New Directions in Orphan Drugs for Rare Diseases: From Clinical Trials to Regulatory Paths

Friday, October 27th / 8.30-11.00
6 Obeticholic acid in primary biliary cholangitis
Domenico Alvaro
20 Clinical Research of drugs for rare disease: Issues and open questions
Marco Scatigna
49 Ethics Committees and orphan drugs: Role and competence of the Ethical Committee for the human clinical investigations in orphan drugs development: procedures for pre-licencing access to the cure of rare diseases
Domenico Tricarico
53 Innovation in orphan drugs: the regulatory path in Italy
Patrizia Popoli
58 The role of regional coordination networks in patients management and access to therapies
Giuseppina Annicchiarico
98 Orphan drugs: post-marketing surveillance in Italy
Annalisa Capuano
164 Olaratumab in rare soft tissue sarcoma
Andrea Marrari
180 Recombinant paratyroid hormone and hypoparathyroidism
Sabrina Corbetta
181 Role of the cooperation among stakeholders in the development of orphan drugs: the example of inherited rare muscle diseases
Sabata Pierno
185 Stem cells in the treatment of limbal stem cell deficiency
Diego Ardig˛