Whereas a rare disease or disorder is one that affects a small number of patients and, in the United States, typically fewer than 200,000 individuals annually are affected by a rare disease or disorder; Whereas, as of February 2017, nearly 7,000 rare diseases affect approximately 30,000,000 people in the United States and their families; Whereas children with rare genetic diseases account for approximately 1/2 of the population affected by rare diseases in the United States; Whereas many rare diseases are serious and life-threatening and lack effective treatments; Whereas, as a result of Federal laws like the Orphan Drug Act ( Public Law 97–414 ; 96 Stat. 2049), there have been important advances made in research on, and treatment for, rare diseases; Whereas the Food and Drug Administration has made great strides in gathering patient perspectives to inform the drug review process as part of the Patient-Focused Drug Development program, an initiative that originated under the Food and Drug Administration Safety and Innovation Act ( Public Law 112–144 ; 126 Stat. 993); Whereas, although nearly 600 drugs and biological products for the treatment of rare diseases have been approved by the Food and Drug Administration, millions of people in the United States have a rare disease for which there is no approved treatment; Whereas lack of access to effective treatments and difficulty in obtaining reimbursement for life-altering, and even life-saving, treatments remain significant challenges for people with rare diseases and their families; Whereas rare diseases and conditions include Von Hippel-Lindau syndrome, fibrous dysplasia, sickle cell anemia, spinal muscular atrophy, Duchenne muscular dystrophy, dermatomyositis, cystic fibrosis, Friedreich's ataxia, many childhood cancers, amyotrophic lateral sclerosis, epidermolysis bullosa, frontotemporal dementia, and metachromatic leukodystrophy; Whereas people with rare diseases experience challenges that include— (1) difficulty in obtaining accurate diagnoses