Rare Diseases: How the new EU rare disease policy framework provides an opportunity to move from building infrastructures to measuring impact

‘Rare 2030’ Foresight Study highlights unmet needs and continued obligation to improve outcomes, explains EURORDIS Health Policy Director Anna Kole.
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By Anna Kole

Anna Kole is Public Health Policy Director - Rare2030 Project Lead at EURORDIS

26 Feb 2021

After two years of broad consultation, the Rare 2030 Foresight Study highlights the outstanding and considerable unmet needs of people living with rare diseases and the continuing need for improvement in the policies, programmes and services that currently address them across Europe.

This study, supported by Parliament and co-funded by the Commission, has collated the input of over 250 patient advocates, practitioners and key opinion leaders as well as thousands of patients to propose policy recommendations that will lead to improved policy and a better future for people living with a rare disease in Europe.

The main recommendations focus on innovation to improve the fulfillment of patients’ unmet needs, which can then be measured by the improvement in health and wellbeing outcomes among the population of people living with rare diseases.

“The Rare 2030 Recommendations will consider the full ecosystem including public health, research and innovation, social affairs, digital efforts as well as all stakeholders”

This includes, but is not limited to, earlier, faster and more accurate diagnosis; taking a holistic approach to the individual’s needs, and improved access to high-quality integrated medical and social care. It also involves a comprehensive approach to care and research, data and core capabilities, improved availability, accessibility, affordability and sustainability of medical products.

The current European Rare Disease policy framework (the Commission Communication in 2008 and the Council Recommendation in 2009) and subsequent strategies have led to tremendous success but require reassessment.

The European Court of Auditors and the European Commission have agreed that this strategy (including the role of the European Reference Networks) “needs to be updated, adapted or replaced.” A new policy framework will provide the vehicle to address these changes with the support of European countries and a new generation of values, technologies and policymakers in Europe today.

The health policy of rare diseases is now mature enough to move from building infrastructures to measuring the impact on improved health and wellbeing outcomes. A new European rare disease policy framework provides an opportunity for this shift and new ambition, prompting a plan for measurable impact at the European and national level. The Rare 2030 Recommendations will consider the full ecosystem including public health, research and innovation, social affairs, digital efforts as well as all stakeholders.

The newly-proposed European Health Union and the EU4Health Programme will be two considerable sources of support addressing the current unmet needs for people living with a rare disease. A new policy framework will complement and guarantee the success of these larger initiatives - ensuring that the health field of rare diseases is addressed by a long-term strategy rather than unsustained projects.

Although the EU is currently limited in its remit on healthcare, it is well placed to contribute to the Sustainable Development Goals to guarantee human rights. A new European rare disease policy framework can continue to most efficiently address the area of rare diseases with concerted EU-level efforts but also combine it with a new focus on ensuring that none of its citizens are left behind.

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