Clinical research is research conducted with Human Participants that are overseen by a Principal Investigator or a Physician Investigator. Clinical research helps doctors and researchers to find new and better ways to understand, detect, control, and treat illness. A clinical research study is a way to find answers to difficult scientific or health questions. For example, the study might explore the best ways to treat Patients with a wide range of health conditions. Clinical research is the main driver of developing new promising therapies.
Clinical Research FAQ
What is clinical research?
What is a protocol?
All clinical studies are based on a set of rules or directions called a protocol. A protocol describes what types of Participants or Patients are eligible to participate in the study. The protocol clearly defines the schedule of tests, procedures, medications, and dosages and sets the length of the study. The Principal Investigator ensures that all study procedures are followed exactly as defined in the protocol.
What is a clinical trial?
According to ClinicalTrials.gov, “in a clinical trial, Participants receive specific interventions according to the research plan or protocol. These interventions may be medical products, such as drugs or devices; procedures; or changes to Participants’ behavior, such as diet.
Clinical trials may compare a new medical approach to a standard one that is already available, to a placebo that contains no active ingredients, or to no intervention. The Physician Investigators in charge to oversee the Study try to determine the safety and efficacy of the intervention by measuring certain outcomes in the Participants. For example, they may give a drug or treatment to Participants who have high blood pressure to see whether their blood pressure decreases.
Clinical trials used in drug development are sometimes described by phase. These phases are defined by the Food and Drug Administration (FDA).
What are clinical trial "phases?"
PHASE 0
Exploratory study involving very limited human exposure to the drug, with no therapeutic or diagnostic goals (for example, screening studies, microdose studies)
PHASE 1
Studies that are usually conducted with healthy volunteers and that emphasize safety. The goal is to find out what the drug's most frequent and serious adverse events are and, often, how the drug is metabolized and excreted.
PHASE 2
Studies that gather preliminary data on whether the drug works in people who have a certain disease or condition. For example, participants receiving the drug may be compared to similar participants receiving a different treatment, usually an inactive substance, called a placebo, or a different drug.
PHASE 3
Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.
PHASE 4
Studies occurring after FDA has approved a drug for marketing. These including postmarket requirement and commitment studies that are required of or agreed to by the study sponsor. These studies gather additional information about a drug's safety, efficacy, or optimal use.
What are "blind" or "masked" studies?
In many clinical trials, one group of Patients will be given an experimental drug or treatment, while a control group is given either a standard treatment for the illness, or a placebo (a harmless “fake” drug), or no treatment at all.
In a “blinded” or “masked” study, Participants do not know whether they are getting the drug being tested, or whether they are in the control group. The goal is to prevent the so-called “placebo effect” from influencing the results of the experiment. The placebo effect is the phenomenon of Patients feeling better simply because they think they are receiving a helpful drug or treatment.
Sometimes, clinical trials are “double-blind” or “double-masked.” That means that neither the Participants, nor the study staff members, know who is receiving the experimental drug and who is in the control group. Studies are performed in this way so that neither the Patients’ nor the doctors’ expectations about the experimental drug can influence the observations and results.
Should I volunteer for clinical research?
Clinical research is a vital part of finding new treatments and cures for diseases. Carefully conducted clinical studies are the fastest way to find treatments that are safe and effective. By volunteering for a clinical study, you would be participating in research that may result in a new treatment for a deadly or debilitating disease.
Before you agree to participate in a study, you must be given complete information about the study, known as “informed consent.” Informed consent involves two essential components: a document and a process. The informed consent document gives a summary of the research project (including the study’s purpose, research procedures, potential benefits and risks, etc.) and explains the individual’s rights as a research Participant. This document is part of an informed consent process, which consists of conversations between the research team and the Participant,and may include other supporting material such as study brochures. The informed consent process provides research Participants with ongoing explanations that will help them make informed decisions about whether to begin or continue participating in the research project. The Food and Drug Administration (FDA) provides details about informed consent with the information page: Informed Consent for Clinical Trials [fda.gov].