CASE STUDY: Patient Recruitment in a Global Phase III Study on Hereditary Angioedema (HAE)
Executive Summary
How Stiris accelerated patient enrollment by applying a proactive recruitment strategy across North and Latin America.
Therapeutic Area: Rare Disease Immunology – Hereditary Angioedema (HAE)
Location: North and Latin America
Challenge: Enrollment was at risk due to the low prevalence of the disease, the availability of established standard-of-care treatments, and specific U.S. enrollment requirements set by the FDA.
Outcome: Stiris completed enrollment 30 days ahead of schedule, helped the sponsor meet regulatory enrollment targets in the U.S., and accelerated participant access to the open-label extension study.
Challenge
A global biotechnology company focused on rare disease therapies selected Stiris to lead study operations across North and Latin America for a Phase III clinical trial in HAE, a genetic condition with an estimated prevalence of 1:10,000 to 1:50,000 individuals.
The study included more than 20 sites across both regions. Recruitment presented three primary challenges:
- Limited eligible population, an inherent characteristic of rare diseases.
- Competition with standard of care: patients already had access to effective treatments, making it more difficult to decide to enroll in a clinical trial.
- Regulatory pressure: the sponsor was required to meet specific U.S. enrollment targets to satisfy FDA requirements.
Solution
Stiris implemented a comprehensive strategy built around four pillars:
- Multichannel Recruitment
Working closely with the sponsor’s advertising vendor, Stiris maximized study visibility to identify potentially eligible patients across both regions.
- Continuous Site Communication
The project management team, CRAs (Clinical Research Associates), and iCRAs (In-house CRAs) maintained frequent contact with investigators to monitor progress, detect barriers early, and respond quickly before they could affect timelines.
- Comprehensive Patient Support
Support services were implemented, including scheduling coordination and travel logistics assistance, reducing the burden of participation and promoting retention throughout the study.
- Flexible Management and Regional Coordination
The team’s ability to adapt quickly, combined with strong coordination between North and Latin America, ensured consistent execution across all regions in the face of the changes inherent to a global study.
Results
- Enrollment completed 30 days ahead of schedule
Early completion helped accelerate the overall development program timeline.
- U.S. regulatory enrollment targets met
The sponsor achieved the participant numbers required by the FDA through the strategies implemented by Stiris.
- Reduction in operational costs
Early enrollment completion generated direct cost savings without compromising the quality of clinical execution.
- Earlier access to the open-label extension
Eligible patients advanced more quickly to the extension phase, with the clinical benefits that this entails.
- Strengthened sponsor relationship
Responsive communication and consistent delivery of results consolidated the long-term relationship between both organizations.
Sponsor Impact
Stiris helped the sponsor overcome the inherent challenges of rare disease recruitment through a combination of operational expertise, consistent communication, and comprehensive support for both patients and sites.
The team’s ability to adapt quickly to the study’s evolving needs allowed them to accelerate enrollment, meet critical regulatory objectives, and optimize overall clinical development timelines.
Key Takeaways
- Recruitment in rare diseases demands proactive, tailored strategies with a strong capacity to respond to change.
- Frequent communication with sites allows teams to anticipate obstacles before they affect timelines.
- Patient support services meaningfully improve both participation and retention.
- Operational flexibility is a differentiating factor in global studies spanning multiple regions.
- Collaborative management strengthens the sponsor relationship and improves overall program outcomes.
Planning a Rare Disease Study?
Rare disease studies require more than operational expertise; they demand creativity, flexibility, and flawless execution.
Stiris Research helps sponsors optimize recruitment, enhance the patient experience, and meet critical clinical development objectives in complex and competitive programs.
Contact us at info@stirisresearch.com to learn how we can support your next study.