- Clinical Trials: What You Need to Know
- Why do we need clinical trials?
- What happens before clinical trials?
- Some facts about clinical trials: Important points to keep in mind
- What are the phases of clinical trials?
- Phase 0 clinical trials: Exploring if and how a new drug may work
- Phase I clinical trials: Is the treatment safe?
- Phase II clinical trials: Does the treatment work?
- Phase III clinical trials: Is it better than what’s already available?
- Submission for FDA approval: New drug application (NDA)
- Phase IV clinical trials: What else do we need to know?
- Who sponsors and runs clinical trials?
- Should I think about taking part in a clinical trial?
- Answers to some common questions about clinical trials
- Other questions you should ask your research team
- What protects the study participants?
- What’s out there? Finding clinical trials
- How do I figure out which study is for me?
- What about cost? Will my insurance cover it?
- What would it be like to take part in a clinical trial?
- What if I’m not eligible for a clinical trial?
- Summing it all up
Answers to some common questions about clinical trials
Most people have some concerns about taking part in a clinical trial, often because they’re not really sure what it will mean for them. Taking time to get as much information as you need before you decide is the best way to be sure that you will make the choice that is right for you.
Will there be risks?
Yes, all clinical trials have risks. But any medical test, drug, or procedure has risks. The risk may be greater in a clinical trial because any new treatment has more unknowns. This is especially true of phase I and II clinical trials, where the treatment has been studied in fewer people.
Perhaps a more important question is whether the risks are outweighed by the possible benefits. 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 realistic about what this chance is. Ask your doctor to give you an idea of what the possible benefits are, and exactly what benefit is likely for you.
With this in mind, you can make a more informed decision. 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.
Will I be a “guinea pig?”
There’s no denying that the ultimate purpose of a clinical trial is to answer a medical question. People who take part in clinical trials may need to do certain things or have certain tests done to stay in the study.
But this doesn’t mean that you will not get excellent, compassionate care while in the study. In fact, most people enrolled in clinical trials appreciate the extra attention they get from their health care team. In 2005, the Coalition of Cancer Cooperative Groups surveyed over 1,700 people with cancer on their awareness and attitudes about clinical trials. Only a few had taken part in clinical trials. But most of those who did were very satisfied: 96% said they were treated with dignity and respect, 92% said they had a positive experience, and 91% would recommend that family or friends take part in a clinical trial if faced with cancer.
Other 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 comfortable with your decision.
Will I get a placebo?
Most cancer clinical trials do not use placebos unless they are given along with an active drug. It’s unethical to give someone a placebo if it would deny the person a chance to get a drug that has already been shown to work.
Unfortunately, there are some types of cancer for which there are no proven effective treatments. 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 offered the standard of care already being used. (See the section, “Phase III clinical trials: Is it better than what’s already available?” for an example of a phase III study using a placebo.)
Can my doctor or I pick which group I’m in?
No. Most studies are randomized. This means that each person who takes part in the study gets assigned to either the treatment group or the control group. Randomization is used in many phase III studies because it helps reduce the chance that one group will be different from the other when they go into the study, which could affect outcomes. This is especially helpful to make sure that the groups contain people in similar states of health, so the results are not skewed in favor of one group. If people were allowed to choose which treatment they got, the study results might not be as accurate. For example, people who were sicker might tend to choose one treatment over the other. If the new treatment was then found not to work as well, doctors couldn’t be sure if this was because the treatment wasn’t as good or because it was tested in sicker people.
Often people have a 50:50 chance of ending up in one group or the other. In some cases, the study may allow for a different ratio, such as 2 out of 3 people getting the new treatment and only 1 out of 3 getting the standard treatment.
Some people find the concept of randomized studies distressing, since neither the patient nor the doctor can choose which group the patient is in. This can be especially true if a study is looking at 2 totally different treatments and a person sees one as better than the other. But remember, doctors are doing the study because they really don’t know which one is better. Unfortunately, taking part in such a study is sometimes the only way a person has a chance of getting a new form of treatment. But even then, that treatment may or may not be the best one for him or her.
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 he or she is getting. In a double-blinded study, neither the patient nor the doctor knows which treatment is being used. Not knowing what you are getting can be hard. Your doctor can always find out which group you are in if there’s an important medical reason (such as a possible drug reaction), but it may result in your being removed from the study. Blinding reduces the risk that the doctors will be biased in their evaluations of the patients’ outcomes. These controls help make the study results more reliable.
The possibility of getting a placebo can also be upsetting to some people. But this almost never means you would get no treatment, unless there was no effective standard treatment to compare with the new treatment. Again, in the example above, Li will definitely get drug D, but he will also get either EX-3 or a placebo.
Will my information be kept confidential?
As much as possible, all of your personal and medical information will be kept confidential. Of course, your health care team needs this information to give you the best possible care, just as they would if you were not in a clinical trial.
Medical information that’s important for the study, such as test results, is usually put on special forms and into computer databases. This is then given to the people who will analyze the study results. Your information is assigned a number or code—your name is not on the forms or in the study database. Sometimes, members from the research team or from the Food and Drug Administration may need to look at your medical records to be sure the information they were given is correct. But your personal information is not given to them and is never used in any published study results.
Last Medical Review: 09/21/2012
Last Revised: 09/21/2012