20th ERTC workshop marks ten years of successful collaboration in moving forward treatment development for rare diseases

20th EURORDIS Round Table of Companies workshopThe EURORDIS Round Table of Companies (ERTC) held its 20th workshop in Brussels on 26 February 2014 with the overarching theme of Unlocking Europe’s Potential in Rare Disease Therapies between now and 2020 - celebrating ten years of dialogue and collaborative efforts between patient advocates, industry, regulatory agencies and national competent authorities to move forward the development of treatments for rare diseases.

The focus was on the research agenda of the new EU Framework Programme Horizon 2020 and the synergies between the European Medicines Agency, the Innovative Medicines Initiative 2 (2014-2020), industry, patients, and science, in order to make sure that the momentum is sustained and new opportunities are optimised. The 90 participants representing all stakeholders discussed how each partner has a vital role to play in paving the way for new treatments development – industry, policy-makers, regulators, patients, and academia

Many important points were raised during the day-long event, including the common view that the EU Regulation on Orphan Medicinal Products is efficient and does not need revising; the utility of rare disease research for personalised medicine and for understanding and treating common diseases; the need to address current huge unmet medical needs rather than developing a third or fourth product for the same therapeutic indication; the pressing need to learn more about the natural history and physiopathology of rare diseases in order to meet the goal of developing new treatments; the elaboration of more guidelines on clinical trials for those diseases for which there are several medicines under development, including earlier identification of clinical endpoints and patient-relevant outcomes for higher approval success rates; the reduction of research and development costs through wider use of adaptive design and statistical methods, and alternatives to animal models and biomarkers in order to reign in prices; promoting Health Technology Assessment approaches adjusted to rarity and involving patient representatives early and throughout the process of product development, authorisation, distribution and assessment.

Such ambitious ground-breaking agendas are not new to the ERTC. Over the years, each ERTC workshop has been scrupulously designed to address the issues of the moment and help shape the rare disease environment in a positive, forward-moving trajectory.

The ERTC was created as a forum for dialogue between rare disease patient advocates, industry, regulators and other stakeholders. It also serves to gather unrestricted funds for EURORDIS activities on behalf of people living with rare diseases. Over the past ten years, the twice yearly round tables have regularly tackled the key issues that can unblock bottlenecks and advance real solutions for more, safe, efficient, effective, affordable, accessible therapy development.

Information on all of the past 20 ERTC workshops is available online. The next ERTC will be held in Barcelona at the end of September. Here’s to another ten years of fruitful collaboration that is valuable to all members of the ERTC – and to people with rare diseases in Europe and beyond.

Louise Taylor, Communications and Development Writer, EURORDIS

Page created: 19/03/2014
Page last updated: 18/03/2014
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