Recruitment procedures for descriptive socio-economic studies in rare diseases. The BURQOL-RD project

Abstract

Background: Patient recruitment is a barrier that limits the power and validity of research findings in rare diseases (RD) research. We report the recruitment strategy carried out for the BURQOL-RD project in eight European countries with their corresponding results to gather socio-economic and health-related quality of life data of patients with 10 RD and their caregivers.

Method: First, literature related to recruitment strategies for RD was reviewed, with a focus on descriptive studies and clinical trials. Second, the recruitment strategy for BURQOL-RD project was designed.

Results: Findings from the literature showed that the most common recruiting strategies were the use of clinical and research networks and patient organizations (POs). The main BURQOL-RD recruitment strategy was driven, in every participant country, by the corresponding National Federation of RD by means of PO registries, e-mail and Internet-based questionnaires; providing a total of 3233 valid responses for the 10 selected RD in all participant countries.

Conclusions: Our results provide inputs to improve the planning of recruitment activities in RD research. Internet-based questionnaires managed by POs using e-mail proved to be a useful strategy for patient recruitment.

Keywords:

• participation
• rare diseases
• recruitment strategies
• socio-economic studies

Acknowledgments

For their valuable assistance in the design and recruitment, the authors wish to thank Federación Española de Enfermedades Raras (FEDER), Spain; Federazione Italiana Malattie Rare (UNIAMO), Italy; National Alliance of People with Rare Diseases (NAPRD), Bulgaria; Allianz Chronishcer Seltener Erkrankungen (ACHSE), Germany; Alliance Maladies Rares, France; Rare Diseases Sweden (Sällsynta Diagnoser), Sweden; Hungarian Federation of People with Rare and Congenital Diseases – Rare Diseases Hungary (HUFERDIS), Hungary; Rare Diseases UK and Centro Estatal de Atención a Personas con ER y sus Familias de Burgos (CREER), Spain.

Declaration of interest

This project was funded by the 2nd Programme of Community Action in the Field of Public Health, promoted by the Executive Agency for Health and Consumers. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.