FDA’s Jesse Goodman, chief scientist and deputy commissioner for Science and Public Health, testified before the US Senate Agriculture, Rural Development, FDA, and Related Agencies’ Appropriations Subcommittee last week. He brought good news regarding the agency’s research and technological efforts toward finding treatments for rare and neglected diseases.

Two days before the hearing, on June 21, FDA launched a database to spark rare-disease drug development, and earlier this week, the agency held a public hearing and webcast to solicit input and ideas regarding the development of medical products for rare diseases.

A bit of background—In 1983, the US adopted the Orphan Drug Act, which created financial incentives, including grants, to developers of drugs for rare disease. There are 6000 rare diseases, defined by the Act as diseases that affect fewer than 200,000 people in the United States. According to this definition, approximately 30 million Americans have a rare disease. Neglected diseases, on the other hand, affect impoverished or disenfranchised populations of the developing world. More than 1 billion people around the world are affected by at least one neglected disease such as TB, leprosy, or malaria.

Since the Orphan Drug Act was passed, 357 drugs and biological products have received orphan-designated market approval. The 2007 FDA Amendments Act gave FDA additional authority to award priority review vouchers to orphan drug applicants. And over the past few years, according to Goodman’s testimony, other regulatory bodies have adopted FDA’s orphan-drug regulatory approaches. Today, orphan-drug applicants are able to file one joint application to FDA and the European Medicines Agency for their new products.

And FDA’s efforts don’t stop there. In February of this year, the agency added a new Associate Director for Rare Diseases to its CDER office to complement the work of FDA’s already existing Office of Orphan Products Development (OOPD). This office is overseeing the new rare-disease database, mentioned above, which includes listings of already approved compounds and products that might help treat rare diseases. These compounds have already receivedorphan-status designation and are market-approved for treatment of other rare diseases.

This coming October, the agency plans to cosponsor the first annual Rare Disease Investigator Training Course in collaboration with NIH and the National Organization for Rare Disorders. FDA and NIH have also partnered to complete a study on the US national policy for rare disease research and related medical product regulation. A report is expected in September before the workshop.

Finally, as part of the 2010 Appropriations Act, FDA is establishing two internal committees to examine rare and neglected diseases. The committees will report to Congress in one year and issue guidance on these diseases, according to Goodman’s testimony.

Considering that 30 million Americans have a rare disease and countless more around the world are in the same situation, or worse, FDA seems to be taking positive steps toward medical research and innovation in this area.