European barometer on rare diseases

Europeans do not accept fatalism! On the occasion of World Rare Disease Day, Ipsos and ASAP FOR CHILDREN present the results of a large survey in 10 European Union countries (11,400 interviews).

The author(s)

  • Etienne Mercier Public Affairs, France
  • Amandine Lama Public Affairs, France
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What proportion of the European population is affected by rare diseases? How do they view the journey of the families of those affected? How do they perceive medical research and access to treatment for patients? What expectations do they have when it comes to improving the care of patients suffering from rare diseases and the development of new treatments?

Main findings of the survey

  • Rare diseases are not so rare: one person in 20 declares to be affected (5%) and 3 out of 10 people are concerned by this problem, being affected themselves, having a very close relative affected (13%) or an acquaintance (13%).
  • Faced with the reality of the painful journey of many parents of children with rare diseases, a majority of Europeans imagine that they would not accept fatalism: if one of their relatives had a rare disease, 72% would not accept the impossibility of obtaining a diagnosis for several years and 72% would also not accept to discover that no research is being carried out to develop a treatment for this disease, considering in both cases that it is possible to act if all the actors concerned are mobilized.
  • They are in favor of measures that could be put in place immediately to improve the care of patients suffering from rare diseases and the support to their families: They believe it is important, even essential, to train health professionals in delivering diagnosis (94% of Europeans), to develop a "culture of doubt" by encouraging health professionals to take more account of the symptoms described to them by parents, in order to reduce diagnostic errors and misdiagnosis in children (91% of Europeans) or to conduct systematic screening at birth for rare diseases for which there is an effective treatment (90% of Europeans). 
  • Europeans are convinced that funding research projects on innovative treatments for rare diseases will help advance research for other diseases: 90% think so.
  • They are also extremely favorable to speeding up the procedures for making innovative treatments available when there is a vital emergency (90%) and for the majority of them it is even essential (54%).

Fact sheet: Ipsos survey for ASAP for Children conducted online among 11,400 Europeans aged 16 and over, interviewed from February 28 to March 24, 2020. National representative samples in each of the country surveyed (quota method).

About ASAP for Children

SMA-SME is a rare and neurodegenerative genetic disease, both muscular and cerebral. Without treatment, the life expectancy of children affected by this disease does not exceed adolescence. Faced with the urgency induced by such a diagnosis and brought about by the incredible advances in gene therapy, the ASAP for Children association was created in 2019 with one and only objective: to cure SMA-SME and Farber's disease, both induced by a mutation of the ASAH1 gene.

To achieve this, the association has set itself the following main missions:

  • To raise awareness of SMA-PME and Farber's disease in order to find other sick children in France and around the world.
  • Raise funds to contribute to the rapid development of a gene therapy treatment and the organisation of a clinical trial.
  • Raise public awareness of this race against time, which is a vital emergency.

Driven by a positive, dynamic, unifying and collaborative approach, ASAP for Children is convinced that it is possible to cure these children as quickly as possible thanks to scientific innovation, solidarity and collective intelligence.

To learn more :

ASAP for children is a recognised association of general interest and is authorised to receive donations.

To make a donation : Click here (

The author(s)

  • Etienne Mercier Public Affairs, France
  • Amandine Lama Public Affairs, France