical methods in the design and analysis of dose response studies. Important procedural steps from a pharmaceutical industry perspective are also examined.
Institute of Medicine,Board on Health Sciences Policy,Committee on Strategies for Small-Number-Participant Clinical Research Trials
Author : Institute of Medicine,Board on Health Sciences Policy,Committee on Strategies for Small-Number-Participant Clinical Research Trials Publisher : National Academies Press Page : 222 pages File Size : 42,6 Mb Release : 2001-01-01 Category : Medical ISBN : 0309171148
Small Clinical Trials by Institute of Medicine,Board on Health Sciences Policy,Committee on Strategies for Small-Number-Participant Clinical Research Trials Pdf
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.
Institute of Medicine,Board on Health Sciences Policy,Forum on Neuroscience and Nervous System Disorders
Author : Institute of Medicine,Board on Health Sciences Policy,Forum on Neuroscience and Nervous System Disorders Publisher : National Academies Press Page : 118 pages File Size : 52,6 Mb Release : 2014-02-06 Category : Medical ISBN : 9780309292498
Improving and Accelerating Therapeutic Development for Nervous System Disorders by Institute of Medicine,Board on Health Sciences Policy,Forum on Neuroscience and Nervous System Disorders Pdf
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.
Pharmaceutical Medicine and Translational Clinical Research by Divya Vohora,Gursharan Singh Pdf
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
National Research Council,Division of Behavioral and Social Sciences and Education,Committee on National Statistics,Panel on Handling Missing Data in Clinical Trials
Author : National Research Council,Division of Behavioral and Social Sciences and Education,Committee on National Statistics,Panel on Handling Missing Data in Clinical Trials Publisher : National Academies Press Page : 163 pages File Size : 51,5 Mb Release : 2010-12-21 Category : Medical ISBN : 9780309186513
The Prevention and Treatment of Missing Data in Clinical Trials by National Research Council,Division of Behavioral and Social Sciences and Education,Committee on National Statistics,Panel on Handling Missing Data in Clinical Trials Pdf
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.
Statistical Methods in Drug Combination Studies by Wei Zhao,Harry Yang Pdf
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
The focus of early drug development has been the submission of an Investigational New Drug application to regulatory agencies. Early Drug Development: Strategies and Routes to First-in-Human Trials guides drug development organizations in preparing and submitting an Investigational New Drug (IND) application. By explaining the nuts and bolts of preclinical development activities and their interplay in effectively identifying successful clinical candidates, the book helps pharmaceutical scientists determine what types of discovery and preclinical research studies are needed in order to support a submission to regulatory agencies.
Institute of Medicine,Board on Health Sciences Policy,Forum on Drug Discovery, Development, and Translation
Author : Institute of Medicine,Board on Health Sciences Policy,Forum on Drug Discovery, Development, and Translation Publisher : National Academies Press Page : 64 pages File Size : 55,8 Mb Release : 2008-08-12 Category : Medical ISBN : 9780309178655
Addressing the Barriers to Pediatric Drug Development by Institute of Medicine,Board on Health Sciences Policy,Forum on Drug Discovery, Development, and Translation Pdf
Decades of research have demonstrated that children do not respond to medications in the same way as adults. Differences between children and adults in the overall response to medications are due to profound anatomical, physiological, and developmental differences. Although few would argue that children should receive medications that have not been adequately tested for safety and efficacy, the majority of drugs prescribed for children-50 to 75 percent-have not been tested in pediatric populations. Without adequate data from such testing, prescribing drugs appropriately becomes challenging for clinicians treating children, from infancy through adolescence. Addressing the Barriers to Pediatric Drug Development is the summary of a workshop, held in Washington, D.C. on June 13, 2006, that was organized to identify barriers to the development and testing of drugs for pediatric populations, as well as ways in which the system can be improved to facilitate better treatments for children.
