U.S. Food and Drug Administration What is a drug as defined by the FDA? Drug Approval Process A drug is any product that is intended for use in the diagnosis, cure mitigation, treatment , or prevention of disease; and that tis intended to affect the structure or any function of the body. Drug Sponsor’s Discovery and Screening Phase Drug Sponsor’s Clinical Studies/Trials P H - A 20 80 3 S The typical number of healthy volunteers used in Phase 1; this phase E emphasizes safety. The goal here in this phase is to determine what the 2 drug's most frequent side effects are and, often, how the drug is 1 metabolized and excreted. IND Application 4 P Drug Developed The sponsor submits an H 100’s Investigational New Drug A The typical number of patients used in Phase 2; this phase emphasizes effectiveness.This goal is to obtain preliminary data on whether the drug works Drug sponsor develops a 1 (IND) application to FDA S in people who have a certain disease or condition. For controlled trials, patients new drug compound and based on the results from E receiving the drug are compared with similar patients receiving a different seeks to have it approved intial testing that include, treatment--usually a placebo, or a different drug. Safety continues to be by FDA for sale in the the drug’s composition and 2 evaluated, and short-term side effects are studied. United States. manufacturing, and develops a plan for testing the drug on humans. At the end of Phase 2, FDA and sponsors discuss how large-scale studies in Phase 3 will be done. Animals Tested DRUG VALUATI E ON G U A SPONSOR R N FDA’s Center for Drug D D R R Sponsor must test new E O S F E Evaluation and Research R A E R T C N drug on animals for H E C (CDER) evaluates new drugs IND REVIEW before they can be sold. toxicity. Multiple species are used to gather basic FDA reviews the IND to assure P information on the safety that the proposed studies, generally referred to as clinical 5 H and efficacy of the 1000’s The center’s evaluation not only prevents quackery, but also trials, do not place human A The typical number of patients used in Phase 3. These studies gather more provides doctors and patients the information they need to compound being subjects at unreasonable risk of S use medicines wisely. CDER ensures that drugs, both investigated/researched. harm. FDA also verifies that E information about safety and effectiveness, study different populations and brand-name and generic, are effective and their health there are adequate informed different dosages, and uses the drug in combination with other drugs. benefits outweigh their known risks. consent and human subject protection. 3 Page 1 Who reviews new drug submissions? What other drug products are regulated by FDA? A team of CDER physicians, statisticians, chemists, pharmacologists, and other Drugs include more than just medicines. For example, scientists review the drug sponsor’s data and proposed labeling of drugs. fluoride toothpastes, antiperspirants (not deodorant), dandruff shampoos, and sunscreens are all considered drugs. FDA’s New Drug Application (NDA)Review FDA’s Post-Approval Risk Assessment Systems FASTER APPROVALS The Accelerated Approval program allows Because it's not possible to predict all of a drug's effects during clinical Drug Labeling earlier approval of drugs that treat serious P diseases and that fill an unmet medical H trials, monitoring safety issues after drugs get on the market is critical. The need. The approval is faster because FDA A role of FDA’s post-marketing safety system is to detect serious unexpected FDA reviews the drug’s professional labeling can base the drug’s effectiveness on a S adverse events and take definitive action when needed. and assures appropriate information is “surrogate endpoint,” such as a blood test 10 or X-ray result, rather than waiting for E communicated to health care professionals 11 results from a clinical trial. and consumers. Facility Inspection 4 Once FDA approves a drug, the The Fast Track program helps reduce the post-marketing monitoring stage begins. time for FDA’s review of products that treat The sponsor (typically the manufacturer) FDA inspects the facilities where serious or life-threatening diseases and Application Reviewed is required to submit periodic safety the drug will be manufactured. those that have the potential to address an unmet medical need. Drug sponsors can updates to FDA. After an NDA is received, FDA has 60 submit portions of an application as the days to decide whether to file it so it can information becomes available (“rolling DAY submission”) instead of having to wait www.fda.gov/medwatch 8-9 be reviewed. If FDA files the NDA, the until all information is available. FDA’s MedWatch voluntary system makes it FDA Review team is assigned to evaluate (800) FDA-1088 (322-1088) phone easier for physicians and consumers to report 60 the sponsor’s research on the drug’s (800) FDA-0178 (322-0178) fax adverse events. Usually, when important new safety and effectiveness. risks are uncovered, the risks are added to the drug's labeling and the public is informed of the new information through letters, public NDA Application health advisories, and other education. In some cases, the use of the drug must be The drug sponsor formally asks FDA to approve substantially limited. And in rare cases, the a drug for marketing in the United States by drug needs to be withdrawn from the market. 7 submitting an NDA. An NDA includes all animal and human data and analyses of the data, as well as information about how the drug behaves in the body and how it is Since the PDUFA was passed in 1992, more manufactured. 12 than 1,000 drugs and biologics have come to the market, including new medicines to treat Review Meeting Drug Approval cancer, AIDS, cardiovascular disease, and 6 life-threatening infections. FDA meets with a drug sponsor prior to FDA reviewers will approve DRUG submission of a New Drug Application. the application or issue a PDUFA has enabled the Food and Drug Administration to bring access to new drugs as fast or faster response letter. SPONSOR than anywhere in the world, all while maintaining the same thorough review process. Under PDUFA, drug companies agree to pay fees that boost FDA resources, and FDA agrees to time frames for its review of new drug applications. Page 2.