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What is the process for approving new drug therapies?

Clinical trials are how we test promising new drugs and therapies to determine if they are safe and effective. Volunteers participate in the research studies that collect data to help investigators demonstrate the safety and effectiveness of therapies to treat the disease or condition. The U.S. Food and Drug Administration (FDA) requires that all drug treatments, therapies, or devices be evaluated in clinical trials before they can be approved for patient use.

Clinical trials, however, are the last phases in the drug development process prior to FDA approval. Before clinical trials are conducted, a significant amount of research has been undertaken. The process starts with Basic Research, where scientists study a disease or condition by using laboratory tests to better understand its cause and to identify targets for potential treatments.

Discovery begins once those targets have been identified. Scientists begin Discovery by developing drug or device prototypes and testing them to identify the best potential new therapy.

Once a promising new therapy has been selected, the researchers conduct additional Pre-clinical studies in the laboratory animals, or cell cultures to determine if the therapy works, how it works, and what the side effects in humans may be. It is important to understand this before the therapy is tested in humans.

When the potential therapy has been shown to be safe for testing in humans, an Investigational New Drug (IND) Application is submitted to the FDA for approval. Once approved, the therapy or device can move into Clinical Trials for further investigation.

Before a drug or therapy is available to patients, it must undergo rigorous testing in three phases of clinical trials. It must pass each phase before it can progress to the next. If the drug or therapy advances through all 3 phases of clinical research and has demonstrated that it is safe and effective, the FDA reviews the data and clinical trial results and makes a determination as to whether it can be made available to the public. Even after the drug has been approved by the FDA, it continues to be evaluated to help further assess the drug’s safety and usage patterns.

The Four Phases of Clinical Trials

Phase 1: Is the treatment safe in humans? A small dose of a new drug or treatment is given to a few very closely observed healthy subjects. The next groups receive a higher dose, and the process continues until reaching the target dose, or adverse effects occur. During this phase, investigators study drugs’ impact on the body and the body’s handling of the medicine, its metabolism, and its disposal. These are the first in human experiments.

Phase 2: Is the treatment likely to work in patients with epilepsy? The drug or treatment is tested with a larger group of patients with epilepsy or seizures to evaluate its potential effectiveness and further assess its safety. This phase also helps establish the optimal dosing regimen.

Phase 3: Is there compelling evidence that this drug is effective and safe in treating epilepsy? The drug or treatment is given to many people to gather additional information that demonstrates that the new medication is as safe and works to treat the symptoms of interest. The FDA will use the information collected at this phase to determine whether a new treatment should be approved and under what conditions.

Phase 4: These are conducted after the drug has been approved and marketed to collect additional information about risks, benefits, and optimal use of the drug or treatment. This phase may also study the effects of the drug on other populations such as children.

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