Title: A resolution designating February 28, 2018, as "Rare Disease Day".
Policy area: Health

Summary:
Designates February 28, 2018, as Rare Disease Day.
Expresses support for a national and global commitment to developing new treatments, diagnostics, and cures for rare diseases and disorders.

[Congressional Bills 115th Congress]
[From the U.S. Government Publishing Office]
[S. Res. 423 Agreed to Senate (ATS)]

<DOC>






115th CONGRESS
  2d Session
S. RES. 423

        Designating February 28, 2018, as ``Rare Disease Day''.


_______________________________________________________________________


                   IN THE SENATE OF THE UNITED STATES

                             March 1, 2018

 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

_______________________________________________________________________

                               RESOLUTION


 
        Designating February 28, 2018, as ``Rare Disease Day''.

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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