What is a clinical trial?
A clinical trial is a research study where volunteers receive investigational treatments under the supervision of a physician and research professionals. These treatments are developed by pharmaceutical and biotechnology companies who select qualified physicians, also known as investigators, to conduct clinical trials to determine the benefits of investigational drugs.
Who can participate in a clinical trial?
All clinical trials have guidelines about who can participate. Before joining a clinical trial, volunteers must qualify for the study. These factors are called “inclusion criteria” and the factors that disallow volunteers from participating are called “exclusion criteria.” These criteria can include age, gender, the type and stage of a disease, previous treatment history, and other medical conditions.
Some research studies seek participants with specific illnesses or conditions to be studied in a clinical trial (like PV or MMP opposed to anyone with pemphigus or pemphigoid). It is important to note that inclusion and exclusion criteria are used to identify appropriate participants, promote participants’ safety, and ensure that researchers learn the information they need.
How does a clinical trial work?
In a clinical trial, a volunteer is generally assigned a specific study group. Volunteers in one study group may receive an investigational treatment (the study drug) while other volunteers may receive a placebo (a treatment already available).
A placebo is an inactive product used to assess the experimental treatment’s effectiveness. The participant, physician, and research staff may not know which volunteer receives a placebo and which receives the active treatment. This is called a “blind study.” Not knowing which participants receive the active treatment lets physician and research staff objectively observe the volunteers during the study. Regardless of which treatment volunteers receive, the level of medical attention and care that each receives is the same.
What questions should be asked before choosing to participate?
Patients thinking about participating in a clinical trial should talk about it with their physicians and medical caregivers. Potential volunteers should know and understand the credentials and experience of the staff and the facility involved in conducting the study.
Questions to ask a physician or medical caregiver:
- How long will the trial last?
- Where is the trial being conducted?
- What treatments will be used and how?
- What is the main purpose of the trial?
- How will patient safety be monitored?
- Are there any risks involved?
- What are the possible benefits?
- What are the alternative treatments besides the one being tested in the trial?
- Who is sponsoring the trial?
- Do I have to pay for any part of the trial?
- What happens if I am harmed by the trial?
- Can I opt to remain on this treatment, even after termination of the trial?
What can volunteers expect if they participate?
Sometimes, participants receive a physical examination and their medical histories are reviewed once they are enrolled in the study. The volunteers’ health continues to be monitored during and after the trial. A detailed description of what is expected of volunteers will be outlined in consent forms and specific clinical trial information.
What is informed consent?
For information about the informed consent process when participating in clinical trials, click here.
What are the benefits and risks of joining a trial?
Volunteers in a clinical trial participate in the development of medical therapies that may offer better treatments (and even cures) for P/P and other chronic diseases. However, there are risks involved.
- Play an active role in their own health care.
- Gain access to research treatments before they are widely available.
- Obtain medical care at health care facilities during the trial.
- Help other P/P patients by contributing to medical research.
- There may be unpleasant, serious, or even life-threatening side effects to experimental treatment.
- The experimental treatment may not be effective.
- The protocol may require more time and attention than a non-protocol treatment. This may include trips to the study site, more treatments, hospital stays, or complex dosage requirements.
Volunteers may withdraw from a study at any time for any reason.
Is my personal and medical information confidential and private?
Access to personal information is usually available to the investigator and research team conducting the clinical trial. In some circumstances, the Institutional Review Board (IRB) overseeing the research and the sponsor or contract research organization coordinating the trial may have access to personal information. This is different in each clinical trial, however, it will be specifically stated in the consent form volunteers are asked to sign. As a clinical trial progresses, researchers report the results of the trial (not personal information) at scientific meetings, to medical journals, and to various government agencies.
What happens after the trial?
After a study phase is complete, the data is collected to determine the drug’s effectiveness, if it is safe, and if there are any side effects. Depending on the results, researchers determine whether to stop testing or move to the next phase of study. Researchers decide if the results are medically important and may submit them to journals for peer-review. Data then may be submitted to the Food and Drug Administration (FDA) for approval.
If a drug is approved, pharmaceutical companies may continue to conduct studies that compare the safety, effectiveness, and cost of the new drug to other drugs already on the market. They may also choose to assess a drug’s long-term effectiveness and its impact on the quality of a person’s life.