Orphan Medicines Regulation for rare diseases on the road to success, but better patient access to approved orphan medicines and additional economic incentives in most Member States needed

Brussels, 28 Jun 2006

Today, the European Commission published its final evaluation (1) - following comments received from stakeholders (2) - on five years of the EU's Orphan Medicines Regulation (EC 141/2000). The report confirms the tangible benefits of the Regulation for patients with rare diseases and its contribution to growth and jobs by stimulating increased investment in R & D to meet a high unmet medical need, which in turn has also led to new start-up companies.

The joint industry task force of EuropaBio - the European association for bioindustries - and of EBE - European Biopharmaceutical Enterprises - welcomes the Commission's Report because it increases awareness about rare diseases in the EU and promotes the continuation and further support of the Orphan Medicines Regulation, an important piece of innovative legislation in Europe.

There are some 8000 rare diseases, many are life-threatening and/or seriously debilitating, and for most of them, there is no access to effective medical treatments. Given that 70-80% of these rare diseases have a genetic origin, biotechnology is an important tool to develop treatments for them. Another consequence of this genetic link is that these diseases affect children, who can thus benefit from more than half of the products developed.

So far the Regulation has delivered 22 approved therapies in the EU, and the portfolio of products in the pipeline, which have been designated as orphan medicines, may promise treatments for more than 200 different conditions (3). This Regulation is transforming the public health environment to the benefit of rare disease patients.

The Commission's Report notes that the total cost of approved orphan medicines today in any given country typically accounts for less than 1% of the national healthcare budgets earmarked for medicines, but timely and equitable access for patients to the approved medicines remains an issue. The Report further notes that some member states are limiting patient access by delaying reimbursement. As of December 2004 only one Member State could demonstrate that all approved orphan medicines were reimbursed and available to patients. Eurordis, the umbrella patient organisation for rare disease patients in Europe, has published a report on the issue of access (4).

"The need for equitable and timely access for patients to innovative therapies, coupled with the need for more incentives by Member States to stimulate research, development and marketing of orphan medicines should be combined." says Erik Tambuyzer, Chair of the Joint EuropaBio / EBE Task Force. "By generating a new set of incentives to tackle access issues to benefit patients, Member States would at the same time help the EU move towards the so-called Lisbon knowledge economy targets." The industry is happy with the Comments from the Public Consultation published by the Commission (2), but misses reference to the access issue and to the suggestion above.

According to the Commission report in the absence of policies at Member State level to stimulate private sector research, the 10 years market exclusivity for an orphan medicine in respect of similar products remains the main industry incentive. However, industry is concerned that the delay in reimbursement and late access for patients cuts broadly into the 10 year market exclusivity, eroding the incentive quite a bit.

One year ago, the Joint EuropaBio / EBE Task Force on Orphan Medicines published a White paper (4) putting forward 9 suggestions for the full implementation of the Orphan Medicines framework in Europe. "We call on the Commission and the Member States to look again at these suggestions, and to consider them," says Erik Tambuyzer. "The full implementation of the Orphan Medicines Framework should include Member States resolving the issues around patient access, and a predictable orphan medicines environment that will foster further R&D in the field, with the typical collaboration between researchers, clinicians, industry and patients which is so typical for this field. Industry is taking up its part of this responsibility, and is listening to other stakeholders as well."

ENDS

For more information, contact

EuropaBio - Adeline Farrelly - Tel: +32 2 735 0313 Direct: +32 2 739 1174 Mobile: +32 475 93 17 24 Email: a.farrelly@europabio.org

European Biopharmaceutical Enterprises (EBE) - Emmanuel Chantelot - Executive Manager - Tel: + 32 2 626 25 61 Email: emmanuel@ebe-biopharma.org

EUROPABIO
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