[Congressional Record Volume 164, Number 37 (Thursday, March 1, 2018)]
[Senate]
[Page S1319]
From the Congressional Record Online through the Government Publishing Office [www.gpo.gov]




SENATE RESOLUTION 423--DESIGNATING FEBRUARY 28, 2018, AS ``RARE DISEASE 
                                 DAY''

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

                              S. Res. 423

       Whereas a rare disease or disorder is one that affects a 
     small number of patients, which in the United States is 
     considered to be a population of less than 200,000 
     individuals;
       Whereas, as of February 2018, nearly 7,000 rare diseases 
     affect approximately 30,000,000 people in the United States 
     and their families;
       Whereas children with rare diseases account for about half 
     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 the Orphan Drug Act (Public Law 97-
     414; 96 Stat. 2049), important advances have been made in the 
     research and treatment of 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 more than 600 drugs and biological 
     products have been approved by the Food and Drug 
     Administration for the treatment of rare diseases, 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 still remain significant 
     challenges for people with rare diseases and their families;
       Whereas rare diseases and conditions include aplastic 
     anemia, porphyria, spina bifida, stiff person syndrome, 
     Gaucher disease, diffuse pulmonary lymphangiomatosis, 
     adrenoleukodystrophy, Noonan syndrome, Rett syndrome, Moebius 
     syndrome, Castleman Disease, epidermolytic ichthyosis, and 
     short bowel syndrome;
       Whereas people with rare diseases experience challenges 
     that include--
       (1) difficulty in obtaining an accurate diagnosis;
       (2) limited treatment options; and
       (3) difficulty finding physicians or treatment centers with 
     expertise in rare diseases;
       Whereas the rare disease community gained important new 
     tools during the 115th Congress with the enactment of the FDA 
     Reauthorization Act of 2017 (Public Law 115-52; 131 Stat. 
     1005), which--
       (1) advanced and facilitated the development and timely 
     approval of drugs and biologics for rare diseases, including 
     diseases affecting children;
       (2) reauthorized user fees to help deliver safe and 
     effective treatments to individuals with rare diseases;
       (3) supported the utilization of real-world evidence;
       (4) supported patient-focused drug development; and
       (5) supported the National Evaluation System for Health 
     Technology;
       Whereas both the Food and Drug Administration and the 
     National Institutes of Health have established special 
     offices to support and facilitate rare disease research and 
     treatments;
       Whereas the National Organization for Rare Disorders 
     (referred to as ``NORD'' in this preamble), 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 2018 marks the 35th 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 that was first 
     observed in the United States on February 28, 2009, and 
     observed in more than 94 countries in 2017; 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, 2018, 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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