Why Washington Should Support ULTRA Act (H.R. 3737)

It’s great to see some representatives in Washington are finally starting to understand the real healthcare issue facing tens of millions of American’s afflicted with rare diseases. You can’t improve health if potential medications remain trapped in research and biotech’s “Valley of Death” and never reach patients.

This week, U.S. Representatives Cliff Stearns (R-FL) and Ed Towns (D-NY), two senior members of the Energy and Commerce Committee, introduced bipartisan legislation to help spur the development of treatments for very rare diseases. Called the Unlocking Lifesaving Treatments for Rare diseases Act or ULTRA ACT (H.R. 3737), this legislation is designed to improve access to the FDA’s Accelerated Approval process for very rare diseases, bring down drug development costs, and spur investment in the development of treatments that could lead to job creation in the U.S.

Over the last 25 years, only two or three drugs for ultra-rare diseases have been approved each year by the FDA and yet there are close to 7,000 different types of rare diseases that impact 30 million American’s.  At the current rate of approval, it will take 150 years to treat 50% of ultra-rare disorders in the database, according to the Kakkis EveryLife Foundation.  We need action in Washington and we need it now.

The ULTRA Act amends Section 506 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 356) and does not make any changes to the Orphan Drug Act. It’s very simple legislation that could make a huge impact for millions of people.

We need a new strategy and clear the path to create new drugs to treat kids like Addi and Cassi, who suffer from a rare genetic cholesterol disease called Niemann Pick Type C.  Please learn more about the ULTRA Act and help us build Congressional support for this critical legislation and join the list of patient organizations supporting this legislation.