Glossary Drug Labeling The drug labeling, or package insert, accompanies each package of drug manufactured by the pharmaceutical company. It is the Food and Drug Administration’s (FDA’s) approved guidance on how the drug should be used and provides the important information on dosage, side-effects, and possible interactions with other drugs. Federal Food, Drug, and Cosmetic Act This Federal law requires FDA to ensure that new drugs developed by pharmaceutical companies are safe and effective before they are marketed. FDA physicians and scientists work with pharmaceutical companies throughout the drug development process to assure that a drug is safe to test in humans, and evaluate all the clinical data to make the ultimate decision of whether the drug can be sold to the public. FDA approves the drug labeling that describes the directions for use, side effects, and warnings. Food and Drug Administration (FDA) The FDA is responsible for protecting the public health by assuring the safety, efficacy, and quality of drugs, biologics and many other medical products. The FDA is also responsible for advancing the public health by helping to speed innovations that make medicines more effective, safer, and more affordable; and helping the public get the accurate, science-based information they need to use medicines to improve their health. More information is available at www.fda.gov. Human volunteers Healthy individuals and patients who volunteer to participate in a clinical study. Hypothesis-testing studies Also known as “confirmatory” clinical studies, hypothesis-testing studies are always well-controlled and are intended to provide meaningful results by examining pre-stated questions (i.e., hypotheses) using predefined statistically valid plans for data analysis, thereby allowing firm conclusions to be drawn to support product claims. Hypothesis-testing studies may occur at any stage of drug development and include all phase III studies, some earlier-phase studies, and many studies of marketed products. Independent third-party PhRMA plans to partner with a non-governmental, non-industry third party to operate and administer the electronic Clinical Study Results Database in the future. Investigational Review Board (IRB) IRBs are independent groups composed of healthcare professionals and community representatives that have the responsibility and authority to review and approve all studies involving human subjects in a particular community or facility. The IRB’s main responsibility is to protect research participants (human volunteers). This includes minimizing the risks to the participants, ensuring that participants are informed of the study protocol, and ensuring that the participant’s privacy is protected. Meaningful Clinical Study Results As used in the PhRMA Principles, this phrase refers to the results of hypothesis-testing studies and to distinguish such studies from those that are merely exploratory in nature. Hypothesis-testing studies typically are the most rigorously conducted and include all phase III studies, some earlier phase studies, and many studies of marketed products. Completed hypothesis-testing studies, by definition and design, always provide “meaningful results.” National Institutes of Health (NIH) NIH is the Federal focal point for medical research in the United States. It comprises 27 separate Institutes and Centers, and is one of eight health agencies of the Public Health Service within the U.S. Department of Health and Human Services. Phase I Clinical Studies The primary purpose of Phase I is to test the safety of the drug in question. The drug is tested on 30 to 100 healthy volunteers and must be deemed safe before its effectiveness can be tested in patients who have the disease. Phase II Clinical Studies Anywhere from 50 to 300 patients (volunteer research participants) who have the disease are tested. The purpose of testing is to determine whether the drug shows some efficacy against the disease, learn of possible side-effects, and determine the drug’s most promising dose and dosing regimen. Phase III Clinical Studies The new drug is tested on many patients – on average 3,000, but up to 10,000 for some drugs – to establish the safety and effectiveness of the drug in a large and varied population. Phase IV Clinical Studies Phase IV includes post marketing studies to delineate additional information including the drug's risks, benefits, and optimal use. PhRMA The Pharmaceutical Research and Manufacturers of America (PhRMA) represents the country's leading research-based pharmaceutical and biotechnology companies, which are devoted to inventing medicines that allow patients to live longer, healthier, and more productive lives. The industry invested an estimated $33.2 billion in 2003 in discovering and developing new medicines. PhRMA companies are leading the way in the search for new cures. Preclinical testing Before a drug is tested on humans, it must first be tested in the laboratory and then on animals. Drug companies try to use as few animals as possible and ensure their humane and proper care during testing. These tests show whether a potential drug has toxic side effects and whether it is safe is at different doses. Principles on Conduct of Clinical Trials and Communication of Clinical Trial Results In 2002, the Executive Committee of PhRMA unanimously adopted a set of principles for the conduct of clinical trials and the communication of results of clinical trials. The voluntary principles describe the relationship of PhRMA member companies with others involved in clinical research and set forth the rules companies follow to protect the safety of research participants wherever the companies conduct clinical trials. In the Principles, the PhRMA companies commit to the timely communication of all meaningful results of clinical trials, whether those results are positive or negative. Further, the results are always to be communicated in an objective, accurate, balanced and complete manner. Standard Format for Presentation of Clinical Study Data PhRMA believes that the synopsis described in the E3 guideline issued by the International Conference on Harmonization (ICH) provides an easy to use template for conveying a summary of clinical study information.