Case Studies on Rare Disease Therapies Development –
A Laboratory of Ideas for European Innovation Leadership

Pharmaceutical small to mid-sized companies and start-ups constitute a laboratory of innovation and should be nurtured through a forward-looking European incentives ecosystem that encourages the development of cutting-edge technologies. The upcoming EU health policy initiatives, including the Pharmaceutical Strategy for Europe and the impact assessment following the evaluation of the OMP and Paediatric Regulations, provide a unique opportunity to harness this potential.