2014

Orphanet Journal of Rare Diseases

Accelerating development, registration and access to medicines for rare diseases in the European Union through adaptive approaches: features and perspectives

There is growing recognition that the current research-and-development (R&D) and innovation-regulation ecosystem could be made more efficient to stimulate and support access to innovative therapies for those patients with rare, life-threatening diseases for which there are no adequate licensed therapies.

Emmanuelle Voisin, David Uguen