Involvement of patient organisations in research and development of orphan drugs for rare diseases in Europe

Molecular Syndromology

Volumn 3, Issue 5, 2012, Pages 237-243

  Mavris, M ^a   Le Cam, Y ^a  

^a Plateforme Maladies Rares   (France)

Author keywords

Patient's organisations; Rare diseases; Research

Indexed keywords

ORPHAN DRUG;

CLINICAL RESEARCH; CONFERENCE PAPER; EUROPE; HUMAN; ORGANIZATION; POLICY; PRIORITY JOURNAL; RARE DISEASE; TECHNOLOGY;

EID: 84870778464     PISSN: 16618769     EISSN: 16618777     Source Type: Journal    
DOI: 10.1159/000342758     Document Type: Conference Paper

References (4)

1. Ayme S, Kole A, Groft S: Empowerment of patients: lessons from the rare disease community. Lancet 371: 2048-2051 (2008).
2. Committee for Orphan Medicinal Products and the European Medicines, Westermark K, Holm BB, Söderholm M, Llinares-Garcia J, Rivière F, et al: European regulation on orphan medicinal products: 10 years of experience and perspectives. Nat Rev Drug Discov 10: 341-349 (2011).
3. Kaye J, Curren L, Anderson N, Edwards K, Ful-lerton SM, et al: From patients to partners: participant-centric initiatives in biomedical research. Nat Rev Genet 13: 371-376 (2012).
4. Polich GR: Rare disease patient groups as clinical researchers. Drug Discov Today 17: 167-172 (2012).