Important steps towards new EU action for improving access to orphan medicines for all patients in the EU

How to promote the sustainable development of valuable orphan drugs while ensuring access to these medicines for all affected citizens in the EU? This question has been at the core of the discussions of the Working Group on Pricing of the EU Pharmaceutical Forum since 2007 and throughout 2008. The fact that effective market access and usage vary strongly between and within Member States has been corroborated by different studies, like the Alcimed Study and the EurordisCare Survey series.

The Working Group, which is facilitated by the European Commission and includes representatives from Member States and patient groups, EURORDIS partnering with the European Patient Forum, has worked hard to produce ideas to improve equal access to orphan drugs across the EU. The results of this work can be found in the Guiding Principles recently adopted by the Group. This document is important because it could help Member States ensure timely and equitable access for all EU citizens to more orphan drugs. Moreover, if accepted by the Council of Ministers, discussion on implementation could take place in 2009.

“Overall the outcome was very positive in my view,” says EURORDIS' Chief Executive Officer Yann Le Cam. “The key bottlenecks identified have been agreed, as well as the way forward. The spirit: European collaboration between Member States and with European authorities is reinforced, and access and quality of care remain the main aims.

The two concrete and most important measures are:

• common scientific assessment report for the clinical added value of orphan drugs to be used by Member States to base and speed up their decisions on reimbursement;
• to promote conditional pricing.

Patients need quick and wide access to innovative medicines. Assessment of clinical added value and pricing will be reviewed at regular periods based on additional 'real life data'.

The first job of the Working Group was to identify the main bottlenecks orphan medicines meet on their way to patients. They concluded that important hurdles exist not only in terms of research and development, but also related to pricing and reimbursement practices by companies and by national authorities, as well as assessment of added clinical value and awareness and expertise-building amongst health professionals.

Of these hurdles, the one on pricing and reimbursement seems to be the most sensitive and problematic, since it largely depends on the economic situation of each Member State. It has to be remembered that national authorities are often reluctant to accept reimbursing orphan drugs which are usually very costly, for which there are sometimes extensions of indications and for which there is often life-long need for treatment. Nevertheless, other factors also come into play, such as the priority given to rare diseases and orphan drugs in the health budget, and the extent to which authorities are able to monitor and manage usage, in order to control budgets despite high prices.

This is why some Member States are already organising the monitored use of orphan medicines through dedicated centres of expertise, to which all patients with a specific orphan disease are referred. Therefore, it can be said that European reference networks between centres of expertise are a way of controlling usage, making better use of limited health budgets, and ultimately reducing variation in access across the EU.

“The concept of European collaboration for scientific assessment of the clinical added value of orphan drugs is now accepted!” explains Yann Le Cam. “Once again rare diseases are leading the way and are promoting innovative approaches to build a better Europe for patients.”

For more information:
Guiding Principles >

This article was previously published in the November 2008 issue of our newsletter.

Author: Paloma Tejada
Photo credits: © EURORDIS