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:

• Committee for Orphan Medicinal Products (COMP) since 2000
• Patients' and Consumers' Working Party (PCWP) since 2006
• Paediatric Committee (PDCO) since 2008
• Committee for Advanced Therapies (CAT) since 2009

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.