FDA 101: Your Essential Guide

Why the FDA Touches Every Aspect of Your Daily Life

The FDA (Food and Drug Administration) is the federal agency responsible for protecting public health by regulating the safety and effectiveness of products Americans use every day. Its oversight covers an estimated $2.7 trillion in products annually.

Key FDA Responsibilities:

• Food safety: From farm to table oversight
• Drug approval: Prescription and over-the-counter medications
• Medical devices: From bandages to pacemakers
• Biologics: Vaccines, blood products, and gene therapies
• Cosmetics: Safety standards for personal care products
• Tobacco products: Health warnings and marketing restrictions

With a $6.5 billion annual budget and over 18,000 employees, the agency operates under the Department of Health and Human Services. The FDA’s funding is unique: 54% comes from federal appropriations, and 46% comes from industry user fees. Pharmaceutical companies, for instance, provide 75% of the drug review budget.

Whether you’re taking medication or eating packaged food, the FDA’s framework affects nearly every product that impacts human health. The agency balances innovation with safety, ensuring new treatments reach patients while maintaining rigorous standards.

As Dr. Maria Chatzou Dunford, CEO of Lifebit, I’ve worked with regulatory bodies like the FDA through our federated genomics platform. My experience in computational biology has shown me how modern data platforms can accelerate regulatory science while upholding the highest safety standards.

What is the FDA and What Does It Regulate?

The FDA acts as a safety net for American consumers, working to ensure that everyday products won’t cause harm. The agency’s mission is to protect public health by making sure drugs work as promised, food is safe to eat, and medical devices function properly. This responsibility covers $2.7 trillion worth of products annually, representing about 20 cents of every dollar spent by U.S. consumers.

With a budget of $6.5 billion and 18,000 employees, the FDA is a key agency within the Department of Health and Human Services. Headquartered in Silver Spring, Maryland, the agency’s strength lies in its nationwide presence, with 223 field offices and 13 laboratories across all 50 states, the U.S. Virgin Islands, and Puerto Rico.

This extensive network allows FDA inspectors and investigators to visit manufacturing plants, review import shipments, investigate consumer complaints, and respond quickly to public health emergencies, from contaminated food outbreaks to medical device recalls.

The Modern FDA: Structure and Key Centers

The FDA is organized into specialized centers, each with deep expertise in a specific product area. The Office of the Commissioner provides overall leadership, while the centers manage the day-to-day regulatory work:

• Center for Drug Evaluation and Research (CDER): As the primary reviewer of new drugs, CDER’s mission is to ensure that prescription and over-the-counter medications are safe and effective. It evaluates both brand-name drugs through the New Drug Application (NDA) process and generic drugs through the Abbreviated New Drug Application (ANDA) process. CDER also publishes the “Orange Book,” which lists approved drug products with therapeutic equivalence evaluations.
• Center for Biologics Evaluation and Research (CBER): CBER is responsible for regulating biologics—complex products derived from living sources like humans, animals, or microorganisms. This includes vaccines, blood and blood products, human tissues, and advanced treatments like cell and gene therapies. Due to their complexity, biologics require a different regulatory approach than traditional small-molecule drugs.
• Center for Devices and Radiological Health (CDRH): CDRH oversees the safety and effectiveness of medical devices, which range from simple tongue depressors to complex life-supporting systems like pacemakers and heart valves. Its authority also extends to radiological health, covering products that emit radiation, such as microwave ovens, X-ray equipment, and laser products.
• Center for Food Safety and Applied Nutrition (CFSAN): CFSAN is responsible for the safety of most of the nation’s food supply. This includes regulating food additives, ensuring the accuracy of food labeling (including the Nutrition Facts panel), and overseeing dietary supplements and cosmetics. It also sets standards for bottled water and manages food safety programs to prevent foodborne illness.
• Center for Veterinary Medicine (CVM): CVM regulates food, food additives, and drugs intended for animals. Its work is crucial for protecting both animal health and the human food supply, as it ensures that drugs administered to food-producing animals do not leave unsafe residues in meat, milk, or eggs.
• Center for Tobacco Products (CTP): Established by the 2009 Family Smoking Prevention and Tobacco Control Act, CTP regulates the manufacturing, marketing, and distribution of tobacco products. Its responsibilities include setting age restrictions, requiring health warnings on packaging, reviewing new tobacco products before they can be marketed, and enforcing rules on advertising and promotion.
• Office of Regulatory Affairs (ORA): ORA is the lead office for all FDA field activities, serving as the agency’s “eyes and ears.” Its investigators, inspectors, and scientists conduct inspections of manufacturing facilities, analyze product samples, and provide evidence to support FDA enforcement actions.

Scope of Regulation: What’s on the Plate?

