Considering clinical trials

Everything that we know about treating cancer and other diseases has been learned from clinical trials. Clinical trials are research studies that involve people. Long before any drug is considered for a clinical trial, it has been studied carefully in the lab and has been tested on animals. It is not true that trials are only available and appropriate for people with advanced disease who have run out of other options. Clinical trials are used to care for cancer patients at all stages of illness.

It is important to understand the basic facts about our system of clinical trials and to remember that they are studies because the answers are not yet known. Occasionally a cancer drug being newly tested turns out to be a home run; more often they are only a bit better than standard treatments, and they may even be less effective. We can learn only because people are willing to participate, hoping that there will be value for them, and hoping to contribute to science and help others in the future. If you are thinking about participating in a clinical trial, it is very important that you understand the system and the probable limits of success.

• Phase 1 clinical trials are the first time that a drug has been used on human beings. The primary concern is safety. As more people are treated, the dose of the drug is increased to learn about side effects and risks. The goal is to identify the highest possible dose with an acceptable number of side effects.
• Phase 2 clinical trials are to learn more about the efficacy of the drug in particular cancers. The goal is to find out if the drug is effective and as safe as standard treatments.
• Phase 3 clinical trials test the new drug vs. existing drugs or drug regimens. The question is whether the new drug is more helpful and/or better tolerated than existing treatments.

Studies have found that only 1 in 10 drugs in a Phase I trial are eventually approved by the FDA. This means that most tested drugs don't turn out to be useful. Read about cancer clinical trials at BIDMC.

Here are some things to talk about with your doctor:

• Will my insurance cover the cost of a trial? Many clinical trials are funded by the government or by industry, so there is no cost to you. Sometimes a trial will even cover transportation or lodging if you have to travel to participate. However, some clinical trials will be billed to your insurance or even directly to you.
• Why do you think this trial might be a good treatment for me?
• What are the potential downsides for me?
• How many additional trips to the hospital would this involve? Would there be extra blood draws or scans? Are those costs covered by the trial?
• What are the alternatives for me right now?
• As hard as this is to remember, clinical trials are not necessarily any better than standard treatments. You aren't signing up for a miracle; you are signing up to help doctors gather information that may be helpful going forward. It is a bonus if it also helps you.
• If you are dealing with advanced cancer and considering a trial, think even more carefully. If you have limited time and energy, you may not want to expend it traveling to a distant center, far from you family and friends.
• Do your homework and think carefully about this decision. Some good sources of more information include:
  • ClinicalTrials.gov: a registry of clinical trials worldwide
  • National Cancer Institute (NCI): information on clinical trials from the NCI
  • American Society of Clinical Oncology (ASCO): information from ASCO's Patient Education site