[Congressional Record (Bound Edition), Volume 162 (2016), Part 2]
[Senate]
[Pages 2368-2369]
[From the U.S. Government Publishing Office, www.gpo.gov]




SENATE RESOLUTION 380--DESIGNATING FEBRUARY 29, 2016 AS ``RARE DISEASE 
                                 DAY''

  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:

                              S. Res. 380

       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;

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       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
       Resolved, 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.

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