[Congressional Bills 113th Congress]
[From the U.S. Government Publishing Office]
[H. Res. 493 Introduced in House (IH)]

113th CONGRESS
  2d Session
H. RES. 493

Expressing support for the designation of February 28, 2014, as ``Rare 
                             Disease Day''.


_______________________________________________________________________


                    IN THE HOUSE OF REPRESENTATIVES

                           February 27, 2014

   Mr. Carson of Indiana (for himself, Mr. Crowley, Mr. Lance, Mrs. 
  Christensen, Mr. Pascrell, and Mr. McCaul) submitted the following 
 resolution; which was referred to the Committee on Energy and Commerce

_______________________________________________________________________

                               RESOLUTION


 
Expressing support for the designation of February 28, 2014, as ``Rare 
                             Disease Day''.

Whereas rare diseases and disorders are those which affect small patient 
        populations, typically populations smaller than 200,000 individuals in 
        the United States;
Whereas nearly 7,000 rare diseases affect nearly 30,000,000 people in the United 
        States and their families;
Whereas over half of all rare diseases affect children;
Whereas many rare diseases are serious, life-threatening, and lack an effective 
        treatment;
Whereas one-third of treatments approved by the Food and Drug Administration in 
        2013 were orphan products intended to treat rare diseases;
Whereas rare diseases and conditions cross the medical spectrum and include a 
        broad range of diseases such as epidermolysis bullosa, progeria, sickle 
        cell anemia, Tay-Sachs, cystic fibrosis, most childhood cancers, and 
        fibrodysplasia ossificans progressiva;
Whereas people with rare diseases experience challenges that include difficulty 
        in obtaining an accurate diagnosis, limited treatment options, and 
        difficulty finding physicians or treatment centers with expertise in 
        their disease;
Whereas the rare disease community made great strides in 2013, including passing 
        the National Pediatric Research Network Act which sets up a network 
        housed within the NIH that facilitates greater collaboration amongst 
        research into pediatric diseases;
Whereas challenges to reimbursement for life-altering and often life-saving 
        treatments still exist, such as in reimbursement for medical foods that 
        treat rare metabolic disorders;
Whereas great strides have been made in research and treatment for rare diseases 
        as a result of the Orphan Drug Act and amendments made by that Act;
Whereas the Food and Drug Administration has taken great strides in involving 
        the patient in the drug review process as part of its Patient-Focused 
        Drug Development program, an initiative that originated in the Food and 
        Drug Administration Safety and Innovation Act;
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, an organization 
        established in 1983 to provide services to, and advocate on behalf of, 
        patients with rare diseases, was a primary force behind the enactment of 
        the Orphan Drug Act and remains a critical public voice for people with 
        rare diseases;
Whereas 2013 marked the 30th anniversary of the Orphan Drug Act and the National 
        Organization for Rare Disorders;
Whereas the National Organization for Rare Disorders sponsors ``Rare Disease 
        Day'' in the United States to increase public awareness of rare 
        diseases;
Whereas ``Rare Disease Day'' is a global event occurring annually on the last 
        day of February;
Whereas ``Rare Disease Day'' was observed in the United States for the first 
        time on February 28, 2009; and
Whereas ``Rare Disease Day'' is anticipated to continue to be observed globally 
        in years to come, providing hope and information for rare disease 
        patients around the world: Now, therefore, be it
    Resolved, That the House of Representatives--
            (1) supports the designation of ``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.
                                 <all>