Michelle LIPUCCI DI PAOLA
Eurordis (European Organisations for Rare Diseases
Dr. Michele Lipucci Di Paola, scientist in Plant Biology at the University of Pisa (Italy). Since 1997 to 2006 Board Member and Former Vice-President EURORDIS ( European Organisation Rare Diseaes ), since 2002 Vice-President AVLT ( Associazione Veneta Lotta alla Talassemia, Rovigo, Italy) , since 2006 Board Member PBSA ( Pan-European Blood Safety Alliance ), since 2000 Coordinator Research& Development and Director External Relations of THAL-LAB (Laboratory for the Development of Genetic and Pharmacogenomics Therapy of Thalassemia)/Center Biotechnology/University of Ferrara ).
Since 2003 to 2006 Member EMA/WGIP/COMP ( European Medicines Agency/ Working Group Interested Parties/ Committee Orphan Medicinal Products ), since 2009 Alternate Member EMA/CAT ( European Medicines Agency/ Committee Advanced Therapy ) representing patients' organizations.
Role of Patients in EU Centralized Procedure for ATMP ( 2.12 MB)
Patients' organizations strongly contributed to the development of EU Regulations on Orphan Medicinal Product, Pediatric Use and Advanced Therapy Medicinal Products. Since 2000, patients' representatives have been involved as full members in various committee and working parties at the European Medicine Agency, bringing the patients' voice, points of view, expertise and perspectives on regulatory procedures. In addition patients' representatives are involved in issues of post-marketing access to the therapies. With the successful implementation of regulation for ATMP in the EU, patients hope to have early access to the innovative products to treat common and rare diseases. However after one year of experience with ATMP regulation, we need a deep reflection with all stakeholders on the emerging challenges, opportunities and risks.