The journey from discovery to FDA approval is one of the most rigorous and comprehensive processes in the pharmaceutical and biotech industries. While drug development promises innovative treatments, from life-saving vaccines to gene therapies, it involves an intensive series of steps and regulatory evaluations. Each stage in the process is designed to guarantee that new therapies meet strict standards for safety, efficacy, and quality before reaching patients.
Understanding the FDA drug approval process is essential for biotech and pharmaceutical companies as they navigate challenges related to clinical trials, regulatory hurdles, and commercialization. This guide will provide an in-depth overview of the FDA’s role, the steps involved in securing drug approval, and the significance of various application types, from New Drug Applications (NDAs) to Emergency Use Authorizations (EUAs).
Founded in 1930, the US Food and Drug Administration (FDA) is a federal agency responsible for protecting public health by ensuring the safety, efficacy, and security of human and veterinary drugs, biological products, medical devices, and other products subject to regulation.
In 1938, Congress passed the Federal Food, Drug, and Cosmetics (FDC) Act, which requires that drugs be safe before they are allowed on the market. The Kefauver-Harris Drug Amendment of 1962 further ensured that drugs not only be safe but also effective before they receive market authorization.
In 1966, the drug division of the FDA was reorganized into the Office of New Drugs (OND), which began reviewing new drug applications. In 1982, the Bureau of Biologics was merged with the FDA. In 1987, two separate entities were established: the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER).
Initially formed with six offices, the Center for Drug Evaluation and Research (CDER) has grown to 13 offices to better fulfill its mission of safeguarding public health. Acting as a consumer watchdog, CDER oversees thousands of prescription and over-the-counter drugs, ensuring their safety and efficacy before they reach the market. This includes assessing new drug applications, monitoring post-market safety, and providing scientific support through the FDA’s review process.
The FDA’s drug approval process involves rigorous scientific and clinical testing to evaluate a drug’s benefits, risks, and side effects. Beginning with preclinical testing and advancing through clinical trials, this multi-step process ensures that new medicines are safe and effective for their intended use. CDER continues to monitor drugs after approval, taking corrective action, including removal from the market, if necessary.
Through these standards, the FDA strives to ensure that approved drugs not only meet safety and efficacy benchmarks but also deliver meaningful patient benefits. Post-approval, the FDA can intervene if new safety concerns arise, prioritizing public health at every step.
The FDA’s drug approval process is carefully designed to ensure that drugs are safe, effective, and appropriate for their intended uses before they reach the public. The process for taking a potential therapeutic compound from initial discovery to final approval spans multiple stages and can last 10-15 years, costing biotech and pharmaceutical companies hundreds of millions, if not billions, of dollars. While estimates vary, studies in publications like the Journal of Health Economics and JAMA Network have placed the median cost of drug development anywhere from $985 million to $2.8 billion.
Here’s a high-level look at the steps involved in the FDA’s drug approval process:
While INDs and NDAs are common, the FDA offers different drug application pathways depending on the drug’s intended use, nature, and novelty. For instance, biologics require a Biologics License Application (BLA), while generic drugs are submitted through an Abbreviated New Drug Application (ANDA). These options, along with mechanisms like Emergency Use Authorizations (EUAs), provide customized routes for drugs to reach the market under various circumstances. Each type of application allows the FDA to cater its review process to the specific drug and its potential role in healthcare.
Through these steps, the FDA balances the need to bring new, effective drugs to patients quickly with the obligation to ensure safety and efficacy, providing essential safeguards for public health.
The FDA’s drug approval process has evolved considerably throughout the 20th and early 21st centuries. This evolution has been largely driven by the need to accelerate and simplify approvals, especially for drugs addressing unmet medical needs. Key shifts have included incorporating new technologies, adjusting regulatory pathways, and refining assessment standards.
