Recruiting caregivers in ultra-rare diseases is one of the biggest challenges in healthcare research. When patient populations are small, caregivers are overburdened, and traditional recruitment methods fall short, even the most important research questions can go unanswered.
This was the case for a client seeking to better understand caregiver perspectives on emerging treatments for Rett Syndrome, an extremely rare neurodevelopmental disorder affecting fewer than 10,000 individuals in the United States.
The Challenge: Limited Reach in an Ultra-Rare Population
The client wanted to explore caregivers’ attitudes, unmet needs, and barriers related to new and emerging Rett Syndrome treatments. However, reaching this audience proved difficult.
Prior to engaging Cadence, the client had attempted a caregiver survey but was only able to recruit 75 respondents—a common limitation in rare disease research, where caregivers are difficult to identify, highly selective about participation, and often fatigued by repeated research requests.
The core challenge wasn’t interest—it was access.
The Cadence Approach: Rethinking Rare Disease Recruitment
Recognizing the unique hurdles of rare disease caregiver recruitment, Cadence implemented a proprietary recruitment strategy inspired by the logistics and rigor of advisory board development, combined with targeted use of social media.
Rather than relying on traditional panels or passive outreach, this approach focused on:
- Meeting caregivers where they already engage
- Building trust through clear communication and study transparency
- Creating an opt-in pathway for ongoing participation, not just a single survey
This methodology was designed not only to answer the immediate research question, but to build a sustainable caregiver resource for future studies.
The Results: Scale, Engagement, and Long-Term Value
Over a two-month period, Cadence successfully surveyed 815 U.S.-based caregivers of children diagnosed with Rett Syndrome.
Importantly, caregivers were given the opportunity to opt in to future research:
- 587 caregivers consented to being contacted for additional studies
Cadence then conducted a follow-up survey with 442 Rett Syndrome caregivers, diving deeper into additional topics and offering caregivers the option to receive communications about available and emerging Rett treatment options.
The response was overwhelmingly positive.
The Outcome: A Living Caregiver Panel
As a result of this multi-phase effort, Cadence has now developed a contact list of 500+ Rett Syndrome caregivers who are:
- Highly engaged
- Interested in ongoing research participation
- Open to receiving information about treatment options and innovations
This caregiver panel represents a powerful, long-term asset—enabling faster, more meaningful research while reducing recruitment friction for future studies.
Why This Matters
In rare diseases like Rett Syndrome, success isn’t just about hitting a recruitment number—it’s about building relationships, trust, and infrastructure that can support research over time.
This case study demonstrates how thoughtful, customized recruitment strategies can dramatically expand reach, deepen insight, and create lasting value for both sponsors and the patient community.
At Cadence, we believe rare disease research requires rare precision—and this project is a powerful example of what’s possible when recruitment is reimagined.