The FDA’s reach is extensive, covering most products that impact health and safety. This includes:

• Human and Veterinary Drugs: The agency reviews both prescription (Rx) drugs, which require a doctor’s order, and over-the-counter (OTC) drugs, which can be sold directly to consumers. This oversight applies to drugs for both people and animals.
• Vaccines and Biologics: These complex products, derived from living sources, include everything from childhood vaccines and blood transfusions to advanced gene therapies that can treat genetic disorders.
• Medical Devices: The FDA regulates a vast range of devices, which are categorized into three classes based on risk: Class I (low risk, e.g., elastic bandages), Class II (moderate risk, e.g., infusion pumps), and Class III (high risk, e.g., artificial hearts). It also oversees products that emit radiation, such as microwave ovens and cell phones.
• Food and Dietary Supplements: The agency oversees about 80% of the U.S. food supply, including produce, seafood, shell eggs, and processed foods. It does not regulate meat from livestock, poultry, or most processed egg products, which are handled by the U.S. Department of Agriculture (USDA). The FDA also monitors the safety and labeling of dietary supplements.
• Cosmetics and Tobacco: Cosmetics must be safe for their intended use and properly labeled. The FDA does not pre-approve cosmetics, with the exception of color additives. A product marketed with a therapeutic claim, such as an anti-dandruff shampoo, is regulated as both a cosmetic and a drug. Tobacco products are regulated to reduce health risks and prevent youth access.

This comprehensive oversight means the FDA is involved in nearly every aspect of daily life, often in unseen ways. For a complete list, see the official page on what does FDA regulate.

The Path to Approval: How Products Get to Market

Getting a new drug or medical device to market requires passing the FDA’s rigorous pre-market evaluation. This process balances innovation with safety, ensuring that new treatments are effective without causing undue harm. The FDA’s work continues long after approval through post-market monitoring to track real-world performance.

The Drug Approval Process

Bringing a new drug to market is a lengthy and expensive journey, often taking 10-15 years and costing over a billion dollars. The process is structured in distinct stages to build a comprehensive profile of the drug’s safety and effectiveness.

• Preclinical Stage: Before any human testing, a drug candidate undergoes extensive laboratory and animal testing to assess its basic pharmacology and potential toxicity. If the results are promising, the sponsor files an Investigational New Drug (IND) application with the FDA, which summarizes the preclinical findings and outlines the plan for human trials. The FDA reviews the IND to ensure human subjects will not be exposed to unreasonable risk.
• Phase I Clinical Trials: Once the IND is cleared, the drug enters Phase I. These trials typically involve a small group of healthy volunteers (20-100) and are designed to assess the drug’s safety, determine a safe dosage range, and identify its side effects.
• Phase II Clinical Trials: If Phase I shows acceptable safety, the drug moves to Phase II. These trials are conducted in a larger group of patients (100-300) who have the condition the drug is intended to treat. The primary goals are to evaluate the drug’s effectiveness (efficacy) and to further assess its short-term side effects and risks.
• Phase III Clinical Trials: This is the most extensive and expensive phase, involving hundreds to thousands of patients. These large-scale trials are designed to confirm the drug’s effectiveness, monitor adverse reactions from long-term use, and compare it to existing treatments. These studies are often randomized, controlled, and double-blind to minimize bias.

After successful completion of all three phases, the sponsor submits a New Drug Application (NDA) to the FDA. The NDA is a massive document containing all the data from preclinical and clinical studies, as well as information on the drug’s manufacturing, composition, and proposed labeling. For generic drugs, companies file an Abbreviated New Drug Application (ANDA), which demonstrates their product is bioequivalent to the brand-name version, a much faster and less expensive path.

Expedited Review Pathways

To speed access to important new drugs, the FDA has several expedited programs:

• Fast Track: For drugs that treat serious conditions and fill an unmet medical need.
• Breakthrough Therapy: For drugs that show substantial improvement over available therapy on a clinically significant endpoint.
• Accelerated Approval: Allows for earlier approval of drugs that treat serious conditions and fill an unmet need based on a surrogate endpoint.
• Priority Review: The FDA aims to take action on an application within 6 months (compared to 10 months under standard review).

• Phase IV (Post-Market Studies): After a drug is approved, the FDA may require the sponsor to conduct post-market safety studies to gather additional information about its long-term risks, benefits, and optimal use.

Medical Device Pathways

Medical devices are classified by risk (Class I, II, or III), which determines their approval pathway.

