Rare disease trials require extraordinary solutions.

Achieve your outcomes with our proven rare disease methodology.

In your next study, leverage our agile rare disease team’s decades of combined experience addressing the nuances of clinical development in rare indications. Our full-service solutions are personalized, flexible, and built to balance your needs with those of your patients, patient communities, and stakeholders.

Want a CRO partner who gives you their full attention?

You’re in the right place.

Let’s Talk

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Therapeutic Experts

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Countries, All Regions

There is no one-size-fits-all in rare disease.

Every indication and patient population is unique. We’ve collected a wealth of diverse experience across indications and cultivated a proven ability to apply lessons learned into strategies for your unique study.

Our expertise is diverse.

We’ve worked on rare disease studies across all therapeutic areas, from metabolic to neurology and oncology. With our wide range of experience, we have the ability to support your study no matter how rare your indication.

Patients are at the core of what we do.

We’re passionate about making trials work for patients and their families to drive better trial experiences and, ultimately, better trial outcomes.

: Every patient counts when it comes to achieving statistical significance. That’s why our solutions are built to access and retain patients throughout the study. Whether you’re looking to work with new sites, utilize cross-border enrollment, or explore new pathways to identify patients, we have you covered.
: We’re constantly exploring new and better ways to develop rare disease trials. We partner with patient advocacy organizations around the world, have specialized partnerships with patient registries, and partner with other likeminded organizations to achieve better treatment options for patients.

: We’re skilled at supporting endpoint selection and development to ensure data quality, and our in-house clinical assessment team has robust experience with clinical assessments management and training.
: With the increased complexity of rare disease research, you can trust we have you covered. Our teams are well-versed in trial logistics, operational solutions, and regulatory engagement, and our scientific and medical expertise is unmatched. No matter the support you need, our experts are accessible, and our senior leadership is available.

Our Experts

Derek Ansel, MS, LCGC

Global Vice President, Therapeutic Strategy, Rare Disease and Oncology

Amy Raymond, PhD, PMP

Executive Director, Therapeutic Strategy Lead, Rare Disease & Cellular and Genetic Medicines

Juliane K. Mills, MS, MPH

Executive Director, Therapeutic Strategy Lead, Rare Disease

Nathan Chadwick

Senior Director, Therapeutic Strategy Lead, Rare Disease

Don’t just take our word for it. Hear what our sponsors have to say:

I liked that they customized the rare disease study team. I liked that they’re thinking about a strong cultural match because often that’s not thought about. That’s attractive to our small biotech company because that’s the way we work, too.

VP of Clinical Science Small Rare Disease Biotech

They (Worldwide) were the best combination of cost, expertise, and fit from a rare disease project management perspective. And one of the other important pieces is the KOL and patient advisory board coordination…Worldwide saying that they actually do the KOL and advisory board coordination is a big selling point. That tells me that they know who the experts are, they work with them, and they can coordinate these advisory boards for us.

Head of Program Operations Small Rare Disease Biopharma

I think the way Worldwide focuses the activity on a rare disease study is quite interesting because they know how to manage this type of study and how to obtain or to recruit rare patients. [They] have access to registries, [can] discuss with patient communities, [and] have access to key opinion leaders.

Director of Project Management Small Rare Disease Biotech

They’ve got the right people in rare disease. They understand the complexities of doing work in rare diseases relative to other diseases that they’ve worked on for decades. They have senior scientific experts to work on the programs… and they give customers access to what they need.

Senior Director of Clinical Operations and Development Rare Disease Biotech

I think they offer good customer experience and service… Very good scientific knowledge from their rare disease team. They have an extensive patient recruitment process, which is really good, and they have access to a large and specific population. They are easy to work with.

Head of QA Small Clinical-Stage Biopharmaceutical in Rare Disease

Searching for the right CRO partner to help with your clinical development needs?

At Worldwide Clinical Trials, we deliver high-quality, reliable, and timely clinical trial services to help optimize the success of your projects. For the second year in a row, data from the Industry Standard Research (ISR) report showed that Worldwide Clinical Trials was the highest-rated Phase II/III CRO across several categories.

Over 80% of respondents agreed that midsize CROs provide a strong cultural fit while being able to pivot quickly based on changing customer needs.

Read the full report here