With insights from 22 countries, the landmark research and resulting reports provide a first-of-its-kind, comprehensive view of the gaps in care for adults living with SMA - from the need for adult-specific care guidelines, to the need for better coordination of care within health systems and more equitable access to disease modifying treatments (DMTs).
Despite an improvement in health outcomes for those living with SMA in recent decades, many concerns around care for adults remain. To address this, SMA Europe and Roche set out to develop a picture of adult care in SMA and issue an urgent call to action for change among policymakers, clinicians and patient advocacy groups (PAGs).
Véronique Van Asche, Chair of the Adult Committee at SMA Europe, said, “Adults living with SMA view this benchmarking report as crucial, as it sheds light on the state of care for their condition in Europe, showing key areas for improvement. They hope the report will serve as a guide for policy changes and actions that will improve their quality of life by addressing critical gaps in care provision.”
Research conducted with PAGs and the clinical care community, combined with a rapid literature review, led to the benchmarking of 19 indicators of care. The resulting Europe-wide report and 22 country-level reports provide rich, evidence-based insights into the status of SMA adult care, common areas of weakness, best practice examples and overall recommendations to improve care provisions and quality of life for adults living with SMA.
The report amplifies the voices of adults living with SMA across Europe. Nicole Gusset, CEO and President of SMA Europe, noted, "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."
In the key areas identified for improvement, the urgency is great.
With more people growing up and growing older with SMA, 50% of people living with SMA are adults, yet adult-specific care guidance is lacking, particularly when it comes to DMTs.1 Many countries rely on 2017 international standards of care or national guidelines, yet these focus mainly on best practice care for children.
The transition from paediatric to adult care can be complex and unnerving, with many people facing a cliff edge as they reach adulthood. Although there are some treatment centres across Europe that provide support at this time, the approach is not unified.
Even when having access to the different specialists they may need, adults with SMA lack the advantage of coordinated day-to-day care through a formal multidisciplinary care team. They often spend a disproportionate amount of time in the healthcare setting and miss out on key aspects of care.
DMTs have demonstrated an important role in stabilising the progression of SMA and are approved for use throughout the majority of Europe, but not everyone has access to them. In some countries, age restrictions are in place which significantly limits access for adults, yet equitable access is essential to enabling people with SMA to achieve more meaningful health outcomes.
Greater support for those living with SMA via social systems is also needed. Funding for personal care assistants, enabling assisted living at home and support for informal caregivers can help people with SMA to live independently and contribute to society in ways that are meaningful to them.
The report outlines how change can be delivered and provides recommendations and best practice examples to draw inspiration from. It calls on policymakers, researchers and clinicians to work with patient organisations to take action with a pragmatic set of international and national recommendations to address the unmet needs. Collectively, we can bring about systemic changes that will empower adults with SMA to achieve meaningful health outcomes and live more independent, fulfilling lives.
Visit SMA Europe’s OdySMA website to learn more:
The reports have been made possible with funding support from Roche.
M-XX-00016421
Date of preparation: February 2024
References
CureSMA, “State of SMA: 2022 Report,” 2022. Available:
Accessed 27 February 2024.
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