Clinical Trials

This is one of the most common questions and misconceptions people have about clinical trials. It is true that oftentimes clinical trials are offered to people when the standard of care hasn’t resulted in clinical benefit, or when there is no standard of care and the therapies being used aren’t yielding good results. However, it is often appropriate to explore clinical trial options earlier in your course of treatment, even when you still have other standard therapies available.

For example, some patients may be eligible for a clinical trial in which a new agent is added to a standard drug in order to improve the standard drug in hope that it will result in greater benefit. Others may be able to try a promising new drug and then go back to standard therapies, which are always available if it wasn’t successful.

Not necessarily. Some clinical trials require standard therapy as a part of the study. Also, there are many nontherapeutic studies that people may not be aware of, many of which can be done while receiving standard therapy.

For example, you may have the opportunity to have your tumor analyzed and archived in a tumor registry, get involved in a study that evaluates data gathered from periodic questionnaires, or participate in a psychosocial trial in which different approaches to counseling are evaluated.

No, you will never receive only a placebo and not the standard of care that is needed for your treatment. At the time of informed consent, it will be made clear if there is a possibility that you might receive a placebo, before enrolling in a clinical trial. However, the vast majority of studies being done are not placebo-controlled trials, and you would know what treatment you’re getting from the very beginning.

In some phase III studies, there is a need to do randomization, in which patients enrolled in the trial are randomly assigned to separate groups that compare different treatments. However, you would never be offered a placebo if there’s a proven therapy of benefit for your particular situation.

A randomized trial does not compare an intervention that’s proven to work with something that might not. Rather, it’s usually offered when there is no standard of care and it would be reasonable for a patient to be placed in either treatment arm of study.

It depends on the therapy being studied, but in many cases trials are set up to continue treatment as long as people are having a beneficial response. Sometimes there’s no evidence that continuing a treatment is better than stopping it, so a trial may call for discontinuation of the drug after a patient receives the prescribed course of treatment.

In general, the only time a drug would be withdrawn from a patient –even if it is working- would be if toxic side effects or other safety issues developed in other patients requiring us to stop administering the drug.

After a clinical trial ends, the data for all participants are reviewed, analyzed, and published in a medical or scientific journal. Results are also posted after a period of time on clinicaltrials.gov, a service of the National Institutes of Health. Everyone has access to the study’s findings either on the website or in the manuscripts.

In most clinical trials, you know right from the beginning what drug you’re going to receive, including the dose and the treatment schedule. However, some trials with two or more treatment groups are blinded, which means participants are not aware of the drug they receive during the course of the study. This is always clearly discussed at the time of informed consent, before you decide whether or not to participate.

Even if a trial provides blinded treatment, it is possible for patients to get their specific results from their treating physician if the type of therapy received in the trial will affect the next choice of treatment.

No. Investigators always publish clinical trial results, whether or not a treatment is found to be safe and effective. It is important to report negative findings because they provide insight on what treatment approaches don’t work and help guide the next steps necessary to move the research forward.

All clinical trials in the United States must be registered and accessible to the public on the clinicaltrials.gov website. It is also now required for investigators to report all trial results by posting them on the site within a specific period of time after the trial ends.

Most people don’t have an increase in the cost of their care by participating in a clinical trial, although there are always exceptions. In general, medical insurance will typically cover charges related to standard-of-care tests and treatments you receive during your participation in a clinical trial, such as blood tests and scans necessary to assess the safety and effectiveness of the investigational therapy.

Patients do not get billed for investigational drugs or for things that are done exclusively for research. The cost section in the informed consent spells out exactly what will be billed to the patient and what won’t.

All clinical trials have participation guidelines. For example, some studies look for volunteers with a specific type of cancer or a certain stage of disease. These factors are called inclusion criteria, in which a person has to meet the requirements on the checklist. Many studies require mandatory screening tests to determine if a person meets all the inclusion criteria.

There are federal rules in place to help ensure the safety and ethics of clinical trials and to protect the rights and welfare of the people who participate in the trials. You will be protected through the informed consent process where the doctor will fully describe the study to you, including the risks. A careful review and approval of the clinical trial protocol will be done by scientific experts, clinical research professionals, and an institutional review board which consists of doctors, health professionals and community members who review studies to protect the rights and welfare of study participants. Also, the Institutional Review Board, Data and Safety Monitoring Boards, the organization sponsoring the trial, and the research team, do ongoing monitoring throughout the duration of the study.

Yes, taking part in a study is completely voluntary and you can leave a study at any time.

Leaving a study early will not result in any penalty or loss of benefits to which you are entitled.

However, if you do decide to stop participation early, talk with your doctor first to ensure your safety as you withdraw from treatments.