Things to Consider Before Taking Part in a Clinical Trial

Deciding to be a part of a clinical trial is personal. Before you decide to be in one, learning about clinical trials and getting all your questions answered is important.

Should I agree to take part in a clinical trial?

This can be a very tough question. Each person’s situation is unique, and each person’s reasons for wanting or not wanting to take part in a trial may be different. There may be more than one trial that looks like an option. It’s important to learn as much as you can.

When trying to decide, first ask yourself some questions.

• Why do I want to take part in a clinical trial?
• What are my goals and what do I expect if I decide to take part? How realistic are these goals?
• How sure are my doctors about my future if I decide to take part (or not take part) in this clinical trial?
• Do I have all the information I need to make an informed decision?
• Have I weighed the benefits against the risks?
• Have I thought about other factors, such as travel, time, and money?
• Have I looked at my other options?

Some of these questions may not have clear answers but should help you start thinking about these issues.

Who should I talk with before deciding?

If your cancer care team is involved with the clinical trial, be sure to ask them your questions. Also, getting a second opinion from a doctor who’s not part of the study may help you decide if a certain study is the best option for you.

If your cancer care team is not involved with the clinical trial you’re interested in, talk with someone linked to the clinical trial. This could be the clinical investigator – the person in charge of the study – or a research coordinator. They should be able to answer your questions about the clinical trial. Be sure to share the details about the clinical trial with your doctor so they can help you decide what might be right for you. Your doctor knows your medical situation best and can probably tell you if the study is worth thinking about. This can take some time, so you might need to make a special appointment.

You might find it helpful to include friends and family members in your decision-making process. They might ask questions you hadn’t thought of and can help you make sure that you’re choosing what's right for you. 

Risks versus benefits

All clinical trials have risks. This is especially true of phase I and II clinical trials, where the treatment has been studied in fewer people.

A bigger question is whether the possible benefits outweigh the risks. People with cancer are often willing to accept a certain amount of risk for a chance to be helped. But it’s always important to be clear on what this chance is. Ask your doctor to give you an idea of what the benefits may be for you. Some people may decide that any chance of being helped is worth the risk, while others may not. Others may be willing to take certain risks to help others.

Each clinical trial has its own benefits and risks. Here are some potential benefits to consider:

• You might help others who have the same disease.
• You could get treatment that’s not available outside of the trial. This treatment might be safer or work better than current options.
• This may increase the number of treatment options.
• You’ll likely see your cancer care team more often so that they can monitor your cancer and check for side effects of the new treatment.
• Some study sponsors (groups or companies that fund the trial) may pay for part or all your medical care and other expenses during the trial. This isn’t true for all clinical trials.

Some possible risks of being in a clinical trial can include:

• The new treatment may have unknown side effects or other risks which might be worse than those from standard treatments.
• The new treatment may not work for you even if it helps others.
• You may need to have more doctor visits or tests which may require more time and travel.
• If you take part in a randomized clinical trial, you may not have a choice about which treatment you get. If the study is blinded, you (and maybe your doctor) won’t know which treatment you’re getting. (This information will be available to the clinical trial team as needed for your safety.)
• Your insurance may not cover all costs of the clinical trial. They usually do cover the costs of what would normally be standard care. Be sure to talk to your insurance provider and someone involved with the clinical trial before you decide to take part.

Clinical trial costs

In most cases, the study sponsor (the group or company that funds the trial) provides the new treatment at no cost and pays for any special tests or extra doctor visits. Some sponsors may pay for more. For example, some might pay you back for travel time and mileage. It’s important to find out what will be paid for before you decide to get involved in a clinical trial.

The Affordable Care Act requires that health insurance covers the routine patient care costs for people who are in approved clinical trials. “Routine patient care costs” are costs that would normally be covered for anyone being treated for your kind of cancer. Insurers are not allowed to drop or limit coverage because a person chooses to take part in a clinical trial. This applies to almost all clinical trials.

The law requires insurance coverage for phase I, II, III, or IV clinical trials related to prevention, detection, or treatment of cancer or other life-threatening disease if the clinical trial meets one of these requirements:

• It’s funded by any US federal agency, such as the National Cancer Institute (NCI), Centers for Disease Control and Prevention (CDC), Department of Defense (DOD), etc.
• It’s covered under an investigational new drug application (IND) that’s reviewed by the US Food and Drug Administration (FDA).
• It does not need an IND application.

Insurers do not need to pay for:

• The treatment, device, or service that’s being studied. This is usually paid for by the trial’s sponsor.
• Items and services only needed for data collection and analysis and not used in direct patient care.
• Any service that’s clearly not in line with standards of care for a certain type of cancer.

It is important to know what the clinical trial may not pay for so you can check if your insurance will.

Medicare and Medicaid coverage for clinical trials

If you have Medicare or Medicaid (as of January 1, 2022), they will likely pay for many of the routine patient care costs for people with cancer who are in approved clinical trials. This is true no matter where in the US you live.

