Clinical trials are an important step in the development of new drugs, devices and other treatments for bleeding disorders. Trials help ensure that new treatments are safe and effective before therapies or devices are approved for wider use. In a trial, a group of study subjects is treated with the new product under close supervision by medical experts. Involvement by members of the bleeding disorders community in research is crucial to the success of clinical trials. Typically, a member of your healthcare team, such as your hematologist, will alert you to the possibility of joining a trial. But before you agree to participate, there are some important issues to consider.
Informed consent rules developed by the Food and Drug Administration (FDA) require that potential study participants be told how they’ll be treated during the trial, what risks and discomforts they may face, how the trial will work and that their participation is voluntary. Be sure to ask questions about any details you don’t understand. As you weigh whether to participate, get answers to the following:
What is the purpose of the trial?
Ask how the treatment or device being tested is different from what’s available now and how it might benefit you more than your current treatment.
What are the possible risks?
The FDA emphasizes that although participants may benefit from a clinical trial, they may also see no benefit or be exposed to unknown risks. It’s important to understand the possible side effects of the treatment being tested and how those side effects will be handled during the trial.
What will be asked of me during the trial?
Trials often involve tests. Thus it’s important to understand what kind of tests and how many you’ll need to undergo during the trial. Some trials may require participants to stop taking other medications. Ask if you’ll have to stop taking your current factor product? In addition, although some treatments may be done at home or in a doctor’s office, some trials require participants to travel to a specific location for treatment. If you need to go to a hemophilia treatment center (HTC), for example, ask how often—and inquire if transportation assistance will be provided.
Who will be in charge of my care?
Ask if you’ll be able to keep seeing your own doctor. It’s also important to know if the doctors involved in the study will keep your doctor informed about your participation in the trial. Ask if you will be informed about the progress of the trial?
What are the costs of participating in the trial?
Ask if you will have to pay for any parts of the study, who will pay if you are injured during the trial and if insurance may cover any of the costs.
CenterWatch, a resource center for information on clinical trials, offers a sample list of questions patients should ask before agreeing to participate in a study. These include questions about the trial itself, the care patients will receive, the clinical trial procedures (such as withdrawing from the trial) and potential costs to participate.
If you are interested in taking part in a clinical trial, speak with your healthcare team at your hemophilia treatment center. You can also search for trials on ClinicalTrials.gov, a US National Library of Medicine database of publicly and privately funded clinical studies around the world.