Clinical trials provide data that prove a new treatment is better than the standard therapy. They may offer alternatives if you have few treatment options or if you’re seeking a treatment with the potential of being more beneficial than the standard treatment. Choosing a clinical trial may mean you can find an option with fewer toxicities than the one being offered or one that is more convenient, such as an oral medication or a shorter treatment time. Although an increasing number of investigational cancer drugs are being approved by the Food and Drug Administration each year, the process is still lengthy and complex. If you can navigate the process you may find a good option, but it’s important to understand the phases involved in clinical trials.

Phase 1 trials enroll a small number of patients to study side effects and establish a safe dosage for a potential treatment. Phase 1 also evaluates how a new treatment should be given (for example, if a new drug is best taken orally or injected into the bloodstream or muscle), how often it should be administered and the most effective dose with the fewest and least severe side effects. Most patients who enter phase 1 trials have limited treatment options or do not improve with standard therapies. This phase is not to determine efficacy.

Phase 2 trials continue to test the safety of a treatment while beginning to evaluate how well it works. These trials are usually limited to a specific cancer that showed benefit with the treatment in earlier trials.

Phase 3 studies compare the experimental drug, combination of drugs, regimen of radiation therapy or surgical procedure with the current standard to determine if it is better. Enrollment is often in the thousands across multiple locations. Typically, a participant is randomly assigned to the standard treatment or the new treatment (called randomization). Patients who are not randomized to the experimental treatment will receive the best standard treatment available.

View Illustration: From the Laboratory to the Clinic

Before enrolling in a clinical trial, you’ll be asked to sign an informed consent document that states you understand the purpose of the research and its risks and benefits, as well as your rights as a patient. The informed consent process also provides an ongoing, open line of communication between you and the researchers to ask questions. Keep a copy of the informed consent document with your medical records. No informed consent document can ask you to waive your legal rights or release the trial’s research team, trial sponsor, drug manufacturer or institution from liability for negligence.

You are allowed time to discuss the informed consent documents with family, friends or your physicians and to ask follow-up questions of the research team. As the trial progresses, the research team will continue to provide information and updates. It is important to understand that because the treatment is experimental, the outcomes and side effects are not always foreseeable, although any predicted risks should be explained to you in detail beforehand.

Discuss the costs associated with the trial with the research team and ask what would be covered by insurance. In most trials‚ the therapy under investigation is provided at no cost to the participant. Routine costs, such as hospital stays, outpatient appointments and tests accrued during a trial, are often covered by insurance or Medicare if the trial meets certain criteria.

Starting in 2014, the recently enacted Patient Protection and Affordable Care Act will prohibit new health plans from denying coverage for routine care that the plan would otherwise provide just because a person with cancer (or other life-threatening illness) is enrolled in a clinical trial. The new law also prohibits an insurer from dropping coverage because a person chooses to participate in a clinical trial. You may also want to consider the cost of travel and lodging if the site of the trial is distant, especially if the trial extends over several weeks or months and frequent trips are needed. Some institutions and nonprofit organizations can help with certain expenses for travel and housing (view Toolbox by visiting curetoday.com/toolbox).

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