SMA Europe launched a Europe-wide survey, aimed at understanding the treatment expectations of people living with SMA, the realities of daily living, as well as their access to clinical trials and therapy. An understanding of how these expectations may vary with parameters such as age and disease severity was also sought.
How we did this
This survey was sent to people of all ages and disease severity, across the SMA community all over Europe. 1,474 completed and validated surveys, from 26 European countries were analysed.
What we found
- Stabilisation of current clinical state is considered to be progress, with 96.6% of all participants in agreement and the proportion similar across stratified groups. This result is in line with findings from our first survey, published in 2017
- Importantly, responses indicated that the current classification of SMA at diagnosis by ‘type’, often does not reflect current mobility level.
- Large gaps in treatment access were identified, with frustrations felt by participants. These varied between age and disease severity groups in particular, despite a high interest in clinical trial participation.
- Alternative treatment options, including combination therapies, are now expected.
What this means
Patient perspectives and expectations must be considered throughout treatment development and subsequent discussions on access.
As this survey shows, stabilisation and minor changes in functional ability can have meaningful impacts on daily life. Our goal must therefore be to gather evidence and further drive research to personalise medicine, so that each individual may have access to the therapy that provides individual optimal therapeutic benefit.