A clinical trial is a carefully designed study which tests the benefits and risks of a specific medical treatment or intervention, such as a new drug or a behavior change (e.g., diet). Once researchers have completed a rigorous screening and preclinical testing process, the company files an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA). This application allows the investigational medicine to be tested in human volunteers in clinical trials.
Every clinical trial is led by a principal investigator, who is usually a doctor, along with a team of nurses and others researchers. The FDA requires a multi-phase clinical trials process to be completed before deciding if the medicine under investigation is safe and effective for a broader patient population. Usually, the number of human volunteers in the trial increases as the treatment moves through these phases, which is why innovative medicines cannot be developed without the help of volunteers who participate in clinical trials.
PhRMA member companies are committed to enhancing diverse participation in clinical trials including identifying and addressing potential barriers to enrollment. PhRMA and its member companies are committed to working with the FDA, patient advocacy organizations and other stakeholders across the research ecosystem to collaborate and employ strategies to encourage greater participation in clinical trials. The FDA Reauthorization Act of 2017, Prescription Drug User Fee Act and the 21st Century Cures Act further mandate the incorporation of science-based approaches, including the use of innovative clinical trial approaches and incorporation of patient perspectives into drug development to advance the innovation of clinically meaningful products for patients.
If the results of all required clinical trials phases show that the investigational new drug is safe and effective, the company submits a New Drug Application or Biologics License Application to the FDA. This application includes reams of data from all the stages of testing, and is a request for FDA approval to market the new medicine.
Scientists at the FDA carefully review all the data from all of the studies on the drug under investigation and, after weighing the benefits and risks of the potential medicine, decide whether to grant approval. Occasionally, the FDA will ask for additional research before granting approval or even convene an independent expert panel to consider data presented by the FDA and the company. The panel will then advise the agency on whether to approve the application and under what conditions.