Announcements

Statement of Support for “The Precision Medicine Answers for Kids Today Act”

Rare diseases affect about 1 in 10 Americans (around 30 million people). More than half of those individuals are children, and a third of those children will die by the age of 5. Tragically, it typically takes rare disease patients years to receive an accurate diagnosis, which can result in physical, emotional, and financial hardship for patients and their families.

Timely and sustainable access to diagnostic testing that rapidly informs appropriate patient care and treatments is fundamental to rare disease patients’ health and well-being.¹ Unfortunately, genetic and genomic sequencing tests are out of reach for thousands of rare disease families. Barriers, such as obtaining health insurance and lack of access to expert clinicians, extend the diagnostic odyssey for children and families with suspected rare diseases. The EveryLife Foundation National Economic Burden of Rare Disease Study in the U.S.² found that the average rare disease diagnosis spans more than six years and includes visits to more than 17 healthcare specialists. As a result, many children do not survive long enough to experience the immense benefits of genetic and genomic sequencing tests.

The Precision Medicine Answers for Kids Today Act (H.R. 5989), included within the 21st Century Cures 2.0 Act and led by Reps. Eric Swalwell (D-CA), Scott Peters (D-CA), would advance patients’ access to lifesaving diagnoses, treatments, and cures; it would equip the globally connected community to eliminate certain challenges of rare diseases; and it would help ensure patients have access to all diagnostic testing, and—by extension—the clinical care, therapy, and treatment they require. We, the undersigned, are proud to support this bill, which could improve health outcomes for tens of thousands of families living with a suspected rare disease by bringing many of them closer to a life-altering diagnosis. The Assistance Fund Global Genes Every Life Foundation

¹ Global Genes and Child Neurology Foundation. (2021). Access to Critical Therapies: Guiding Principles of Rare Disease Care and Patient Access. 22 Jan. 2021, https://globalgenes.happyfox.com/kb/article/235-access-to-critical-therapies-guiding-principles-of-rare-disease-care-and-patient-access/

² EveryLife Foundation for Rare Diseases. (2021). The National Economic Burden of Rare Disease Study. 25 Feb. 2021, https://everylifefoundation.org/burden-study/

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