By Scott Gottlieb, M.D., Commissioner of Food and Drugs
(FDA)– Scientists have identified nearly 7,000 rare diseases ranging from rare cancers to metabolic disease and in the United States alone, more than 30 million people—or 1 out of every 10 Americans—have at least one rare disease. Tragically, more than half of these people are children.
The United States observes the last day of February as Rare Disease Day to raise awareness among the public and decision-makers about rare diseases and their impact on patients’ lives. Here at FDA, the observance has been expanded to include a full week of activities to reflect on what progress we have made—and what more needs to be done.
Thirty-five years ago, there were few drugs and fewer devices to treat these rare diseases. To spur drug development, Congress enacted the Orphan Drug Act in 1983, which provided financial incentives and other inducements. This was followed in 1990 with legislation establishing a rare disease path to market for medical devices. Since the original Act was passed, we’ve made great progress. Over the last 35 years, drugs and biologics have been developed and approved for 650 rare disease indications, and 72 devices have received approval for orphan indication under the humanitarian device program. In 2017, FDA had its most approvals yet for rare disease indications.
Despite our successes, there are still no treatments for the vast proportion of rare diseases or conditions. FDA is committed to do what we can to stimulate the development of more products by improving the consistency and efficiency of our reviews, streamlining our processes and supporting rare disease research. We have developed a series of new videos and other materials that explain the scope of what FDA is doing to encourage the development of medical products for rare diseases. I invite you to take a look at them.