In this guest blog post, Sarah Khor, Director, International Government Affairs & Policy at Amgen (EUCOPE member) sets out why the recently adopted WHA Resolution on rare diseases is an opportunity not to be missed for Europe.

The adoption of the World Health Assembly (WHA) Resolution on rare diseases on May 24, 2025 marks a milestone moment for the global rare disease community. It signifies a strong commitment to improving the lives of the over 300 million people living with a rare disease worldwide. For the EU, this Resolution is more than just a milestone – it is a call to action urging both EU Institutions and Member States to step up their game in addressing rare diseases.

The goal: To create smoother and faster pathways to diagnosis, care, and treatment, ensuring that those affected receive the support they need without unnecessary delays.

The call for a more ambitious EU

When considered together, rare diseases are not rare at all: more than 30 million people in the EU are currently living with a rare disease. Aside from their enormous impact on physical and mental health, as well as quality of life, rare diseases also have a substantial economic impact. In fact, they cost each person affected an estimated €121,900 per year in direct and indirect costs, more than twice the average income.[i] To put this into perspective, the overall economic cost burden of rare diseases has been estimated to be over 40% higher than that of cardiovascular diseases. [ii]

The EU has taken great strides in addressing rare diseases in recent years. Perhaps the most notable achievement has been the establishment of European Reference Networks (ERNs) – cross-border networks bringing together European hospital centres of expertise and reference to tackle rare conditions. ERNs are a prime example of how the EU can add value. However, it cannot stop there. Improving access to the best diagnosis, treatment, and care available for those affected by rare diseases remains a critical challenge. This is an area where the EU can truly make a difference by setting standards and fostering cooperation between Member States. It is time to build on our progress and push for even greater achievements.

The WHA Resolution, which provides for a Global Action Plan on rare diseases, should be the perfect catalyst for the EU to step up and take ambitious action, to the benefit of people living with rare diseases, those close to them, as well as our societies and economy.

Putting innovation at the heart

Innovation plays a critical role in bringing life-saving and life-prolonging medicines to patients and improving outcomes. It enhances efficiency, effectiveness, quality, sustainability, safety, and affordability of healthcare.

Patients deserve access to first-in-class therapies that have been developed for their specific condition and evaluated as safe and effective to treat it. For those with rare diseases, where treatment options are often non-existent, these innovative therapies can be life-changing.

Healthcare systems need to recognise the value of new and innovative products, especially in underserved areas, and ensure patients can access them. This recognition is crucial not only for patient care but also to encourage ongoing innovation, in alignment with current legislative discussions. In today’s competitiveness-driven environment, fostering innovation and ensuring patients benefit from the latest treatments can significantly improve health outcomes for those with rare diseases now and in the future, as well as bolster the EU’s global competitiveness in the sector.

A plan to make a difference

A robust rare disease plan can have enormous benefits, reducing the burden of disease on those affected and on wider society. For example, a dedicated plan can include measures to raise awareness of rare disease, which in turn helps ensure earlier and more accurate diagnoses. It can also promote better coordination of care, access to innovative treatments, and enhanced data collection – which can ultimately improve research and understanding of rare disease, and better support for patients and their families and carers.

National rare disease plans across EU Member States currently vary in maturity and effectiveness. The WHA Resolution should trigger change at the national level, in every country. Every Member State should put in place an ambitious and concrete rare disease plan, and those that already have one should take this global action as a call to ensure it is updated and in line with the latest innovations in diagnosis, treatment and care. Publishing a plan is only the first step. Proper implementation is vital to achieve actual impact and lead to tangible, measurable improvements in the timely diagnosis, care, and treatment of those living with a rare disease.

A dedicated EU Action Plan on Rare Diseases would provide the necessary framework to harmonise efforts, set the standard, and drive progress across the region. An EU Action Plan would allow for more coordination and sends a strong political message that the EU is prioritising rare diseases. Backed by a diverse array of stakeholders including patient advocates, the EU Institutions, Members of the European Parliament, Member States, and industry leaders, to name a few, such a plan would provide the above-country coordination needed to make a difference throughout the EU. By advancing and going beyond the provisions in the WHA Resolution through coordinated EU and national action, the EU has the opportunity to be at the forefront in rare diseases.

Conclusion

The adoption of the WHA Resolution is a unique opportunity which the EU should seize to deliver meaningful change for rare disease patients. This is the EU’s chance to lead the way globally and set an example for other regions, as it has done in the past and across various policy domains. By committing to ambitious actions at both the EU and national levels, we can ensure that rare disease patients receive the care, treatment, and support they deserve. Let’s work together to make this vision a reality.

 

[i] Charles River Associates (2024). The economic cost of living with a rare disease across Europe.

[ii] Charles River Associates (2024). The economic cost of living with a rare disease across Europe.

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