[Congressional Record Volume 169, Number 38 (Tuesday, February 28, 2023)]
[Senate]
[Page S537]
From the Congressional Record Online through the Government Publishing Office [www.gpo.gov]




SENATE RESOLUTION 84--DESIGNATING FEBRUARY 28, 2023, AS ``RARE DISEASE 
                                 DAY''

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

                               S. Res. 84

       Whereas a rare disease or disorder is a disease or disorder 
     that affects a small number of patients;
       Whereas, in the United States, a rare disease or disorder 
     affects fewer than 200,000 individuals;
       Whereas, as of the date of the adoption of this resolution, 
     more than 25,000,000 individuals in the United States are 
     living with at least 1 of the more than 7,000 known rare 
     diseases or disorders;
       Whereas children with rare diseases or disorders account 
     for a significant portion of the population affected by rare 
     diseases or disorders in the United States;
       Whereas many rare diseases and disorders are serious and 
     life-threatening;
       Whereas this year marks the 40th anniversary of the 
     enactment of the Orphan Drug Act (Public Law 97-414; 96 Stat. 
     2049), a landmark law enabling tremendous advances in the 
     research and treatment of rare diseases and disorders;
       Whereas, in 2022, the Center for Drug Evaluation and 
     Research, in the Food and Drug Administration (referred to in 
     this preamble as ``FDA''), established the Accelerating Rare 
     disease Cures program with a vision of speeding and 
     increasing the development of effective and safe treatment 
     options to address the unmet needs of patients with rare 
     diseases;
       Whereas the 117th Congress passed into law as part of the 
     Consolidated Appropriations Act, 2023 (Public Law 117-328; 
     136 Stat. 4459), provisions creating the rare disease 
     endpoint advancement pilot program in the FDA to support the 
     development of novel efficacy endpoints to help facilitate 
     the development and timely approval of rare disease 
     treatments;
       Whereas, although the FDA has approved more than 1,100 
     drugs and biological products for an orphan indication for 
     the treatment of a rare disease or disorder, approximately 90 
     percent of rare diseases do not have a treatment approved by 
     the FDA for their condition;
       Whereas limited treatment options and financing life-
     altering and lifesaving treatments can be challenging for 
     individuals with rare diseases or disorders and their 
     families;
       Whereas rare diseases and disorders include sickle cell 
     anemia, spinal muscular atrophy, amyotrophic lateral 
     sclerosis, thyroid eye disease, myotonic dystrophy, t-cell 
     prolymphocytic leukemia, Sanfilippo syndrome, microtia, 
     cystinosis, meatal atresia, and conductive deafness;
       Whereas individuals with rare diseases or disorders can 
     experience difficulty in obtaining accurate diagnoses and 
     finding physicians or treatment centers with expertise in 
     their rare disease or disorder;
       Whereas the 116th Congress passed the Medicaid Services 
     Investment and Accountability Act of 2019 (Public Law 116-16; 
     133 Stat. 852), which included provisions for improving 
     access to coordinated, patient-centered health care for 
     children with complex and rare medical conditions in 
     Medicaid, and became effective October 1, 2022;
       Whereas the FDA and the National Institutes of Health 
     support innovative research on the treatment of rare diseases 
     and disorders;
       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 was 
     observed in more than 100 countries in 2022; and
       Whereas Rare Disease Day is expected to be observed 
     globally for years to come, providing hope and information 
     for rare disease and disorder patients around the world: Now, 
     therefore, be it
       Resolved, That the Senate--
       (1) designates February 28, 2023, as ``Rare Disease Day''; 
     and
       (2) recognizes the importance of, with respect to rare 
     diseases and disorders--
       (A) improving awareness;
       (B) encouraging accurate and early diagnosis; and
       (C) supporting national and global efforts to develop 
     effective treatments, diagnostics, and cures.

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