Nearly all treatments, including drugs and medical devices, require clinical trials and approval by the government’s Food and Drug Administration (FDA). The journey from a scientific breakthrough to a treatment you can access is a long one — taking years. (The recent approvals of COVID-19 vaccines are welcome exceptions to that timeframe.)
A clinical trial is a research study using human volunteers, referred to as participants, to answer specific health questions. In general, clinical studies are designed to add to medical knowledge related to the treatment, diagnosis, and prevention of diseases or conditions. These studies are conducted to evaluate whether the new treatment, procedure or device is safe and effective. Clinical trials can be sponsored by the government, by academic institutions and by industry and usually involve at least three phases.
Phase 1 clinical trials evaluate the safety and dosage of a drug or treatment to determine how well it works. This testing normally takes place with a small group of healthy volunteers. If the trial meets the primary outcomes, as proposed in the application to the FDA, then the FDA permits the trial to proceed to Phase 2.
Phase 2 clinical trials test the efficacy and side effects in treating a particular disease, with several hundred participants with the disease.
Phase 3 clinical trials are the ultimate test of whether a treatment is safe and effective for a wide variety of people, and typically involve a much larger group of volunteers for a longer period of time than Phase 1 and 2 trials. Just like Phase 2 trials, the plan normally involves assigning participants to treatment or control groups.
Choosing to participate in a clinical trial is an important personal decision. If you or someone you know is interested in learning more, it is important to talk to your doctor and family members. All clinical trials have guidelines about who can participate. Each has its own protocol or set of guidelines, and volunteers must meet certain criteria to qualify for inclusion. Because small groups and select doctors are involved with trials, don’t expect your doctor to have specific details on each study. Be sure to understand that expressing trial interest does not guarantee involvement nor does it guarantee that you will be included in the treatment group. If you want to learn more, try these resources:
An easy-to-use website that provides regularly updated information about federally and privately supported clinical trials. You can find specific clinical trials for a wide range of diseases. Glaucoma-related trials globally are accessible in one section – with such details as location, sponsor and status (completed, active, recruiting, etc.).
Offers a clinical trial database with currently enrolling trials, information on the trials process, and on drugs and new medical therapies.
A free and secure registry that brings together people who are trying to find research studies and researchers who are looking for people to participate in their studies.
This NIH Clinical Trials and You website is a resource for people who want to learn more about clinical trials.