PRESS RELEASE Rare Disease Day 2013 Policy Event

Faster access to medicines for Rare Disease patients “The Transparency Directive” Brussels, 26 February 2013 10:00-14:00 Radisson Blu EU Hotel, Rue d’Idalie 35, Brussels Streamed LIVE on www.eurordis.org/tv 21 February 2013 --- In celebration of Rare Disease Day 2013 and in line with this year’s Rare Disease Day slogan: Rare Disorders without Borders, EURORDIS is co-hosting, together with Members of the European Parliament Antonyia Parvanova and Cristian Silviu Buşoi, a Policy Event in Brussels on 26 February to examine how policy measures can help improve access to therapies for rare disease patients. Enhancing transparency around medicinal product pricing and reimbursement decisions in order to obtain faster, more equitable access to medicines for patients will be discussed in the context of the EU Transparency Directive on medicinal products, in view of the Parliament’s second reading. Co-host of this multi-stakeholder event, Rapporteur on the Transparency Directive Antonyia Parvanova (Environment, Public Health and Food Safety Committee) says, "Updated rules for a fair and transparent process on the pricing and reimbursement of medicines is to benefit all patients, and in particular the ones suffering from rare diseases. Availability and access to treatment is of crucial importance when we talk about rare diseases, and we should keep on upholding this principle throughout the upcoming legislative process. Our goal is to bring more transparency but also to support Member States for an efficient and evidence-based decision making process, which should ultimately support the sustainability of national healthcare systems, delivering for all." "The Commission proposal for a revised Transparency Directive is an important and necessary step towards more efficient decision-making by Member States in pricing and reimbursement (P&R) of medicines. At a time when countries across the EU are seeking improved management of their healthcare systems, it is essential that the procedural aspects of P&R decision-making are brought towards similar standards of quality and efficiency. However, what is not covered, as it is outside the remit of the Commission, is the growing problem of unequal access to new medicines within the EU. New policies are needed to maximise access, while providing support for innovation. Models for differential pricing, based on countries' ability to pay, should be explored,” adds meeting Co-chair Richard Bergström, Director General of the European Federation of Pharmaceutical Industries and Associations (EFPIA). “We are grateful to all the event participants for contributing their experience to the process of improving access to orphan medicinal products in Europe. Their presence is sure to make for a lively debate that will hopefully move forward a faster and more equitable, transparent mechanism for bringing approved rare disease treatments to the market.” says EURORDIS Chief Executive Officer Yann Le Cam.

More about Rare Disease Day Rare Disease Day is held on the last day of February– a rare day for rare people. Each year on Rare Disease Day, hundreds of patient groups and partners organise activities to raise awareness for the

issues facing people affected by a rare disease. 28 February 2013 marks sixth Rare Disease Day. Over 6,000 different rare diseases have been identified to date, and while all together they affect millions of people around the world, when taken separately, each individual disease affects only a very small percentage of the population. This rarity causes problems. Rare Disease Day was created in 2008 and is coordinated by the European Organisation for Rare Diseases (EURORDIS), a non-governmental patient-driven alliance representing more than 550 individual rare disease patient organisations in over 50 countries. This year, a variety of unique events are being held in over 60 countries and regions around the world, including all 27 European Union Member States, Argentina, Australia, Bahrain, Brazil, Burkina Faso, Cameroon, Canada, China, Iceland, Iran, Israel, Japan, Lebanon, New Zealand, Pakistan, Palestine, Russia, Singapore, Taiwan, and the USA, amongst others. At the European level, this year’s Rare Disease Day brings attention to the disparities that exist between countries when it comes to patient access to diagnostics, care and treatment. These disparities are caused in part by a lack of coordination between and amongst Europe and the Member States. Stakeholders in the field are working hard to eliminate these disparities, and this year’s Rare Disease Day slogan “Rare Disorders without Borders” emphasises that all rare disease patients deserve access to diagnostics, care and treatment. There are an estimated 30 million people affected by rare diseases across Europe, and no individual country has the resources to understand, diagnose and treat all of the individual rare diseases that have been identified. That is why European and international cooperation and collaboration are essential in order to meet the challenge of treating rare diseases.

Rare Disease Day 2013 Video As part of this year’s Rare Disease Day campaign, EURORDIS has released its official Rare Disease Day video, highlighting this year’s Rare Disease Day theme “Rare Disorders without Borders”. This magical and poignant video, created by director Carlo Hintermann, mixes animation with live cinematography, and is available in 12 languages. Watch the video and learn more about the patients who appear in it: rarediseaseday.org For more information about Rare Disease Day, the events planned in participating countries and communication material, visit: www.rarediseaseday.org

About rare diseases A rare disease, also referred to as an orphan disease, is any disease that affects a small percentage of the population. The European Union considers a disease as rare when it affects fewer than 1 in 2,000 citizens. Rare diseases are frequently chronic, progressive, degenerative, and often lifethreatening. Over 6,000 different rare diseases have been identified to date, affecting 30 million Europeans. Despite their great overall number, rare disease patients are the orphans of health systems, frequently lacking access to diagnosis, treatment and the benefits of research.

About EURORDIS EURORDIS is a non-governmental patient-driven alliance of patient organisations and individuals active in the field of rare diseases. We are dedicated to improving the quality of life of all people living with rare diseases in Europe. EURORDIS represents 561 rare disease organisations in 51 countries (including 25 EU Member States) covering more than 4,000 rare diseases. More information is available on www.eurordis.org

Press Contact: Lara Chappell Communications Manager, EURORDIS Plateforme Maladies Rares 96 rue Didot - 75014 Paris Tel : 33 (0)1.56.53.52.60 [email protected]