Interest in rare diseases has expanded in recent years, as evidenced by politicians' and health authorities' agendas, yet ultra-rare diseases still receive far too little attention. Although no legal classification of an "ultra-rare" disease has been created, the National Institute for Health and Care Excellence has informally designated this subcategory for medications having indications for disorders with a prevalence of less than one in per 50 000 people. The approach by which ultra-rare disease research should be conducted, as well as the question of quality, are both critical considerations. Although less stringent criteria may be used for orphan pharmaceuticals than for drugs treating more prevalent diseases, this should not be used as an excuse to deny people with rare diseases the best available treatment. When a patient is diagnosed with a rare or ultra-rare condition, having the correct physician and treatment plan is just as vital as having a support system.