Understanding Cancer Clinical Trials

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What patients need to know when deciding whether a clinical trial is right for them.

Clinical trials provide data that prove whether a new treatment is better than the standard therapy. They could 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 might 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 one with a shorter treatment time. Although an increasing number of investigational cancer drugs are being approved by the Food and Drug Administration (FDA) each year, the process is still lengthy and complex. If patients can navigate the process, they might find a good option, but it’s important to understand the phases involved in clinical trials and the potential benefit.

Phase 0 trials are the first to move a drug out of the laboratory and into people. Their goal is to determine how the body handles a specific drug. These trials are very small, including only 10 to 15 people, usually healthy individuals.

Phase 1 trials enroll a small number of patients with diverse tumor types to study side effects and establish a safe dosage for a potential treatment, usually by increasing the dose serially and observing for side effects. 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 work, sometimes in combinations with approved drugs. These trials are usually limited to a specific cancer that showed benefit with the treatment in earlier trials.

Phase 3 trials 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 thou- sands 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.

Phase 4 trials are sometimes referred to as real-world studies, because they illustrate how a drug works outside of a strictly regulated clinical trial. Some, but not all, of these trials occur after a drug has been approved by the FDA. Some goals of phase 4 trials are to learn more about a drug’s effectiveness, safety and side-effect profile. These trials also help determine how the medication works when given alongside other treatments, how it compares to other therapies on the market and whether or not it is cost-effective. Phase 4 investigations are large, including several hundreds or thousands of patients.


Compassionate drug use is the use of a new, unapproved drug to treat a seriously ill patient when no other treatments are available.

Many people with life-threatening diseases can’t find suitable clinical trials. This might be because they live too far from cancer research centers where the studies are being done, or because they’re not eligible for some reason.

People who aren’t in clinical trials might be able to get access to an unapproved drug from the company that makes it in two ways:

  • Through expanded access programs (EAPs).
  • Through “Right to Try” laws.

Expanded Access Programs (EAPs). In general, EAPs are for patients who meet all of these conditions:

  • Have a serious and life-threatening condition.
  • Are not eligible for any current clinical trial that’s using the drug.
  • Have no other comparable treatment options.
  • Are likely to experience benefits that outweigh the risks involved.

The FDA approves or denies EAP requests based on available reports from the company sponsoring the drug in clinical trials, including safety information and data about how the drug may treat cancer. To learn more, see the FDA’s information about EAPs at tinyurl.com/y9woscn3.

“Right to Try.” “Right to try” came about when a federal law was passed in 2018 that allows patients another path to access unapproved drugs, without needing the review and approval of the FDA. “Right to try” laws do not replace EAPs, but provide another way to possibly be able to access unapproved drugs.

It’s important to understand that “right to try” does not actually give patients the right to try any unapproved drug. Instead, it gives them the right to request access to an unapproved drug from the company that makes it, without having to go through the FDA.

Bypassing the FDA does not necessarily mean that such access will be granted. To be eligible for “right to try,” a person must:

  • Be diagnosed with a life-threatening disease or condition.
  • Have tried all approved treatment options for the disease or condition.
  • Be certified by a doctor to be unable to participate in a clinical trial for the investigational drug.
  • Give written informed consent that he or she understands the risks of taking the investigational drug. 4Receive permission from the maker of the drug and approval from the institutional review board of a plan to administer and monitor effects from the drug.

To learn more, see the FDA’s information about “right to try” at fda.gov/patients/learn-about-expanded- access-and-other-treatment-options/right-try.


Before enrolling in a clinical trial, patients are asked to sign an informed consent document that states they understand the purpose of the research, its risks and benefits, the study procedures and their rights as patients. 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.


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 done during a trial, are often covered by insurance or Medicare if the trial meets certain criteria.

A provision in the Affordable Care Act, effective in 2014, prohibits 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 might 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 (visit curetoday.com/journey).


No single resource lists every clinical trial, but ClinicalTrials.gov is the most comprehensive and allows searching by tumor type, drug type, location and other factors. The process might involve searching the web, calling pharmaceutical companies or asking a doctor or cancer center for information. Patient advocacy groups can also help patients find appropriate trials.

Patients should begin with their oncologist‚ who not only can tell them if something is available locally‚ but also give them resources on what is happening in other parts of the country. Although it could be time-consuming to search for clinical trials at each location, patients will probably want to start with facilities closest to home. Those who are looking for a particular drug might 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 might 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 of the patients‚ previous treatments received and current health status. Patients should have their medical histories accessible when searching for clinical trials because eligibility requirements could disqualify them from participating. Once they have found a number of trials for which they possibly qualify, patients should discuss them with their doctors and contact the study coordinators.