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One Year On: The EU’s progress against the UN Resolution on rare diseases

December 2022

One year ago, the United Nations General Assembly adopted the Resolution: ‘Addressing the challenges of persons living with a rare disease and their families’, which set 193 countries the direction for bettering the lives and wellbeing of those living with a rare disease globally.

By endorsing it, all 27 EU Member States made a political commitment to protect the rights of people living with a rare condition and to build an infrastructure of health and other public services that address the full breadth of their currently unmet needs.

However, due to their complexity and low prevalence, no country alone holds the knowledge and capacity to treat all types of rare diseases. By working on their own, they cannot successfully make good on the promises they committed to in the UN Resolution.

A comprehensive, goals-based European Action Plan for Rare Diseases – which ties together the progress made across all EU countries and policy areas – is the only way to ensure that Europe completely delivers on the UN Resolution.

Given the necessity of broader action at the European level, we must pose the question: What progress has the EU made in the past year in bringing about improved rare disease policies and legislation?

We break down the UN Resolution’s calls into three broad themes – advancing healthcare services, improving mental health and wellbeing, and creating equitable societies – and recap the EU’s progress over the past 12 months in these areas.

Advancing healthcare services

2022 has seen decisive political steps forward in terms of policy development. However, achieving truly personalised and integrated care must involve tying together these areas of progress through a European Action Plan for Rare Diseases.

Valentina Bottarelli, European Public Affairs Director & Head of European Advocacy, EURORDIS

The UN Resolution calls on countries to “strengthen health systems” that are “safe, of quality, accessible, available and affordable.”

In 2022, the EU saw several legislative developments relating to the development of medicines, data-sharing, diagnostics, and cross-border cooperation.

The European Commission spent the last year continuing to draft its Revision of the Orphan Medicinal Products and Paediatric Regulations, which should improve the system built to incentivise the development of rare disease medicines. Poised for release in early 2023, the Revision is assured to be undergoing final internal scrutiny. While the Revision does not present the only opportunity to greater encourage the development of more transformative medicines, it will make significant progress if it takes on many of our recently published recommendations.

In May, the Commission began the legislative passage of the European Health Data Space (EHDS). As we enter 2023, the Commission should consider our recommendations on how to unlock the potential of health data for advancing research.

In July, the Czech Presidency of the EU Council joined our call for harmonised newborn screening programmes across Europe, paving the way for greater uniformity in Member States’ application of the great scientific leaps forward that have been made in diagnostics.

Although these developments have been positive, they have not resulted from an overarching strategy aimed at meeting all of the medical needs of rare disease patients through more integrated and holistic care.

Improving mental health and wellbeing

The psychological burden of living with a rare disease is going unrecognised and unaddressed for too many. Challenges like lengthy diagnostic journeys and a lack of available treatments exert their own strains on mental health. An EU Action Plan on mental health must respond to the needs of people from our rare disease community.

Matt Bolz-Johnson, ERN & Healthcare Advisor, EURORDIS

The UN Resolution urges the implementation of “effective programmes to promote mental health and psychosocial support” for persons living with a rare disease.

The World Health Organisation (WHO) warned earlier this year that the COVID-19 pandemic had a grave impact on the mental health of those with a pre-existing physical health condition. Reflecting the WHO’s finding, EURORDIS’ had found that two third of people living with a rare disease suffered from depression, or a feeling of not being able to overcome their problems, subsequent to the onset of the pandemic. Of those rare disease patients who experienced disruption to their health care during COVID-19, six in ten had specifically seen their psychiatry sessions interrupted.

Mental health resultantly rose on the EU policy agenda in 2022, with Commission President Ursula von der Leyen promising a new strategy on mental health in her State of the European Union address. Next year’s Action Plan on mental health must address the very particular pressures on the psychological wellbeing of those with a rare condition, as these have thus far not been sufficiently appreciated by policymakers. Alongside promoting more specialised psychiatric services, the Commission’s mental health strategy must explicitly acknowledge that the availability of medical treatments – which directly and effectively treat a condition – is a key factor of the wellbeing of someone living with a rare disease.

Creating equitable societies

Most people who live with a rare disease, live with a disability. This disability can be visible, invisible, degenerative, or vary from one day to the next. We must break down the many existing barriers to ensure their full participation in society, on an equal basis with others.

Raquel Castro, Social Policy and Initiatives Director, EURORDIS

Meeting the commitments of the UN Resolution must involve recognition that the needs of people living with a rare disease are not purely medical. Policy actions beyond the sphere of physical or mental health services are vital to making sure people living with a rare condition or disability are not deprived of the same opportunities for personal fulfilment that others enjoy.

In 2022, the European Commission progressed actions from its new Strategy for the Rights of Persons with Disabilities, which are essential for the rare disease community. As part of this, the Commission published its Disability Employment Package, which includes crucial measures such as guidelines on reasonable accommodation at work. However, as noted by the European Disability Forum, several issues that negatively affect the labour inclusion of persons with disabilities are not currently tackled by the Package.

In 2022, the Commission continued working towards its 2023 proposal on the EU Disability Card, to facilitate free movement for people with disabilities across the EU.

Despite these advances, access to disability benefits and support remains a challenge, often due to the lack of recognition by national assessment procedures. A EURORDIS survey found that 34% of people living with a rare disease face inadequate assessments, and 19% do not receive an assessment at all.

The EU (still) needs an Action Plan for Rare Diseases

The EU has seen major progress, but this progress has been too confined to specific spheres of rare disease policy. An overarching strategy on rare disease, to which all EU countries are bound, is vital.

In 2022, we saw the two successive Council of EU presidencies (France and the Czech Republic), Members of the European Parliament, and the European Economic and Social Council all allying themselves to the call for a European Action Plan for Rare Diseases. Last Friday 9 December, a new total of 22 Member States endorsed the call for an Action Plan.

The political momentum is demonstrably growing, but with the Commission’s current mandate ending in 2024, an Action Plan has never been more urgent. The rare disease community needs a European Action Plan for Rare Diseases, and it needs it now.

Julien Poulain, Communications Manager