FDA Takes Steps to Provide Clarity on Developing New Drug Products in the Age of Individualized Medicine

The following is attributed to Patrizia Cavazzoni, M.D., acting director of the Center for Drug Evaluation and Research

Advances in scientific knowledge and drug development technology have provided an opportunity for new approaches to drug development, including the development of drugs for the treatment of rare diseases. These advances have contributed to an increase in development and approval of drugs for the treatment of rare diseases in recent years. In fact, in the past eight years, the U.S. Food and Drug Administration has approved more than twice as many drugs for rare diseases, often referred to as orphan drugs, as in the previous eight years.

For genetic diseases, recent approaches to testing and molecular diagnosis have allowed us to pinpoint, in some cases, the exact cause of a patient’s disease. For a patient with a very rare genetic disease...