What Are Clinical Trials?
Before any new medication, treatment, or medical procedure is approved for use by the American public, the Food and Drug Administration (FDA) requires a series of clinical trials to ensure its safety and effectiveness. Clinical trials take place in a variety of settings, including universities, hospitals, doctors’ offices, and community clinics, and are usually the result of an observational study (the first step in clinical research). Each study includes a principal investigator (usually a medical doctor) who leads a team of additional doctors, nurses, and health care experts.
While many people mistakenly believe clinical trials are private or closed, any eligible patient who meets the conditions of the study may enter. These studies continue to offer new ways to diagnose, treat, and prevent mesothelioma and other cancers in veterans and their families.
The Four Phases
Generally, new mesothelioma treatments require three phases of testing before receiving initial FDA approval for public use. At each stage, a medication’s dosage is reevaluated for effectiveness and the presence/severity of any side effects. If at any point in the study, the medication does not meet the standards for medical efficacy, the FDA can determine the medicine unfit for prescription.
At times, medications go through multiple trials as their formulations and doses are adjusted. In some cases, new drugs or procedures undergo an additional fourth phase of prolonged observation for drug interactions and complications among specific groups (such as pregnant women).
The table below describes the typical progression of clinical trials.
|Phase||# of Participants||Length of Phase||Objective|
|Phase 1||20 to 80||Several months||Test safety, discover side effects, determine dosage (often, on healthy people).|
|Phase 2||100 to 300||Several months to years||70% of drugs reach this phase. Focuses on whether drug is effective in people with certain disease or condition while continuing to test safety.|
|Phase 3||300 to 3,000||1 year to 4 years||33% of drugs reach this phase. Studies drug on different populations, with different dosages, and combinations with other drugs. FDA may approve for experimental drug if results are positive.|
|Phase 4||General population||2+ years||Up to 30% of drugs reach this phase. After approval, surveillance conducted of patients prescribed drug for long-term effects.|
Veterans who were diagnosed with mesothelioma may be eligible for VA compensation. To find out if you qualify, speak with a patient advocate today.
Clinical Trials for Mesothelioma
Although mesothelioma is a rare disease, clinical trials are continually available to patients across the United States. In addition to testing new medications for treatments like chemotherapy and immunotherapy, mesothelioma clinical research studies how to better detect and prevent the disease in healthy patients.
While a clinical trial may attempt to treat your type of mesothelioma, it is important to remember that not every patient is eligible to participate in every trial. Each study has its own standards in order to test the drug under specific conditions. Some experiments focus on healthy participants, while others are open to patients of a specific age or background with a certain diagnosis. In some instances, the principal investigator may invite specific patients to take part in the study.
Trials may include or exclude patients based on factors like:
- Race or ethnicity
- Type of mesothelioma
- Stage of cancer
- Tumor cell type
- Previous treatment history
- Other medical conditions
Currently, immunotherapy medications and gene therapy are among the most common clinical trials being conducted, because their side effects are often more manageable than standard chemotherapy or radiation. In addition to new medications, drugs used to treat other forms of cancer (such as non-small cell lung cancer) are being tested on mesothelioma patients like the combination of atezolizumab, pemetrexed disodium, cisplatin, and surgery with (or without) radiation therapy. These medicines include:
- Nivolumab (Opdivo®)
- Engineered CAR T-cell therapy
- TC-210 gene therapy
- Nintedanib (OFEV®)
- Pevonedistat (NEDD8 inhibitor)
- Avelumab (Bavencio®)
- Ipilimumab (Yervoy®)
Are Clinical Trials Safe?
Clinical trials test new drugs and treatments on human patients, and they do involve a certain amount of risk. However, an institutional review board (IRB) exists to review, approve, and monitor all federally-funded or conducted clinical trials. IRBs consist of doctors, researchers, and community members who have experience with the disease being studied. Members of the board are there to ensure the trial is conducted safely and ethically, to protect the rights and health of patients, and to minimize risk.
Additionally, data-monitoring committees and various government agencies, including the Office of Human Subjects Research Protections and Data and Safety Monitoring Boards (DSMBs), monitor clinical trials. These boards oversee and protect all participants.
Patients who wish to participate in a clinical trial must meet or speak with the lead researcher, who will explain the trial process. They will also sign a document (known as giving your “informed consent”) that indicates their desire to join the study. The form is designed to protect patients from unethical research practices.
Researchers will provide detailed information about the trial, the potential risks and benefits, and treatment alternatives. Signing an informed consent is not a contract and clinical trial participants may withdraw at any time.
Veterans with mesothelioma can take action without affecting their benefits.
What If I’m Not Eligible?
Investigational drugs that are not yet FDA-approved are usually only available to people who have elected to participate in clinical trials, but there are some exceptions. “Compassionate drug use” is an option for patients who live too far away from a trial location, or if some other factor (including age, gender, or disease stage) precludes them from participating.
Though compassionate use is heavily restricted, a mesothelioma specialist will help patients determine if compassionate use is an option for palliative or curative treatment.
Questions to Ask Your Doctor
Patients may ask questions at any time before, during, or after the clinical trial. Below is a list of questions from the National Institutes of Health (NIH):
- What drug or treatment does this trial study?
- Has this treatment been tested previously?
- Why will this particular be effective? Why might it not be effective?
- What treatments will I receive during this trial?
- What determines which treatments I receive?
- Will I know which treatment I receive?
- What will I need to do?
- How long will this clinical trial last?
- What type of tests and procedures are involved in this study?
- Considering the possible risks, benefits, and side effects, how does this trial compare to my current treatment?
- How often should I plan to visit the clinic or hospital?
- Who pays for me to participate?
- Are travel expenses or accommodations reimbursed?
- If the treatment is effective, may I continue using it after the trial is complete?
- Who oversees participant medical care during the trial?
- Is there an option for long-term follow-up care?
- Do I have access to the results?
- If I am injured, what are my options?
- What are my options if I am injured during the study?
Although there are risks, mesothelioma patients may find clinical trials extremely beneficial, especially if their cancer hasn’t responded well to conventional therapies. These studies may also lead to significant breakthroughs in treatment options or diagnostic procedures for future patients.