Clinical research is the study of health and illness in people. Clinical research is how we develop new treatments and gather knowledge for better health and care. Clinical research is also how we build the evidence for potential new approaches to find the ones that are safe and effective.
Every day around the world, researchers and scientists are investigating thousands of potential drugs, devices, and procedures. A very small percentage of those under investigation show enough promise to begin testing in humans. A clinical trial is a research study involving human participants, which tests and evaluates the investigational drug, device, or procedure to find out how safe it is and how well it works. Before any investigational drug, device, or procedure can be approved for use, it must be evaluated in a series of clinical trials. Clinical trials are carefully monitored by various regulatory agencies to ensure the safety of all trial participants who agree to take part. Those regulatory agencies include the Food and Drug Administration (FDA) in the United States, the European Medicines Agency (EMA) in Europe, and other Ministries of Health or regulatory authorities in countries around the world. All of them are government agencies that are responsible for issuing rules and regulations in the country of their jurisdiction regarding the conduct of clinical trials.
Clinical trials are divided into 4 different phases. Each phase is designed to collect specific information about the investigational drug, device, or procedure.
Phase 3 trials further test the safety and how well the investigational drug, device, or procedure works in hundreds, or even many thousands, of participants with the target disease being studied. Phase 3 clinical trials often compare the investigational drug, device, or procedure with a placebo (which contains no active medicine) or an existing standard treatment in a randomized fashion. Participants in a randomized trial have a chance to receive the investigational drug, device, or procedure, but also may have a chance to receive the comparator, which could be a placebo (inactive) or the existing standard of care.
A Clinical Trial Protocol is a clinical trial’s blueprint, describing in detail the steps of the trial:
The protocol provides the methods and instructions that investigators need to precisely follow, and details the steps that must be taken to ensure the safety of the trial participants and the integrity of the data collected throughout the trial. The protocol is used to gain ethics approval by local ethics committees (ECs) or institutional review boards (IRBs) for the clinical trial. Local ECs and IRBs are committees of physicians, scientists, statisticians, community members, and others who are not involved in the trial. Local ECs and IRBs review the protocol or trial design to make sure it will not cause unnecessary risk to participants, and review reports of side effects throughout the trial. It is their responsibility to confirm that appropriate steps are taken to protect the rights and welfare of trial participants.
People may be interested in clinical trials for a variety of reasons. Some people participate in clinical trials to contribute to medical research and to help doctors find other ways to help patients. Others participate in clinical trials because they hope to receive investigational drugs, devices, or procedures due to their illness not responding to standard treatment. These participants hope that the investigational drug, device, or procedure will work for them. However, there is no guarantee that the investigational drug, device, or procedure will work, that the participants won’t experience unexpected side effects, or that they won’t receive a comparator drug or placebo. For these reasons, everyone who participates in a clinical trial undergoes trial-related medical exams and tests to monitor their safety.
Yes, there are clinical trials designed specifically to look at investigational drugs, devices, or procedures in children. These trials are called pediatric clinical trials. It is often specified in the protocol title if the patient population is solely children. There are also clinical trials that look at the safety and efficacy of investigational drugs, devices, or procedures in children as well as in adults; the age range of the patient population will be detailed in the trial information.
That depends on the clinical trial. Some clinical trials offer payment, which can vary depending on what is involved and what is expected of the participants. Other trials do not offer payment, but may compensate participants for the time they spend taking part in the trial; generally, this amount is given to cover expenses for parking, transportation, meals, and possibly for lost work time.
Funding for clinical research trials comes from the federal government, such as the National Institutes of Health, and/or private industry, such as pharmaceutical and biotech companies, medical institutions, and foundations.
IQVIA is a contract research organization (CRO), and we work closely with pharmaceutical and biotech companies to get investigational drugs, devices, and procedures through the complexities of clinical development. We are a full-service provider, from strategic design and planning through feasibility and start-up, recruitment, laboratories, and all aspects of worldwide clinical trial execution, providing global, dedicated resources across the Research and Development lifecycle.