Regulatory Focus™ > News Articles > FDA Grant to Support Neglected Orphan Disease Trials

FDA Grant to Support Neglected Orphan Disease Trials

Posted 03 August 2012 | By Alexander Gaffney, RAC 

The US Food and Drug Administration (FDA) is looking to help fund the development of products intended to treat orphan diseases in areas where no treatments currently exist or the existing treatments are inferior.

In a Federal Register post on 3 August, FDA noted the grant money will total $14.1 million, with $4.1 million going toward the development of between five and 10 new awards during the first year and the remaining $10 million supporting continuation awards thereafter.

The grants, administered by FDA's Office of Orphan Product Development (OPD), will be used to support clinical studies on safety or effectiveness that will, "Either result in, or substantially contribute to, market approval of these products."

Phase 1 studies will be eligible for grants of up to $200,000 per year for three years, while Phase 2 and 3 studies may receive up to $400,000 per year for four years. Continued funding beyond the first year will be contingent upon performance factors, compliance with regulations and the availability of continued funding-far from certain with impending budget cuts set to come into effect in January 2013.

All entities-public, private, academic, government, for-profit and nonprofit-will be allowed to apply for the grants, though for-profit entities will be required to exclude profit margins in their request. Further, any entity that uses funds to lobby the government will be disqualified from receiving an award, likely eliminating many larger companies.

Orphan diseases are defined as any condition affecting fewer than 200,000 people in the US. Many diseases have few treatments available to those suffering from the condition, and those without any treatments at all are referred to as "neglected" orphan diseases.

Under the Orphan Drug Act, orphan diseases are eligible for special incentives meant to make the development of treatments financially palatable to pharmaceutical manufacturers and investors, many of whom once shied away from developing drugs due to the low financial returns or possible losses. Under the Act, sponsors may receive financial incentives, expedited reviews for approval and additional years of marketing exclusivity.

Read more:

FDA - Research Project Grants: Clinical Studies of Safety and Effectiveness of Orphan Products


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