New Drug Development


Book Description

Drug development is complex and costly, requiring the testing of numerous chemical compounds for their potential to treat disease. Before a new drug can be marketed in the United States, a new drug application (NDA), which includes scientific and clinical data, must be approved by the Food and Drug Administration (FDA). Recent scientific advances have raised expectations that an increasing number of new and innovative drugs would soon be developed to more effectively prevent, treat, and cure serious illnesses. However, industry analysts and the FDA have reported that new drug development, and in particular, development of new molecular entities (NMEs)-potentially innovative drugs containing ingredients that have never been marketed in the United States-has become stagnant. GAO was asked to provide information on (1) trends in the pharmaceutical industry's reported research and development expenses as well as trends in the number of NDAs submitted to, and approved by, FDA; and (2) experts' views on factors accounting for these trends and their suggestions for expediting and enhancing drug development. GAO analyzed data







New Drug Development


Book Description




Clinical Trials Handbook


Book Description

Best practices for conducting effective and safe clinical trials Clinical trials are arguably the most important steps in proving drug effectiveness and safety for public use. They require intensive planning and organization and involve a wide range of disciplines: data management, biostatistics, pharmacology, toxicology, modeling and simulation, regulatory monitoring, ethics, and particular issues for given disease areas. Clinical Trials Handbook provides a comprehensive and thorough reference on the basics and practices of clinical trials. With contributions from a range of international authors, the book takes the reader through each trial phase, technique, and issue. Chapters cover every key aspect of preparing and conducting clinical trials, including: Interdisciplinary topics that have to be coordinated for a successful clinical trialData management (and adverse event reporting systems) Biostatistics, pharmacology, and toxicology Modeling and simulation Regulatory monitoring and ethics Particular issues for given disease areas-cardiology, oncology, cognitive, dementia, dermatology, neuroscience, and more With unique information on such current issues as adverse event reporting (AER) systems, adaptive trial designs, and crossover trial designs, Clinical Trials Handbook will be a ready reference for pharmaceutical scientists, statisticians, researchers, and the many other professionals involved in drug development.




Patenting of Pharmaceuticals and Development in Sub-Saharan Africa


Book Description

This book critically investigates the patent protection of medication in light of the threats posed by HIV/AIDS, malaria and tuberculosis epidemics to the citizens of countries in Sub-Saharan Africa (hereinafter “SSA” or “Africa”). The book outlines the systemic problems associated with the prevailing globalized patent regime and the regime’s inability to promote access to life-saving medication at affordable prices in SSA. It argues that for pharmaceutical patents to retain their relevance in SSA countries, human development concepts must be integrated into global patent law- and policy-making. An integrative approach implies developing additional public health and human development exceptions/limitations to the exercise of patent rights with the goal of scaling up access to medication that can treat epidemics in SSA. By drawing on multiple perspectives of laws, institutions, practices, and politics, the book suggests that SSA countries adopt an evidence-based approach to implementing global patent standards in domestic jurisdictions. This evidence-based approach would include mechanisms like local need assessments and the use of empirical data to shape domestic patent law-making endeavors. The approach also implies revising patent rules and policies with a pro-poor and pro-health emphasis, so that medication will be more affordable and accessible to the citizens of SSA countries. It also suggests considering the opinions of individuals and pro-access institutions in enacting crucial pieces of health-related statutes in SSA countries. The approach in this book is sensitive to the public health needs of the citizens affected by epidemics and to the imperative of building local manufacturing facilities for pharmaceutical research and development in SSA.




Compound Solutions


Book Description

Claiming 1.5 million lives in 2015, tuberculosis is the world’s most deadly infectious disease. Because of the population it overwhelmingly affects, however, pharmaceutical companies are uninterested in developing better drugs for the disease. Compound Solutions examines Product Development Partnerships (PDPs), which arose early in the twenty-first century to develop new drugs and vaccines for infectious diseases in low-income countries. Here, for the first time, is a sustained examination of PDPs: the work they do, the partnerships they form, their mission, and their underlying philosophy of addressing global health needs—with implications that extend well beyond tuberculosis. Focusing on two PDPs for tuberculosis—the Global Alliance for TB Drug Development (TB Alliance) and Aeras (a nonprofit focused on vaccine development)—Susan Craddock argues that PDPs do much more than product development. As innovative sites of humanitarian pharmaceutical production, they are contravening mainstream pharmaceutical production by tying drug and vaccine research to global health needs rather than shareholder demand. In largely untethering the profit incentive from pharmaceutical production, Craddock shows, PDPs exhibit more creative and efficient scientific practices, reshaping regulatory norms and implementing more ethical forms of clinical trials that enhance community engagement and capacity building. An unparalleled, interdisciplinary analysis of PDPs as politically, socially, scientifically, and economically innovative sites of pharmaceutical production, Compound Solutions is a must for readers in the fields of public health, science and technology studies, and medical social science.