Several factors have influenced these changes. Federal regulations have expanded, drug and device complexities have increased, and the pharmaceutical industry has grown into a major economic force in the United States. To keep pace, the FDA has adopted electronic submissions and integrated real-world data to streamline review processes. Additionally, the agency has introduced a priority review program, expediting approvals for drugs that offer significant advantages over existing treatments. Another major step is the accelerated approval process, which allows drugs to be approved based on surrogate endpoints—early indicators of efficacy—without waiting for complete clinical results.
In recent years, the FDA has also shifted toward a risk-based approach. This approach emphasizes balancing benefits and risks rather than solely evaluating safety and efficacy. The agency has further bolstered international collaboration, working closely with global regulators to enhance access to new drugs worldwide.
A recent study in the Journal of Family Medicine and Primary Care highlights trends in drug approvals over the last two decades. The study notes an uptick in the expedited approval of anticancer drugs and biologics, contrasting with slower approval rates for drugs treating neurological disorders (such as depression and psychosis) and lifestyle diseases like obesity and diabetes. This trend reflects the heightened research focus on developing anticancer therapies and biologics.
While opinions vary on whether these FDA changes are ultimately beneficial or pose risks, they have undoubtedly accelerated the approval timeline, bringing innovative treatments to patients faster while maintaining standards of safety and efficacy.
We all know what a drug is, but to appease the SEO best practices laid out by Google, let's include a quick definition. Who knows, it might even be helpful to review the exact definition, in the eyes of the FDA. According to the agency, a drug is any substance "intended to diagnose, treat, cure, or prevent a disease or condition." This includes prescription medications, over-the-counter remedies, and biologics, such as vaccines and blood products. Importantly, the definition covers substances for human and animal use, as both fall under the FDA’s purview.
However, the FDA does make some distinguishments. For example, it considered drugs to be different from dietary supplements, a category that includes herbal products, vitamins, and complementary therapies. These products, regulated by the Center for Drug Evaluation and Research (CDER) under the Dietary Supplement Health and Education Act of 1994, do not undergo the same rigorous approval process as drugs. Instead, dietary supplements are evaluated based on guidelines for safe manufacturing practices and labeling but are not required to demonstrate efficacy in treating diseases or conditions.
There are some common types of drug applications the FDA reviews that sponsors can submit to receive approval to sell and market a medical product, including the New Drug Application (NDA), Abbreviated New Drug Application (ANDA), Investigational New Drug application, Biologics License Application (BLA), and Over-the-Counter (OTC) Drug Review.
Additionally, there are mechanisms the FDA can use to quickly authorize the use of a medical product or modify an approved drug that include the Emergency Use Authorization (EUA) and Supplement to a Previously Approved Application.
An NDA is submitted to gain approval to market a new drug. Typically, the sponsor, often a pharmaceutical company, files an NDA when they believe sufficient data on safety and efficacy has been collected to meet FDA requirements.
The process is comprehensive, often taking years and substantial resources to complete, and involves submitting extensive preclinical and clinical data. This application must include study results, detailed manufacturing information, and a proposed drug label. The NDA process works as follows:
NDAs are essential, providing the FDA with crucial data to make informed decisions about new drugs.
An ANDA is filed to market a generic drug equivalent to an existing brand-name drug. Unlike NDAs, ANDAs typically do not require preclinical and clinical data for safety and efficacy, allowing a faster, more cost-effective approval process.
To obtain approval, the application must prove the generic drug’s equivalence to the brand-name product in safety, efficacy, and quality. Some cases may require bioequivalence studies or additional trials. Once approved, generic drugs provide affordable alternatives and increase competition, lowering prices for consumers.
The ANDA process generally works as follows:
ANDAs play a vital role in increasing access to medications and reducing healthcare costs.
An IND application is submitted to gain approval for testing an investigational drug in human clinical trials, marking the first step toward market authorization. The application, submitted by the drug’s sponsor, provides the FDA with detailed preclinical data on the drug’s safety, pharmacology, and manufacturing processes to confirm its suitability for human trials.
