The RealiseD project launched a new multi-stakeholder survey that invites the rare disease community to share insights and help improve clinical trial enrolment

New survey launched to improve rare diseases clinical trial enrolment

Clinical trials are essential to advancing new therapies; however, recruiting participants for rare and ultra‑rare conditions remains a significant challenge. The RealiseD project is launching a survey that seeks to identify and understand the factors that influence enrolment in rare disease clinical trials. This consultation invites health care professionals, academia, industry representatives, regulators, trial sponsors and patient advocates to share their perspective on barriers and opportunities and contribute to shaping a more inclusive and efficient future for rare disease research. The online survey, which can be accessed through this link, is divided into three sections: 

  • The first explores trial site and structural factors, looking at logistics, staffing, and data-sharing tools that impact accessibility.  
  • The second focuses on trial design and operational factors, including aspects such as trial location, duration, and participant experience.  
  • The third section delves into patient-level and disease factors, examining how age, treatment options, and disease severity influence participation. 

All responses are strictly anonymous, and only aggregated data will be shared—individual answers will never be disclosed. Insights from multiple stakeholders will help us understand the structural, operational, and patient-level factors that impact enrolment and create solutions that make trials more inclusive, patient-centric and effective. By participating, respondents can play a vital part in advancing enrolment strategies for rare and ultra-rare disease trials worldwide.  

This consultation process will close on 28 January 2026.

About RealiseD

RealiseD (compRehensive mEthodological and operational Approach to cLinical trIalS in ultra rarE Diseases) is an Innovative Health Initiative (IHI) project focused on developing and supporting methodological and operational approaches for clinical trials in rare and ultra‑rare diseases, including the design, analysis and interpretation of data from very small patient populations. An overview of the project’s scope is available via the IHI project factsheet for RealiseD.
ERDERA (the European Rare Diseases Research Alliance) works to strengthen rare disease research across Europe, including through its Clinical Research Network and a strong focus on meaningful patient engagement (Patient and Public Involvement and Engagement (PPIE)). RealiseD’s work aligns with these themes and, according to the IHI project factsheet, RealiseD’s tools and systems are intended to be tested and initiated within ERDERA.

News & Updates

You might also be interested in

Publicaciones (1)

EU rare disease action plan- European Added Value Assessment

This study investigates possible measures that could be taken at EU level to address these challenges. It finds significant European added value in harmonising coordination and access across the 27 Member States, mainly in terms of improved diagnostic tools and availability of medical treatment, better health outcomes, particularly lower infant mortality, and improved well-being of family members and caregivers.
Rare Disease Day 2026 visual representation

One year of ERDERA: the European alliance built to accelerate rare disease research reports first results

28 February, across Europe and beyond: one year into delivery, ERDERA is advancing towards shorten diagnostic journeys and improved therapies for people living with a rare disease.
the power behind erdera2 (1)

Undiagnosed Day 2026 | Where Diagnosis Becomes Hope: The Power of Collaboration and Technology in Rare Diseases

This meeting will focus on practical approaches to phenotyping and diagnosis in undiagnosed conditions, including how to define next steps when a diagnosis remains uncertain, and how to strengthen pathways and collaboration around undiagnosed care.
Genetic Mapping

ERDERA unites 10 000 rare disease datasets in a single, secure European resource

In its first year, ERDERA has brought together 10 000 harmonised genomic and phenotypic datasets from unsolved rare disease cases across Europe, creating a secure, standardised and scalable resource.

ERDERA's

search form