Clinical trials are scientific studies in which new treatments — drugs, diagnostic procedures, and other therapies — are tested in people to find out if they are safe and effective. Nearly all cancer drugs in use today were tested in clinical trials.
If you have been diagnosed with cancer, the benefits of participating in a clinical trial are potentially numerous. In addition to the chance that the trial will aid your own treatment, you can also help doctors develop better therapies for current and future patients.
Having a larger care team is another advantage because clinicians, researchers, research coordinators, research nurses, technicians, social workers, support staff, and other health-care professionals work together in planning your care. Studies show that this extra attention can enhance patient outcomes.
While new treatments being evaluated through clinical trials could potentially be better than standard treatments, there are no guarantees. That’s why the new therapy is being tested. But, given improvements in technology and a better understanding of cancer at the molecular level, there has been gradual improvement, through clinical trials, in our ability to develop drugs that work more effectively and reduce toxicity.
Because of the sheer number of trials, and the unfamiliar terminology in which they are often described, it can sometimes be difficult for patients to determine which trial might be right for them. You can speak with your oncologist or view some common questions and answers about clinical trials.
Patients with blood cancers such as leukemia or lymphoma now have added opportunities to participate in clinical trials, nationwide. Learn more.
Watch a video to understand the risks and benefits of being in a clinical trial. See a list of clinical trials available at Dana-Farber. See a list of clinical trials available to cancer patients nationwide.
How do I know if I am eligible for a clinical trial?
Because trials are designed to answer specific questions — what percentage of patients benefit from a new therapy? which patients are most likely to benefit? what are the most common side effects and how severe are they? — they tend to be open to specific sets of patients.
Each trial has detailed requirements for participation, referred to as eligibility criteria. Eligibility is based on the type or subtype of cancer a patient has, and may be further limited based on:
- age
- gender
- stage of disease
- co-existing health problems
- the type of number of previous treatments
Before enrolling, prospective participants are thoroughly briefed on the trial — its purpose, the potential benefits and risks of the therapy being tested, the schedule on which it will be administered — by a clinician. A detailed written informed consent is made available to patients for their review and consideration. Enrollment requires signing a formal consent document witnessed by their physician.
As a general rule, trials evaluating new treatments are open to patients who haven’t previously been treated or have already received standard therapy for their disease. Some trials compare a standard regimen already shown to be effective to a newly developed regimen that clinical investigators believe may improve patients’ outcome, a process called randomization.
The National Institutes of Health maintains a website — clinicaltrials.gov —listing more than 300,000 trials in more than 200 countries. The site is searchable by type of disease and lists the eligibility criteria for each trial, along with detailed information on the treatment protocol, where the trial is available, and whom to contact for more information. People with questions about their eligibility for a particular trial can seek guidance from their oncologist. Because new trials are opening constantly, it makes sense to check the website regularly to see if a trial for which you are eligible has recently begun recruiting participants.
How can I find the right clinical trial for me?
At most major cancer centers, oncologists themselves can often direct patients to specific clinical trials open at that site or nearby institutions. At Dana-Farber, a computer application called MatchMiner enables physicians to quickly link patients with clinical trials based on the set of genetic mutations in patients’ tumor cells. Ultimately, the program’s developers hope to make it fully open source and available to patients and clinical trial leaders at other institutions.
Patients can also sort through information on clinical trials themselves. Advocacy groups such as the Leukemia & Lymphoma Society and the American Cancer Society have services that assist patients through cancer treatment. Many of these organizations also host online discussions to share information about clinical trials. Some patients hire patient advocates, also known as patient navigators, to guide them through the health-care system and, if necessary, help identify appropriate clinical trials.
Can patients participate in more than one clinical trial at a time?
As a general rule, patients can enroll in only one therapeutic clinical trial at a time. This is because trials are designed to focus on a specific therapy or treatment regimen. Participation in multiple trials would make it difficult for researchers to identify the risks and benefits of each therapy on its own.
Patients may, however, participate in a tumor-banking trial at the same time as they’re taking part in a trial of a new therapy. A tumor-banking trial involves collecting and storing tumor tissue for later analysis. Such analysis may help determine which tumors are most likely to respond to particular drugs, for example, or which tumors are at greatest risk of metastasizing.
In some cases, patients can participate simultaneously in a therapeutic trial and a lifestyle intervention trial, which studies the effect of changes in diet or exercise. It is important to speak with your doctor and care team about clinical trials you qualify for, and whether you can participate in more than one at a time.
What can be learned from clinical trials that fail?
When a clinical trial shows that a new treatment is no better than the standard, it can be disappointing. But such outcomes yield valuable, potentially lifesaving information.
Trials can “fail” if the experimental therapy doesn’t work better than current treatments. These “negative” outcomes are important for several reasons. They can spare patients false hope or exposure to drugs with no value, and they can save researchers time and money spent pursuing treatments that are ineffective or too toxic.
Some trials have found that standard therapy itself is ineffective. For example, it had long been common practice for women with early-stage breast cancer to have axillary lymph node dissection – removal of lymph nodes near the breast — to prevent the cancer’s spread. This procedure can lead to arm swelling known as lymphedema and other painful side effects. But clinical trial results published in 2011 revealed that women who had had their lymph nodes removed didn’t survive longer than those who didn’t have the procedure. These negative findings quickly led to a 50 percent drop in use of the procedure in such patients, according to a report published in 2016.