While laboratory-based preclinical research may help to understand the possible mechanisms of action and safety of a medicine, it cannot substitute studies that show how the drug will interact with the human body.
The phrases “clinical trials” and “clinical research” refer to study on human beings. Researchers design drug studies to investigate specific difficulties with a prospective treatment candidate.
Clinical trials follow a precise timeline, beginning with small-scale Phase 1 studies and ending with large-scale Phase 3 research. If the initial phase of treatment is successful, the therapy progresses to the next phase.
The clinical trial method is successful until the developer files a marketing application to the US Food and medicine Administration (FDA) or a regulatory authority in another country for the medicine to be licenced for doctors to prescribe to patients.
While the phases and designs of clinical trials may differ for certain ailments and specialist treatments, such as cancer medications or gene therapies, below is a basic synopsis of each step for the majority of pharmaceuticals:
Healthy volunteers (people) are often utilised in Phase 1 studies to investigate prospective drug options. Typically, 20 to 80 healthy people participate in Phase 1.
A new medicine candidate’s safety is largely tested in a Phase 1 trial before going on to additional clinical research. A Phase 1 research may offer information on how much of the medicine is present in the blood after administration, how the drug acts in the body, and the side effects associated with greater doses, in addition to safety.
Phase 2 trials include researchers administering the medicine to a larger patient group (typically a few hundred) with the sickness or condition being studied in order to evaluate the treatment’s first effectiveness and further explore its safety. The optimal strategy to distribute a drug candidate in order to optimise possible benefits while minimising risks is to determine the optimum dosage or dosages, which is a major focus of Phase 2 studies.
Phase 3: For disorders that affect a significant number of people, Phase 3 studies typically include 300 to 3,000 volunteers from the patient groups for whom the medicine will eventually be used. In the control group, participants are randomly assigned to either the medicine under research or a placebo (a substance having no therapeutic effect), or to the current standard of care treatment. Researchers conduct phase 3 trials to determine if a medicine candidate gives a therapeutic benefit to a specific population, to provide more detailed safety information, and to serve as the basis for product labelling.
After one or more Phase 3 trials are completed, researchers review the data to see whether the medicine is beneficial and has an acceptable safety profile when used to treat a disease. If this is the case, the company may submit a New Drug Application (NDA), which contains all data and information gathered throughout the process up to and including the results of the Phase 3 clinical trial(s), as well as any extra data required by the appropriate regulatory authorities. To get marketing approval, the NDA is submitted to the FDA for assessment (or analogous applications may be presented to regulatory agencies outside of the US). Because their results typically serve as the basis for approval, phase 3 trials are sometimes referred to as critical studies. If the medication is approved, doctors may suggest it to their patients.