A Blueprint for Modern Rare Disease & Pediatric Trials

Gooseberry Research | 2025

1. Why Rare and Pediatric Trials Struggle With Enrollment

Rare-disease and pediatric studies consistently face:

  • Small and geographically scattered patient populations

  • High travel burden for medically fragile participants

  • Heavy caregiver time requirements

  • Limited site capacity and expertise

  • High early-dropout rates due to logistical burnout

Traditional site-centric models simply cannot reach enough qualified participants, nor keep them consistently engaged.

2. The Case for Decentralized & Hybrid Approaches

Decentralized trial (DCT) elements remove the barriers that historically block enrollment by:

  • Bringing study procedures to the participant

  • Reducing dependence on single geographic hubs

  • Easing caregiver burden

  • Increasing accessibility for medically complex or mobility-restricted families

  • Enabling participation from regions previously excluded from research

In rare and pediatric trials, the value is not theoretical - it directly determines feasibility.

3. The Gooseberry Model: In-Home Research Nursing at Scale

Gooseberry Research delivers decentralized trial execution through a trained, specialty in-home research nursing workforce.

Key elements include:

  • Pediatric-trained and rare-disease-trained nurses

  • Protocol-specific certification and competency assessment

  • In-home visits covering: sample collection, dosing, vitals, diaries, device setup, remote assessments

  • Caregiver training and study-equipment management

  • 24/7 on-call clinical support for families

  • HIPAA-aligned documentation and chain-of-custody procedures

This model is built to integrate smoothly with sponsors, CROs, and site networks.

4. Enrollment Impact: Removing Barriers at the Start

Gooseberry’s decentralized support improves recruitment by enabling families to join studies without:

  • Multiple long-distance site trips

  • Lost wages or childcare challenges

  • Repeated hospital visits for routine assessments

Families who would have declined participation often convert to enrolled candidates when the trial meets them at home.

5. Retention Impact: Keeping Participants Through Database Lock

Retention improves when families experience:

  • Fewer missed days of work or school

  • Stronger rapport with a dedicated home-health nurse

  • Simplified scheduling

  • Lower travel and lodging burden

  • Support for complex medical needs in a familiar environment

Consistent in-home engagement keeps families in the study through the final visit, reducing the risk of expensive protocol deviations and data gaps.

6. What DCT Looks Like in Practice: Example Visit Types

Core in-home procedures include:

  • PK/PD sample collection

  • Safety labs

  • IMP administration (where permitted)

  • Vitals, ECG, and anthropometrics

  • Clinical outcome assessments

  • Device training and troubleshooting

  • Caregiver-reported and patient-reported outcome coaching

  • Wearable and sensor deployment

  • Concomitant medication verification

Hybrid workflows combine in-home visits with periodic in-clinic visits for imaging, specialty assessments, or complex procedures.

7. Where Sponsors Gain Efficiency

Sponsors gain value through:

  • Faster recruitment timelines

  • Lower screen-fail and dropout rates

  • Fewer missed visits and deviations

  • More inclusive patient access

  • Reduced caregiver and participant burden

  • Increased confidence in data continuity across global regions

These gains translate to shorter timelines, cleaner datasets, and fewer rescue efforts.

8. Global Capability via Gooseberry + FutureMeds@Home

Gooseberry’s partnership with FutureMeds expands decentralized capabilities across 24 European research-home-health sites, supporting:

  • Cross-border rare-disease studies

  • Multi-regional pediatric trials

  • Long-term follow-up studies

  • Decentralized gene-therapy, neuromuscular, metabolic, and neurodevelopmental protocols

This is one of the only trans-Atlantic decentralized research nursing capabilities operating today.

9. Where DCT Works Best in Rare Disease

Decentralized support is most impactful in:

  • Pediatric neuromuscular disorders

  • Metabolic and endocrine conditions

  • Genetic neurodevelopmental disorders

  • Ultra-rare conditions with geographically dispersed families

  • Longitudinal follow-up phases

  • Studies with frequent safety checks or routine low-complexity procedures

Any protocol requiring frequent check-ins benefits materially from in-home operations.

10. Blueprint for Implementation: Step-by-Step

  1. Feasibility & protocol review
    Assess visit schedules, procedures, and geographic distribution needs.

  2. Country and state regulatory assessment
    Define what can be decentralized in each operating region.

  3. Site collaboration plan
    Integrate home-based visits with site workflows and PI oversight.

  4. Nurse recruitment & training
    Protocol-specific competency, device training, safety procedures, documentation.

  5. Logistics & supplies management
    Kit delivery, sample shipping, IMP coordination, cold-chain when required.

  6. Visit execution & real-time documentation
    Secure reporting through site-approved eSource or sponsor templates.

  7. Quality oversight
    Chart review, deviation prevention, safety monitoring, and audit readiness.

11. Summary

Decentralized trial solutions aren’t an add-on in rare and pediatric research - they’re critical infrastructure.
Bringing care to families rather than the other way around reduces burden, expands access, and keeps participants engaged to study completion.

Gooseberry Research offers an experienced, compliant, and scalable model that improves feasibility, enrollment, and data reliability across rare-disease and pediatric trials.

To explore whether Gooseberry can support your upcoming study, contact:
info@gooseberryresearch.com.

Or request a proposal