Patients Access to Treatment

EU level legislation and initiatives encourage the development of rare disease medicines and foster equitable access across Europe.


Orphan Medicine Regulation

Orphan medicines legislation aims at providing incentives for pharmaceutical companies to develop and market medicinal products to treat rare diseases.


Orphan Medicines Designation

Medicines intended for rare diseases can receive an orphan designation in the European Union and benefit from a number of regulatory incentives.


Committee for Orphan Medicinal Products (COMP)

The Committee for Orphan Medicinal Products (COMP) was created within the European Medicines Agency to review orphan designation applications.


Paediatric Committee (PDCO)

The Paediatric Committee at the European Medicines Agency provides opinions on the development of medicines for children, in accordance with Regulation (EC) 1901/2006.


Committee for Advanced Therapies (CAT)

Advanced-therapy medicines may offer new treatments for many rare diseases. The Committee for Advanced Therapies (CAT) at the European Medicines Agency assesses their quality, safety and efficacy.



EURORDIS promotes medicines safety by gathering crucial information for identifying and reporting adverse effects from medicines


Clinical Added Value of Orphan Medicinal Products Information Flow (CAVOMP-INF)

The CAVOMP-INF considers how to coordinate the respective procedures for marketing authorisation and for reimbursement decisions.


Mechanism of Coordinated Access to orphan medicinal products (MoCA)

The MoCA seeks collaborative ways to identify and assess the added value of orphan medicinal products.


Transparency Directive

Transparency Directive 89/105/EEC is being revised in order to streamline, reduce delays and improve transparency in the Member State decision process for the pricing and reimbursement of medicines.

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