Eurordis - Rare Disease Europe

Cite this report

This report presents the results of the H-CARE Pilot Survey for the four European Reference Networks (ERNs) that initated this study conducted by Rare Barometer: ERN GENTURIS, ERKNet, ERN Lung and eUROGEN.

The goal of this study was to test the feasibility of measuring rare disease patients’ and carers’ experience with the care delivered by healthcare providers who are part of an ERN. A Topic Expert Committee composed of patient representatives, clinicians and managers from ERNs GENTURIS, ERKNet, Lung and eUROGEN selected a patient reported Experience Measure (PREM) questionnaire validated for chronic conditions, the Patient Assessment of Care for Chronic Conditions Short Form (PACIC-S). This questionnaire was administered by Rare Barometer, with the participation of hospital units that were members of the four ERNs of the pilot.

This report presents the results of the pilot study for the 1,319 patients and carers who assessed European healthcare providers treating rare diseases affecting kidneys, lungs, the urogenital area and genetic tumour risk syndromes:

• Patients had a better healthcare experience when they were treated by hospital units that are part of a ERN.
• The PACIC-S showed good internal consistency and good construct validity in German, French, English and Spanish. But content validity was low as all aspects of care experience for rare diseases were not covered by the scale, showing that the PACIC-S did not fully capture the healthcare experience of people living with a rare disease.
• Recommendations for the development and validation of a new scale for RDs include planning onsite distribution of paper questionnaires in specialised hospital units, in addition to online distribution towards patient organisations and on social media, in order to reach a sufficient number of respondents for each ERN and language.

Jessie Dubief, EURORDIS-Rare Diseases Europe (survey design, statistical analyses, report writing)

Rare Barometer is a not-for-profit survey initiative run independently by EURORDIS-Rare Diseases Europe.