SIXTH WORKSHOP ON PARTNERING FOR RARE DISEASE THERAPY DEVELOPMENT
London, 25-27 October 2005
“People with Rare Diseases – No Longer Alone in the World”

Encouraging research, facilitating development, and securing take up of innovation and treatments for rare diseases through a supportive regulatory framework and functional partnering to ensure equal access to treatments for all.

The EPPOSI London workshop in context

The EU orphan medicinal products Regulation EC 141/2000 entered into force in April 2000 upon unanimous approval from the European Parliament. By April 2005, 254 orphan designations have been granted by the European Commission and 20 orphan medicinal products have been given marketing authorisation in the EU. Stakeholders agree that this positive start is a source of hope for patients and their families, but much remains to be done to ensure that more products reach those that need them. The impact of the “orphan drug” Regulation is currently being assessed after the first 5 years of existence. This is an ideal time to compare the situation in Europe with that in other regions of the world, such as the USA, where similar regulations has been in place for a longer period of time. This is why this workshop is organised in partnership with NIH and FDA, and other people, active in work related to rare diseases from other regions in the world, are being invited.

It is also time to look at the R&D process as part of a broader priority framework, to identify bottlenecks and possible solutions and to benchmark the initiatives which have shown to be efficient in providing an appropriate framework for the development and provision of innovative therapies for rare diseases. With the European Commission’s next Research Framework and Public Health programmes both under discussion this year, this conference will provide an excellent opportunity to discuss the impact of EC support for basic research and the development of information systems in the field of rare diseases. The USA has also supported several important initiatives to boost R&D in the field of rare diseases and the results can useful as input in the discussion of the EU approach.

There is a major European conference in Luxembourg in June 2005, organised by EURORDIS in collaboration with the EC and the Luxemburg EU Presidency, to present their case. The views of patient organisations will be an important basis for discussion at the London EPPOSI workshop. The industry groups EBE/EFPIA and EuropaBio have published a White Paper “Towards an Optimal Orphan Medicinal Products Framework in Europe” with suggestions which equally will contribute to the workshop.

Topics to be debated during the workshop and compared for Europe and the USA will include priority setting in research, facilitating development, the importance of accurate diagnostic procedures, facilitating clinical trials and encouraging transatlantic collaboration, harmonisation of reference frames, facilitating access to innovative therapies, and care provision for rare diseases in the context of political and economic globalisation.

These same topics are currently the subject of active debate amongst stakeholders, sometimes with very conflicting views. During the workshop, participants will be asked to actively contribute to an action plan of key recommendations, addressed to specific stakeholders, which will be debated in the final session. By providing such a forum for open and interactive debate, the 2005 EPPOSI workshop will give all stakeholders the opportunity to impact on the future of patients with rare diseases and contribute to advancing international cooperation.