A clinical trial is designed to answer specific questions about new drugs, medical devices, or new ways of using known treatments. Clinical trials are used to determine whether the new drug or treatment is safe, and whether it works.
Clinical trials consist of four phases: Phase I tests a new treatment on a small group, and concentrates on safety; Phase II deals with safety and efficacy, and expands the study to a larger group of people (several hundred); Phase III expands the study to an even larger group of people (thousands), and is designed to determine conclusively whether or not the treatment is effective; Phase IV takes place after the drug has been licensed, to monitor the drug for long-term effects.
The randomized, double-blind, placebo-controlled (or active-comparator-controlled) trial offers the strongest evidence that a treatment is effective. The number of participants also considerably effects how reliably the trial can determine the effects of a treatment.
Clinical trials must be consistent with good clinical practice (GCP), a rigorous set of guidelines designed to protect the participants’ safety and the integrity of the trial data. The FDA requires pharmaceutical companies and contract research organizations to conduct rigorous clinical trials verifying the safety and efficacy of the new drugs before granting approval for marketing.
The trial objectives and design are usually documented in clinical trial protocols. Once the objectives are determined, case report forms must be carefully designed to gather complete, unambiguous data from the trial.
During the trial, the data management team must continually monitor and verify the data to ensure that they are accurate and consistent. Any missing or inconsistent data must be investigated and corrected.
21 CFR Part 11 Checklist (tool to verify FDA compliance of studies using computer systems to gather data electronically)
Center Watch (resources for investigative sites, including industry news, standard operating procedures, educational books and publications, events, organizations, etc.)