Patient advocates involvement

Patient advocates are actively involved in orphan medicines development and regulatory affairs.

Patient advocates participate in the work of the European Medicines Agency (EMA) as members of the following scientific committees and working party:


Three patients' representatives sit on the Committee for Orphan Medicinal Products (COMP) and the position of Vice-Chair of the COMP has to date always been held by a patients' representative.

Patients' representatives in EMA scientific committees are permanent and full members with equal voting rights.

Rare disease patients are also solicited as experts on the development of products related to their disease e.g. via the process of protocol assistance with the Scientific Advice Working Party (SAWP) and the Scientific Advisory Groups.

Further to their regulatory role, activities of patient representatives (advocates) in the framework of therapeutic development include:

  • Promoting drug development
  • Ensuring clear information to patients
  • Attempting to achieve equal access to treatments both at European and national levels

An annual EURORDIS Summer School aims to support patient representatives at the EMA and train other patient advocates to participate in the drug development process. The Summer School provides an overview of clinical trials, drug development and regulatory procedures.

Our work has enabled rare disease patient representatives to participate in activities related to the orphan drug development process.

Page created: 07/02/2012
Page last updated: 16/01/2018
The voice of rare disease patients in EuropeEURORDIS The international voice of people living with rare diseases, Rare Diseases International is a EURORDIS initiativeRare Disease International Bringing together patients, families and experts to share experiences in a moderated multilanguage forum, RareConnect is a EURORDIS initiative RareConnect The Rare Barometer Programme is a EURORDIS initiative that carries out surveys to transform rare disease patients' experiences into figures and facts that can be shared with decision-makers.Rare Barometer An international awareness raising campaign taking place on the last day of February each year, Rare Disease Day is a EURORDIS initiativeRare Disease Day Join the largest gathering of rare disease stakeholders across Europe, at the biennial European Conference on Rare Diseases and Orphan Products. ECRD is a EURORDIS initiativeEuropean Conference on Rare Diseases