Before Treatment: Understanding Clinical Trials

Clinical trials provide data that prove a new treatment is better than the standard therapy. They may offer alternatives if patients have few treatment options or if they’re seeking a treatment with the potential of being more beneficial than the standard treatment. Choosing a clinical trial may mean patients 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 patients can navigate the process, they may find a good option, but it’s important to understand the phases involved in clinical trials and the potential benefit.

Phase 1 trials enroll a small number of patients to study side effects and establish a safe dosage for a potential treatment. Phase 1 studies also evaluate 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 or 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 designed to determine the effectiveness of the treatment.

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 hundreds to 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 identified standard treatments.

Informed Consent

Before enrolling in a clinical trial, patients are asked to sign an informed consent document that states they understand the purpose of the research and its risks and benefits, as well as their rights as a patient. Patients should keep a copy of the informed consent document with their medical records. No informed consent document can ask patients to waive their legal rights or release the trial’s research team, trial sponsor, drug manufacturer or institution from liability for negligence.

Patients are allowed time to discuss the informed consent documents with family, friends or their 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 in detail beforehand.

Covering the Costs

Patients should 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 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 is enrolled in a clinical trial. The law also prohibits insurers from dropping coverage because a person chooses to participate in a clinical trial. Patients considering a clinical trial may also want to calculate 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.

Searching for a Clinical Trial

No single resource lists every clinical trial (clinicaltrials.gov is the most comprehensive and allows searching by tumor type, drug class and other factors). The process may involve searching the Web, calling pharmaceutical companies or asking a doctor or cancer center for information.

Patients should begin with their oncologist‚ who can not only tell them if something is available locally‚ but also give them resources on what is available in other parts of the country. Although it may be time-consuming to search for clinical trials at each location, patients will probably want to start with facilities closest to home. Patients who are looking for a particular drug may want to contact the pharmaceutical company directly for the best information.

Fewer trial locations will be available for drugs in early-phase testing, so patients may have better luck with late-phase trials, which are conducted in multiple sites across the country.

Each trial has its own eligibility criteria and often has restrictions based on the type and stage of cancer‚ age‚ previous treatment received and current health status. Patients should have their medical history accessible when searching for clinical trials because eligibility requirements may disqualify them from participating in certain trials. Once they have found a number of trials they may qualify for, patients should discuss them with their doctor and contact the study coordinators.