Anyone who has been diagnosed with mesothelioma may be wondering about their options for participating in a clinical trial. Perhaps a health care provider mentioned the possibility of a clinical trial to you, or a friend or family member inquired about your ability to take part in a clinical trial. The term “clinical trial” is used frequently in discussions concerning new research and treatments for cancer patients, but it may not always be clear what different people mean when they talk about clinical trials, or how they can benefit patients.
There are different types of clinical trials, and there are various ways in which clinical trials are regulated and evaluated in the United States. There are four phases that you should know about if you or a loved one has mesothelioma. Ultimately, once you learn more about clinical trials, you can find out about eligibility for clinical trial participation. Not all cancer patients—so not all malignant mesothelioma patients—will be able to participate in every clinical trial. Not only are clinical trials tailored to specific types of patients, but it will also be important for a patient to be able to show that they are healthy enough to take part in the clinical trial.
Learning More About Clinical Research
In order to understand what clinical trials are and how they work, it is critical to learn more about clinical research more broadly. According to the National Institutes of Health (NIH), clinical research is, quite simply, “medical research involving people.” The U.S. National Library of Medicine discusses clinical research through the terminology of “clinical study,” and explains that a “clinical study involves research using human volunteers (also called participants) that is intended to add to medical knowledge.” Within the area of clinical research, there are two types of clinical research that can be undertaken: observational studies and clinical trials (which can also be known as interventional studies).
Observational research, which may also be described as observational studies, involve practices that are distinct from clinical trials. Thus, the U.S. National Library of Medicine uses the opposing language of observational studies versus interventional studies to discuss the differences between observational research and clinical trials.
To be clear, clinical research is the umbrella term, and both observational studies and clinical trials are types of clinical research that are housed by that umbrella term.
Understanding Clinical Trials as a Part of Clinical Research: Observational Studies Versus Clinical Studies
To understand how clinical trials (or interventional studies) are distinct from observational studies, it is important to understand the kinds of practices and methods that are involved in observational studies. The NIH defines observational studies as research in which people are observed “in normal settings.” The NIH clarifies that, in observational studies, “researchers gather information, group volunteers according to broad characteristics, and compare changes over time.” In observational studies, for instance, “researchers may collect data through medical exams, tests, or questionnaires about a group of older adults over time to learn more about the effects of different lifestyles on cognitive health.” Ultimately the information gleaned through an observational study may be used to develop a specific clinical trial.
The U.S. National Library of Medicine explains observational studies in a manner that focuses more on medical terminology that you might hear when you are considering being part of clinical research: “In an observational study, investigators assess health outcomes in groups of participants according to a research plan or protocol. Participants may receive interventions (which can include medical products such as drugs or devices) or procedures as part of their routine medical care, but participants are not assigned specific interventions by the investigator (as in a clinical trial).”
How do clinical trials differ from observational studies? The NIH explains that clinical trials are specific types of research studies “that are aimed at evaluating a medical, surgical, or behavioral intervention.” When a clinical trial is conducted, it is usually the “primary way that researchers find out if a new treatment, like a new drug or diet or medical device (for example, a pacemaker) is safe and effective in people.” Clinical trials are also used with some frequency to determine if a particular type of medical treatment, including for a specific type of cancer, can be effective, and whether it produces side effects that are more or less severe than treatments that are currently in common use. Clinical trials can also be used to assess methods of identifying diseases early on (such as assessing the value or effectiveness of a particular screening tool or method).
Clinical Trials Are Regulated by the U.S. Food and Drug Administration (FDA) and Undergo Approval and Monitoring by Institutional Review Boards (IRBs)
Researchers cannot automatically start a clinical trial when they have an idea for a study or when they want to evaluate a specific type of intervention in mesothelioma patients. Rather, clinical trials are carefully regulated and undergo significant attention and evaluation prior to their approval.
You should know that researchers must receive approval for a clinical trial from the U.S. Food and Drug Administration (FDA). Before the FDA will approve a clinical trial, researchers have to go through a number of steps. As the NIH explains, researchers will first conduct tests in animals to determine if the plan for the clinical trial is potentially safe for human participants. It is only once the clinical trial is safe for humans that the FDA will approve it. Yet FDA approval is not the only process for evaluation and approval.
Before human participants take part in any kind of clinical trial, an institutional review board (IRB) will also be involved in the clinical trial process. As the U.S. National Library of Medicine clarifies, “each federally supported or conducted clinical study and each study of a drug, biological product, or medical device regulated by FDA must be reviewed, approved and monitored by an institutional review board.” What is an IRB? It is “made up of doctors, researchers, and members of the community,” and it is designed to ensure that the human participants in the clinical trial are properly protected and are not put at unnecessary risk.
Four Phases of Clinical Trials
Clinical trials are classified in four different phases, and the phases progress from Phase I to Phase IV. The idea is that, through each phase of the clinical trial process, researchers will be able to better understand the effectiveness and safety of the intervention being tested as part of the clinical trial. During the first three phases of a clinical trial, researchers will use only a specific number of participants, and the participants will be specifically monitored by the researchers. After Phase III, the FDA can approve the intervention—the drug, the treatment, the device, etc.—for clinical use in patients, but it will continue to be monitored by the FDA for both safety and effectiveness. Before the FDA will approve a new mesothelioma or other cancer drug for treatment in a broader patient population, it will usually need to progress from Phase I to Phase II and through Phase III before reaching approval at Phase IV. These are how the four phases of clinical trials progress, according to the NIH:
- Phase I trial: In a Phase I trial, healthy people are usually used to assess the safety of the intervention and any potential harmful side effects. Typically, a Phase I trial will involve anywhere from 20 to 80 participants. In some cases, Phase I trials will also involve mesothelioma or other cancer patients for whom all other treatments and options have been ineffective. In short, in a Phase I trial, researchers are using only a small number of participants to see how the drug or treatment is tolerated by human patients. The focus is on safety in Phase I.
- Phase II trial: In a Phase II trial, researchers will increase the number of participants, usually up to anywhere from 100 to 300 participants. In a Phase II trial, researchers are usually seeking to determine how effective the intervention will be. Accordingly, Phase II trial participants will often include many patients with the type of mesothelioma or other cancer that the clinical trial drug is intended to test. Researchers will be seeking to determine if the drug or treatment may be effective will continuing to assess participant safety. Phase II trials can last quite a long time, sometimes many years.
- Phase III trial: A Phase III trial is designed to gather more information about the intervention (i.e., the drug or treatment) and to assess it within a broader population. At a Phase III trial, researchers may be assessing how the intervention affects various types of participants, as well as how the new drug or treatment being tested compares with existing drugs or treatments. If the results of a Phase III clinical trial are positive, the FDA may then approve the drug or treatment for clinical use in patients.
- Phase IV trial: Phase IV means that the drug or treatment has been approved, and it will continue to be monitored for years in a broad patient population.
You should speak with your health care team about any questions you have concerning clinical trials.