This book proposes and investigates a universal framework, and accompanying documentation system, to facilitate and catalogue benefit-risk decisions; a valuable addition to the benefit-risk toolbox. Over the past decade, pharmaceutical companies and regulatory agencies have been reviewing the benefit-risk assessment of medicines with a view to developing a structured, systematic, standardized approach. Examining the evaluation of such an approach by several mature regulatory authorities ensures that the reader gains a unique insight into the ongoing debate in this area. The field of benefit-risk assessment continues to evolve at a rapid pace due to political and societal pressure, as is reflected in the recent FDA PUDFA agreement as well as in the EMA 2015 Roadmap. Rather than provide a comprehensive snap-shot of this constantly changing environment, this book evaluates selected current approaches to benefit-risk assessment. The strengths and weaknesses of publicly available documents in communicating benefit-risk decisions to stakeholders are reviewed and these evaluations are used to inform development of a prospective framework that could be used to harmonise procedures globally.
Many practitioners in the pharmaceutical industry are still largely unfamiliar with benefit-risk assessment, despite its growing prominence in drug development and commercialization. Helping to alleviate this knowledge gap, Benefit-Risk Assessment in Pharmaceutical Research and Development provides a succinct overview of the key considerations rele
This classic, field-defining textbook, now in its sixth edition, provides the most comprehensive guidance available for anyone needing up-to-date information in pharmacoepidemiology. This edition has been fully revised and updated throughout and continues to provide a rounded view on all perspectives from academia, industry and regulatory bodies, addressing data sources, applications and methodologies with great clarity.
Therapeutic risk management of medicines is an authoritative and practical guide on developing, implementing and evaluating risk management plans for medicines globally. It explains how to assess risks and benefit-risk balance, design and roll out risk minimisation and pharmacovigilance activities, and interact effectively with key stakeholders.A more systematic approach for managing the risks of medicines arose following a number of high-profile drug safety incidents and a need for better access to effective but potentially risky treatments. Regulatory requirements have evolved rapidly over the past decade. Risk management plans (RMPs) are mandatory for new medicinal products in the EU and a Risk Evaluation and Mitigation Strategy (REMS) is needed for certain drugs in the US.This book is an easy-to-read resource that complements current regulatory guidance, by exploring key areas and practical implications in greater detail. It is structured into chapters encompassing a background to therapeutic risk management, strategies for developing RMPs, implementation of RMPs, and the continuing evolution of the risk management field.The topic is of critical importance not only to the pharmaceutical and biotechnology industries, but also regulators and healthcare policymakers.Some chapters feature contributions from selected industry experts. - An up-to-date practical guide on conceiving, designing, and implementing global therapeutic risk management plans for medicines - A number of useful frameworks are presented which add impact to RMPs (Risk Management Plans), together with regional specific information (European Union, United States, and Japan) - A comprehensive guide for performing risk management more effectively throughout a product's life-cycle
This is a comprehensive major reference work for our SpringerReference program covering clinical trials. Although the core of the Work will focus on the design, analysis, and interpretation of scientific data from clinical trials, a broad spectrum of clinical trial application areas will be covered in detail. This is an important time to develop such a Work, as drug safety and efficacy emphasizes the Clinical Trials process. Because of an immense and growing international disease burden, pharmaceutical and biotechnology companies continue to develop new drugs. Clinical trials have also become extremely globalized in the past 15 years, with over 225,000 international trials ongoing at this point in time. Principles in Practice of Clinical Trials is truly an interdisciplinary that will be divided into the following areas: 1) Clinical Trials Basic Perspectives 2) Regulation and Oversight 3) Basic Trial Designs 4) Advanced Trial Designs 5) Analysis 6) Trial Publication 7) Topics Related Specific Populations and Legal Aspects of Clinical Trials The Work is designed to be comprised of 175 chapters and approximately 2500 pages. The Work will be oriented like many of our SpringerReference Handbooks, presenting detailed and comprehensive expository chapters on broad subjects. The Editors are major figures in the field of clinical trials, and both have written textbooks on the topic. There will also be a slate of 7-8 renowned associate editors that will edit individual sections of the Reference.
