In the largest study of its kind across the Nordic region, our 2025 survey reveals a compelling story of challenge and hope within the rare disease community.
Nordic rare disease patients urgently need better coordinated care and psychological support, with 95% calling for improved collaboration between healthcare professionals.
View the survey highlighs here.
Despite being widely used, there isn’t one consistent definition of ‘patient empowerment’ with full endorsement from the communities it is meant to represent.
In 2015, the European Patient’s Forum worked to develop a possible definition of empowerment as “a multidimensional process that helps people gain control over their own lives and increases their capacity to act on issues that they themselves define as important.”
In 2008, the WHO described empowerment as a “prerequisite for health” and “a proactive partnership and patient self-care strategy to improve health outcomes and quality of life among the chronically ill ”
As policy expands to include increasing references to patient empowerment in national rare disease strategies, it is important to ensure that people living with rare diseases themselves are the ones shaping the meanings and uses of this term. So that it may be used correctly, reflect the needs of the people it relates to, and most importantly become measurable so that it might be improved.
We explored six dimensions of empowerment through our survey. You can find out more about these six dimensions and the survey itself through these posters [link to posters used in session]:
While 95% of rare disease patients need specialist access and coordinated care, more than one in three lack holistic long-term treatment plans and face significant barriers accessing medical expertise, highlighting a critical gap between essential care needs and current healthcare delivery.
Despite 96% of patients prioritising trust in healthcare systems, one in four lack consistent treatment access and nearly one in three struggle with inadequate financial support, revealing fundamental gaps in healthcare security and trust for rare disease patients.
While 96% of patients consider knowledgeable medical specialists vital, nearly one in three face an information vacuum about their conditions and treatment options, with many discovering support networks years after diagnosis.
While 87% of rare disease patients consider social inclusion vital, critical gaps persist in psychological support (unmet for 41%) and community connection (unmet for 11%), with many patients facing isolation and lacking access to specialised mental health care that understands their unique challenges.
While nearly all patients (97%) emphasize the critical importance of being heard and believed by healthcare providers, significant gaps remain in shared decision-making, with one in three lacking access to second opinions and a quarter excluded from their own treatment planning, highlighting a persistent disconnect between patient expertise and healthcare delivery.
While over 80% of rare disease patients prioritize pursuing personal goals and interests, they face significant barriers in accessibility and support systems, from educational and workplace inclusion to basic infrastructure needs, with the constant burden of advocating for essential aids across different jurisdictions.
We surveyed 1,607 individuals, including 953 people living with rare diseases and 751 carers across five Nordic countries, revealing significant gaps between aspiration and reality.
The findings paint a stark picture: 95% of respondents call for improved collaboration between healthcare professionals, while 41% report unmet psychological support needs. Years of misdiagnosis or feeling dismissed have left deep gaps in confidence, with 28% reporting lack of access to medical specialists and 38% citing the absence of long-term treatment plans as their most pressing unmet need. People living with rare diseases often know more about their condition than those treating them, yet their expertise frequently goes unrecognised in treatment decisions. While patients overwhelmingly stress the importance of being heard and believed by healthcare providers (97%), significant gaps remain in shared decision-making, with one in three lacking access to second opinions and one in four excluded from their own treatment planning.
The emotional toll of living with a rare disease extends far beyond medical care into every aspect of daily life. Over 80% of patients prioritise the ability to pursue personal goals yet face persistent barriers. These challenges manifest in education, workplace inclusion, and even basic infrastructure needs. The impact on quality of life is significant, with 14% reporting their educational inclusion needs as unmet, 11% for the workplace. Trust in the healthcare system remains fragile, with 16% of respondents reporting a lack of confidence in their care providers.
Mental health support remains a critical gap in the current system. While the emotional burden of living with a rare disease affects both patients and carers profoundly, psychological support services are often out of reach or inadequate.
The survey captured insights from a substantial sample of 1,607 participants across the Nordic region, comprising 953 people living with rare diseases (PLWRDs) and 751 carers or parents. The response distribution demonstrated strong regional engagement, with Denmark contributing 788 respondents (448 PLWRD, 384 carers), Norway providing 237 respondents (178 PLWRD, 67 carers), Sweden representing 338 respondents (229 PLWRD, 138 carers), Finland adding 103 respondents (79 PLWRD, 33 carers), and Iceland contributing 123 respondents (7 PLWRD, 121 carers). To ensure accessibility and accurate representation, the survey was professionally translated into Danish, Icelandic, Norwegian, Swedish, and Finnish, with native speakers from patient organisations verifying the translations. The survey was conducted from May 6th to June 10th, 2025
Alexion: DK/UNB-U/0025 | Takeda: C-ANPROM/BE/NON/0022 | Sobi: NP-42181
