Pediatric rheumatic diseases (PRD) encompass an heterogeneous group of rare diseases, ranging from juvenile idiopathic arthritis (JIA), juvenile systemic lupus erythematosus (JSLE) to juvenile dermatomyositis (JDM) and others. The PRD constitute a small market which is of little interest for pharmaceutical industries. Difficulties in operating in this specific area relate to problems which are peculiar to children, such as specific methods for the assessment of the primary outcome, or paediatric formulations, with the need of setting large networks in order to collect in a reasonable time frame an adequate number of patients.
Ruperto, N. (2010). Needs in paediatric Rheumatology: The PRINTO perspective. PHARMACEUTICALS POLICY AND LAW, 12(1-2), 105-107 [10.3233/PPL-2010-0279].
Needs in paediatric Rheumatology: The PRINTO perspective
Ruperto N
2010
Abstract
Pediatric rheumatic diseases (PRD) encompass an heterogeneous group of rare diseases, ranging from juvenile idiopathic arthritis (JIA), juvenile systemic lupus erythematosus (JSLE) to juvenile dermatomyositis (JDM) and others. The PRD constitute a small market which is of little interest for pharmaceutical industries. Difficulties in operating in this specific area relate to problems which are peculiar to children, such as specific methods for the assessment of the primary outcome, or paediatric formulations, with the need of setting large networks in order to collect in a reasonable time frame an adequate number of patients.
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