The U.S. Food and Drug Administration (FDA) operates as a federal agency dedicated to safeguarding public health. This mission encompasses ensuring the safety, efficacy, and security of human drugs, veterinary drugs, biological products, and medical devices. The FDA also oversees the safety of the nation’s food supply, cosmetics, and products that emit radiation. This broad responsibility allows the agency to enforce laws and regulations designed to protect consumers.
How Drugs Gain Approval
The journey for a new drug to receive FDA approval is a rigorous, multi-stage process. It begins with preclinical research, involving laboratory and animal testing to gather initial data on the drug’s safety and efficacy. This stage determines if a drug is safe enough for human trials by evaluating its biological effects, potential toxicity, and pharmacological profile. Studies at this phase include pharmacodynamics, which examines how the drug affects the body, pharmacokinetics, which looks at how the body processes the drug, and toxicology studies, which assess potential toxic effects to identify safe dosage ranges.
After successful preclinical testing, an Investigational New Drug (IND) application is submitted to the FDA to begin human trials. The FDA reviews this application, which includes preclinical data, manufacturing information, and proposed clinical trial plans. The FDA has 30 days to either approve the IND or issue a clinical hold, ensuring human subjects are not exposed to unreasonable risks. Once an IND is approved, the drug proceeds to clinical trials, conducted in three sequential phases.
Phase 1 clinical trials are the first human studies, involving a small group of 20 to 100 healthy volunteers or patients with the disease. These trials primarily focus on safety, assessing potential side effects, and understanding how the drug is metabolized and excreted. These studies establish initial safety profiles and determine a safe dosage range for further investigation.
Phase 2 clinical trials involve a larger group of participants, ranging from a few dozen to about 300 individuals who have the condition the drug is intended to treat. Objectives of this phase are to evaluate the drug’s efficacy and determine the optimal dosage. These studies also continue to monitor safety and further characterize adverse events.
Phase 3 clinical trials are large-scale studies, involving several hundred to 3,000 participants or more, all with the target disease or condition. These trials confirm the drug’s efficacy, gather extensive data on its effectiveness across a broader population, and identify less common adverse events. They last between one to four years and are often randomized and controlled, meaning participants are assigned to different groups to compare the new drug’s performance against a placebo or existing treatment.
Upon completion of Phase 3 trials, the drug sponsor submits a New Drug Application (NDA) for small-molecule drugs or a Biologics License Application (BLA) for biological products. This application includes all preclinical and clinical trial data, manufacturing information, and proposed labeling. The FDA has 60 days to decide whether to file the application for review. The review process takes 10 months for a standard review and 6 months for a priority review, involving a team of experts who evaluate the submitted data. Advisory committees, composed of independent experts, may also convene to discuss issues and provide non-binding recommendations that can influence the FDA’s final decision.
Ensuring Drug Safety After Approval
FDA oversight of a drug does not conclude with its approval; post-market surveillance activities continue to monitor its safety. The FDA Adverse Event Reporting System (FAERS) is a computerized database that collects adverse event reports, medication error reports, and product quality complaints submitted by healthcare professionals, patients, and manufacturers. This system supports the FDA’s safety surveillance program for drugs and biologic products.
Reports submitted to FAERS are evaluated by clinical reviewers to identify new safety concerns, unusual side effects, or emerging trends. While FAERS is a tool for detecting safety issues, reports do not always establish a causal relationship between a drug and an event, and not all adverse events are reported. If a potential safety concern is identified, the FDA may perform further evaluation, which can lead to regulatory actions such as updating the drug’s labeling, restricting its use, or communicating new safety information to the public.
For certain medications with safety concerns, the FDA may require a Risk Evaluation and Mitigation Strategy (REMS) to help ensure that the benefits outweigh its risks. REMS are drug safety programs designed to reinforce safe medication use. Components of a REMS can include Medication Guides for patients, communication plans for healthcare providers about risks, and “Elements to Assure Safe Use” (ETASU), which might involve prescriber training, patient registries, or specific laboratory testing. The FDA may require a REMS before or after a drug is approved.
The FDA also monitors manufacturing quality and inspects facilities where drugs are produced to ensure compliance with current good manufacturing practices (CGMPs). These inspections verify the accuracy of submitted information and ensure the quality, safety, and effectiveness of drugs. In response to new safety information or manufacturing issues, the FDA can require drug recalls or mandate label changes. Drug recalls remove or correct products that violate federal laws and can be initiated voluntarily by manufacturers or at the FDA’s request, especially in urgent situations posing a health risk. Label changes are updates to prescribing information to reflect new safety data, such as adverse reactions or contraindications, and must be implemented upon FDA approval.
Accessing FDA Drug Information
The FDA provides public resources for accessing information about approved drugs. The Drugs@FDA database offers information on prescription and over-the-counter drugs. This database includes drug information, regulatory history, the most recent FDA-approved prescribing information, patient labeling, and reviews by FDA staff evaluating the drug’s safety and effectiveness. This resource allows individuals to verify a drug’s approval status and find official prescribing details.
Patient information leaflets, also known as Medication Guides or Patient Package Inserts, are FDA-approved documents designed to provide patients with clear information about their medications. These leaflets detail safety information, common side effects, and instructions for safe use. The FDA regularly updates these materials to reflect new safety data.
For information on generic drug equivalents, the Orange Book is a resource. This publication lists drug products approved by the FDA, along with therapeutic equivalence evaluations for approved prescription drug products. It helps prescribers and pharmacists identify generic drugs that can be substituted for brand-name drugs, promoting cost containment in healthcare.
The Purple Book serves a similar function for biological products, including biosimilar and interchangeable biological products. This searchable online database provides information on FDA-licensed biological products, including whether they have been determined to be biosimilar or interchangeable to a reference product. While the Orange Book focuses on small-molecule drugs, the Purple Book is the resource for biologicals, reflecting their unique regulatory pathway. These resources collectively offer the public official drug information.