IV 113th CONGRESS 2d Session H. RES. 493 IN THE HOUSE OF REPRESENTATIVES February 27, 2014 Mr. Carson of Indiana (for himself, Mr. Crowley , Mr. Lance , Mrs. Christensen , Mr. Pascrell , and Mr. McCaul ) submitted the following resolution; which was referred to the Committee on Energy and Commerce RESOLUTION Expressing support for the designation of February 28, 2014, as Rare Disease Day .

Whereas rare diseases and disorders are those which affect small patient populations, typically populations smaller than 200,000 individuals in the United States; Whereas nearly 7,000 rare diseases affect nearly 30,000,000 people in the United States and their families; Whereas over half of all rare diseases affect children; Whereas many rare diseases are serious, life-threatening, and lack an effective treatment; Whereas one-third of treatments approved by the Food and Drug Administration in 2013 were orphan products intended to treat rare diseases; Whereas rare diseases and conditions cross the medical spectrum and include a broad range of diseases such as epidermolysis bullosa, progeria, sickle cell anemia, Tay-Sachs, cystic fibrosis, most childhood cancers, and fibrodysplasia ossificans progressiva; Whereas people with rare diseases experience challenges that include difficulty in obtaining an accurate diagnosis, limited treatment options, and difficulty finding physicians or treatment centers with expertise in their disease; Whereas the rare disease community made great strides in 2013, including passing the National Pediatric Research Network Act which sets up a network housed within the NIH that facilitates greater collaboration amongst research into pediatric diseases; Whereas challenges to reimbursement for life-altering and often life-saving treatments still exist, such as in reimbursement for medical foods that treat rare metabolic disorders; Whereas great strides have been made in research and treatment for rare diseases as a result of the Orphan Drug Act and amendments made by that Act; Whereas the Food and Drug Administration has taken great strides in involving the patient in the drug review process as part of its Patient-Focused Drug Development program, an initiative that originated in the Food and Drug Administration Safety and Innovation Act; Whereas both the Food and Drug Administration and the National Institutes of Health have established special offices to advocate for rare disease research and treatments; Whereas the National Organization for Rare Disorders, an organization established in 1983 to provide services to, and advocate on behalf of, patients with rare diseases, was a primary force behind the enactment of the Orphan Drug Act and remains a critical public voice for people with rare diseases; Whereas 2013 marked the 30th anniversary of the Orphan Drug Act and the National Organization for Rare Disorders; Whereas the National Organization for Rare Disorders sponsors Rare Disease Day in the United States to increase public awareness of rare diseases; Whereas Rare Disease Day is a global event occurring annually on the last day of February; Whereas Rare Disease Day was observed in the United States for the first time on February 28, 2009; and Whereas Rare Disease Day is anticipated to continue to be observed globally in years to come, providing hope and information for rare disease patients around the world: Now, therefore, be it

That the House of Representatives— (1) supports the designation of Rare Disease Day ; (2) recognizes the importance of improving awareness and encouraging accurate and early diagnosis of rare diseases and disorders; and (3) supports a national and global commitment to improving access to, and developing new treatments, diagnostics, and cures for, rare diseases and disorders.