What is a clinical trial?
Before a new drug (or device) is made available to the public, it’s important to conduct clinical trials to determine if it reduces seizures and is safe to use. A clinical trial is a research study conducted with human volunteers and is used to evaluate a new treatment’s safety and effectiveness. Clinical trials can also be used to determine if a new treatment is better than an existing one.
The FDA is responsible for evaluating all potential therapies, using the data collected by a clinical trial to determine if the benefits outweigh the potential risks. If they do, the FDA will approve the treatment so that it can be made available for prescription use.
The Five Phases of a Clinical Trial
Before a drug can be requested approval from the FDA, it must be studied comprehensively in clinical trials. These trials are grouped into five separate phases. Each phase has a different purpose and helps answer a different scientific question. Devices go through a similar process, but they have three stages of clinical trials (pilot/feasibility, pivotal, and post-marketing) instead of 5 stages.
For simplicity, we explain only the drug clinical trial phases below.
Phase 0: Does the treatment warrant continued exploration?
This phase, which is done on a very small number of healthy people (usually less than 15), uses a very small dose of the treatment to ensure that it isn’t harmful. The goal is to determine that the drug acts as expected in people. This phase can help eliminate treatments that don’t behave as predicted, saving time and money. This phase is also sometimes called Pre-phase 1 or a “proof-of-concept” trial.
Phase 1: Is the treatment safe in people?
A small dose of a new drug is administrated to a few closely observed healthy patients. The next groups receive a higher dose, and the process continues until reaching the target dose, or adverse effects are observed. During this phase, investigators study the impact of the drug on the body, the stability of the drug in the body, how the body breaks the drug down (metabolism), and how the drug is cleared from the body. These are the first-in-human clinical studies.
Phase 2: Is the treatment likely to work in patients with epilepsy?
The drug 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 with the specific disorder that is being studied (eg. focal seizures, absence seizures, Dravet syndrome, etc.) to gather additional information that demonstrates that the new medication is safe and works to reduce seizures. 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: Post-marketing
After the drug has been approved and marketed, additional studies may be conducted to collect more information about risks, benefits, and optimal use of the drug. This phase may also study the effects of the drug on other populations that weren’t initially studied such as children.
Who participates in clinical trials?
Clinical trials need volunteers that are both healthy and with the disorder being studied. Decisions on who is ultimately selected are based on the trial’s eligibility criteria, which are designed to help identify the most appropriate candidates to participate in the trial safely. Criteria may include age, gender, type and stage of a disease, previous treatment history, and other medical conditions.
Many different types of people can participate in clinical trials. For each person, the reason may be different, but the most common reasons are:
- To provide a path to help move science forward
- To allow the participant to play a more active role in their health care
- Early access to new treatments that are not yet available to the public
- To help researchers learn more about a certain health problem
- To offer hope for patients and an opportunity for researchers to find better treatments in the future
Is a clinical trial right for you?
To determine if a particular trial is right for you, do as much research as you can. Every patient – and every clinical trial – is different. Each clinical trial has its own set of qualifications, time commitments, treatment regimen, and more. To determine if a particular trial is right for you, do as much research as you can.
We’ve compiled a list of frequently asked questions to help you research and prepare to meet with a clinical trial’s research coordinator.