What is a clinical study?
A clinical study is a research program used to evaluate a new medical treatment or device for two things: 1) safety and 2) effectiveness. Regulatory agencies such as the United States Food and Drug Administration (FDA) and Health Canada require multiple rounds, or Phases, of clinical testing before approving a new treatment for use in humans. A clinical study is a research study involving volunteer human subjects who are assigned to one or more interventions (including placebo or other controls) to evaluate the effects of these interventions on health-related biomedical or behavioral outcomes (i.e. whether or not the intervention affects health or behavior).
What are clinical study phases?
In the U.S., the FDA requires multiple rounds of clinical study testing before approving a new drug or device for human use. These are called clinical study Phases and are numbered (i.e. Phase 1 study, Phase 2 study, etc). Here are the different types clinical study Phases:
- Preclinical testing: all research performed before the clinical study process begins. This includes basic research experiments and early animal testing to assess how the new drug or device works as well as early safety and toxicology testing.
- Phase 1: Screening for safety – Often the first time a drug or device is tested in humans, Phase 1 studies evaluate safety and safe dose levels of a drug in a small population (20-80) of often healthy individuals. Although in clinical studies of cancer treatment, drugs are never tested in healthy volunteers due to their side effects and high toxicity. The outcomes of these studies are often safety measures such as reported side effects.
- Phase 2: Establishing effectiveness – After safety is determined in a small population, Phase 2 studies involve initial tests of whether or not a drug is effective. Testing is performed on a larger group of humans (100-300) , often patients rather than healthy individuals, in order to assess effectiveness and to measure side effects not seen with the small Phase 1 testing population.
- Phase 3: Final confirmation of safety and effectiveness – Phase 3 clinical studies involve large study groups (200-1000) to confirm safety and effectiveness. These studies require comparisons between treatment groups including either placebo or other controls.
Who organizes and oversees clinical studies?
Clinical studies are designed and overseen by a number of different groups. Studies are primarily organized and designed by academic research teams or biopharma companies with oversight from regulatory agencies. In the U.S., the FDA is responsible for approving clinical study design including protocols and endpoints as well as reviewing final data from the study to assess whether or not it was successful. Since clinical studies can involve many people at multiple different locations, the day-to-day details of a clinical study can be outsourced to a Contract Research Organization (CRO). Since a clinical study often involves multiple locations, at each location (often a hospital or other medical facility) there is a Principal Investigator, the doctor or scientist in charge of the individual site, a clinical study coordinator, who heads administrative aspects of the study as well as all of the doctors, nurses and other medical professionals who administer the new therapy or device to patients in the study. Clinical studies are overseen at different levels by all of these parties: the principal investigator oversees the study at a single site, the CRO oversees the study at many sites and the FDA/company/research team is responsible for taking all evidence gathered during the study and assessing whether or not it was effective.
How do I become part of a clinical study?
All clinical studies have pre-defined eligibility criteria for people interested in taking part in the study. These are determined before the study by both the regulatory agencies and the scientists/researchers designing the study. Eligibility criteria often include age ranges, medical history, current medications and other factors. People who meet all eligibility requirements can contact the coordinator of a clinical study at a study site to ask about being screened for inclusion in the study. All active clinical studies, their eligibility requirements and a list of clinical study locations with contact information of study coordinators and principal investigators can be found at the NIH-based website, https://ClinicalTrials.gov/.
For more information about the AGENT study please see: https://clinicaltrials.gov/ct2/show/NCT03750786