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FDA sets up rare disease research fund

Will invest $14m across 15 orphan drug projects
FDA headquarters White Oak

The US Food and Drug Administration (FDA) is to support the research of treatment for rare diseases through a new $14m fund.

The money will support 15 research projects that are investigating new ways to tackle rare diseases, defined in the US conditions that affect less than 200,000 people in the country.

The fund will be administered through the Orphan Products Grants Program, part of the Orphan Drug Act.

This Act was passed in the US to stimulate the development of 'orphan drugs' to treat rare conditions - traditionally seen as an unattractive market for pharma companies due to the limited market and complexity of many rare conditions.

Interest has increased substantially in recent years, however, due in part to more attractive regulatory environments in both the US and EU, with respective regulators providing incentives, such as tax benefits, fee reductions and an extended period of marketing exclusivity.

These incentives are encouraging companies to invest more in the area and tap the 30 million people in the US that are thought to be affected by at least one rare disease.

The FDA is also investing directly in the area, and its Orphan Products Grants Program has provided more than $300m to fund more than 530 new clinical studies that have supported the marketing approval of 50 products.

The latest round of grants includes funding for projects investigating treatments for cystic fibrosis; sickle cell anaemia; and the vision loss disorder Stargardt disease.

“The FDA is committed to fostering and encouraging the development of products for rare diseases, most of which have no available or adequate treatments,” said Dr Gayatri Rao, director of the FDA's Office of Orphan Product Development.

“The grants awarded this year support studies in very vulnerable, difficult-to-treat populations who have no available options.”

Article by
Thomas Meek

22nd October 2013

From: Research, Regulatory



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