Clinical trials offer an opportunity for patients to participate in scientific and industry research. In a clinical trial, participants receive specific interventions according to the research plan or protocol created by the investigators. These interventions may be medical products, such as drugs or devices, procedures, or changes to participants’ behavior, such as diet.
National Institutes of Health PSC Trials
The National Institutes of Health (NIH) offers a web site of clinical trials specific to PSC. You can visit the site by clicking here.
What should a patient consider before enrolling in a clinical study?
Christopher L. Bowlus, MD, University of California Davis, Co-Chair, PSC Partners Scientific/Medical Advisory Committee
Participating in clinical studies is critical to understanding PSC and to developing effective treatments. The opportunities for PSC patients to participate in studies has increased significantly in the last decade and hopefully will continue to increase as we make progress towards improving the lives of PSC patients. Although there are unique challenges to PSC given its rarity and the slow and variable progression of the disease, the general rules and ethics of conducting clinical studies are the same as in any other disease. The following are some general principles to consider before participating in a clinical study.
What does participation require?
Clinical studies can be generally divided into two basic types of studies, observational and interventional. Observational studies may involve the collection of data such as medical records, surveys, and biologic materials such as blood and DNA. The collection may be retrospective (already existing) and/or prospective (to be collected in the future). Likewise, interventional studies involve the collection of data and biologic materials, but also involve treatment with a medication, procedure, or device. Interventional studies may be open-labeled, meaning that everyone knows who is getting treatment and typically do not involve a placebo. Other interventional studies are double-blind. Some participants in these studies receive placebo but neither patients nor doctors know who is getting the placebo versus the drug in order to prevent any bias in the results.
What are the risks of participation?
All clinical studies have risks. Observational studies typically have minimal risk including loss of privacy and if blood is to be drawn, bruising and minor discomfort. Interventional studies involve the same risks as well as additional risks related to the intervention. In most cases, the safety of a medication will have been established either in a small number of patients or possibly in a large number of patients with a different disease. The specific risks of the intervention should be clearly explained and understood prior to agreeing to participate. In addition, there may be procedures required of the study, for example a liver biopsy, which should also be similarly explained.
What are the benefits of participation?
For observational studies, there are generally no direct benefits to the individual participating in the study. The major benefit of participation is related to an altruistic act that may help others. In contrast, there may be a potential benefit in participating in an interventional study if the treatment is found to be effective.
What are the costs of participation?
There should be no costs to you or your insurance for any tests or procedures that are done only for research purposes. If a test or procedure is done as part of routine care but the data is collected for research purposes, then the study will usually not pay for those tests and procedures.
Travel expenses and compensation for a participant’s time may be covered by some research studies.
What information should be provided before participating in a study?
All clinical studies should only be conducted after they receive approval from an independent Institutional Review Board (IRB). A major function of the IRB is to ensure that participation in a clinical study includes a process of informed consent. The informed consent process should include 1) explanation of the study; 2) an opportunity to read the informed consent form that described the study and its risks, benefits, and alternatives; 3) answering all questions about the study; 4) time to consider participation; and 5) signature and dating of the informed consent form. You may also be asked to sign a HIPAA waiver to allow the research team to have access to your medical records for research purposes.
What are your rights? Questions you should ask
How will your health information be kept private?Who will see the information that can identify you? (Name, address, phone number, date of birth)What happens if you decide to leave the trial?
Other questions you may want to ask
- If the study will use a placebo, what are the chances that a participant will receive a placebo versus the study drug?
- How often will you have to go to the hospital or clinic where the clinical trial is being conducted?
- Who will be responsible for your care?
- Will you have to stay in the hospital during the clinical trial?
- If so, how often and for how long? Will you have to travel long distances?
- Will you have check-ups after the trial?
- Who can you speak with about questions you have during and after the trial?
- Who will have access to your medical records?
- Will you be able to take your regular medications while in the clinical trial?
- What will happen at the end of the study?
- How will the doctor know whether the drug is working?
- Will the participant have an opportunity to stay on the drug if it appears to be effective?
- Will participants assigned to placebo have the opportunity to take the drug at the end of the study?
(These questions frequently do not have answers at the start of a study, but are worth asking.)
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