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Cross-border initiative to empower rare disease researchers

There are as many as 7,000 rare diseases affecting 360 million people around the globe. Clinical trials are essential to develop treatments for rare diseases, but they often face challenges related to small patient populations and limited resources. By their nature, rare diseases trials will only have a limited number of potential participants and those participants are often widely dispersed. Cross-border enrollment, where patients from different countries participate in clinical trials conducted outside their home country, is a potential solution to overcome these challenges. 

 

Rare disease research requires uncommon approaches

Rare diseases are characterised by small patient populations and limited treatment options. This poses significant challenges for clinical research and drug development. Traditional clinical trial recruitment strategies may not be feasible due to the scarcity of eligible participants within a single geographical area or country. Cross-border enrollment presents a promising approach to address these challenges. By recruiting patients from multiple countries to participate in clinical trials it expands the pool of participants and improves trial feasibility.

While all clinical trials require a collaborative approach, cross-border clinical trials come with additional collaborators in multiple locations. The combined efforts of multiple stakeholders, including researchers, regulators, industry partners, and patient advocates, are crucial to harness the full potential of cross-border enrollment in rare diseases clinical trials. Ultimately those trials may lead to improve outcomes for patients with rare diseases.

 

European cross-border initiative

As a company dedicated to clinical studies of rare diseases, we recognise the importance of cross-border enrollment in clinical studies. ICON experts are actively involved in the EU-X-CT European initiative which was established to enable cross-border access to trials for patients when there is no option to join a clinical trial in their own country. The European Forum for Good Clinical Practice (EFGCP) and European Federation of Pharmaceutical Industries and Associations (EFPIA) set up a multistakeholder collaboration platform. This allows cross-border collaboration between patient organisations, academic researchers and institutions, research networks, CROs and pharmaceutical companies. Its aim is to systematically collect sparse information on the requirements, options and obstacles to cross-border participation in clinical trials from all European countries within, and outside, the European Union. Based on a gap analysis, EU-X-CT will develop multistakeholder recommendations for different aspects of cross-border access to clinical trials. It will provide all collected information for each country on a publicly available website.

 

Our involvement

ICON’s Bojana Mirosavljevic, Patient Advocacy Strategy Directo, Joanna Sprague, Regulatory Affairs Associate Director, and Helena Luning, Senior Manager, Study Start Up Site Activation are actively involved in this initiative and will contribute their considerable expertise and spread awareness about this important topic. The ICON team also participated at the recent EU-X-CT Public Stakeholders' Forum 2024 which took place in Brussels on 12 April 2024. This forum presented a great opportunity to jointly review the national conditions for patients, clinical investigators, academia and public funders and industry sponsors in need to cross-border participation of patients in clinical trials. 

By pooling resources and information, this Europewide initiative has plenty of potential benefits. For researchers it opens the door to a wider pool of potential clinical trial participants and collaborators. For patients with rare diseases, it may offer the hope of improved outcomes and new treatment options sooner.

Download our whitepaper on cross-border enrollment of rare disease patients. 

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