Monday, October 20, 2014
Medical oncologist Paul Sabbatini demystifies common misconceptions surrounding clinical trials.
In our last blog post about clinical trials, medical oncologist Paul Sabbatini shed light on how phase I trials are conducted at Memorial Sloan Kettering and how to know if you should consider participating in one. In this Q&A, we asked Dr. Sabbatini to demystify common myths and misconceptions surrounding clinical trials.
Myth: Clinical trials are only appropriate for people who have exhausted all of their standard treatment options.
That is one of the most common misconceptions people have about clinical trials. It’s true that we often offer clinical trials when the standard of care hasn’t resulted in clinical benefit, or when there’s no standard of care and the therapies being used aren’t yielding good results. However, it’s 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 we try to improve a standard drug by adding a new agent to it. In this way, it’s possible to receive the standard of care with the hope that the addition of a novel strategy 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.Back to top
Myth: I must give up standard therapy in order to participate in a clinical trial.
Some clinical trials contain standard therapy as a part of the study.
Also, there are a whole host of 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.Back to top
Myth: I won’t have access to the experimental treatment once a clinical trial ends, even if it’s working.
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 was working — would be if toxic side effects or other safety issues developed in other patients requiring us to stop administering the drug.Back to top
Myth: I’ll never know the results of the clinical trial in which I participate.
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’s 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.Back to top
Myth: Investigators conceal evidence when an experimental treatment doesn’t perform as well as expected.
Investigators always publish clinical trial results, whether or not a treatment is found to be safe and effective. It’s important to report negative findings because they tell us 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. We are also now required to report all trial results by posting them on the site within a specific period of time after the trial ends.Back to top
Myth: I will get a placebo instead of actual treatment during a clinical trial.
Our informed consents are very clear, and you would be made aware before enrolling in a clinical trial if there was a possibility that you might receive a placebo. However, the vast majority of our studies 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. For example, we know that adjuvant chemotherapy reduces the risk of disease recurrence in people who’ve had certain treatments for breast cancer. There would never be a trial in which some of these patients would not be given adjuvant chemotherapy in addition to their primary treatment.
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 the study. For instance, there has never been any data to show whether someone who is in remission after treatment for recurrent ovarian cancer should continue to receive chemotherapy. That would be an ideal setting to consider a randomized trial.Back to top
Myth: I must pay out-of-pocket for the cost of receiving treatment in a clinical trial because insurance doesn’t cover experimental therapy.
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.
We don’t bill patients 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.Back to top
Myth: Children with cancer aren’t given the opportunity to participate in clinical trials.
In fact, the majority of our pediatric patients participate in some form of clinical research during their care and, as a result, our pediatric specialists have made some remarkable improvements in the treatment of pediatric cancers over the years. These advances are proof of the impact of successful trials. We need to continue to educate and — when appropriate — encourage adult patients to participate in clinical trials so that we can achieve similar improvements for them.Back to top