EUPESMA Series
One Goal
Together we prioritise: results from EUPESMA on Clinical Trial Participation
With the patient experience data from the pan-European community survey on SMA and clinical trials we provide insights into clinical trial participation and the future wishes in relation to clinical trials so that patient perspectives can inform the planning of future clinical trials.
98% say there is a need for new clinical trials
- New treatments for SMA
- 71.3%
- Muscle treatments
- 70.2%
- Regenerative medicine
- 62.7%
- Improved versions of existing treatments
- 53%
What should future clinical trials in SMA focus on?
When asked what clinical trials in SMA should focus on in the future, the most frequently chosen areas of preference were:
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.
