European Society for Phenylketonuria and Allied Disorders Treated as Phenylketonuria
hosted a panel during this year`s European Health Forum in Gastein titled ‘Defining unmet
medical need: what counts?’
The panel featured experts in various fields: Steffen Thirstrup from the European Medicines
Agency (EMA), Anita MacDonald from Birmingham Women’s and Children’s Hospital, Rosa
Castro from EURORDIS – Rare Diseases Europe, and Thomas Bols from PTC Therapeutics,
Inc.
In the insightful and substantive discussion, panelists presented their views. Anita
MacDonald described the everyday realities of living with Phenylketonuria, outlining the
challenges patients face and the risks that a restrictive definition of unmet medical need
could create. Rosa Castro stressed that whether the definition is narrow or broad will directly
influence how well patient needs are addressed and emphasized it should be inclusive.
Steffen Thirstrup emphasized the European Medicines Agency’s long-standing commitment
to involving patients in its procedures and expressed hope that new legislation can improve
development and products coming to the European market. Thomas Bols highlighted that
companies develop therapies specifically for patient groups with unmet medical needs and
presented his point of view on the various elements of the definition of unmet medical needs
and how they influence companies’ decisions. Watch the full panel discussion from the
European Health Forum Gastein 2025 here.