Considering a clinical trial? What you should know when making the decision
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If you or someone in your family has been diagnosed with a serious or complex illness, you may have the option of taking part in a clinical trial of a new medication, surgical approach, or medical device. Before you decide to be part of a clinical trial, however, it’s important to understand how clinical trials work and the possible benefits and risks of taking part in one.
What is a clinical trial?
Most people have heard about clinical trials for new treatments for cancer, but there are trials for a much wider range of diseases and conditions. For example, researchers are exploring the safety and effectiveness of a drug called valmorolone (AGAMREER) in people with muscular dystrophy. Another study focuses on a device that delivers mild electrical pulses to treat painful diabetic neuropathy. There are also trials studying new approaches to the prevention of disease and the accuracy of diagnostic tests.
Clinical trials recruit volunteers for research studies to answer specific questions about promising new or experimental drugs, vaccines, and other treatments or diagnostic tools. These trials are required as part of the process of getting Food and Drug Administration (FDA) approval to allow the treatment, test, or device to be used in the United States.
Like any medical treatment or procedure, clinical trials can offer benefits and also pose potential
risks to the people who take part in them. If you have a serious, life-threatening, or rare health problem and none of the standard treatments have improved your condition, clinical trials can provide you with access to new treatments that may offer hope. In addition, when you take part in a clinical trial, you usually receive a higher level of attention from the research team than you might from a medical provider while undergoing standard treatment.
In terms of risks, there is the potential for unforeseen serious side effects and the treatment being investigated may be less effective than the current standard treatment or not effective at all. While it’s not a risk per se, you may receive a placebo as part of some trials, so you would not receive the new treatment that’s being studied.
How do clinical trials work?
Every clinical trial has a protocol—a detailed, written action plan that specifies the objectives and design of the trial and its eligibility criteria (who can take part).
After you volunteer for a trial, the research team will ask you questions and perform tests to determine if you are eligible to participate in the trial. As part of the decision about who is eligible to participate, researchers consider inclusion and exclusion criteria. These criteria can include factors such as the stage of your condition, your family history, whether or not you have a certain gene mutation, and whether you have other health conditions or take medications that may interact or interfere with the treatment that is being tested in the trial.
It’s important to know who will oversee your care during the trial and what their qualifications are, who you can ask questions of during and after the trial, and whether you will be responsible for the cost of any of the required tests or treatments, although in most cases the cost of your care is covered.
The phases of clinical trials explained
Clinical trials follow a strict set of criteria that protect patients. The trials are conducted in a series of steps known as phases.
The four phases of clinical trials are:
- Phase I: This is the first investigation of a treatment in humans. The goal is to gather information on dosage, timing, and the safety of the treatment. The researchers examine how the drug is absorbed and metabolized and whether it is altered by those processes. These trials are usually small and use healthy adults as test subjects.
- Phase II: These trials focus on examining the safety and clinical effectiveness of a treatment. Test subjects are people who have been diagnosed with the disease that the treatment is designed for. This stage of testing also explores short-term side effects of the treatment.
- Phase III: At this point in the clinical trial process, researchers work to evaluate the effectiveness of the new treatment compared to standard treatment for the disease. The treatment is tested in a large number of patients who have been diagnosed with the disease. Participants are randomly assigned to either the investigational group, where they will receive the treatment under study, or a control group, where they will receive the standard treatment or a placebo. The goal is to confirm any positive findings from the smaller Phase II trials. If the results of the Phase III trial are positive and show that the treatment is safe and effective, the researchers compile the data from all three trial phases and send it to the FDA for review with a request for a license for clinical use.
- Phase IV: This phase usually takes place after the treatment has been approved for standard use, with the goal of evaluating the long-term safety and effectiveness of a treatment. It’s less common than the other phases of trials.
Questions to ask when considering a clinical trial
The following questions can be helpful to ask so you can gather the information you need to make an informed decision:
- What is the scientific rationale for conducting the trial?
- What are the objectives?
- In what phase is the trial? How many participants will there be?
- What are the eligibility requirements?
- What is the intervention and what is its duration and schedule?
- Will some participants be part of a control group that receives standard treatment or a placebo instead of the experimental treatment?
- What are the possible risks, side effects, and benefits?
- What medical tests and follow-up tests will participants undergo? How often?
- What are the endpoints (measurable outcomes that indicate an intervention’s effectiveness)?
- Who is sponsoring the trial?
Finding a clinical trial
You can find detailed information about available trials at ClinicalTrials.gov, a registry and results database of publicly and privately supported trials. Other resources include the FDA and the National Cancer Institute.
Connect with PinnacleCare for health support
Before joining a clinical trial, carefully review the protocol with your physician and talk with your PinnacleCare Health Advisor, who can help you understand what the trial entails and what potential risks and benefits it includes. Your PinnacleCare Health Advisor can also be a good resource for the latest information on promising trials that are enrolling patients and can help you decide if a clinical trial is appropriate for your particular health situation.
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