About Clinical Trials

What is a Clinical Trial

Living with a disease or condition impacting your health, or the health of a loved one, can be stressful or even life-altering. But new experimental interventions are constantly in development for hundreds of indications – many of which are being tested today in patients and healthy volunteers through clinical trials. These treatments have already passed initial safety screenings and have the potential for improving quality of life. If you’re living with one of these conditions, taking part in a clinical trial could be the hope you’re looking for. Participation also gives you the opportunity to play a critical role in medical research leading to better healthcare outcomes for yourself or others in the future. And if you’re currently healthy, voluntarily joining a trial is equally as important to medical progress.

Types of Clinical Trials

Clinical trials are part of the final steps for researchers before an experimental intervention can be approved for general public use among patients. They can study new drugs, vaccines, medical devices, medical procedures, or behavioral interventions as well as existing ones that warrant further study. Both safety and effectiveness are considered, and all are carried out through well-controlled, time-refined methods with highly trained researchers and medical professionals.

If you decide to participate in a trial, it will be one of two kinds:

Interventional

This is the experimental study that most likely comes to mind when you think about clinical trials. It’s part of research needed before a drug, device, or procedure is approved. To test safety and effectiveness, participants are provided the experimental intervention, typically to compare treated subjects with those receiving no treatment, an inactive placebo, or the standard of care. In some studies, such as those testing dosages, all participants receive the experimental intervention. The data from the study is then analyzed and sent to regulatory agencies for review. (Subsequent references to clinical trials here will refer primarily to interventional studies.)

Observational

In this type of study, investigators observe participants throughout their existing, ongoing treatment – or regular daily lives – and track their specific health outcomes over time. Unlike interventional trials, observational trial participants are not assigned to a specific group based on a research plan, such as being divided into treatment and placebo groups, but instead any treatment received would be as a part of their routine medical care. Observational study designs are used for reasons such as better understanding a disease or potential correlations for a disease that may be independent of any treatment (e.g., the effects of lifestyle on cardiac health, or ethical considerations. Consider a study involving patients with diabetes, where it would be unethical to ask one group to stop taking insulin. Or a study of alcohol’s impact on pregnant women, where you would never require one group to drink. This design is often used to investigate cause and effect associations before interventional trials are finished.

Clinical Trial Phases

Clinical trials typically go through a series of phases. Before progressing to the next phase, regulatory agencies must approve both the safety and effectiveness of the intervention. The entire clinical trial process for an approved intervention takes an average of about a decade, though some could take 15 years or more.

All new treatments must go through extensive study in labs – called pre-clinical development – before testing on patients. If these initial results are promising, researchers seek permission from regulatory authorities and independent ethics committees to study the treatment in humans.

Phase

The main focus of Phase 1 studies is to examine an intervention’s safety and see how it interacts with the body. If the test is for a drug, it can include different dosages. About 58% of interventions successfully advance to Phase 2*.

Often includes less than 100 participants
Participants are healthy volunteers or patients with a specific condition
Analyzes how the intervention interacts with the body and any potential side effects
Takes on average about 2 years to complete

Phase

Phase 2 studies focus on intervention effectiveness while also collecting data about safety and side effects. About 31% of Phase II trials are successful enough to move to Phase 3*.

Usually includes 100-500 participants
Participants have a specific condition
Participants are sometimes given a placebo
Helps better determine ideal dosage
Takes on average about 3 years to complete

Phase

This is the last stage of research before an intervention can be approved for general public use. Phase 3 studies determine the effectiveness and safety of interventions as compared to those already existing. About 60% of Phase 3 trials are successful enough to be submitted for regulatory approval, and about 90% of those receive approval (which takes around an additional 1.5 years)*

Usually includes 300-3,000 participants
Participants have a specific condition
Takes on average about 3 years to complete

Phase

Testing continues after an intervention is approved. Phase 4 studies determine the effectiveness and safety of interventions when used by the public in daily life, and collect additional data on benefits, risks, and quality of life.

Usually includes several thousand participants with a specific condition
Helps determine the optimal way to deliver the treatment
Takes many years to complete

Phase

Often includes less than 100 participants
Participants are healthy volunteers or patients with a specific condition
Analyzes how the intervention interacts with the body and any potential side effects
Takes on average about 2 years to complete

Outcomes & Benefits of Clinical Trials

If you’re living with a condition, taking part in a trial could offer an opportunity for better health and also potentially help thousands or millions of others impacted by similar conditions. And if you’re a healthy volunteer, you’ll also be a crucial part of medical progress.

After a clinical trial’s completion, regulatory agencies review all data to make a decision based on the observed safety and effectiveness of the intervention.

Participation Benefits:

Use of new drugs, devices, or therapies not yet available to the public
A specialized team of healthcare professionals focused on you
Access to the latest medical research and information on the condition
The privilege of knowing you are advancing medicine and helping others – present and future – who are similarly affected

Anyone thinking about joining a clinical trial must weigh the costs and benefits. It’s important to discuss participation with your doctor so you are fully aware of all considerations.

Clinical Trials Safety

Are you apprehensive about joining a clinical trial? It’s completely understandable, but be reassured that all studies are designed with the highest ethical and safety standards. Participants are valued and treated with the best in care and closely monitored throughout – both to ensure your health and the success of the study. And if you do decide to sign up, it’s always voluntary and you can drop out at any time if you change your mind. That said, you do need to consider some potential risks:

You cannot choose the intervention you receive, including the dosage level or if you get a placebo
The experimental intervention might not be better than available interventions or could have worse side effects
Taking part in a trial may require more doctor visits than usual
Trial interventions might not be fully covered by health insurance