National Academies of Sciences, Engineering, and Medicine,Health and Medicine Division,Board on Health Care Services,National Cancer Policy Forum
Author : National Academies of Sciences, Engineering, and Medicine,Health and Medicine Division,Board on Health Care Services,National Cancer Policy Forum Publisher : National Academies Press Page : 145 pages File Size : 48,5 Mb Release : 2018-02-12 Category : Medical ISBN : 9780309457972
The Drug Development Paradigm in Oncology by National Academies of Sciences, Engineering, and Medicine,Health and Medicine Division,Board on Health Care Services,National Cancer Policy Forum Pdf
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.
New Approaches to Drug Discovery by Ulrich Nielsch,Ulrike Fuhrmann,Stefan Jaroch Pdf
This volume gives an overview of state of the art technologies and future developments in the field of preclinical pharmaceutical research. A balanced mix of experts from academia and industry give insight in selected new developments in the drug discovery pathway. The topics cover the different parts of the drug discovery process, starting with new developments in the target identification and validation area. The lead generation part as a next step focuses on the requirements and technologies to identify new small molecules as lead compounds for further optimization; in a second section the technologies to identify biologics as leads are addressed. The final part focuses on the pharmacological models and technologies to characterize new compounds and the impact of biomarkers to facilitate the transfer of drug candidates into the development phase.
A Comprehensive Guide to Toxicology in Preclinical Drug Development by Ali S. Faqi Pdf
A Comprehensive Guide to Toxicology in Preclinical Drug Development is designed for toxicologists who need a thorough understanding of the drug development process. This multi-contributed reference will provide a detailed picture of the complex and highly interrelated activities of preclinical toxicology in both small molecules and biologics --
Theory of Drug Development by Eric B. Holmgren Pdf
Theory of Drug Development presents a formal quantitative framework for understanding drug development that goes beyond simply describing the properties of the statistics in individual studies. It examines the drug development process from the perspectives of drug companies and regulatory agencies. By quantifying various ideas underlying drug development, the book shows how to systematically address problems, such as: Sizing a phase 2 trial and choosing the range of p-values that will trigger a follow-up phase 3 trial Deciding whether a drug should receive marketing approval based on its phase 2/3 development program and recent experience with other drugs in the same clinical area Determining the impact of adaptive designs on the quality of drugs that receive marketing approval Designing a phase 3 pivotal study that permits the data-driven adjustment of the treatment effect estimate Knowing when enough information has been gathered to show that a drug improves the survival time for the whole patient population Drawing on his extensive work as a statistician in the pharmaceutical industry, the author focuses on the efficient development of drugs and the quantification of evidence in drug development. He provides a rationale for underpowered phase 2 trials based on the notion of efficiency, which leads to the identification of an admissible family of phase 2 designs. He also develops a framework for evaluating the strength of evidence generated by clinical trials. This approach is based on the ratio of power to type 1 error and transcends typical Bayesian and frequentist statistical analyses.
Optimization of Pharmaceutical R&D Programs and Portfolios by Zoran Antonijevic Pdf
Very little has been published on optimization of pharmaceutical portfolios. Moreover, most of published literature is coming from the commercial side, where probability of technical success (PoS) is treated as fixed, and not as a consequence of development strategy or design. In this book there is a strong focus on impact of study design on PoS and ultimately on the value of portfolio. Design options that are discussed in different chapters are dose-selection strategies, adaptive design and enrichment. Some development strategies that are discussed are indication sequencing, optimal number of programs and optimal decision criteria. This book includes chapters written by authors with very broad backgrounds including financial, clinical, statistical, decision sciences, commercial and regulatory. Many authors have long held executive positions and have been involved with decision making at a product or at a portfolio level. As such, it is expected that this book will attract a very broad audience, including decision makers in pharmaceutical R&D, commercial and financial departments. The intended audience also includes portfolio planners and managers, statisticians, decision scientists and clinicians. Early chapters describe approaches to portfolio optimization from big Pharma and Venture Capital standpoints. They have stronger focus on finances and processes. Later chapters present selected statistical and decision analysis methods for optimizing drug development programs and portfolios. Some methodological chapters are technical; however, with a few exceptions they require a relatively basic knowledge of statistics by a reader.