Approval Process

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

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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 Page 2 Page Once FDA approves a drug, the a drug, FDA approves Once post-marketing stage begins. monitoring sponsor (typicallyThe the manufacturer) submit periodic safety is required to FDA. to updates FDA’s MedWatch voluntary makes system it MedWatch FDA’s report to and consumers physicians easier for when important new Usually, events. adverse the the risks are added to risks are uncovered, of drug's labeling and the public is informed public through letters, the new information and other education. In health advisories, the use of drug must be some cases, the cases, And in rare substantially limited. from the market. be withdrawn drug needs to nitive actionnitive when needed. fi Since the PDUFA was passed in 1992, more passed in 1992, more was the PDUFA Since to come than 1,000 drugs and biologics have treat including new medicines to the market, and disease, AIDS, cardiovascular cancer, infections. life-threatening FDA’s Post-Approval Risk Systems Assessment Post-Approval FDA’s Because it's not possible to predictBecause it's not possible to all of a drug's effects during clinical issues after safety drugs get on the market monitoring is critical. The trials, detect is to post-marketing serious unexpected system safety role of FDA’s and take de events adverse www.fda.gov/medwatch (800) FDA-1088 (322-1088) phone (800) FDA-0178 (322-0178) fax PDUFA has enabled the Food and Drug Administration to bring access to new drugs as fast or faster new drugs as fast or faster to bring access to and Drug Administration has enabled the Food PDUFA Under PDUFA, process. review all while maintaining the same thorough in the world, than anywhere its for time frames and FDA agrees to that boost FDA resources, fees pay agree to drug companies of new drug applications. review fluoride toothpastes, antiperspirants (not deodorant), (not deodorant), antiperspirants fluoride toothpastes, Drugs include more than just medicines. For example, example, For than just medicines. Drugs include more E S P A H 4 dandruff shampoos, and sunscreens are all considered drugs. drugs. all considered are and sunscreens dandruff shampoos, program allows allows program What other drug products are regulated by FDA? by other drug products regulated are What program helps reduce the helps reduce program Fast Track Accelerated Approval Approval Accelerated time for FDA’s review of products that treat of products that treat review FDA’s time for diseases and serious or life-threatening an address to the potential those that have Drug sponsors can unmet medical need. submit portions of an application as the (“rolling available becomes information wait to of having instead submission”) is available. until all information FASTER APPROVALS FASTER The serious of drugs that treat earlier approval diseases and that fill an unmet medical because FDA is faster approval The need. on a effectiveness can base the drug’s such as a blood test endpoint,” “surrogate rather than waiting for result, or X-ray a clinical trial. from results The Drug Approval FDA reviewers will approve will approve FDA reviewers the application or issue a response letter. 12 Facility Inspection Facility FDA inspects the facilities where the drug will be manufactured. 11 FDA’s New Drug Application (NDA)Review Drug Application New FDA’s le it so can fi les the NDA, fi Who reviews new drug submissions? new reviews Who NDA Application Review Meeting Review The drug sponsor formally asks FDA to approve approve asks FDA to drug sponsor formally The by marketing States in the United a drug for submitting an NDA. An NDA includes all animal and human data analyses of the data, as the drug about how as information well it is in the body and how behaves manufactured. Application Reviewed Application FDA meets with a drug sponsor prior to FDA meets with a drug sponsor prior to submission of a New Drug Application. Drug Labeling After an NDA is received, FDA has 60 an NDA is received, After decide whether to to days be reviewed. If FDA be reviewed. FDA Review team is assigned to evaluate evaluate is assigned to FDA Review team research on the drug’s the sponsor’s and effectiveness. safety FDA reviews the drug’s professional labeling professional FDA reviews the drug’s is information and assures appropriate health care professionals to communicated and consumers. scientists review the drug sponsor’s data and proposed labeling of drugs. labeling of drugs. data and proposed the drug sponsor’s scientists review A team of CDER physicians, statisticians, chemists, pharmacologists, and other pharmacologists, chemists, statisticians, of CDER physicians, A team 9 - 7 6 8 10 DRUG

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