ORIGINAL ARTICLES  TRENDS IN MOLECULAR DIAGNOSIS AND THERAPY OF β-THALASSEMIA AND SICKLE CELL ANEMIA 

Minerva Biotecnologica 2003 June;15(2):161-6

Copyright © 2003 EDIZIONI MINERVA MEDICA

language: English

The orphan medicinal products: an international challenge

Meyers A. ¹, Lipucci di Paola M. ²

¹ National Organization for Rare Disorders (NORD), Danbury, CT, USA; ² European Organization for Rare Disorders (EURORDIS), Paris, France

Here we analyse the American experience on the Orphan Medicinal Products together with the European one. In particular, we considered the procedures for Orphan designation and development, the availability of financial resources and incentive measures for the development, the access and availability of orphan medicines designated and authorized for the market, the role of patients’ representatives, the legislation and health policies. The great challenge in the near future is expected to be the improvement of trans-national cooperation and the building of an active and international community able to act and address the economic and intellectual efforts towards the concrete solutions of the priorities, including information, development, access, availability of therapies for rare diseases over the national borders.