Expressing support for the designation of February 28, 2023, as "Rare Disease Day".

IV 118TH CONGRESS 1ST SESSION H. RES. 181 Expressing support for the designation of February 28, 2023, as ‘‘Rare Disease Day’’. IN THE HOUSE OF REPRESENTATIVES FEBRUARY 28, 2023 Mr. CARSON (for himself, Mr. BACON, Ms. BROWNLEY, Mr. CARTER of Lou- isiana, Mr. FITZPATRICK, Mr. GARCI´A of Illinois, Mr. GRIJALVA, Ms. NORTON, Mr. HUDSON, Ms. JACOBS, Ms. KAMLAGER-DOVE, Mr. MULLIN, Mr. PAYNE, Ms. SEWELL, Mr. SWALWELL, Ms. TLAIB, Mr. TONKO, Mr. BISHOP of Georgia, Mr. KEATING, and Ms. MATSUI) sub- mitted the following resolution; which was referred to the Committee on Energy and Commerce RESOLUTION Expressing support for the designation of February 28, 2023, as ‘‘Rare Disease Day’’. 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 af- fects 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; VerDate Sep 11 2014 01:15 Mar 01, 2023 Jkt 039200 PO 00000 Frm 00001 Fmt 6652 Sfmt 6300 E:\BILLS\HR181.IH HR181 kjohnson on DSK79L0C42PROD with BILLS 2 •HRES 181 IH 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 enact- ment of the Orphan Drug Act (Public Law 97–414; 96 Stat. 2049), a landmark law enabling tremendous ad- vances in the research and treatment of rare diseases and disorders; Whereas, in 2022, the Center for Drug Evaluation and Re- search, 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 sup- port 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 orphan indications for drugs and biological products for the treatment of rare diseases and disorders, more than 90 percent of rare diseases do not have a treatment ap- proved by the FDA for their condition; VerDate Sep 11 2014 01:15 Mar 01, 2023 Jkt 039200 PO 00000 Frm 00002 Fmt 6652 Sfmt 6300 E:\BILLS\HR181.IH HR181 kjohnson on DSK79L0C42PROD with BILLS 3 •HRES 181 IH Whereas limited treatment options and financing life-altering and lifesaving treatments can be challenging for individ- uals with rare diseases or disorders and their families; Whereas rare diseases and disorders include sickle cell ane- mia, spinal muscular atrophy, amyotrophic lateral scle- rosis, thyroid eye disease, myotonic dystrophy, t-cell prolymphocytic leukemia, Sanfilippo syndrome, microtia, meatal atresia, and conductive deafness; Whereas individuals with rare diseases or disorders can expe- rience difficulty in obtaining accurate diagnoses and find- ing 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 condi- tions in Medicaid, and became effective October 1, 2022; Whereas the FDA and the National Institutes of Health sup- port 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 glob- ally for years to come, providing hope and information for rare disease and disorder patients around the world: Now, therefore, be it Resolved, That the House of Representatives— 1 VerDate Sep 11 2014 01:15 Mar 01, 2023 Jkt 039200 PO 00000 Frm 00003 Fmt 6652 Sfmt 6201 E:\BILLS\HR181.IH HR181 kjohnson on DSK79L0C42PROD with BILLS 4 •HRES 181 IH (1) expresses support for the designation of 1 ‘‘Rare Disease Day’’; and 2 (2) recognizes the importance of, with respect 3 to rare diseases and disorders— 4 (A) improving awareness; 5 (B) encouraging accurate and early diag- 6 nosis; and 7 (C) supporting national and global efforts 8 to develop effective treatments, diagnostics, and 9 cures. 10 Æ VerDate Sep 11 2014 01:15 Mar 01, 2023 Jkt 039200 PO 00000 Frm 00004 Fmt 6652 Sfmt 6301 E:\BILLS\HR181.IH HR181 kjohnson on DSK79L0C42PROD with BILLS