Whereas rare diseases and disorders are those which affect small patient populations, typically populations smaller than 200,000 individuals in the United States; Whereas nearly 7,000 rare diseases affect nearly 30,000,000 people in the United States and their families; Whereas over half of all rare diseases affect children; Whereas many rare diseases are serious, life-threatening, and lack an effective treatment; Whereas rare diseases and conditions cross the medical spectrum and include a broad range of diseases such as epidermolysis bullosa, progeria, sickle cell anemia, Tay-Sachs, Leigh’s disease, WAGR syndrome, cystic fibrosis, most childhood cancers, fibrodysplasia ossificans pro­gres­siva, Friedreich’s ataxia, and tuberous sclerosis; Whereas people with rare diseases experience challenges that include difficulty in obtaining an accurate diagnosis, limited treatment options, and difficulty finding physicians or treatment centers with expertise in their disease; Whereas more than 450 drugs and biologics have been approved for the treatment of rare diseases according to the Food and Drug Administration, millions of people in the United States have rare diseases for which there is no approved treatment; Whereas challenges to reimbursement for life-altering and often life-saving treatments still exist, such as in reimbursement for medical foods that treat rare metabolic disorders; Whereas great strides have been made in research and treatment for rare diseases as a result of the Orphan Drug Act and amendments made by that Act; Whereas the Food and Drug Administration has taken great strides in involving the patient in the drug review process as part of its Patient-Focused Drug Development program, an initiative that originated in the Food and Drug Administration Safety and Innovation Act; Whereas both the Food and Drug Administration and the National Institutes of Health have established special offices to advocate for rare disease research and treatments