Whereas a rare disease or disorder is one that affects a small number of patients, which in the United States is considered to be a population of less than 200,000 individuals; Whereas, as of February 2018, nearly 7,000 rare diseases affect approximately 30,000,000 people in the United States and their families; Whereas children with rare diseases account for about half 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 the Orphan Drug Act ( Public Law 97–414 ; 96 Stat. 2049), important advances have been made in the research and treatment of 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 more than 600 drugs and biological products have been approved by the Food and Drug Administration for the treatment of rare diseases, 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 still remain significant challenges for people with rare diseases and their families; Whereas rare diseases and conditions include aplastic anemia, porphyria, spina bifida, stiff person syndrome, Gaucher disease, diffuse pulmonary lymphangiomatosis, adrenoleukodystrophy, Noonan syndrome, Rett syndrome, Moebius syndrome, Castleman Disease, epidermolytic ichthyosis, and short bowel syndrome; Whereas people with rare diseases experience challenges that include— (1) difficulty in obtaining an accurate diagnosis; (2) limited treatment options