Patient-Reported Experience Measures for People Living with Rare Diseases (RD-PREM)

Why the RD-PREM Project Matters

Rare diseases pose unique challenges:

• Low prevalence and fragmented expertise across healthcare systems
• Limited access to coordinated care
• Barriers to consistent patient feedback collection

Despite progress through the European Reference Networks (ERNs)—collaborative hubs for rare disease care involving over 300 hospitals—there remains no validated PREM framework that can be used across all rare diseases.

RD-PREM fills this gap, enabling healthcare systems to:

• Systematically measure patient experience
• Compare care across regions and conditions
• Improve healthcare quality, governance, and accountability

Project Goals

We aim to develop validated, accessible RD-PREM questionnaires that:

1. Enable adult rare disease patients to self-report their healthcare experiences
2. Allow family members/carers to report experiences on behalf of patients unable to self-report
3. Develop child-friendly versions for patients under 18

Project Methodology

The project employs a participatory, multiple-method, and multi-phase approach. This ensures diverse and continuous involvement from:

• People living with rare diseases
• Family carers and advocates
• Clinicians and hospital administrators
• Researchers and policymakers

To maximise reach and inclusion, we use both in-person and online engagement, leveraging EURORDIS’ established platforms for survey distribution and expert recruitment.

Acknowledgments

The RD-PREM Project is made possible by the Health Research Board and the passionate efforts of our research partners, clinical collaborators, patient advocates, and—most importantly—individuals living with rare diseases who share their stories and insights to shape a better future for all.