Developing Innovative New Medicines for Patients
What Are Clinical Trials?
Once researchers have completed a rigorous screening and preclinical testing process, the company files an Investigational New Drug (IND) application with the Food and Drug Administration (FDA) to allow the investigational drug to be tested in human volunteers in clinical trials. A clinical trial is a study that is carefully designed to test the benefits and risks of a specific medical treatment or intervention, such as a new drug or a behavior change (e.g., diet). Every clinical trial is led by a principal investigator, who is usually a medical doctor, and a research team of nurses and others. Because the FDA requires a multi-phase clinical trials process to be completed before deciding if an investigational medicine is safe and effective for a broader patient population, progress in the development of innovative medicines cannot be made without the people who volunteer to participate in clinical trials.
How Are Drugs Discovered?
Developing a new medicine begins with understanding the disease or condition as thoroughly as possible. Basic research provides clues about how to treat diseases and potential ways to target the symptoms or underlying causes. Armed with an idea, researchers work to understand biological targets for a potential medicine. A drug target can be a protein, RNA, DNA or other molecule that is somehow involved in the disease. Researchers conduct studies in cells, tissues and animal models to determine whether the target can be influenced by a medicine. Then they look for a lead compound—a promising molecule that could influence the target and, potentially, become a medicine. This is the first step in the discovery and development process, a process that spans from initial research to the delivery of life-saving or life-enhancing new medicine.
FDA Review and Approval
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, which includes reams of data from all the stages of testing, 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 investigational drug 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 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.