Role of EURORDIS

EURORDIS plays an important role in the orphan drug development process through participation in the Committee for Orphan Medicinal Products at the European Medicines Agency.

EURORDIS was instrumental in the development of and advocating for the adoption of the:

• EU Regulation on Orphan Medicinal Products in 1999
• EU Regulation on Paediatric Drugs in 2006
• EU Regulation on Advanced Therapy Medicinal Products in 2007

Orphan drug development

We also play an important role in the orphan drug development process by participating in the Committee for Orphan Medicinal Products (COMP), the Paediatric Committee (PDCO), the Committee for Advanced Therapies (CAT) and the Patients' and Consumers' Working Party (PCWP) at the European Medicines Agency (EMA).

Advocacy for incentives

In the case of orphan drug development, we successfully advocated for the implementation of incentives resulting in:

• Fee waivers relating to the marketing approval process,
• A 2-year extension of market exclusivity for paediatric orphan drugs (12 years instead of 10 for other orphan drugs,
• Parallel EU-US submission of orphan drug designation applications to speed up development and access to new drugs based on a single dossier,
• Creation by the Commission, within the EU Research Framework Programme 7, of a Clinical Research Programme for orphan drugs in support of designated products,
• National incentives such a research grants and tax credits.

We continually advocate for patient access to authorised orphan drugs through:

• Regular surveys assessing and comparing the real situation regarding availability of orphan drugs for patients in the various Member States,
• Promotion of European common policy and criteria for orphan drug access. Read more on the Clinical Added Value of Orphan Drugs (CAVOD)

Quality and consistency of information

In our close collaboration with the EMA, we ensure the quality and consistency of information produced on orphan drugs.

When new drugs receive orphan designation, a Public Summary of the Orphan drug opinion (PSO) is produced and we review opinions prior to their publication on the EMA website.

We also facilitate the review of EPAR summaries (European Public Assessment Reports) by rare disease patient groups. EPAR summaries are produced at the time of marketing authorisation and are documents destined for the general public.

Patient empowerment

EURORDIS identifies and supports rare disease patient representatives for participation in:

• Patients’ representatives involved in EMA scientific committees and working parties
• Protocol assistance
• Scientific Advisory Groups (SAG) at the Committee for Human Medicinal Products
• Other meetings such as discussions on guidelines and risk management programmes

The role played by EURORDIS is financially independent from the pharmaceutical industry. All orphan drug activities are made possible through the work of EURORDIS volunteers and the financial support from its members, AFM-Téléthon and the EMA.