[Congressional Record (Bound Edition), Volume 163 (2017), Part 3]
[Senate]
[Pages 3150-3151]
[From the U.S. Government Publishing Office, www.gpo.gov]




SENATE RESOLUTION 73--DESIGNATING FEBRUARY 28, 2017, AS ``RARE DISEASE 
                                 DAY''

  Mr. BROWN (for himself, Mr. Barrasso, Mr. Whitehouse, Ms. Warren, Mr. 
Markey, Mr. Coons, Mr. Wicker, Mr. Van Hollen, Ms. Stabenow, Mrs. 
Feinstein, Ms. Klobuchar, Mr. Hatch, and Mr. Booker) submitted the 
following resolution; which was considered and agreed to:

                               S. Res. 73

       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 February 2017, nearly 7,000 rare diseases 
     affect approximately 30,000,000

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     people in the United States and their families;
       Whereas children with rare genetic diseases account for 
     approximately \1/2\ of the population affected by rare 
     diseases in the United States;
       Whereas many rare diseases are serious and life-threatening 
     and lack effective treatments;
       Whereas, as a result of Federal laws like the Orphan Drug 
     Act (Public Law 97-414; 96 Stat. 2049), there have been 
     important advances made in research on, and treatment for, 
     rare diseases;
       Whereas the Food and Drug Administration has made great 
     strides in gathering patient perspectives to inform the drug 
     review process as part of the Patient-Focused Drug 
     Development program, an initiative that originated under the 
     Food and Drug Administration Safety and Innovation Act 
     (Public Law 112-144; 126 Stat. 993);
       Whereas, although nearly 600 drugs and biological products 
     for the treatment of rare diseases have been approved by the 
     Food and Drug Administration, millions of people in the 
     United States have a rare disease for which there is no 
     approved treatment;
       Whereas lack of access to effective treatments and 
     difficulty in obtaining reimbursement for life-altering, and 
     even life-saving, treatments remain significant challenges 
     for people with rare diseases and their families;
       Whereas rare diseases and conditions include Von Hippel-
     Lindau syndrome, fibrous dysplasia, sickle cell anemia, 
     spinal muscular atrophy, Duchenne muscular dystrophy, 
     dermatomyositis, cystic fibrosis, Friedreich's ataxia, many 
     childhood cancers, amyotrophic lateral sclerosis, 
     epidermolysis bullosa, frontotemporal dementia, and 
     metachromatic leukodystrophy;
       Whereas people with rare diseases experience challenges 
     that include--
       (1) difficulty in obtaining accurate diagnoses;
       (2) limited treatment options; and
       (3) difficulty finding physicians or treatment centers with 
     expertise in the rare diseases;
       Whereas the rare disease community gained important new 
     tools during the 114th Congress with the passage of the 21st 
     Century Cures Act (Public Law 114-255), which--
       (1) streamlines the review by the Commissioner of Food and 
     Drugs of genetically targeted therapies;
       (2) incentivizes the development of rare pediatric disease 
     therapies;
       (3) strengthens pediatric medical research; and
       (4) adds billions of dollars of funding for the National 
     Institutes of Health;
       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 
     (referred to in this preamble 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 2017 marks the 34th anniversary of the enactment of 
     the Orphan Drug Act (Public Law 97-414; 96 Stat. 2049) 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 was observed 
     in more than 85 countries in 2016; 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 28, 2017, 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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