III 113th CONGRESS 2d Session S. RES. 368 IN THE SENATE OF THE UNITED STATES February 27, 2014 Mr. Brown (for himself, Mr. Barrasso , Mr. Wicker , Mr. Coons , Mr. Bennet , Mr. Whitehouse , Mrs. Feinstein , Mr. Pryor , and Ms. Warren ) submitted the following resolution; which was considered and agreed to RESOLUTION Designating February 28, 2014, as Rare Disease Day .

Whereas a rare disease or disorder is one that affects a small number of patients – in the United States, typically less than 200,000 individuals annually; Whereas as of the date of approval of this resolution, nearly 7,000 rare diseases affect approximately 30,000,000 people in the United States and their families; Whereas children with rare genetic diseases account for more than half of the population affected by rare diseases in the United States; Whereas many rare diseases are serious, life-threatening, and lack an effective treatment; Whereas great strides have been made in research and treatment for rare diseases as a result of the Orphan Drug Act ( Public Law 97–414 ); Whereas the Food and Drug Administration has made 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 ( Public Law 112–144 ); Whereas a third of all treatments approved by the Food and Drug Administration in 2013 were orphan products intended to treat rare diseases; Whereas lack of access to effective treatments and difficulty in obtaining reimbursement for life-altering, and even life-saving, treatments still exist and remain significant challenges for the rare disease community and their families; Whereas rare diseases and conditions include epidermolysis bullosa, progeria, sickle cell anemia, spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD), Tay-Sachs, cystic fibrosis, pulmonary fibrosis, many childhood cancers, and fibrodysplasia ossificans progressiva; Whereas people with rare diseases experience challenges that include difficulty in obtaining accurate diagnoses, limited treatment options, and difficulty finding physicians or treatment centers with expertise in their diseases; Whereas the rare disease community made great strides in 2013, including the passage of the National Pediatric Research Network Act ( Public Law 113–55 ), which calls special attention to rare diseases and directs the National Institutes of Health to facilitate greater collaboration among researchers; 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, 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 observed each year on the last day of February; Whereas Rare Disease Day is a global event, first observed in the United States on February 28, 2009, and observed in 60 countries in 2013; and Whereas Rare Disease Day is expected to be observed globally for years to come, providing hope and information for rare disease patients around the world: Now, therefore, be it

That the Senate— (1) designates February 28, 2014, as 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.