Cancer clinical trials must meet certain criteria to qualify for coverage. Clinical trials must focus on the prevention, detection, and treatment of cancer or another life-threatening disease. The clinical trial must also be approved, done by, or paid for by certain government agencies or another accepted group.

If you’re not sure if your trial meets all the requirements, discuss these concerns with your doctor, call Medicare (1-800-633-4227) or contact your state Medicaid agency.

How to find out more about costs

Gather as much information as you can about the clinical trial and contact your insurance provider to find out about costs. Many insurers may not be able to give you a simple yes or no answer, because they may review claims on a case-by-case basis. They’ll also want to be sure that the doctors supplying the main part of your cancer care are “in network.”

If your insurance will not pay for parts of the clinical trial, ask your doctor or the research coordinator about other options. Sponsors may be willing to cover some of the costs if your insurance does not.

Common questions and concerns about clinical trials

Most people have some concerns about taking part in a clinical trial because they’re not sure what it will mean for them. Get as much information as you need to make the choice that’s right for you.

Will I be a “guinea pig?”

You will not be a guinea pig, but it's true that the role of a clinical trial is to answer a medical question. People who take part in clinical trials may need to do extra things or have certain tests done as part of the clinical trial. But this doesn’t mean that you won’t get excellent care. In fact, most people enrolled in clinical trials welcome the extra attention they get from their cancer care team.

Studies have shown that people with cancer who felt well informed before they took part in a clinical trial had less regret after the study than those who felt unsure. That’s why it’s important to take your time, ask questions, and feel good about your decision.

How do I know if I qualify for a clinical trial?

Before you can join a clinical trial, you must meet the requirements (eligibility criteria). All clinical trials have criteria about who can and can’t be in them.

• Inclusion criteria: The factors that allow a person to sign up for a study.
• Exclusion criteria: The factors that can prevent a person from joining a study.

Criteria are different for each study. For cancer clinical trials, the inclusion and exclusion criteria usually have to do with:

• The type and stage (extent) of cancer a person has
• Previous treatments a person had
• The length of time since a person last had treatment
• Results of certain lab tests
• The medicines a person is taking
• Other medical problems the person has, including a prior history of another cancer
• A person’s activity level (performance status)

Talk with your doctor or someone on the clinical trial team to see if you may be eligible for a clinical trial you are interested in.

Will I get a placebo?

A placebo is an inactive treatment used in some types of clinical trials – sometimes called a “sugar pill.” Researchers compare it to the new treatment to help make sure any results are actually from the new treatment. Most clinical trials for cancer don’t use placebos unless they are given along with the standard treatment. It’s unethical to give someone a placebo instead of a treatment that’s known to work.

There are some types of cancer that no treatments have been proven to help. In rare cases, testing a new treatment against a placebo might be needed to prove that the treatment is better than nothing at all. The very least you should expect from any clinical trial is to be given the standard treatment.

Can my doctor or I pick which group I’m in?

No, not for studies that are randomized. This means that each person in the study gets assigned by chance to either the treatment group (who gets the new treatment) or the control group (who gets the best standard treatment). Randomization helps to make sure that the groups have people in similar states of health, so the results are not affected by differences between the groups. If people could choose which treatment they got, the study results might not be as accurate.

Will I know which group I’m in? Will my doctor know?

Each study is different. In a blinded study, the patient doesn’t know which treatment they’re getting. In a double-blinded study, neither the patient nor the doctor knows which treatment is being used.

Not knowing what treatment you’re getting can be hard. This can be especially true if a trial is looking at totally different treatments and a person believes that one is better than the other. But remember, doctors are doing the study because they really don’t know which one is better. Sometimes, taking part in such a study is the only way a person has a chance of getting a new treatment.

Your doctor can always find out which group you’re in if there’s an important medical reason (like a medication reaction), but it may result in you being removed from the study. Blinding reduces the risk that the doctor or patient’s beliefs about the new treatment will affect their evaluation of response or side effects.

Will my information be kept confidential?

As much as possible; your cancer care team needs some personal and medical data to give you the best possible care, just as they would if you were not in a clinical trial.

Information that’s needed for the clinical trial, like test results, is put in special forms and into computer systems. This is only shared with the people who analyze the results. Your data is given a number or code so your name isn’t on the forms or in the study system. Sometimes, members from the research team or from the FDA might need to look at your records to be sure the data they were given is correct. But your personal information isn’t given to them and is never used in any published clinical trial results.

What if I’m not eligible for a clinical trial?

Some people are too sick or have other problems that keep them from taking part in clinical trials.

Other people may be interested in a certain treatment that’s only available in clinical trials, but they don’t meet the eligibility criteria. Sometimes, a person’s doctor may ask the study sponsor for a special exception. The trial's clinical investigator will decide whether to grant the request. If approved, the person gets the new treatment, but their results aren’t included in the final analysis.

In some cases, studies may have already enrolled enough people and aren’t taking any more participants.

Sometimes, there are ways to get access to treatments that are in late-phase clinical trials but not yet approved by the FDA. These are called compassionate use programs. In recent years, the FDA has broadened these programs to allow some patients who urgently need these treatments to be able to get them. For more, see Compassionate Use.