Session 20

Simulation: the innovation gap in rare diseases

Bridging the scientific and economic challenges

Organised by Ipsen

The recent example of COVID-19 has demonstrated that innovative solutions are essential to healthcare systems and that political priorities impact drug development. This political will is particularly important for the 7,000+ rare and ultra-rare diseases, most of which have no approved treatments.  Yet the development of ‘orphan medicines’ is impeded by the limited scientific understanding and challenging economics of rare diseases.

This interactive session – involving a simulation, roundtable discussions followed by a panel discussion featuring key stakeholders - will shed light on the particularities of orphan drug development, how pharmaceutical companies make investment decisions, and the importance of an environment supportive of innovation to foster resilient health systems and ensure access for people with rare and ultra- rare diseases.