IV 118TH CONGRESS 1ST SESSION H. RES. 276 Expressing support for the designation of April 5, 2023, as ‘‘Barth Syndrome Awareness Day’’. IN THE HOUSE OF REPRESENTATIVES APRIL 3, 2023 Mr. TONKO (for himself, Mr. NORMAN, and Mr. BILIRAKIS) submitted the fol- lowing resolution; which was referred to the Committee on Energy and Commerce RESOLUTION Expressing support for the designation of April 5, 2023, as ‘‘Barth Syndrome Awareness Day’’. Whereas Barth syndrome is a rare, life-threatening, genetic disorder which primarily affects males; Whereas Barth syndrome is caused by a mutation in the tafazzin gene (TAFAZZIN, also called G4.5), resulting in an inborn error of phospholipid metabolism, affecting many systems of the body; Whereas Barth syndrome is a multisystem disorder with characteristics including abnormalities of heart and skel- etal muscle, low levels of certain white blood cells called neutrophils that help to fight bacterial infections, and delays in growth; VerDate Sep 11 2014 23:43 Apr 03, 2023 Jkt 039200 PO 00000 Frm 00001 Fmt 6652 Sfmt 6300 E:\BILLS\HR276.IH HR276 kjohnson on DSK79L0C42PROD with BILLS 2 •HRES 276 IH Whereas with probably fewer than 10 new Barth infants identified each year in the United States, the incidence of Barth syndrome is likely only 1 in every 300,000 to 400,000 births and could be as few as 1.5 in 1 million; Whereas globally there are approximately 300 individuals di- agnosed with Barth syndrome, and, in the United States, there are fewer than 150 individuals diagnosed with Barth syndrome; Whereas Barth syndrome can be fatal in childhood due to heart failure or uncontrollable infection, with approxi- mately 50 percent of deaths due to Barth syndrome oc- curring within the first year of life and 85 percent before the fifth year of life; Whereas individuals with Barth syndrome who survive to adulthood often have a severely limited life expectancy; Whereas individuals with Barth syndrome, like many other rare disorders, experience challenges with obtaining a di- agnosis, limited treatment options, and difficulty finding and accessing treatment centers and physicians with ex- pertise in Barth syndrome; Whereas because the disorder affects multiple systems of the body, a patient with Barth syndrome often requires ac- cess to care from experts across a range of medical spe- cialties; Whereas the Barth Syndrome Clinic at Kennedy Krieger In- stitute is the only interdisciplinary clinic dedicated to the diagnosis and treatment of Barth syndrome in the United States; Whereas, to date, there is no treatment for Barth syndrome that is approved by the Food and Drug Administration (FDA); VerDate Sep 11 2014 23:43 Apr 03, 2023 Jkt 039200 PO 00000 Frm 00002 Fmt 6652 Sfmt 6300 E:\BILLS\HR276.IH HR276 kjohnson on DSK79L0C42PROD with BILLS 3 •HRES 276 IH Whereas there is a critical need for research and development to advance treatments for Barth syndrome; Whereas, as a result of the Orphan Drug Act, there have been important advances in research on and treatment for rare diseases, including development efforts in Barth syndrome; Whereas the FDA established the Accelerated Approval Path- way in 1992 and Congress codified the pathway in 2012; Whereas the Accelerated Approval Pathway is an important pathway for rare and ultrarare diseases as it allows for ‘‘earlier approval of drugs that treat serious conditions, and fill an unmet medical need. . .’’; Whereas Congress and the FDA have affirmed the impor- tance of incorporating the patient perspective throughout the drug review process through the FDA’s Patient-Fo- cused Drug Development program; Whereas the Barth Syndrome Foundation, a nonprofit orga- nization established in 2000 to accelerate progress through collaboration between families and scientists, is dedicated to educating, advancing treatments, and find- ing a cure for Barth syndrome; Whereas the Barth Syndrome Foundation sponsors ‘‘Barth Syndrome Awareness Day’’ in the United States to in- crease public awareness and generate additional support for Barth syndrome; and Whereas ‘‘Barth Syndrome Awareness Day’’ is expected to be observed in the United States for years to come, pro- viding hope and information for patients, caregivers, and families around the country: Now, therefore, be it Resolved, That the House of Representatives— 1 VerDate Sep 11 2014 23:43 Apr 03, 2023 Jkt 039200 PO 00000 Frm 00003 Fmt 6652 Sfmt 6201 E:\BILLS\HR276.IH HR276 kjohnson on DSK79L0C42PROD with BILLS 4 •HRES 276 IH (1) supports the designation of ‘‘Barth Syn- 1 drome Awareness Day’’; and 2 (2) recognizes the importance of, with respect 3 to Barth syndrome— 4 (A) improving awareness; 5 (B) encouraging accurate and early diag- 6 nosis; 7 (C) advancing research; 8 (D) developing new treatments, 9 diagnostics, and cures; and 10 (E) identifying regulatory pathways for 11 drug development of ultrarare diseases like 12 Barth syndrome. 13 Æ VerDate Sep 11 2014 23:43 Apr 03, 2023 Jkt 039200 PO 00000 Frm 00004 Fmt 6652 Sfmt 6301 E:\BILLS\HR276.IH HR276 kjohnson on DSK79L0C42PROD with BILLS