IND applications also permit the legal transport of an investigational drug across state lines for testing. These applications include a proposed trial design, a risk management plan, and an outline of potential benefits, which together assure the FDA that human testing will prioritize safety. The two primary IND types are Commercial INDs (intended for market approval) and Research INDs (aimed at safety and efficacy data collection). Specialized IND categories include:
The IND application process generally involves:
This process is a cornerstone of drug development, ensuring that only drugs with sufficient safety data progress to human testing.
A Biologic License Application (BLA) is submitted to obtain FDA approval for biologic products—complex molecules produced from living cells, such as vaccines, blood products, and gene therapies. Governed by the Public Health Service Act (PHS Act), BLAs focus on demonstrating the product’s safety, efficacy, and consistent quality for its intended use.
Unlike drugs synthesized chemically, biologics are produced through living systems, making their development and manufacturing more complex. To ensure product integrity, sponsors must provide the FDA with detailed information on the product’s development, manufacturing processes, and clinical trials that establish safety and effectiveness.
The BLA process generally includes the following steps:
BLAs are critical for bringing innovative biologics to the public, allowing treatments for complex and previously untreatable conditions.
OTC drugs are non-prescription medications used to treat common ailments, such as headaches, colds, and allergies. To ensure public safety, the FDA runs an OTC Drug Review program, which assesses each drug’s safety, efficacy, and labeling to make certain it’s suitable for self-medication.
OTC drugs evaluated under this program are subject to OTC monographs—guidelines for each therapeutic category that outline ingredients, dosages, labeling, and testing requirements. Drugs that comply with a monograph can be marketed without an NDA, while those that don’t require separate FDA approval.
The OTC Drug Review process typically includes:
The OTC Drug Review allows manufacturers to bring safe, effective non-prescription drugs to market, enhancing consumer access to affordable healthcare options.
Beyond traditional drug applications, FDA approvals can also be categorized by product type, including name-brand drugs, generic drugs, and over-the-counter drugs.
In addition to drug approvals, the FDA oversees the approval of medical devices. Device testing often mirrors that of drugs, beginning with laboratory assessments, progressing to animal studies, and finally undergoing human trials. Approved devices are classified by risk level, with higher-risk devices facing more stringent requirements.
This classification system enhances the FDA’s ability to regulate a wide range of products, ensuring safety and effectiveness across pharmaceuticals and medical devices.
In addition to traditional applications, the FDA has pathways to authorize medical products more quickly, including the Emergency Use Authorization (EUA) and Supplement to a Previously Approved Application.
The FDA provides a variety of resources to assist sponsors in navigating the complex submission process, from early research stages through to final application. These resources include guidance documents, regulatory requirements, forms, and specific information on pathways like accelerated approval. Together, they establish clear standards for every aspect of drug development, from preclinical studies and clinical trials to submission requirements for New Drug Applications (NDAs), Biologics License Applications (BLAs), and more.
With these resources, sponsors can prepare applications that not only meet FDA standards but also reduce the likelihood of delays or denials. Utilizing the available guidance and tools allows sponsors to streamline the submission process, increase compliance, and enhance the chances of a successful review.
Approval decisions are complex and influenced by multiple critical factors. Primarily, the FDA assesses a drug's safety and efficacy as demonstrated through robust clinical trials. If a drug shows compelling evidence of effectiveness in treating its target condition with manageable risks, it stands a stronger chance of approval. However, several other elements can affect the outcome:
Approval, delay, or denial is a dynamic process where thorough evaluation safeguards public health, ensuring that only safe, effective, and high-quality drugs reach the market. By meeting FDA requirements and preparing for potential issues, sponsors can improve their chances of a timely and favorable outcome.
Navigating the FDA’s drug approval process demands significant resources, from preclinical research through clinical trials. Excedr’s leasing program offers a cost-effective way to secure high-quality lab equipment without depleting capital reserves. By leasing instead of purchasing, labs can maintain financial flexibility, avoid large upfront expenses, and allocate funds more strategically to advance critical research milestones.
If you’re looking for a streamlined approach to acquiring the equipment necessary for rigorous FDA testing and compliance, contact Excedr to learn how our leasing program can support your lab’s success in every phase of the approval process.
