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Finding the Few: Accelerating Clinical Research for HAE
When time-critical research meets an ultra-rare disease population, traditional recruitment approaches fall short. This was the exact challenge facing a leading pharmaceutical company seeking to advance a groundbreaking treatment for Hereditary Angioedema (HAE) – a rare condition affecting just one in 50,000 Americans.
The mission was complex: identify and enroll 80 patients who had experienced documented HAE attacks in the previous six months, spread across 25 research sites nationwide, all within a 12-month window. Adding to the challenge, these participants needed to meet stringent inclusion criteria for a Phase III clinical trial that could potentially transform HAE treatment.
By combining sophisticated digital targeting with deep community engagement, we didn’t just meet our goals – we surpassed expectations, completing enrollment ahead of schedule while setting new sponsor benchmarks for efficiency and cost-effectiveness.
This is the story of how precision strategy, authentic patient engagement, and innovative technology came together to accelerate the path to a potential breakthrough treatment.
The 1-in-50,000 Challenge: Racing to Advance HAE Options
Support Rapid Patient Identification
Find and engage 80 qualified HAE patients across the United States, focusing specifically on those with documented attacks in the past six months. Time was of the essence – our 12-month window demanded precision targeting and swift action.
Optimize Site-Level Recruitment
Strategically direct potential participants to 25 research sites nationwide, ensuring efficient distribution of candidates and maximizing each site’s enrollment potential.
Accelerate Screening-to-Enrollment Pipeline
Develop a pre-qualification strategy that would identify the most promising participants early, streamlining the path from initial contact to successful patient enrollment.
Maintain Cost Efficiency at Scale
Despite the rare disease challenge, execute a recruitment strategy that would deliver qualified participants while maintaining reasonable cost-per-enrollment benchmarks.
Build Trust Within the HAE Community
Create authentic connections with both patients and advocacy groups, recognizing that in rare disease communities, trust and credibility are paramount to success.
A Multi-Channel Strategy for Rare Disease Trial Success
Digital Innovation Meets Community
Connection We engineered a precision-targeted digital ecosystem that went beyond traditional patient recruitment methods. By crafting custom audience profiles based on HAE- specific indicators and advocacy connections, we could identify potential participants with unprecedented accuracy.
Building Trust Through Advocacy Partnerships
Recognizing that the HAE community is tight-knit and trust-dependent, we forged strategic alliances with the US Hereditary Angioedema Association (HAEA) and other key advocacy groups. These partnerships became the cornerstone of our outreach, providing direct access to engaged patients while building crucial credibility.
Patient-First Communication Strategy
Our messaging focused on what mattered most to HAE patients:
+ Clear explanation of the treatment’s potential impact
+ Comprehensive support including travel assistance
+ Access to dedicated study coordinators
+ Streamlined study participation process
Real-Time Optimization Engine
We built a sophisticated patient recruitment pipeline that included:
+ Custom-built online pre-screening portal for rapid candidate evaluation
+ Geographic targeting aligned with 25 study sites
+ Dynamic performance analytics driving continuous optimization
+ Agile resource allocation based on channel effectiveness
Delivering 85 Randomized Patients at 30% Lower Costs
Unprecedented Speed to Full Enrollment
In a field where every day counts, we didn’t just meet enrollment goals – we exceeded them, completing recruitment a full month ahead of schedule. This acceleration put life-changing research on a faster track to potential breakthrough treatments.
Record-Breaking Performance Metrics
+ Surpassed study enrollment goal with 85 randomized patients
+ Generated 250+ qualified pre-screened referrals
+ Achieved 27% qualification-to-randomization rate
+ Delivered 30% cost savings per randomized patient
Channel Performance Analysis
Our strategic multi-channel approach proved transformative:
+ Social media outreach: 45% of enrolled patients
+ Advocacy partnerships: 35% of enrolled patients
+ Physician referrals: 20% of enrolled patients
The success of our digital-first strategy, combined with deep community engagement, demonstrates a new model for rare disease trial recruitment. This approach didn’t just f ill enrollment – it created a blueprint for accelerating research while maintaining rigorous qualification standards and cost efficiency.
Beyond the numbers, these results represent something more significant: proof that with the right strategy, even the most challenging rare disease clinical trials can achieve exceptional recruitment success.
Innovation in Action: Elevating Clinical Trial Recruitment Standards
This innovative campaign demonstrates that even in the challenging landscape of rare disease trials, strategic innovation can dramatically accelerate patient recruitment. By combining precision digital targeting with authentic community engagement, we didn’t just meet enrollment goals – we redefined what’s possible in rare disease clinical trial recruitment.
The results speak volumes: full enrollment one month ahead of schedule, costs 30% below industry benchmarks, and a qualification rate that exceeded expectations. But beyond these metrics lies a more significant achievement: we’ve established a repeatable model for rare disease trial recruitment that balances speed, precision, and cost-efficiency while maintaining the highest standards of patient qualification.
Most importantly, this success advances our ultimate mission: accelerating the development of breakthrough treatments for patients who need them most. Every day saved in recruitment is a day closer to potentially life-changing therapies for the HAE community.
This case study proves that with sophisticated strategy, deep patient understanding, and innovative execution, we can overcome even the most challenging recruitment barriers – setting new standards for what’s possible in patient recruitment.
Impact at a Glance
+ Full enrollment achieved 1 month ahead of schedule
+ 85 patients randomized, exceeding 80-patient target
+ 250+ qualified referrals generated
+ 30% below industry cost benchmarks