III 114th CONGRESS 2d Session S. RES. 380 IN THE SENATE OF THE UNITED STATES February 29, 2016 Mr. Brown (for himself, Mr. Barrasso , Mr. Wicker , Mr. Whitehouse , Ms. Warren , Mr. Coons , and Mr. Hatch ) submitted the following resolution; which was referred to the Committee on the Judiciary RESOLUTION Designating February 29, 2016 as Rare Disease Day .

Whereas a rare disease or disorder is one that affects a small number of patients and, in the United States, typically fewer than 200,000 individuals annually are affected by a rare disease or disorder; 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 about 1/2 of the population affected by rare diseases in the United States; Whereas many rare diseases are serious and life-threatening and lack an effective treatment; Whereas, as a result of the Orphan Drug Act ( Public Law 97–414 ; 96 Stat. 2049), there have been important advances made in the research of and treatment for rare diseases; Whereas the Food and Drug Administration (in this preamble referred to as the FDA ) has made great strides in involving the patient in the drug review process as part of the Patient-Focused Drug Development program, an initiative that originated in the Food and Drug Administration Safety and Innovation Act ( Public Law 112–144 ; 126 Stat. 993); Whereas, although approximately 500 drugs and biological products for the treatment of rare diseases have been approved by the FDA, millions of people in the United States have a rare disease for which there is no such approved treatment; 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 people with rare diseases and their families; Whereas rare diseases and conditions include epidermolysis bullosa, progeria, sickle cell anemia, spinal muscular atrophy, Duchenne muscular dystrophy, Tay-Sachs disease, cystic fibrosis, pulmonary fibrosis, many childhood cancers, fibrodysplasia ossificans progressiva, Smith-Magenis syndrome, Batten disease, and hemophilia; 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 the rare diseases; Whereas the rare disease community made significant progress during the 113th Congress, including the passage of the National Pediatric Research Network Act of 2013 ( Public Law 113–55 ; 127 Stat. 644), which calls special attention to rare diseases and directs the National Institutes of Health (in this preamble referred to as the NIH ) to facilitate greater collaboration among researchers; Whereas the rare disease community continued this progress through the first session of the 114th Congress, including the passage of the Ensuring Access to Clinical Trials Act of 2015 ( Public Law 114–63 ; 129 Stat. 549) and through increased funding for orphan products and rare disease research; Whereas both the FDA and the NIH have established special offices to advocate for rare disease research and treatments; Whereas the National Organization for Rare Disorders (in this preamble referred to as NORD ), a nonprofit 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 2016 marks the 33rd anniversary of the enactment of the Orphan Drug Act and the establishment of NORD; Whereas NORD sponsors Rare Disease Day in the United States and partners with many other major rare disease organizations 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 more than 80 countries in 2015; 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 29, 2016, 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.