Involvement of Patient Organisations in Research and Development of Orphan Drugs for Rare Diseases in Europe

Patients' representatives have an increasingly present voice in all aspects of drug development from fundamental research through regulatory processes to health technology assessment. Although major advances have been made in raising awareness and increasing funding for rare diseases, important challenges remain in terms of best use of resources, coordinating efforts and improving policy. This article describes actions taken by rare disease patients' organisations as well as initiatives at the national and European levels to promote research into rare diseases. A survey conducted by EURORDIS (European Organisation for Rare Diseases) on the support (financial and non-financial) provided by patients' organisations in rare disease research is described as well as the involvement of patients' representatives in regulatory processes for medicinal products at the European Medicines Agency. The importance of including patients' groups in fundamental and clinical research as equal partners has become a fact that clearly contributes to the success of an application and the research conducted. Copyright (C) 2012 S. Karger AG, Basel