The pharmaceutical industry develops, produces, and markets drugs or pharmaceuticals (also known as conventional therapies) licensed for use as medications. The biopharmaceutical research sector invests tens of billions of dollars each year to support research that advances the boundaries of scientific knowledge and brings new medicines to patients. This research leads to treatments that save lives and improve health for patients around the world.
How Drugs are Developed and Approved
Biopharmaceutical researchers look for a molecule or “lead compound” that may alter the disease course. They may screen compound libraries, develop a molecule from scratch or use some substance from nature as the starting point. After many safety and efficacy tests, they often redesign the most promising compounds to optimize their disease-fighting properties. Often, hundreds of variations are pursued.
The next step is to test the optimized compounds in the laboratory to find the most effective lead with a safety profile that supports initial introduction into humans. Scientists try to determine how a compound works and describe its safety profile. If the compound appears to be safe and effective, the company submits an Investigational New Drug Application to the FDA to seek approval to begin clinical trials.
SOURCE: Pharmaceutical Research and Manufacturers of America, Drug Discovery and Development: Understanding the R&D Process, www.innovation.org.