SUMMARY: SMA Europe takes great pride in the launch of the benchmarking report which is proof of our collective commitment to drive progress and enhance the lives of all those living with spinal muscular atrophy (SMA). The project is a collaborative effort of the SMA Europe’s international community and has been supported by MEP Stelios Kympouropoulos (EPP, Greece).

SMA Europe is the European umbrella for national SMA patient organisations. Among its main strategic pillars is to improve access to diagnosis, optimal treatment, and care for all SMA patients in Europe, as well as to accelerate progress in the diagnosis, treatment, and care of people living with SMA. SMA Europe bases its actions on evidence-based advocacy. SMA Europe’s membership extends over 25 countries and is made up of 27 patient organisations.

SMA has historically been considered a children’s disease. Yet today, adults make up an estimated 50% of the population. And with the increasing availability of innovative treatments that improve quality of life and survival rates, those numbers are set to grow. It remains crucial not to overlook the needs of those living with SMA in adulthood. Yet, many essential initiatives focus predominantly on the needs of children living with SMA, leaving adults, and those transitioning into adulthood, relatively neglected.

This benchmarking study was designed to measure and compare how care for adults living with SMA is provided in 23 countries across Europe. Categorised under three overarching themes, the results were mapped against 19 indicators. The report provides two international and six national recommendations to provide an advocacy tool for any European patient advocate in the field of SMA. Apart from the main benchmarking report, 22 short country reports are also provided to support national patient organisations in their advocacy actions.

This complements the atlas of SMA Europe’s “OdySMA initiative”. In this atlas, data on access to diagnosis, medicines and care is collected systematically to create visualisations that illustrate access pathways across Europe, providing a comprehensive and organised representation of information related to SMA, to ensure that no one is left behind.

Dr Nicole Gusset, CEO and President of SMA Europe:

“This report is more than just a compilation of facts and figures; it is a powerful advocacy tool, representing an opportunity to amplify our voices and advocate for the needs of adults living with SMA. This part of our community has long been underserved and deserves nothing less than comprehensive care and support.”

Stelios Kympouropoulos MEP (EPP, Greece):

“One of my credos in politics is that our policies and laws should be based on sound data. The present study does exactly that by shedding a light on the state of care for adults living with SMA in Europe. On 6-9 June 2024, European citizens will go to vote and elect their representatives at the European Parliament. Major changes will also be made at the leadership of the main EU Institutions. I am convinced that the study in hand needs to feed into the reflection stage the European Institutions are getting into. A reflection stage about how the next mandate’s social and healthcare policies will reflect the needs of people with rare diseases and in particular, SMA patients.”

This benchmarking report has been made possible thanks to the invaluable contributions and support provided by several organisations and individuals, namely the Expert Advisory Group, the SMA Europe member organisations, and the healthcare professionals who responded to the survey. 

The project is a collaborative effort and shared outcome of a partnership between SMA Europe and F. Hoffmann-La Roche Ltd, driven by their shared vision to improving care in the field of SMA. This report has been made possible with funding provided by Roche as part of this partnership.

---

---