• Premarket Approval (PMA): This is the most stringent pathway, required for high-risk Class III devices like pacemakers and artificial hearts. Companies must submit extensive scientific and clinical evidence to prove the device’s safety and effectiveness. Products that pass this review are considered “FDA approved.”
• 510(k) Clearance: Most Class II (moderate-risk) devices and some Class I devices fall into this category. Manufacturers must submit a premarket notification, or 510(k), demonstrating that their new device is “substantially equivalent” to a legally marketed predicate device. This means it is at least as safe and effective as the existing device. These products are “FDA cleared,” not approved.
• De Novo Classification: This pathway is for novel, low-to-moderate-risk devices that have no existing equivalent on the market. It provides a route to market without having to go through the more rigorous PMA process.

Post-Market Surveillance: Ensuring Ongoing Safety

The FDA’s oversight continues long after a product is on the market. The MedWatch program allows healthcare professionals and patients to Report a problem with a product to the FDA. This data, collected in an adverse event reporting system, helps the agency identify emerging safety concerns.

For products with known serious risks, the FDA may require Risk Evaluation and Mitigation Strategies (REMS) to ensure the benefits outweigh the risks. If a product poses a significant health threat, the agency works with companies on product recalls and safety alerts. Recalls are classified by risk:

• Class I Recall: A situation where there is a reasonable probability that the use of or exposure to a violative product will cause serious adverse health consequences or death.
• Class II Recall: A situation where use of or exposure to a violative product may cause temporary or medically reversible adverse health consequences.
• Class III Recall: A situation where use of or exposure to a violative product is not likely to cause adverse health consequences.

A Century of Safeguarding: The History and Evolution of the FDA

The FDA‘s history is one of evolution, often driven by public health crises and scientific progress. It began as a small division in the U.S. Department of Agriculture, but its mission was cemented by pioneers like Dr. Harvey Washington Wiley. His famous “Poison Squad” experiments, where volunteers consumed food with common but questionable additives like borax and formaldehyde, exposed the dangers in unregulated products and, along with muckraking journalism like Upton Sinclair’s The Jungle, built public support for federal oversight.

Public Health Crises and Legislation that Shaped Policy

The FDA’s authority grew in response to tragedies that revealed gaps in consumer protection:

• 1906 Pure Food and Drugs Act: This landmark law prohibited interstate commerce in misbranded and adulterated foods and drugs, giving the government power to seize illegal products and prosecute offenders.
• 1938 Food, Drug, and Cosmetic (FD&C) Act: Sparked by the Elixir Sulfanilamide tragedy, where a new sulfa drug formulation used a toxic solvent (diethylene glycol) and killed over 100 people, this act overhauled U.S. food and drug law. For the first time, it required companies to prove their drugs were safe before marketing them and expanded FDA oversight to include cosmetics and medical devices.
• 1951 Durham-Humphrey Amendment: This amendment to the FD&C Act formally established the two classes of drugs we have today: prescription (Rx), which could only be dispensed with a doctor’s order, and over-the-counter (OTC).
• 1962 Kefauver-Harris Amendments: Following the global thalidomide crisis, where a sedative caused severe birth defects in thousands of children, these amendments were passed. Championed by FDA medical officer Dr. Frances Oldham Kelsey, who famously resisted pressure and blocked the drug’s approval in the U.S., the law required manufacturers to prove that drugs were not only safe but also effective for their intended use.
• 1976 Medical Device Amendments: Prompted by safety failures with devices like the Dalkon Shield IUD, this legislation gave the FDA clear authority to regulate medical devices, establishing the three-class, risk-based system still in use today.
• 1983 Orphan Drug Act: To encourage the development of treatments for rare diseases (affecting fewer than 200,000 Americans), this act created financial incentives for manufacturers, including tax credits for clinical testing and seven years of market exclusivity after approval.
• The AIDS Epidemic: In the 1980s, intense pressure from patient advocacy groups pushed the FDA to create expedited review pathways, allowing faster access to promising treatments for life-threatening diseases. This fundamentally changed the agency’s approach to balancing speed and safety.
• 1990 Nutrition Labeling and Education Act (NLEA): This act mandated the now-ubiquitous “Nutrition Facts” panel on most packaged foods and standardized the use of health claims like “low-fat” and “high-fiber.”
• 2009 Family Smoking Prevention and Tobacco Control Act: This law granted the FDA comprehensive authority to regulate the manufacturing, marketing, and sale of tobacco products for the first time.

Funding the Mission

The FDA’s $6.5 billion annual budget is funded through a hybrid model that has both supporters and critics.

• Federal appropriations from Congress cover about 54% of the budget, supporting core activities like food safety inspections, research, and enforcement.
• Industry user fees provide the remaining 46%. Through programs like the Prescription Drug User Fee Act (PDUFA), companies pay fees to support the review of their own product applications.

This user fee model has significantly reduced review times for new drugs, giving patients faster access to treatments. However, it has also raised concerns about potential conflicts of interest. The FDA maintains that its rigorous scientific standards remain independent of funding sources, with its commitment to public health as the top priority.

