Abstract
BackgroundRare rheumatologic diseases are a heterogeneous group of conditions associated with high morbidity. As a whole group, rare rheumatologic diseases afflict millions of people demanding for effective therapies. Therefore, we analyzed the impact of the US Orphan Drug Act on the development of anti-rheumatic orphan drugs.MethodsAnalysis of the FDA database for orphan drug designations.ResultsIn the last three decades, out of 77 orphan drug designations, 14 orphan drug approvals were granted by the FDA for the treatment of rare rheumatologic disorders, i.e. juvenile idiopathic arthritis (N = 5), cryopyrin-associated periodic syndromes (N = 3), uveitis (N = 3), familial Mediterranean fever (N = 1), anti-neutrophil cytoplasmic antibody-associated vasculitis (N = 1), and xerostomia and keratoconjunctivitis sicca in Sjögren’s syndrome (N = 1). Mean time (standard deviation) from designation to approval was 3.9 (2.81) [range 1 … 12] years. Number of FDA-approved small molecules (N = 6, 43 %) and biologics (N = 8, 57 %) was comparable. Almost every fifth (19 %) orphan drug designation was withdrawn. Despite the rarity of conditions, 13/14 pivotal studies were randomized controlled trials.ConclusionsOrphan drug development is challenging: thirty years of US orphan drug act supported the development and FDA approval of 14 orphan drug programs with anti-rheumatic compounds for six rheumatologic diseases.
Highlights
Rare rheumatologic diseases are a heterogeneous group of conditions associated with high morbidity
Rheumatologic diseases are associated with high morbidity leading to reduced quality of life, potentially life-long disability, and premature death
Disease terms with more than one word were searched with the full disease term and with each individual component of the term, e.g., “juvenile idiopathic arthritis” was searched as “juvenile”, “idiopathic”, “arthritis”, and “juvenile idiopathic arthritis”
Summary
Rare rheumatologic diseases are a heterogeneous group of conditions associated with high morbidity. Rare rheumatologic diseases afflict millions of people demanding for effective therapies. We analyzed the impact of the US Orphan Drug Act on the development of anti-rheumatic orphan drugs. Rheumatologic diseases are associated with high morbidity leading to reduced quality of life, potentially life-long disability, and premature death. As a whole group rare rheumatologic disorders afflict a large group of people [1, 2]. The impact of the US orphan drug act on successful drug development for the treatment of rare rheumatologic diseases has not been systematically analyzed. We analyzed how many orphan drugs were designated and subsequently approved by the FDA between 1983 and 2013 to treat rare rheumatologic diseases
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