Home > Search Results: Le Cam, Yann
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Articles, 2 records found
Articles 2 records found  
1.
15 p, 1.4 MB Recommendations from the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL) / Annemans, Lieven (Ghent University) ; Aymé, Ségolène (ICM, Paris) ; Le Cam, Yann (EURORDIS-Rare Diseases Europe, Paris) ; Facey, Karen (University of Edinburgh) ; Gunther, Penilla (Swedish Parliament, Stockholm) ; Nicod, Elena (Bocconi University, Milan) ; Reni, Michele (IRCCS Ospedale San Raffaele Scientific Institute, Milan) ; Roux, Jean-Louis (EURORDIS-Rare Diseases Europe, Paris) ; Schlander, Michael (Institute for Innovation & Valuation in Health Care, Wiesbaden, Germany) ; Taylor, David (University College London (UCL)) ; Tomino, Carlo (Clinical Research at IRCSS San Raffaele, Rome) ; Torrent-Farnell, Josep (Universitat Autònoma de Barcelona. Departament de Farmacologia, de Terapèutica i de Toxicologia) ; Upadhyaya, Sheela (NICE, Manchester) ; Hutchings, Adam (Dolon Ltd, London) ; Le Dez, Lugdivine (European government relations and public policy at Celgene, Brussels)
Rare diseases are an important public health issue with high unmet need. The introduction of the EU Regulation on orphan medicinal products (OMP) has been successful in stimulating investment in the research and development of OMPs. [...]
2017 - 10.1186/s13023-017-0601-9
Orphanet Journal of Rare Diseases, Vol. 12 (march 2017)  
Detailed record - Creative Commons
2.
10 p, 1.0 MB The International Rare Diseases Research Consortium : Policies and Guidelines to maximize impact / Lochmüller, Hanns (Newcastle Upon Tyne, UK) ; Torrent i Farnell, Josep (Universitat Autònoma de Barcelona. Departament de Farmacologia, de Terapèutica i de Toxicologia) ; Le Cam, Yann (Paris, France) ; Jonker, Anneliene H. (IRDiRC Scientific Secretariat, Inserm US-14, Paris, France) ; Lau, Lilian P. L. (IRDiRC Scientific Secretariat, Inserm US-14, Paris, France) ; Baynam, Gareth (Western Australian Register of Developmental Anomalies, WA Perth, Australia) ; Kaufmann, Petra (Bethesda, MD USA) ; Dawkins, Hugh J. S. (Perth, WA Australia) ; Lasko, Paul (Montréal, QC Canada) ; Austin, Christopher P. (Bethesda, MD USA) ; Boycott, Kym M. (Ottawa, ON Canada)
The International Rare Diseases Research Consortium (IRDiRC) has agreed on IRDiRC Policies and Guidelines, following extensive deliberations and discussions in 2012 and 2013, as a first step towards improving coordination of research efforts worldwide. [...]
2017 - 10.1038/s41431-017-0008-z
European Journal of Human Genetics, Vol. 25 (november 2017) , p. 1293-1302  
Detailed record - Creative Commons

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