Gene Patents and Collaborative Licensing Models


Book Description

The cost of patent licenses needed to design a new genetic test or treatment may ultimately prevent research projects getting started, as individual components are protected by different patent owners. This book examines legal measures which might be used to solve the problem of fragmentation of patents in genetics.




Global Pharmaceutical Policy


Book Description

There is a strong argument that people throughout the world have a right to receive the medicines they need in an appropriate, affordable, and timely way. Global Pharmaceutical Policy describes the laws, policies, and customs relating to the development and provision of medicines, identifies their strengths and weakness, and then proposes global solutions for getting things better. Here is a masterpiece written in a clear and elegant style. Together, Dukes and Abbott have experience and insight that are unrivalled. Joe Collier, Emeritus Professor of Medicines Policy, St George s, University of London, UK Pharmaceuticals play a central role in health care throughout the world. The pharmaceutical industry is beset with difficulties as increasing research and development expenditure yields fewer new treatments. Public and private budgets strain under the weight of high prices and limited access. The world s poor see little effort to address diseases prevalent in less affluent societies, while the world s wealthy are overusing prescription drugs, risking their health and wasting resources. As the global economic crisis exacerbates pressure on health care budgets, a new presidential administration in Washington, DC has committed to broad health care reform. These circumstances form the backdrop for this extraordinarily timely examination of the global system for the development, production, distribution and use of medicines. The authors are acknowledged experts in the fields of pharmaceutical law and policy, with many years experience advising governments, multilateral organizations and policy-makers on issues involving innovation, access and use of medicines. Supported by a team of independent scientists, doctors and lawyers, they take an insightful look at the issues surrounding global regulation of the pharmaceutical sector, and offer pragmatic suggestions for reform. This book will be of interest to government policy-makers, members of industry, healthcare professionals, teachers, students and lawyers in the fields of public health, intellectual property and international trade.




New Drug Approval


Book Description

Before approving a drug, the FDA assesses a drug's effectiveness. This assessment may be based on evidence showing that a drug has a positive impact on a surrogate endpoint (SE) -- a laboratory measure, such as blood pressure -- instead of more direct clinical evidence, like preventing strokes. After approval, FDA requires a drug sponsor to further study the drug. There have been concerns about FDA's reliance on SE and its oversight of post-marketing studies (PS). This report provides info. on: (1) all drug applications approved based on SE in FDA's accelerated approval process; (2) a subset of applications for potentially innovative drugs approved based on SE under FDA's traditional process; and (3) FDA's oversight of PS. Illustrations.




Genetic Resources and Traditional Knowledge


Book Description

This fascinating study describes efforts to define and protect traditional knowledge and the associated issues of access to genetic resources, from the negotiation of the Convention on Biological Diversity to the Declaration on the Rights of Indigenous Peoples and the Nagoya Protocol. Drawing on the expertise of local specialists from around the globe, the chapters judiciously mix theory and empirical evidence to provide a deep and convincing understanding of traditional knowledge, innovation, access to genetic resources, and benefit sharing. Because traditional knowledge was understood in early negotiations to be subject to a property rights framework, these often became bogged down due to differing views on the rights involved. New models, developed around the notion of distributive justice and self-determination, are now gaining favor. This book suggests – through a discussion of theory and contemporary case studies from Brazil, India, Kenya and Canada – that a focus on distributive justice best advances the interests of indigenous peoples while also fostering scientific innovation in both developed and developing countries. Comprehensive as well as nuanced, Genetic Resources and Traditional Knowledge will be of great interest to scholars and students of law, political science, anthropology and geography. National and international policymakers and those interested in the environment, indigenous peoples' rights and innovation will find the book an enlightening resource.