Looking for cross-border clinical trials?

In the Global Access section of this platform, patients with rare diseases, their care teams, and stakeholders dedicated to expanding global research access will find information on the importance of cross-border trials—and guidance on navigating and accessing rare disease clinical trials worldwide.

Our mission is to break down participation barriers and advance healthcare equity across the globe. Please use the bookmarks bar above to explore more detailed resources.

Cross-border clinical trials essential for the treatment of rare diseases

For the vast majority of rare diseases, no approved treatments exist. Clinical trials and patient registries are essential for rare diseases because they

🔬 Are often the only route to discovering, testing, and approving treatments for

rare diseases.

👀 Help define how the disease behaves, identify biomarkers for diagnosis and

treatment, and establish clinical outcomes to evaluate—crucial for future

diagnostics and care of frequently under-researched diseases.

💊 Offer the only access to potentially life-saving therapies for many patients.

💡 Contributes to broader discoveries, including repurposing existing drugs or

identifying new therapeutic targets.

Hence, clinical trials are not only a pathway to therapies but are foundational to understanding these diseases, accelerating innovation, and providing hope to patients whom traditional research models have long neglected.

Because each rare disease impacts only a small number of patients—often dispersed across multiple countries—conducting

robust, timely, and inclusive clinical trials

in such conditions requires cross-border collaboration. Cross-border clinical trial participation is not a luxury—it's a necessity in rare disease research. It enables equity, accelerates innovation, and ensures

no patient is left behind due to geography.

The practice of traveling to a country to seek specialized medical diagnosis and interventions is called Medical Tourism. For rare diseases, medical tourism through cross-border clinical trial participation can accelerate trial completions and increase the inclusion of diverse patients, thereby increasing confidence in the relevance of the trial data.1

Practical tips for finding cross-border clinical trials and resources

🔗 Connect with patient advocacy groups focused on the disease of interest or rare

diseases, such as the National Organization for Rare Disorders (NORD). They are

often a rich source of information on available resources, including clinical trials.

🤝 Collaborate with your medical team to determine whether a clinical trial is

appropriate and to help identify relevant trials and support services for

participation.

🖥️ Leverage technological advancements, such as AI tools, with guidance from the

care team to explore treatment options and stay informed.

🔍 Check searchable websites that list clinical trials—both in general and specifically

for rare diseases—and provide information on how to participate (for country-

specific details, refer to the dedicated country pages linked here).

Some of the key resources include:

Orphanet: A European reference portal for rare diseases and orphan drugs, Orphanet provides detailed information on clinical trials specific to rare conditions. It includes both ongoing and completed interventional studies across various countries.
EU Clinical Trials database: Website that supports the running of clinical trials for human medicines in the European Union (EU) and European Economic Area (EEA)
The RD-Connect Registry: An integrated platform connecting databases, registries, biobanks and clinical bioinformatics for rare disease research.
World Health Organization International Clinical Trials Registry Platform (ICTRP): The ICTRP aggregates data from multiple registries worldwide, offering a global perspective on clinical trials, including those targeting rare diseases. It facilitates searching for interventional studies across different regions.
The U.S. ClinicalTrials.gov: A website and online database of clinical research studies and information about their results with a purpose to provide information about clinical research studies to the public, researchers, and health care professionals. Database includes studies that take place in all 50 states and over 200 countries. The U.S. government does not review or approve the safety and science of all studies listed on this website.
Australian Clinical Trials
National Organization for Rare Disorders (NORD): Maintains a curated list of clinical trials for various rare diseases, with a primary focus on studies conducted in the United States, and provides information on interventional trials and guidance for patient participation.
Rare Diseases Europe (EURORDIS): A non-profit alliance of over 1000 rare disease patient organisations from 74 countries that work together to improve the lives of over 30 million people living with a rare disease in Europe.

An example of global map of the distribution of all rare disease clinical trials registered on the ClinicalTrials.gov in 2021

Map source: Building cross-border collaborations to increase diversity and accelerate rare disease drug development – meeting report from the inaugural IndoUSrare Annual Conference 2021Therapeutic Advances in Rare Disease October 2022 DOI:10.1177/26330040221133124

1Rare Diseases at FDA access date: 4-13-2025.

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