[Benefit assessment in special situations--rare diseases].
The term rare disease is often used to justify why special study designs and, in particular, randomisation cannot be implemented and therefore cannot be requested. Definitions of rare diseases are not uniform, and absolute numbers of common definitions are large enough to perform clinical trials. Due to smaller patient numbers in common clinical trials patients with rare diseases are subject to a higher risk for imprecise results. Increasing this risk by using inadequate methodological designs is not justified. A compromise regarding the special situation of these patients is acceptable for certain details but not for the main study concept. It should be made clear that patients with rare diseases have the same right to receive adequately tested treatments as all other patients.
Windeler
Jürgen
J
Medizinischer Dienst der Spitzenverbände der Krankenkassen e.V. (MDS), Essen, Germany. j.windeler@mds-ev.de
Lange
Stefan
S
D002986:Clinical Trials as Topic; D006801:Humans; D054990:Idiopathic Pulmonary Fibrosis / Q000628:therapy; D009369:Neoplasms / Q000628:therapy; D009965:Orphan Drug Production; D035583:Rare Diseases / Q000145:classification / Q000188:drug therapy / Q000628:therapy*; D018570:Risk Assessment