What is a clinical trial?
How therapies for rare diseases are approved
Clinical trials are research studies that test if a treatment is safe and effective in human patients. Through clinical trials, doctors and scientists can search for ways to potentially improve treatments and increase the quality of life for people with different diseases.
There are gene therapy clinical trials available for some individuals living with certain rare genetic conditions.
Gene therapy for rare diseases: Clinical trial timeline (3-5 years or more)
Because of the urgent need for treatment and the small number of potential patients, clinical trials for rare diseases may combine the steps of conventional clinical trials for more timely approval.
These trials may include adults and adolescents, young children, even infants, and smaller patient groups. Only patients with a confirmed diagnosis are enrolled; healthy volunteers are not eligible. Many studies include long-term follow-up to monitor patient safety.
Preclinical
Lab and animal studies
- Using animal models of a particular disease, determine dose range and optimal administration factors, such as delivery (by mouth or injections) and timing
- Establish the initial safety profile of the therapy
Phase 1&2
People with disease or condition
- Determine safe and effective dose, plus patient benefit and safety
Phase 3
People with disease or condition
- Establish safety and efficacy in patients with the disease or condition
- Used by regulatory agencies to determine approval
Are clinical trials safe?
Connect with your healthcare provider to help you make the right decision. Your decision can only be made after you and your doctor discuss the goals of the trial, risks, benefits, and expectations. Before taking part in a trial, you will need to read and then sign an informed consent document, which also describes the potential risks of participating in a clinical trial.
Talking with your care team about participating in a clinical trial
If you are interested in learning more about participation in a clinical trial, reach out to your treatment team to get more information. Your healthcare provider can help find answers to questions you may have and help you decide if a trial is right for you.
