About Lupus Trials
The Lupus Research Alliance developed this brochure in partnership with the Center for Information and Study on Clinical Research Participation (CISCRP) to provide an all-in-one resource to learn about clinical trials.
We also developed this brochure about why participation by African Americans in lupus clinical trials is necessary to understand if potential new therapies are safe and effective among this community. African Americans are disproportionately affected by lupus in both the percentage of people who develop the disease and the severity of its symptoms.
For further information, below are answers to common questions people have about clinical trials.
What is a Clinical Trial?
Clinical trials help to answer the question, “Will this drug help people and is it safe?” In clinical trials, people volunteer to test new treatments, interventions or tests, so researchers can determine what does and doesn’t work. Clinical trials also help researchers and doctors decide if the benefits outweigh possible side effects. Lupus research has been greatly advanced by the caring people who participate in clinical trials. In these studies, scientists and participants work together to find safer, more effective ways to diagnose, prevent, and ultimately cure lupus.
Sometimes people refer to clinical trials as clinical research. Here are some key terms to know as you think about participating in clinical research.
Clinical research is the study of health and illness in people intended to add to our medical knowledge. This type of research is done via clinical studies that involve human volunteers (or participants). There are two main types of clinical studies: interventional studies (also called clinical trials) and observational studies. The type of clinical study design will depend on what question the researcher is trying to answer.
Interventional studies (also known as clinical trials) are studies where researchers test new ways to prevent, detect, or treat disease. These are trials in which participants are assigned to groups that receive one or more intervention/treatment (or no intervention) so researchers can evaluate the effects of the interventions on health outcomes. What treatment each participant receives is determined by the study’s protocol, or the written description of the clinical study, that includes the study’s objectives, design, and methods.
Observational studies, the other main type of clinical study, allow scientists to find an association between things, but they cannot prove that one thing causes another. They can help researchers come up with a hypothesis to later test in a clinical trial.
How does a new medication become available to patients?
Despite much progress, there is still no cure for lupus and a continued need for new treatments. Developing these new therapies takes many years to complete, and patient involvement in clinical trials is necessary to make any new therapy a reality.
The development of a new drug begins many years prior to its being tested in people. The idea for what eventually becomes a clinical trial often starts in the laboratory. The typical timeframe from discovery in the laboratory until receiving approval for use in people is approximately 12 years.
Learn more about steps in the drug development process from the Food and Drug Administration – https://www.fda.gov/forpatients/approvals/drugs/
Once a potential new drug is ready for testing, it goes through many phases of clinical trials to ensure that it is safe and effective. Learn more about the phases of a clinical trial.
How do I know if a clinical trial is safe?
Data collected during clinical trials is used by regulatory agencies such as the U.S. Food and Drug Administration (FDA) to determine whether the drug should be approved and for which illnesses. All trials are governed by well-defined ethical rules and standards to protect safety of people.
These trials follow a specific study plan (known as a protocol), developed by researchers. The protocol outlines key information including:
- The goal of the study
- The number of patients who will take part
- Who is eligible to take part. This is known as the eligibility criteria and can include specific requirements such as persons of a specific age range, gender, type and stage of the disease, previous treatments taken for the disease, and exclusion of other health problems
- What tests patients will get and how often they will get them (a schedule of events)
- What type of information will be collected
- Detailed information about the treatment plan
Clinical trials follow strict scientific standards. These standards protect patients and help produce reliable study results. Clinical trials may find that a new therapy improves patient outcomes, offers no benefit, or causes unexpected harm. All of these results are important as they advance medical knowledge and help to improve patient care.
What are the benefits and risks of participating in a clinical trial?
All clinical trials have benefits and risks, and it is important to understand these when deciding whether to take part in a clinical trial. The risks associated with a study are described in the informed consent document that each potential study participant receives. Participants also can ask the study team any questions about potential risks and benefits.
When deciding whether to participate in a trial, it is important to consider the following:
- You may gain access to new treatments before they are widely available and could be among the first to benefit.
- You will have the support of a team of healthcare providers who understand your disease and will monitor your health closely.
- The chance to play an active role in your health and to get a better understanding of your disease.
- Information gathered can help others and provide additional scientific knowledge about new treatments. It’s important that drugs are tested to make sure they work well in people of different sex, age and race.
- The satisfaction of helping improve lupus treatment for yourself and future generations.
- The new therapy being studied may not be better than the current standard of care.
- The new therapy may not work for you or you may not be in the treatment group and may continue to receive only standard of care.
- The new therapy may have side effects or risks that were not expected; this is especially true during Phase 1 and Phase 2 clinical trials.
- The trial could take more of your time than your usual treatment; you may have more doctor visits and tests.
Why is it important for drugs to be tested in different populations?
Medicines can affect people differently and must be tested among each potential patient group—by race, gender, age, and ethnic background so doctors can know how to use the products safely and effectively. Clinical trial participants should represent the patients that will eventually use the medications; however, this is not always the case. Racial and ethnic minorities are under-represented in clinical trials. For example, while African Americans make up 13% of the U.S. population only 5% of clinical trial participants are African American. Latinos make up 17% of the U.S. population but make up about 7% of participants in clinical trials. It is especially important for people of color with lupus, who tend to have more severe complications, to participate in trials so potential lupus treatments can be tested.
I’m interested in participating in a clinical trial, so what do I do now?
Talk to your doctor to see if the study may be right for you. If so, the next step is to go through a screening process to see if you meet the inclusion and exclusion criteria for enrollment. Every clinical trial has “inclusion” and “exclusion” criteria that determine who is eligible to participate. These criteria are determined before a trial begins enrolling participants.
- Inclusion criteria are characteristics you must have to participate in the trial. They define the characteristics of the population needed to answer the question being tested.
- Exclusion criteria are characteristics you cannot have to participate in the trial. These criteria are characteristics of participants that could interfere with the success of the study or increase their risk for an unfavorable medical occurrence.
Examples of criteria that are sometimes considered include gender, age, lupus complications and/or medications.
What questions to should I ask my healthcare team about clinical trials?
It is important for individuals who are considering participation in a trial to understand what will be involved. The National Institutes of Health (NIH) asking the healthcare team overseeing the study about possible risks and benefits, care during the trial, personal concern, and financial issues. Some of the recommended questions are:
- What is the purpose of the study?
- Who has reviewed and approved the study?
- What are the possible benefits, risks, and side effects?
- What kinds of treatments, procedures, and/or tests will I have during the study?
- How might the trial affect my daily life?
- How long will the trial last?
- Who will pay for the tests and treatments? Will I have to pay for any part of the trial?
- Will there be reimbursement for other expenses (e.g., travel and child care needed to participate)?
- Who will be in charge of “my” care? Who should be contacted if there is a problem, question, or concern?
- What will happen after the trial?
The Office for Human Research Protections, a part of the U.S. Department of Health & Human Services, has resources for people interested in learning more about clinical research –
For more questions to ask when deciding whether to volunteer for research, click here.
To view a series of short videos with basic information about clinical research, click here.