There is an urgent need for the Ataxia community to be “clinical trial ready” because many therapies are in the drug development pipeline. To successfully bring a treatment to the market, patient involvement is crucial. The Ataxia community is needed to participate in research studies and clinical trials, but that is not their only role in drug development. Drug developers need to understand how Ataxia symptoms impact a person’s daily life in order to find meaningful solutions. NAF developed PrepRARE as a program to educate members about all stages of the clinical trial process. Check out our series of webinars, articles, and other information to make sure that you are ready to make informed decisions about participation when a new clinical trial begins recruitment.
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A clinical trial is a process to test a potential new treatment’s safety and effectiveness. The treatment, often referred to as an “intervention,” may be a medicine, device, procedure, or changes to the participants behavior – such as diet. The clinical trial is designed to measure the outcome for participants when the intervention is introduced. In the United States, the Food and Drug Administration (FDA) is the regulatory agency that oversees all clinical trials. The FDA is also responsible for reviewing applications for new interventions to treat a disease or condition. The FDA gives approval if the data from the clinical trial proves that benefits of the intervention outweigh the known risks.
Before an intervention is tested in people, it must be successful in animal tests and proven that it will likely be safe for humans. After a clinical trial is ready for human participants, how does the process work? Keep reading to learn!
When a researcher or pharmaceutical company want to test their intervention on humans, they must submit an Investigation New Drug (IND) application to the FDA. After they receive approval, they can begin recruiting volunteers to participate in their clinical trial.
After IND approval, the clinical trial can start enrolling patients to try their intervention. Clinical trials are listed at ClinicalTrials.gov. The listing will include detailed information about where the study will take place, for how long, and eligibility criteria for participants.
Researchers and pharmaceutical companies will often contact a patient advocacy organization for the disease or condition to help with their recruiting efforts. Staying connected with NAF is a great way to learn about new clinical trials for Ataxia. Become a member to receive notices when new clinical trials that might interest you are recruiting participants.
The next thing to know is that clinical trials are classified by their phase, which include Phase I, Phase II, and Phase III. Phase I is typically a small number of participants to determine safety of the intervention. Phase II begins after a successful Phase I and evaluates effectiveness of the intervention. If the treatment benefits enough patients in Phase II, then the trial moves to Phase III. Phase III tests the treatment in a larger population and compares it against current treatments available.
Learn More:
Clinical Trial Phases
Even if there aren’t clinical trials available right now, there are steps you can take to be ready when a new trial starts recruiting!
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