Why participate in a trial?
Deciding whether to get involved in a clinical trial or not is a highly personal decision. The PWA will likely need to talk it over with close friends, doctors, or other family members before they feel comfortable deciding. And the reasons for participating in clinical trials are as varied as the people who enroll.
For the PWA, clinical trial participation can be:
- An opportunity to try new therapies that aren’t yet approved
- A way to contribute to research that could ultimately improve their condition
- A chance to help others in the future
- A chance to find other treatment options when current treatments haven’t worked or the PWA suffers from side effects that negatively affect their life
Learning the types of trials
There are many different types of clinical trials. However, the kind of trial you research and discuss with the PWA depends on their personal goals and comfort level.
Here are some examples of different kinds of trials you may encounter as you investigate:
- Preventive: Evaluates new medications, vitamins, minerals, supplements, vaccines, and/or lifestyle changes that might lower a person’s risk of certain diseases or conditions.
- Screening: Attempts to find the best ways to detect certain diseases or conditions before patients experience symptoms. This trial also tries to determine if earlier detection of disease decreases a person’s risk of severe harm or death.
- Diagnostic: Tries to discover new ways to diagnose and detect certain medical conditions and to improve testing methods, and the effectiveness of procedures and tools
- Treatment/intervention: Finds new treatments for a disease or condition. This trial can involve new and/or existing drugs, psychotherapy, a new device, or a new surgical approach. Assuring the safety of participants is the priority.
- Therapeutic: Investigates a treatment, not necessarily pharmacological, that is believed likely to benefit participants who receive the intervention
- Non-therapeutic: Obtains knowledge that may contribute to the future development of treatments and procedures.
Understanding the phases of a trial
Technically there are five potential phases of a trial, though only three are required for all programs.
Phase 0: Researchers determine a drug’s safety before it’s approved to study in humans
Phase 1 (required): Identifies the most effective (and safest) dosage levels of the new treatment
Phase 2 (required): Participants in this phase are people who have the disease or condition (such as acromegaly). This phase focuses on learning how the treatment affects the body and works for a specific disease. The study continues if the treatment is deemed safe and has some benefit.
Phase 3 (required): During this phase, researchers want to study the drug’s effects in a larger population of participants (can range from hundreds to thousands) who have the disease. Researchers may also use a placebo. This phase is also when the regulatory agency reviews the whole development program and the results of the clinical trials before deciding whether to approve the new drug.
Phase 4: This phase is usually conducted after a drug is approved and marketed to monitor a specific possible safety concern or monitor side effects in a particular population.