Session 14

Defining unmet medical need: what counts?

Going beyond clinical criteria in the EU’s Pharma Legislation

Organised by the European Society for Phenylketonuria and Allied Disorders Treated as Phenylketonuria

European policymakers and Member States are revising the General Pharmaceutical Legislation (GPL) and debating a new definition of Unmet Medical Need (UMN). This new definition will influence how diseases are categorised, how innovation is incentivised, and how funding is allocated. Many patient communities are concerned that if the final definition focuses only on clinical criteria of morbidity or mortality, patient critical outcomes and factors such as quality of life, burden of treatment on patients and caregivers, and the broader socio-economic impacts of the disease will be overlooked.

The session will examine where we are in the GPL process and what is at stake. It will explore how narrow, clinically-focused definitions risk systematically under-recognising diseases like Phenylketonuria (PKU), where clinical morbidity is low but the disease and its treatment impose a significant burden, potentially stalling the development of new treatments and locking patients into suboptimal care.

Further speakers to be announced