By looking into this, am I encouraging my loved one to sign up to be experimented on like a guinea pig?

Before a drug can be approved for use, it needs to undergo a rigorous screening and preclinical testing process to ensure that it is safe and effective. There is a period—often years—of testing done before a clinical trial can even begin. The entire process can take up to six years to complete. Additionally, every clinical trial has a thorough informed consent process to make sure that you and your loved one understand what’s involved as a participant and what, if any, side effects previous participants have experienced. Participants are also able to leave the trial at any time and for any reason.

If we go through all this, are we going to find out that my loved one received a placebo—so it was a waste of our time?

Some clinical trials measure the effectiveness of a potential new drug or treatment against a placebo—an inactive substance or treatment used as a control when testing new drugs or treatments--but placebos aren’t used in every trial. Some other trials test a potential new drug or treatment against the existing standard of care. The decision about whether to use a placebo in a clinical trial is based on how serious the illness is, whether an existing treatment is available, and other considerations to ensure a high standard of ethics. For serious or life-threatening diseases, the best available treatment is used instead of a placebo.

I don’t want my loved one to receive some dangerous, experimental drug. How safe is this?

Because clinical trials are done for research purposes, there is some level of risk. However, the drugs being studied are given to clinical trial participants only after the drugs have gone through a rigorous testing process and scientific evidence indicates that the drug is likely to be effective and safe for use in humans. The safety of the patients is a top priority for everyone involved in the trial. Before a clinical trial can even begin, it’s reviewed by an institutional review board (IRB)—a committee of doctors, scientists, and community members who have the responsibility to protect clinical trial participants. This review makes sure that both before and during the trial, steps are taken to protect the rights and safety of the trial participants. During the clinical trial, the safety of participants is closely monitored.

How much time is involved? Am I going to have to use up all my sick leave to take my loved one to appointments?

The amount of time required to participate in a clinical trial varies widely. For some, your loved one may only need to make a few visits every couple of months. Others may require weekly visits. Some trials require overnight visits. Before your loved one enrolls in a trial, the trial schedule will be explained and you will have an opportunity to ask questions or share any concerns that you have.

Are there a lot of out-of-pocket costs? How can we afford this?

Clinical trial participants are rarely required to pay for costs related to participating in a trial. Those research costs are covered by the sponsor of the study. There may be some patient care costs that are not covered by the sponsors of the research which are often covered by the patient’s health insurance carrier. Before enrolling in a clinical trial, you should ask the research team what costs your loved one would be expected to pay and then check with your loved one’s health insurance carrier to see if they will cover it.

After all this trouble, is my loved one going to benefit in any way?

Participants in a clinical trial may have access to an investigational drug that is not yet available to people outside of the trial. Participants are closely monitored by clinical trial researchers—maybe even more closely than their regular doctor. Depending on the clinical trial treatment plan (or protocol), participants may receive additional tests and lab work that are outside of their usual care.

We don’t live in New York, Chicago, Los Angeles, or any other large city with a major hospital or research facility. Is it still possible for my loved one to participate in a clinical trial?

Regional hospitals and smaller research facilities also run clinical trials. In addition, some clinical trials—particularly those for rare diseases—offer compensation for any patients who need to travel in order to participate. 

My loved one is doing okay now. Aren’t clinical trials for people who have run out of all other treatment options?

While participating in a clinical trial can be an option for patients who tried the other options without success, there are clinical trials for patients at all different stages of their diagnosis. Some trials that target newly-diagnosed patients. Others may recruit patients who exhibit specific symptoms or who were diagnosed many years ago.

What is a Clinical Trial?

According to the National Institutes of Health (NIH), the goal of clinical trials is to determine if a new treatment—such as a new drug or medical device—prevention, or behavior approach is safe and effective. Often a clinical trial is used to learn if a new treatment is more effective and/or has less harmful side effects than the standard treatment.

Another type of clinical trial tests ways to find a disease early, sometimes before there are symptoms. Others test ways to prevent a health problem. A clinical trial may look at how to make life better for people living with a life-threatening disease or a chronic health problem. There are even clinical trials to study the role of family caregivers or support groups.

Before the U.S. Food and Drug Administration (FDA) will approve the start of a clinical trial, laboratory tests have been performed to test a potential therapy’s safety and efficacy. If these studies show favorable results, the FDA gives approval for the intervention to be tested on people.

Clinical trials advance through four phases to test a treatment, find the appropriate dosage, and look for side effects. After the first three phases, if researchers find a drug or other intervention to be safe and effective, the FDA approves it for clinical use and continues to monitor its effects.

Phases of Clinical Trials

Phase 1:

20-100 healthy volunteers or people with the disease/condition being studied. The purpose is to study the safety and determine the proper dosage. This phase lasts several months. The FDA reports that 70% of new drugs move to the next phase.

Phase 2:

Up to several hundred people with the disease/condition. The purpose is to determine the short-term safety and efficacy and to see if there are any side effects. A small group is given a placebo for comparison. This phase lasts from several months to 2 years. The FDA reports that 33% of drugs move to the next phase.

Phase 3:

300-3,000 people with the disease/condition. The purpose is to further study the efficacy, look out for any adverse reactions, and compare the new drug with other treatments (another drug or placebo). This phase lasts 1-4 years. Because this phase is larger and longer, side effects that take a while to appear or are rare, are more likely to be seen. The FDA reports only 25-30% of drugs move to the next phase.

Phase 4:

Several thousand people who have the disease/condition. The purpose is to further study the efficacy and safety.