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R&D ecosystem

Need 1: Improve the R&D ecosystem for basic research and company take up of development

Basic research and clinical development are the backbone of OMP development. Without a solid understanding of underlying disease mechanisms, biomarkers and targets, coupled with a strong research pipeline, no OMP development can take place. In fact, the lack of basic research is among the key reasons for the absence of development in certain disease areas. While dispersed and small patient populations make research in rare diseases challenging in itself, the lack of a functioning R&D ecosystem in Europe hampers further research activity and company take-up.

Although various initiatives, such as the ERNs, have enabled a clustering of knowledge on rare diseases, rare disease research is still dispersed across many different institutions and the respective expertise is held by few specialists, who also operate at different geographical locations. This implies that research activity lacks scale and visibility among researchers and companies with potential commercial interests for development.

In addition, research is often not ‘development ready’ and securing funding for research is difficult. Therefore, policy makers should take measures to improve the R&D ecosystem for rare diseases in Europe. Policy solutions should build on the many existing structures and initiatives that already make up the EU rare disease R&D infrastructure – and include better funding, incentives for development-ready research and the necessary collaborative infrastructures for all stakeholders in R&D.

The recommendations

  • Form an EU rare disease hub for large-scale collaboration, data sharing and generation, and diagnosis.
  • Form a Rare Disease PPP fund for basic research and early development
  • Provide guidance and incentives for translational basic research
  • Establish a coherent policy framework for the use of RWE