Drug Approval Process

Extracted Attributes

• Economic Goals

  Lowering drug prices via Most Favored Nation Status and biosimilar competition

• Governing Laws

  Federal Food, Drug, and Cosmetic Act (FD&C); Public Health Service (PHS) Act

• Reform Strategies

  Reducing pivotal trials, replacing animal testing, Bayesian statistics, AI surveillance

• Clinical Trial Phases

  4 Phases (Phase 1: Safety, Phase 2: Effectiveness, Phase 3: Pivotal/Safety & Efficacy, Phase 4: Post-market)

• Standard Review Timeline

  6 to 10 months (PDUFA dates)

• Average Development Duration

  8.5 to 12 years

• Primary Regulatory Authority

  Food and Drug Administration (FDA)

Timeline

• The Public Health Service (PHS) Act is enacted, providing a framework for biological products. (Source: Pharmacy Times)

  1944-01-01

• JAMA publishes a study estimating the R&D investment needed to bring new medicines to market. (Source: Drugs.com)

  2020-03-03

• FDA updates guidelines for designating Orphan Drug Products. (Source: Drugs.com)

  2022-07-08

• FDA updates official documentation regarding the drug development and approval process. (Source: FDA.gov)

  2022-08-08

• Commissioner Marty Makary outlines major reforms to accelerate drug approval timelines at the JP Morgan Healthcare Conference. (Source: Document 065d2e96-4d40-49bd-8511-d8d35f8b01f4)

  2025-01-15

Food and Drug Administration

The Food and Drug Administration (FDA) is a federal agency of the United States Department of Health and Human Services. The FDA is responsible for protecting and promoting public health through the control and supervision of food safety, tobacco products, caffeine products, dietary supplements, prescription and over-the-counter pharmaceutical drugs (medications), vaccines, biopharmaceuticals, blood transfusions, medical devices, electromagnetic radiation emitting devices (ERED), cosmetics, animal foods & feed, and veterinary products. The FDA's primary focus is enforcement of the Federal Food, Drug, and Cosmetic Act (FD&C). However, the agency also enforces other laws, notably Section 361 of the Public Health Service Act as well as associated regulations. Much of this regulatory-enforcement work is not directly related to food or drugs but involves other factors like regulating lasers, cellular phones, and condoms. In addition, the FDA takes control of diseases in the contexts varying from household pets to human sperm donated for use in assisted reproduction. The FDA is led by the commissioner of food and drugs, appointed by the president with the advice and consent of the Senate. The commissioner reports to the secretary of health and human services. Marty Makary is the current commissioner. The FDA's headquarters is located in the White Oak area of Silver Spring, Maryland. The agency has 223 field offices and 13 laboratories located across the 50 states, the United States Virgin Islands, and Puerto Rico. In 2008, the FDA began to post employees to foreign countries, including China, India, Costa Rica, Chile, Belgium, and the United Kingdom.

Web Search Results

• FDA Drug Approval Process - Drugs.com

  Skip to main content # FDA Drug Approval Process ## What is the FDA approval process? The 4 phases of a drug approval process includes: Pre-clinical, IND (Investigational New Drug) Application Clinical New Drug Application (NDA) Review Post-marketing risk assessments The U.S. Food and Drug Administration's (FDA's) Center for Drug Evaluation and Research (CDER) is a science-led organization in charge of overseeing the drug approval process before a drug is marketed. CDER ensures that both brand and generic drugs work correctly and that the health benefits outweigh the known risks. They review each drug closely using an independent team of clinicians and scientists who evaluate safety, efficacy and labeling of the drug product. [...] 1. Phase 1: Phase 1 focuses on safety. About 20 to 80 healthy volunteers are recruited to establish a drug's safety and side effects and takes about 1 year. Absorption, metabolism and excretion of the drug are also emphasized. 2. Phase 2: Phase 2 focuses on effectiveness. Roughly 40 to 300 patient volunteers are used to assess the drug's effectiveness in those with a specific condition or disease. This phase runs about 2 years. Groups of similar patients may receive the actual drug compared to a placebo (inactive pill) or other active drug to determine if the drug is effective. Safety and side effects are also reviewed. [...] ## Sources NDA and BLA Approvals. US Food and Drug Administration (FDA). Accessed July 29, 2024 at Wouters OJ, McKee M, Luyten J. Estimated Research and Development Investment Needed to Bring a New Medicine to Market, 2009-2018. JAMA. 2020 Mar 3;323(9):844-853. doi: 10.1001/jama.2020.1166 Development & Approval Process - Drugs. US Food and Drug Administration (FDA). 08/08/2022. Accessed July 29, 2024 at The FDA's Drug Review Process: Ensuring Drugs Are Safe and Effective. US Food and Drug Administration (FDA). Accessed July 29, 2024 at Designating an Orphan Drug Product. Drugs and Biologic Products. 07/08/2022. US Food and Drug Administration (FDA). ## Further information

• Drug Approval Process - Friends of Cancer Research

  FOCR 30 Year Anniversary # Drug Approval Process # Drug Approval Process The drug approval process is rigorous. Only a small portion of the drugs submitted to the U.S. Food and Drug Administration (FDA) are approved and it takes an estimated 8.5 years on average for a drug to go from early laboratory discovery to approval for human use. [...] Throughout this process, a Sponsor and the organizations responsible for developing the new drug, must coordinate with the Center for Drug Evaluation and Research (CDER) or the Center for Biologics Evaluation and Research (CBER) on the clinical trial design, analysis of results, and permission to conduct and escalate human testing. For cancer drugs, sponsors engage with CDER’s Oncology Center of Excellence (OCE). Sponsors submitting new drugs are also required to pay user fees in order to support FDA in operating efficiently. FDA identifies twelve steps in the complete development and review collaboration. Once a drug has been approved, sponsors are often responsible for additional post-market research to confirm their drug’s safety, effectiveness, and optimal use.

