Accompanied by supplements.

Rare diseases collectively affect millions of Americans of all ages, but developing drugs and medical devices to prevent, diagnose, and treat these conditions is challenging. The Institute of Medicine (IOM) recommends implementing an integrated national strategy to promote rare diseases research and product development.

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.

Identifies drug products approved on the basis of safety & effectiveness by the FDA under the Federal Food, Drug, & Cosmetic Act. Composed of 4 parts: approved prescription drug products with therapeutic equivalence evaluations; over-the-counter (OTC) drug products that require approved applications as a condition of marketing; drug products with approval under Sect. 505 of the Act; & products that have never been marketed, have been discontinued from marketing, or that have had their approvals withdrawn for other than safety or efficacy reasons.

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