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Policymakers Act on Rare Disease Community’s Behalf

Introductory image: Health Professional and Patient Looking at a Tablet in a Hospital Setting

When the federal government is handing out money, it’s all too common for rare diseases to be overlooked since, by definition, they affect far fewer people. But December was a good month for our community. The U.S. Senate and House of Representatives passed bipartisan legislation, along with a spending bill, which both will benefit patients with bone marrow failure disease.

On Dec. 16, Congress finalized the reauthorization of a bill on stem cells sponsored by U.S. Rep. Chris Smith (R-NJ), who also introduced the original legislation in 2005. The Stem Cell Therapeutic and Research Reauthorization Act (H.R. 2820) will continue to fund two key donor networks that support treatment and therapies using adult stem cell lines.

For the next five years, the C.W. Bill Young Cell Transplantation Program will receive $30 million annually, and its companion program, the National Cord Blood Inventory, will receive $23 million per year, enabling more patients to have access to life-saving treatments.

And on Dec. 18, Congress passed the annual omnibus spending bill that appropriates funds to run the government for another year. The bill included a $2 billion boost to fund NIH research programs and an additional $132 million for the FDA, including new money to increase its grants program for orphan drugs, the kind our patients need to treat their rare conditions.