Modern Challenges and Future Directions

The FDA today faces challenges unimaginable a century ago, from globalized supply chains to rapidly emerging technologies like artificial intelligence. To keep pace, the agency is collaborating with international partners, responding to public health crises with greater speed, and embracing technology to make its oversight more effective.

The FDA’s Role in Public Health Emergencies

During health emergencies, the FDA uses special tools to get life-saving products to the public quickly. The COVID-19 pandemic highlighted the importance of the Emergency Use Authorization (EUA) process. The EUA pathway allowed the agency to authorize vaccines, tests, and treatments in record time based on robust safety and effectiveness data, though on a shorter timeline than full approval.

Beyond pandemics, the Medical Countermeasures (MCMs) initiative prepares the nation for threats ranging from bioterrorism to natural disease outbreaks. This includes work on the first FDA-approved Ebola vaccine, Ervebo. The agency also actively combats fraudulent products that emerge during crises. Lessons learned from these events, as detailed in scientific research on Emergency Use Authorizations during COVID-19, help refine future responses.

Adapting to Scientific and Technological Change

The FDA is actively embracing innovation to modernize its regulatory processes.

• Data Modernization: The openFDA initiative makes vast amounts of anonymized data, such as adverse event reports, publicly available. This allows external researchers to generate new insights that can improve public health.
• Digital Health: The agency is developing clear guidance for mobile medical applications and other digital health tools to ensure their safety and effectiveness without stifling innovation.
• Artificial Intelligence (AI): AI and machine learning are changing drug findy and safety monitoring. The FDA’s precisionFDA platform, a collaborative cloud environment, is expanding the agency’s ability to analyze complex biological data. This is part of the broader Critical Path Initiative, which aims to modernize regulatory science by incorporating real-world data and real-world evidence into decision-making, as seen in the Oncology QCARD Initiative for cancer drug development.

Frequently Asked Questions about the FDA

Here are answers to some common questions about the FDA’s regulatory processes.

How does the FDA regulate dietary supplements?

Dietary supplements are regulated differently from drugs. Under the Dietary Supplement Health and Education Act (DSHEA) of 1994, manufacturers are responsible for ensuring their products are safe and properly labeled before they are sold. They do not need to get FDA approval beforehand.

The FDA’s role is primarily post-market enforcement. The agency monitors for unsafe products, investigates complaints, and takes action against supplements that are adulterated or make false disease-related claims. Unlike drugs, supplements do not have to be proven effective to be sold.

What is the difference between “FDA approved” and “FDA cleared”?

These terms refer to different regulatory pathways for medical devices and are not interchangeable.

• “FDA Approved” is used for high-risk devices and new drugs that have undergone the FDA’s most rigorous review process (PMA), which includes extensive clinical data to prove safety and effectiveness.
• “FDA Cleared” applies to most medical devices that have gone through the 510(k) process. This means the manufacturer has demonstrated the device is “substantially equivalent” to another legally marketed device.

Both pathways ensure products meet safety standards, but the level of review differs based on risk.

What is the FDA’s role in food safety?

The FDA oversees most of the U.S. food supply, excluding meat, poultry, and some egg products (which are handled by the USDA). The Food Safety Modernization Act (FSMA) of 2011 shifted the agency’s focus from responding to contamination to preventing it.

Key responsibilities include:

• Setting science-based safety standards for how food is grown, processed, and stored.
• Inspecting domestic and international food facilities.
• Regulating food additives and labeling to ensure consumers are informed.
• Coordinating with the CDC and state agencies to respond to foodborne illness outbreaks and manage recalls.

This work ensures the food on your table is safe, properly labeled, and free from harmful contaminants.

Conclusion

The FDA is a vital public health agency that touches nearly every aspect of daily life, overseeing $2.7 trillion worth of products to ensure their safety and effectiveness. For over a century, it has evolved in response to public health crises and scientific advances, from the Elixir Sulfanilamide tragedy to the COVID-19 pandemic.

Today, the FDA faces the challenge of regulating in an era of unprecedented technological change. The agency is embracing AI and machine learning to revolutionize how drug safety and effectiveness are understood, using real-world data to gain new insights through initiatives like precisionFDA.

The future of regulation depends on leveraging large-scale data for real-time insights. Secure, compliant data analysis is the backbone of modern pharmacovigilance and regulatory science. When researchers and regulators can safely access and analyze vast biomedical datasets, they make better, faster decisions.

Lifebit’s federated AI platform powers the secure, compliant data analysis essential for this new era. By enabling real-time access to global biomedical and multi-omic data, we support the crucial research that underpins the FDA‘s mission of protecting public health. Learn how Lifebit can power next-generation public health initiatives.