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Rare Disease Clinical Development at Worldwide

Rare is extraordinary. Explore what our unique rare disease clinical development methodology, passion for empowering rare disease communities, and advancing treatments can do for your rare disease trial.

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Learn about our rare disease clinical development services

Every patient in a rare disease trial is important to us.

We’re ready to take your rare disease therapeutic into the next phase, bringing it one step closer to the patients and families waiting. Through our innovative partnership with patient-led organizations and communities across the globe, we harness the power of shared goals to develop patient-partnered and caregiver-driven solutions that drive success in your rare disease trial.

Rare is different. So is your study.

Patient- & Caregiver-Driven Solutions

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Our Rare Disease Clinical Development Services

For specific information about how we can support your study, get in touch

We’re continuously expanding our services and exploring innovative new solutions to the challenges rare disease trials face. From endpoint selection to patient-optimized protocols, we bring the accessible, global expertise needed to supplement your team’s needs.

Advocacy Engagement

  • Partnered with more than 115+ patient organizations
  • Focus groups and panels
  • Satisfaction Surveys
  • Patient & caregiver interviews
Learn more about how we partner with patient organizations

Patient-Focused Solutions

  • Patient & caregiver strategy
  • Patient education & transparency
  • Burden assessments
  • Outreach & collaboration

Differentiated Partnerships

  • More than 115+ patient organizations
  • Like-minded nonprofit partnerships through Rare Matters Worldwide
Driving new treatment options through EveryCure

Genetic Screening & Referral Network Partnerships

  • Access to global genetic testing laboratories
  • Medical geneticists & genetic counselors
  • Coordination of Rare Diseases Sanford (CoRDS) Registry
  • Participation with International Conference on Newborn Sequencing (ICoNS)

Clinical Development Plans

  • Endpoint selection
  • Regulatory engagement
  • Access & reimbursement

Regulatory Guidance & Planning

  • Global regulatory expertise
  • Established agency relationships
  • Application support
Learn more about our regulatory expertise

Clinical Operations & Logistics

  • Operational Phase I-IV strategy
  • Preferred rare disease-focused vendor partnerships
  • Experienced and tenured project teams
Cross-border enrollment

Real-World Evidence

  • Operational expertise in registries and NHS natural history studies
  • Access to medical claims through sister company, Trinity Analytics
  • Global operational strategy for access to historical medical records
Learn more about our RWE expertise

Cellular & Genetic Medicines

  • Modality-specific approach
  • Adult & pediatric experience across phases and LTFU
  • Diverse routes of administration
Learn more about our expertise in cell and gene therapies

Clinical Assessment Training and Surveillance (CATS) Solutions

  • Endpoint adjudication
  • Mitigation of placebo effect
  • COA/eCOA data capture
  • Licensing and translations
Learn more about our CATS solutions

Cell & Gene Therapies

  • Complex cell & gene therapy programs
  • Nurtured site relationships
  • Modality-specific, strategic approach
Learn more about our cell & gene therapy capabilities
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We bring an unwavering commitment to personalized attention.

Diverse & Accessible Expertise

We’ve collected a wealth of experience across indications and are well-versed in the complexities of rare disease research from trial logistics and regulatory engagement to scientific and medical expertise.

Patient-Informed & Caregiver Driven Solutions

We work in rare diseases because we are inspired by the work the patient communities have done for rare diseases, and we’re passionate about bringing them into the clinical research conversation.

See our awards

Our Rare Methodology

Our seasoned strategic rare disease team partners with your team, bringing our in-house scientific experts, rare disease community partnerships, and KOL relationships to offer integrated solutions for your study that address the needs of all your stakeholders.

See what our clients have to say

Your experience is important to us.

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.

Small Rare Disease Biotech VP of Clinical Science

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.

Small Rare Disease Biopharma Head of Program Operations of Clinical Operations

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.

Small Biopharmaceutical Customer Director

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.

Rare Disease Biotech Senior

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.

Small Clinical-Stage Biopharmaceutical in Rare Disease Head of QA

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