IV 

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; 


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•HRES 493 IH 

Whereas rare diseases and conditions cross the medical spectrum and include a broad range of diseases such as 

epidermolysis bullosa, progeria, sickle cell anemia, TaySachs, 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; 


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•HRES 493 IH 

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 

1 Resolved, That the House of Representatives— 

2 (1) supports the designation of ‘‘Rare Disease 

3 Day’’; 

4 (2) recognizes the importance of improving 

5 awareness and encouraging accurate and early diag6 nosis of rare diseases and disorders; and 

7 (3) supports a national and global commitment 

8 to improving access to, and developing new treatVerDate Mar 15 2010 23:42 Feb 27, 2014 Jkt 039200 PO 00000 Frm 00003 Fmt 6652 Sfmt 6201 E:\BILLS\HR493.IH HR493

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•HRES 493 IH 

1 ments, diagnostics, and cures for, rare diseases and 

2 disorders. 

Æ 


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