Joint EBE-EuropaBio Postition Paper European Commission Public Consultation Rare Diseases: Europe’s Challenges

14.02.2008

Joint EBE-EuropaBio Postition Paper European Commission Public Consultation Rare Diseases: Europe’s Challenges

Re: Public Consultation – Rare Diseases: Europe’s Challenges

Dear Mr. Montserrat,

Thank you for the opportunity to comment on the European Commission consultation paper “Rare Diseases: Europe’s Challenges”.

EuropaBio and EBE’s joint Taskforce on Orphan Medicinal Products welcomes the initiative of the European Commission to conduct a public consultation on rare diseases. Patients with rare diseases have the same right to treatment and prevention as any other patients. The combined focus in the consultation paper on research, development and market access for Orphan Medicinal Products (OMPs) is also welcomed – as it is the latter that is creating the current bottleneck.

The spirit of the EU Orphan Medicinal Product Regulation has always been to provide timely and equitable access to therapies for patients with rare diseases and to balance risk by providing economic and other incentives to industry to develop such therapies. Patients with rare disorders deserve the same care and the same safety, efficacy and quality of products as patients with more common diseases. But, while access to new orphan medicinal products is discussed in the consultation paper, the companies involved in the orphan drugs field believe that more emphasis should be placed on why access to existing treatments for rare diseases is a problem. Implementation of the OMP Regulation can only be truly successful if patients who need the products have access to them.