Clinical Trial FAQ

If you are an individual who is interested in learning more about clinical trials, below are some frequently asked questions that may be helpful.

What is a Clinical Trial?

Clinical trials help to answer the question, “Will a treatment help people and is it safe?” In clinical trials, people agree or consent to try new treatments, interventions, or tests, so clinical researchers can determine what is or is not effective. Clinical trials also help researchers and providers decide if the benefits outweigh possible side effects. Lupus research has been greatly advanced by the people who participate in clinical trials.

What is Clinical Research?

Clinical research is the study of human health and illness in people intended to add to medical knowledge. This type of research can be done in the setting of clinical studies that involve human participants or in the context of research that involves human biological samples or experiences. There are two common types of clinical studies: interventional studies and observational studies, but other approaches are done as well. The type of clinical study design will depend on what question the researcher is trying to answer. The study framework is defined in the written description, or clinical study protocol.

Interventional Studies

Interventional studies are studies which test new ways to prevent, detect, or treat disease. In these studies 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.

Observational Studies

Observational studies allow researchers to observe participants or evaluate data that was already collected to examine how characteristics or outcomes may change over time. In these studies, no interventions are given or assigned. Observational studies may help inform the use of a treatment or support a hypothesis to be further evaluated in another clinical trial.

How does a new medication become available to patients?

The development of a new treatment begins many years prior to it being available for use by those living with lupus, often starting in a laboratory. The typical timeframe from discovery in the laboratory until receiving approval for use in people is approximately 12 years.

Once a potential new treatment is ready for testing, it begins the journey through clinical development—Phase 1, Phase 2, Phase 3, and Phase 4 (also known as post-approval). Each phase of clinical development is done to ensure that the treatment is safe and effective. Learn more about the phases of a clinical trial.

Learn more about steps in the drug development process from the Food and Drug Administration

Despite much progress, there are still few approved treatments for lupus with the continued need for further therapies to provide options to those living with lupus. Developing these new treatments for the diverse lupus community takes time and patient involvement in clinical trials is necessary to make any new therapy a reality.

How do participants 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 for patient use 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 participate
The eligibility criteria, which is set of standards a person must meet to participate in the study. These 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
What type of information will be collected
Detailed information about the treatment plan
Background information that describes potential risks and benefits

Clinical trials follow strict 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 care and the lived experience.

All the potential risks must be explained to a participant before they agree to be in a trial. Providers will monitor the health of participants throughout a trial, and a participant can leave a trial at any time for any reason.

How can potential participants learn more about available clinical trials?

Patient advocacy groups like Lupus Therapeutics and the Lupus Research Alliance can offer information about trials and provide resources to help individuals decide if they should participate. Individuals should consult their healthcare provider(s) about their possible involvement, either as part of their care or as another option. Through the Lupus Clinical Investigators Network (LuCIN), an innovative network of clinical trial sites, Lupus Therapeutics helps connect individuals with clinical trials.

Additionally, the NIH has a website you can search to find trials: www.clinicaltrials.gov.

What are the next steps for individuals interested in participating in a clinical trial?

Participants should speak with their provider to see if the study may be right for them. If so, the next step would be a screening process to see if an individual meets 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 a person 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 an individual 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 should an individual ask their 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) suggests 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 are a part of the study?
How might the trial affect daily life?
How long will the trial last?
Who will pay for the tests and treatments? Will there be costs for any part of the trial?
Will there be reimbursement for other expenses (e.g., travel and childcare needed to participate)?
Who will be in charge of an individual’s care during the study? Who should be contacted if there is a problem, question, or concern?
What will happen after the trial?

The Office for Human Research Protections (OHRP), a part of the U.S. Department of Health & Human Services, has resources for people interested in learning more about clinical research.

OHRP also provides a list of questions to ask when deciding whether to participate in research.