FH Europe Foundation Ambassadors have collaborated as part of the European Patients’ Forum (EPF) Taskforce on unmet needs, specifically the definition of 'unmet medical needs' and how it will shape the future of healthcare. This urgent and important issue arose in the context of the reform of the EU pharmaceutical legislation. In the spring of 2023, the European Commission adopted a proposal for a new Directive and a new Regulation, which revises and replaces the existing general pharmaceutical legislation (Regulation 726/2004 and Directive 2001/83/EC) and the legislation on medicines for children and for rare diseases (Regulation 1901/2006 and Regulation 141/2000/EC, respectively).
In a defining moment for pharmaceutical regulation in the European Union, Article 83 of the European Commission's proposal for a Directive on medicinal products for human use introduces the pivotal concept of "unmet medical need."
Over a number of weeks, the international patient advocates community brought together by the EPF, worked together to highlight the need for more inclusive approaches beyond morbidity and mortality. To develop a fair and equitable definition, they wanted to ensure the patient's voice was heard. The Taskforce believes that patients should play an active role in shaping the definition of unmet medical needs as they are the ones directly affected by it. By incorporating their perspectives and experiences, a deeper understanding of the challenges they face and the gaps in healthcare that need to be addressed is gained. Through this collaborative effort, the Taskforce hoped to create a definition that truly reflected the diverse needs of patients and paves the way for a more patient-centred healthcare system.
“If the EU only looked at morbidity and mortality as signs of unmet medical need, they would miss other vital life-changing signs that may need significant therapeutic improvements from a patient's point of view, like how well current treatments are working or how they affect the quality of life”
We thank the Ambassadors involved in this project, ensuring the needs of the community affected with inherited dyslipidaemias are well represented, especially in the context of primary and secondary prevention, quality of life and precision medicine delivery for patients with FH, HoFH, elevated Lp(a) and FCS.
A position paper on this crucial topic prepared by the EFP has now been published.
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