Our European Patient Expectation Survey Series – EUPESMA

Our European Patient Expectation Survey SeriesOur European Patient Expectation Survey Series (EUPESMA) are designed to ascertain the needs and wants of people living with SMA, to help us advocate for better access to treatments and care. ​

We are currently working on our 3rd edition.

 

 


Background

  1. Our first survey, published in 2017, sought to obtain a view on the clinical state of European people living with Type II and Type III SMA, the impact of this situation on their quality of life and their expectations regarding clinical development. A compelling majority of respondents indicated that stabilisation of their current clinical state would represent therapeutic progress. These results allowed us to inform drug developers and the European Medicines Agency (EMA), the latter culminating with a dedicated workshop on SMA, which was held on 11th November 2016 in London, prior to the first treatment for SMA being approved.
  2. In 2021, our second survey was published and this one 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. In line with findings from our 2017 study, participants considered stabilisation of their condition to be progress. Notably, 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 that varied in particular between age and disease severity groups, yet there was high interest in clinical trial participation. In addition, alternative treatment options, including combination therapies, are now expectations. These perspectives should be central considerations in the research and development processes of new SMA therapies, through data generation and discussions on access to therapies. Results from this survey indicate that collaboration between stakeholders is essential to the foundation upon which innovative approaches for SMA treatments and access can be explored.

Current EUPESMA survey (2021)

Our newest survey is carried-out in collaboration with Tilburg University in the Netherlands and supported by the Cure SMA Industry Collaboration.

Aim

To gain an in depth understanding of the experience people living with SMA have, with nutrition, physiotherapy and treatment, to help us advocate for better access to treatments and care.

Method

A survey has been designed, translated and distributed over 20 countries throughout Europe. Please help us by answering it. All information can be found here.

Our Team


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