• Understanding the FDA Approval Process and PDUFA Dates

  The FDA’s drug approval process involves several key components and essential information for evaluation. To initiate the review of new medications, drug sponsors submit NDAs or BLAs. This allows the FDA to assess the drug’s safety and effectiveness, packaging labeling, and manufacturing processes. The FDA is required to decide by the established PDUFA date, which can be either 6 or 10 months after submission. If the application is approved, this date becomes the official approval date. However, if a CRL is issued, sponsors can either withdraw their application or resubmit it for review. ###### REFERENCES [...] In 1944, the Public Health Service (PHS) Act was enacted, providing a legislative framework for the provision of public health services. This act encompasses biological products and defines them as a “virus, therapeutic serum, toxin, antitoxin, vaccine, blood, blood component or derivative, allergenic product, and analogous product, applicable to the prevention, treatment, or cure of a disease or condition of human beings.” Biologics also meet the definition of drugs under the FD&C Act.3 ## Approval Process To ensure that medications marketed in the US are safe and effective, the FDA’s Center for Drug Evaluation and Research (CDER) reviews new drug applications (NDAs). CDER oversees both prescription and nonprescription or OTC drugs, with [...] OTC drugs being brought to the market following the NDA process or through an OTC monograph. It is the responsibility of the company seeking approval to analyze the medication and subsequently submit supporting evidence for review.4 This evidence is derived from data collected from human clinical trials, including information on safety, efficacy, dosages, indications, and drug metabolism of a medication. Based on this research, the FDA decides whether to approve the application to market the medication.5 ## Types of Drug Applications Types of applications submitted to CDER include investigational new drug, NDA, abbreviated NDA, OTC drug, and biologic license application (BLA).6 This article specifically focuses on NDAs and BLAs.

• From Idea to Market: The Drug Approval Process

  Results: The process starts with preclinical testing. For drugs that appear safe, an investigational new drug application is filed with the FDA. If approved, clinical trials begin with phase 1 studies that focus on safety and pharmacology. Phase 2 studies examine the effectiveness of the compound. Phase 3 is the final step before submitting a new drug application (NDA) to the FDA. An NDA contains all the information obtained during all phases of testing. Phase 4 studies, or postmarketing studies, are con-ducted after a product is approved. Recent changes in legislation have streamlined the approval pro-cess. Critics contend that these changes have compromised public safety, resulting in the need to recall several products from the market. Proponents claim that changes in the approval [...] This application can either be approved or rejected, or the FDA might request further study before making a decision. Following acceptance, the FDA can also request that the manufacturer conduct additional postmarketing studies. Overall, this en-tire process, on average, takes between 8 to 12 years.2 Figure 1 summarizes the drug approval pro-cess. [...] The purpose of this article is to present a concise overview of the drug approval process. It will briefly review the history of the FDA and follow the journey of a new product from early develop-ment until approval by the FDA for prescription use. Methds We describe the drug development process based on a review of the literature and Web sites address-ing FDA processes and policies. Key words used for the searches included “Food and Drug Administra-Submitted, revised, 18 January 2001. From the Department of Family Medicine (MSL, LKS), Northwestern University Medical School, Chicago. Address reprint requests to Martin S. Lipsky, MD, Department of Family Medicine, Morton 1–658, 303 East Chicago Ave, Chicago, IL 60611.

• Development & Approval Process | Drugs - FDA

  Before a drug can be tested in people, the drug company or sponsor performs laboratory and animal tests to discover how the drug works and whether it's likely to be safe and work well in humans. Next, a series of tests in people is begun to determine whether the drug is safe when used to treat a disease and whether it provides a real health benefit. For more information about the drug development and approval process, see How Drugs Are Developed and Approved. ### FDA Approval: What it means FDA approval of a drug means that data on the drug’s effects have been reviewed by CDER, and the drug is determined to provide benefits that outweigh its known and potential risks for the intended population. The drug approval process takes place within a structured framework that includes: [...] In some cases, the approval of a new drug is expedited. Accelerated Approval can be applied to promising therapies that treat a serious or life-threatening condition and provide therapeutic benefit over available therapies. This approach allows for the approval of a drug that demonstrates an effect on a “surrogate endpoint” that is reasonably likely to predict clinical benefit, or on a clinical endpoint that occurs earlier but may not be as robust as the standard endpoint used for approval. This approval pathway is especially useful when the drug is meant to treat a disease whose course is long, and an extended period of time is needed to measure its effect. After the drug enters the market, the drug maker is required to conduct post-marketing clinical trials to verify and describe the [...] Drug companies seeking to sell a drug in the United States must first test it. The company then sends CDER the evidence from these tests to prove the drug is safe and effective for its intended use. A team of CDER physicians, statisticians, chemists, pharmacologists, and other scientists reviews the company's data and proposed labeling. If this independent and unbiased review establishes that a drug's health benefits outweigh its known risks, the drug is approved for sale. The center doesn't actually test drugs itself, although it does conduct limited research in the areas of drug quality, safety, and effectiveness standards.