EUPESMA: European Patient Experience Surveys in SMA
One Goal Series
Together we prioritise: results from EUPESMA on SMA medicines, access, and treatment journeys
The European Patient Experience Survey (EUPESMA) series maps the experiences and expectations of people living with SMA and to ascertain their needs and wants. With this evidence, SMA Europe advocates for equitable access to optimal treatment and care in Europe.
Patient journeys in SMA are not linear - they are punctuated by disruptions, delays, or redirections.
The experiences reported by our European SMA community in the latest EUPESMA survey shed light on the complexities of treatment journeys in SMA. Better understanding these experiences can guide healthcare professionals, policy makers, and the advocacy community at large to build the best support for individuals and families living with SMA.
The SMA Treatment Journey at a Glance
- Diagnosis: 58% had no treatment or clinical trials available at diagnosis
- Treatment decision-making: Only 31% had a choice between SMA medicines
- Starting treatment: 85% saw starting treatment as an opportunity
- Living with treatment: 89.6% consider stabilisation to be meaningful progress
- Switching and interrupting treatment: 19% experienced treatment interruption
- Looking ahead: 82% think there is a need for new medicines in SMA
The question
Why do we need Pan-European patient surveys?
Patient experience data provides essential real-world insights into the daily realities, challenges, and expectations of people living with SMA. Data from clinical studies and registries are vital, but they cannot fully capture how treatment access, care pathways, and quality of life vary across countries and the continuum of the condition. By collecting this data directly from the community, we ensure that lived experience becomes visible, measurable, and actionable, forming a credible evidence base that supports equitable, person-centred care and strengthens the community’s voice in decision-making.
SMA Europe conducts Pan-European patient experiences surveys to
- understand how access to medicines and care differs across Europe
- identify disparities and unmet needs
- ensure that diverse experiences across the continuum and domains are represented
- generate robust, comparable evidence that supports advocacy for fair and consistent care
- provide stakeholders, including policymakers, clinicians, pharmaceutical industry, regulators, and payers, with clear insights into what works well and where improvements are needed
- strengthen the unified Pan-European voice of the SMA community and guide efforts toward equitable access.
An SMA Europe series
Our SMA patient expectations surveys
Our EUPESMA-2019
Our second survey, published in 2021, sought to understand the treatment expectations of people living with SMA after the approval of the first therapy, the realities of daily living and access to clinical trials and therapy, and how this varied according to parameters such as age and disease severity.
