Advice From a Doctor: Choosing Clinical Trials
Daily, more than 1,500 mothers, fathers, sisters, daughters and sons die from a cancer diagnosis in the U.S alone. That is equivalent to five jets carrying 300 passengers crashing every day in our country.
Clinical trials are the cornerstone of progress in disease treatment. As a cancer doctor of more than 15 years who has taken care of thousands of patients, I believe a scientifically sound – and ethically and expertly designed – clinical trial is the gold standard in treatment. While clinical trials are key in cancer research, they’re also important in a range of diseases and conditions.
Selecting the right trial is not an easy process for those involved. It’s certainly not easy for the patient, who is trying to navigate the complicated decision-making process. It’s not easy for many doctors either.
Patients often have questions. Below are a few common inquiries doctors of all specialties find themselves addressing:
1. Why should I sign up to be a “guinea pig”?
Every medicine available today, whether for headaches, heart attacks or cancer, was developed through a clinical trial. Someone like you and me, after weighing the risks and benefits, signed the informed consent to take that medicine in a trial. Now, each one of us and our family members are reaping the benefits of that patient’s willingness to go the extra mile in the name of science.
A scientifically sound and ethically designed clinical trial undergoes many checks and balance to protect the patient. Patient safety, from beginning to end, is key to the decision making for the design of any trial. Therefore, anyone who signs up to participate in a trial can be assured that they are not considered a guinea pig, but rather, part of an exciting new treatment that could change the treatment of that condition forever.
2. Am I going to get just a sugar pill?
In clinical trials, no patient will be given a sugar pill when there is a proven effective treatment. When there is no effective standard treatment for patients in very advanced stages, trials may be designed with placebo only. But, in general, placebo-controlled trials will include a standard treatment option. If there is a placebo, that will be discussed with you up front, and you should ask your doctor about the pros and cons of that approach.
3. Are clinical trials the “last resort”?
In oncology, specifically, there are clinical trials for stage I through stage IV cancers. Since cancer treatment is far from perfect, we’re always looking for opportunities to improve treatment options for every stage of a cancer diagnosis, not just late stage. Many stage I or II cancers can become metastatic stage if we don’t treat or prevent it early on, which is why clinical trials are important for early-stage cancer patients, too. Clinical trials are never a last resort; they’re the gold standard treatment for any stage of cancer.
4. What is the difference between a phase I/II and phase III trial?
Trials are divided into phase I, II and III. There are also phase IV (post-marketing) trials. Phase I trials, in general, focus on a drug’s side effects, how it is metabolized in our body and the optimal dose by itself or in combination with another drug. Phase II trials are designed to find the efficacy of a new drug in a particular disease (lung cancer, breast cancer or lymphoma, for example). Phase III trials are mature trials; by the time a drug reaches phase III, it has already passed phase I and phase II. In phase III trials we are trying to compare the new drug, new approach or new combination to the current standard treatment. If the new drug is found to be better than the standard treatment, it’ll be filed for Food and Drug Administration approval.
5. How do I find the best trial for me?
The first step is to talk to your doctor and find out if there are trials available at the center where you’re being treated. If not, ask about the most exciting development with that particular diagnosis (in patients with BRCA mutation, look for a PARP inhibitor trial; in patients with melanoma, look for an immunotherapy trial). Once you find out the promising treatments for that diagnosis, you can go to ClinicalTrials.Gov to find existing trials in the U.S. At that site, you can chose phase I, II or III trials; your doctor can determine that. It’s OK to get a second opinion from a large center and from an expert in that field.
6. Who will pay for my trials?
That’s an important question. The sponsor of the trial will pay for most trial-related expenses. You should ask the research team that question upfront; they can clarify. Typically, the insurance company covers the standard-care aspect of the clinical trial; however, this can vary among insurance plans and states. We encourage you to inquire, as participating in clinical trials should not cause a patient extra financial burden.
7. What about the unknown side effects?
Phase I trials focus on side effects. Those trials are “first-in-human” trials. However, for any new drug, we have a very good idea of potential side effects before we introduce it to patients. In addition, the clinical trial team – especially the research nurses – will be watching you like a hawk to make sure you’re not experiencing any serious side effects. Once the side effects are noted, various mechanisms are in place to make sure they’re promptly addressed and, if needed, that the dose is modified. Clinical trial teams take side effects very seriously to ensure patients are protected.
8. Am I going to know the results of the trial?
Yes. There are many mechanisms for informing patients; it varies with different trials. If a trial is closed early due to a major benefit from the new treatment, the patients on the control arm may be informed or allowed to cross over to the new treatment. After a trial is completed, sometimes the study team will write individual letters to patients with the results of the study. Your research team can always update you on the progress of the trial. When there’s an exciting result or breakthrough new medicine, and you hear that in the evening news, you can feel proud to have been part of history!
9. How do I know if it’s going to help me?
There is an element of uncertainty in most of the trials. If we know the answers completely, there’s no need to do the trials. The level of uncertainty could vary, depending upon the phase of the trial. Your study team, doctor and research nurse can help you understand the level of uncertainty of the trial.
10. What is consent?
The informed consent process is the most important aspect of participating in a trial, prior to you joining it. Usually the investigator, research nurse or another member of the study team will review the consent in detail with you. You will be given ample time to review the document, ask questions and take notes. This is the time to ask the questions I listed above. All clinical trials are completely voluntary, and without your full informed consent, you will not be entered into a trial.
The thought of participating in a clinical trial can be burdensome for patients, but if they know the questions to ask and have an open mind, it will become clear whether or not to participate. As an oncologist working to advance medical treatment, I encourage patients to consider them, and ultimately make a decision aligned with their situation.
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