WASHINGTON, DC— U.S. Senators Bob Casey (D-PA), Pat Toomey (R-PA) and Ron Wyden (D-OR) introduced the Preserving Access to Orphan Drugs Act of 2011, bipartisan legislation to ensure that the development of treatments for rare diseases is not jeopardized. This legislation ensures that unnecessary regulatory hurdles are not placed in front of innovative new therapies for rare diseases and conditions.
“With 30 million Americans suffering from a rare disease, it is important to ensure that the development of treatments and cures for these diseases is not inhibited,” Senator Casey said. “This is a responsible step to make certain that current law won’t threaten the development of treatments or jeopardize care.”
“The government should be encouraging the development of treatments for rare diseases, not imposing additional fees on those badly-needed medicines,” Senator Toomey said. “In Pennsylvania, many of our life sciences companies are working on developing orphan drugs, but due to a narrow provision in the Affordable Care Act, some will be hit with additional fees. This bipartisan legislation will rectify this problem, and we hope it will lead to the development of many new treatments.”
“When it comes to therapies for rare diseases, the ‘first past the post’ mentality can be devastating for innovation,” Senator Wyden said. “Incentivizing only the first drug to come to market throws a wet blanket over the creation of newer and better drugs to treat the same disease. Development of these drugs is already at a disadvantage because their very nature implies a large investment to treat a small population. This bill allows manufacturers to continue to search for the next best therapy to help those diagnosed with these rare diseases or conditions by incentivizing innovation.”
The Affordable Care Act created a fee on the sales of brand name drugs. While the legislation intended to exempt drugs targeting rare diseases, or orphan drugs, the Treasury Department issued a narrow interpretation that left some orphan drugs susceptible to the fee. The Preserving Access to Orphan Drugs Act of 2011 corrects this inequity and would exempt all orphan drugs with an FDA indication solely for one or more rare diseases from this unintended fee.