Few people realize that prescription drugs have become a leading cause of death, disease, and disability. Adverse reactions to widely used drugs, such as psychotropics and birth control pills, as well as biologicals, result in FDA warnings against adverse reactions. The Risks of Prescription Drugs describes how most drugs approved by the FDA are under-tested for adverse drug reactions, yet offer few new benefits. Drugs cause more than 2.2 million hospitalizations and 110,000 hospital-based deaths a year. Serious drug reactions at home or in nursing homes would significantly raise the total. Women, older people, and people with disabilities are least used in clinical trials and most affected. Health policy experts Donald Light, Howard Brody, Peter Conrad, Allan Horwitz, and Cheryl Stults describe how current regulations reward drug companies to expand clinical risks and create new diseases so millions of patients are exposed to unnecessary risks, especially women and the elderly. They reward developing marginally better drugs rather than discovering breakthrough, life-saving drugs. The Risks of Prescription Drugs tackles critical questions about the pharmaceutical industry and the privatization of risk. To what extent does the FDA protect the public from serious side effects and disasters? What is the effect of giving the private sector and markets a greater role and reducing public oversight? This volume considers whether current rules and incentives put patients' health at greater risk, the effect of the expansion of disease categories, the industry's justification of high U.S. prices, and the underlying shifts in the burden of risk borne by individuals in the world of pharmaceuticals. Chapters cover risks of statins for high cholesterol, SSRI drugs for depression and anxiety, and hormone replacement therapy for menopause. A final chapter outlines six changes to make drugs safer and more effective. Suitable for courses on health and aging, gender, disability, and minority studies, this book identifies the Risk Proliferation Syndrome that maximizes the number of people exposed to these risks. Additional Columbia / SSRC books on the privatization of risk and its implications for Americans: Bailouts: Public Money, Private ProfitEdited by Robert E. Wright Disaster and the Politics of InterventionEdited by Andrew Lakoff Health at Risk: America's Ailing Health System-and How to Heal ItEdited by Jacob S. Hacker Laid Off, Laid Low: Political and Economic Consequences of Employment InsecurityEdited by Katherine S. Newman Pensions, Social Security, and the Privatization of RiskEdited by Mitchell A. Orenstein
If you believe that the latest blockbuster medication is worth a premium price over your generic brand, or that doctors have access to all the information they need about a drug’s safety and effectiveness each time they write a prescription, Dr. Jerry Avorn has some sobering news. Drawing on more than twenty-five years of patient care, teaching, and research at Harvard Medical School, he shares his firsthand experience of the wide gap in our knowledge of the effectiveness of one medication as compared to another. In Powerful Medicines, he reminds us that every pill we take represents a delicate compromise between the promise of healing, the risk of side effects, and an increasingly daunting price. The stakes on each front grow higher every year as new drugs with impressive power, worrisome side effects, and troubling costs are introduced. This is a comprehensive behind-the-scenes look at issues that affect everyone: our shortage of data comparing the worth of similar drugs for the same condition; alarming lapses in the detection of lethal side effects; the underuse of life-saving medications; lavish marketing campaigns that influence what doctors prescribe; and the resulting upward spiral of costs that places vital drugs beyond the reach of many Americans. In this engagingly written book, Dr. Avorn asks questions that will interest every consumer: How can a product judged safe by the Food and Drug Administration turn out to have unexpectedly lethal side effects? Why has the nation’s drug bill been growing at nearly 20 percent per year? How can physicians and patients pick the best medication in its class? How do doctors actually make their prescribing decisions, and why do those decisions sometimes go wrong? Why do so many Americans suffer preventable illnesses and deaths that proper drug use could have averted? How can the nation gain control over its escalating drug budget without resorting to rationing or draconian governmental controls? Using clinical case histories taken from his own work as a practitioner, researcher, and advocate, Dr. Avorn demonstrates the impressive power of the well-conceived prescription as well as the debacles that can result when medications are misused. He describes an innovative program that employs the pharmaceutical industry’s own marketing techniques to reduce use of some of the most overprescribed and overpriced products. Powerful Medicines offers timely and practical advice on how the nation can improve its drug-approval process, and how patients can work with doctors to make sure their prescriptions are safe, effective, and as affordable as possible. This is a passionate and provocative call for action as well as a compelling work of clear-headed science.
The Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) were designed to encourage more pediatric studies of drugs used for children. The FDA asked the IOM to review aspects of pediatric studies and changes in product labeling that resulted from BPCA and PREA and their predecessor policies, as well as assess the incentives for pediatric studies of biologics and the extent to which biologics have been studied in children. The IOM committee concludes that these policies have helped provide clinicians who care for children with better information about the efficacy, safety, and appropriate prescribing of drugs. The IOM suggests that more can be done to increase knowledge about drugs used by children and thereby improve the clinical care, health, and well-being of the nation's children.
Drug overdose, driven largely by overdose related to the use of opioids, is now the leading cause of unintentional injury death in the United States. The ongoing opioid crisis lies at the intersection of two public health challenges: reducing the burden of suffering from pain and containing the rising toll of the harms that can arise from the use of opioid medications. Chronic pain and opioid use disorder both represent complex human conditions affecting millions of Americans and causing untold disability and loss of function. In the context of the growing opioid problem, the U.S. Food and Drug Administration (FDA) launched an Opioids Action Plan in early 2016. As part of this plan, the FDA asked the National Academies of Sciences, Engineering, and Medicine to convene a committee to update the state of the science on pain research, care, and education and to identify actions the FDA and others can take to respond to the opioid epidemic, with a particular focus on informing FDA's development of a formal method for incorporating individual and societal considerations into its risk-benefit framework for opioid approval and monitoring.
In spite of recent progress in the harmonization of terminology and processes affecting work on the clinical safety of medicines consensus is needed on standards for many difficult aspects of day-to-day pharmacovigilance that continue to pose problems for both the pharmaceutical industry and drug regulators. The CIOMS V Working Group has generated proposals for pragmatic approaches to dealing with such issues as: classification and handling of individual safety case reports from a variety of sources (spontaneous consumer reports solicited reports literature the Internet observational studies and secondary data bases disease and other registries regulatory ADR databases and licensor-licensee interactions); new approaches to case management and regulatory reporting practices (proper clinical evaluation of cases incidental vs other events patient and reporter identifiability seriousness criteria expectedness criteria case follow-up criteria and the role and structure of case narratives); improvements and efficiencies in the format content and reporting of periodic safety update reports (PSURs) (including results of an industry survey on PSUR workloads and practices; proposals for high case volume and long time-period reports simplification of certain PSURs summary bridging reports addendum reports license renewal reports for EU and Japan dealing with old products and other technical details); determination and use of population exposure (denominator) data (sources of data and a guide to analytical approaches for a variety of circumstances).The Group has also taken stock of the current state of expedited and periodic clinical safety reporting requirements around the world with summary data on regulations from more than 60 countries. Recommendations are made for enhancing the harmonization steps already taken as a result of previous CIOMS publications and the ICH process. In addition to dealing with unfinished and unresolved issues from previous CIOMS initiatives the report covers many emerging topics such as those involving new technologies. Its 20 Appendices provide a wealth of detailed explanations and reference information. It is the most comprehensive and recent treatment of difficult pharmacovigilance issues affecting the working practices and systems of drug safety and other pharmaceutical professionals.
