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.
In this section
-
Digital Disruption
- AI and clinical trials
-
Clinical trial data anonymisation and data sharing
-
Clinical Trial Tokenisation
-
Closing the evidence gap: The value of digital health technologies in supporting drug reimbursement decisions
-
Digital disruption in biopharma
-
Disruptive Innovation
- Remote Patient Monitoring
-
Personalising Digital Health
- Real World Data
-
The triad of trust: Navigating real-world healthcare data integration
-
Patient Centricity
-
Agile Clinical Monitoring
-
Capturing the voice of the patient in clinical trials
-
Charting the Managed Access Program Landscape
-
Developing Nurse-Centric Medical Communications
- Diversity and inclusion in clinical trials
-
Exploring the patient perspective from different angles
-
Patient safety and pharmacovigilance
-
A guide to safety data migrations
-
Taking safety reporting to the next level with automation
-
Outsourced Pharmacovigilance Affiliate Solution
-
The evolution of the Pharmacovigilance System Master File: Benefits, challenges, and opportunities
-
Sponsor and CRO pharmacovigilance and safety alliances
-
Understanding the Periodic Benefit-Risk Evaluation Report
-
A guide to safety data migrations
-
Patient voice survey
-
Patient Voice Survey - Decentralised and Hybrid Trials
-
Reimagining Patient-Centricity with the Internet of Medical Things (IoMT)
-
Using longitudinal qualitative research to capture the patient voice
-
Agile Clinical Monitoring
-
Regulatory Intelligence
-
An innovative approach to rare disease clinical development
- EU Clinical Trials Regulation
-
Using innovative tools and lean writing processes to accelerate regulatory document writing
-
Current overview of data sharing within clinical trial transparency
-
Global Agency Meetings: A collaborative approach to drug development
-
Keeping the end in mind: key considerations for creating plain language summaries
-
Navigating orphan drug development from early phase to marketing authorisation
-
Procedural and regulatory know-how for China biotechs in the EU
-
RACE for Children Act
-
Early engagement and regulatory considerations for biotech
-
Regulatory Intelligence Newsletter
-
Requirements & strategy considerations within clinical trial transparency
-
Spotlight on regulatory reforms in China
-
Transfer of marketing authorisation
-
An innovative approach to rare disease clinical development
-
Therapeutics insights
- Endocrine and Metabolic Disorders
- Cardiovascular
- Cell and Gene Therapies
- Central Nervous System
-
Glycomics
- Infectious Diseases
- NASH
-
Oncology
- Paediatrics
-
Respiratory
-
Rare and orphan diseases
-
Advanced therapies for rare diseases
-
Cross-border enrollment of rare disease patients
-
Crossing the finish line: Why effective participation support strategy is critical to trial efficiency and success in rare diseases
-
Diversity, equity and inclusion in rare disease clinical trials
-
Identify and mitigate risks to rare disease clinical programmes
-
Leveraging historical data for use in rare disease trials
-
Natural history studies to improve drug development in rare diseases
-
Patient Centricity in Orphan Drug Development
-
The key to remarkable rare disease registries
-
Advanced therapies for rare diseases
-
Transforming Trials
-
Accelerating biotech innovation from discovery to commercialisation
-
Ensuring the validity of clinical outcomes assessment (COA) data: The value of rater training
-
Linguistic validation of Clinical Outcomes Assessments
-
Optimising biotech funding
- Adaptive clinical trials
-
Best practices to increase engagement with medical and scientific poster content
-
Decentralised clinical trials
-
Biopharma perspective: the promise of decentralised models and diversity in clinical trials
-
Decentralised and Hybrid clinical trials
-
Practical considerations in transitioning to hybrid or decentralised clinical trials
-
Navigating the regulatory labyrinth of technology in decentralised clinical trials
-
Biopharma perspective: the promise of decentralised models and diversity in clinical trials
-
eCOA implementation
- Flexible delivery models
-
Implications of COVID-19 on statistical design and analyses of clinical studies
-
Improving pharma R&D efficiency
-
Increasing Complexity and Declining ROI in Drug Development
-
Innovation in Clinical Trial Methodologies
- Partnership insights
-
Risk Based Quality Management
-
Transforming the R&D Model to Sustain Growth
-
Accelerating biotech innovation from discovery to commercialisation
-
Value Based Healthcare
-
Strategies for commercialising oncology treatments for young adults
-
US payers and PROs
-
Accelerated early clinical manufacturing
-
Cardiovascular Medical Devices
-
CMS Part D Price Negotiations: Is your drug on the list?
-
COVID-19 navigating global market access
-
Ensuring scientific rigor in external control arms
-
Evidence Synthesis: A solution to sparse evidence, heterogeneous studies, and disconnected networks
-
Global Outcomes Benchmarking
-
Health technology assessment
-
Perspectives from US payers
-
ICER’s impact on payer decision making
-
Making Sense of the Biosimilars Market
-
Medical communications in early phase product development
-
Navigating the Challenges and Opportunities of Value Based Healthcare
-
Payer Reliance on ICER and Perceptions on Value Based Pricing
-
Payers Perspectives on Digital Therapeutics
-
Precision Medicine
-
RWE Generation Cross Sectional Studies and Medical Chart Review
-
Survey results: How to engage healthcare decision-makers
-
The affordability hurdle for gene therapies
-
The Role of ICER as an HTA Organisation
-
Strategies for commercialising oncology treatments for young adults
-
Blog
-
Videos
-
Webinar Channel