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Voice of the patient

How ExtendMed Helped Imbue Partners Capture Rare Disease Patient Voices for Regulatory Success

ExtendMed partnered with Imbue Partners to support a biotech’s FDA submission for a rare leukodystrophy treatment, using the Health Expert Connect™ platform to gather patient and caregiver insights through forums, surveys and interviews.

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At a glance

INDUSTRY
Life Sciences – Rare Disease

AUDIENCE
Patients and caregivers affected by CSF1R/ALSP

CHALLENGE
Capturing FDA-ready patient experience data in a rare, cognitively impairing disease

SOLUTION
Multi-modal, asynchronous engagement combining forums, surveys, and interviews

RESULTS
Regulatory-grade data, improved participation and unexpected community building

What Imbue needed

  • A flexible, accessible platform to engage patients and caregivers affected by a rare, progressive disease
  • Tools to separate patient and caregiver discussions for honest, burden-of-care reporting
  • Support for validated clinical assessments (CATS, NPI-12, CBFS)
  • Asynchronous participation options to accommodate cognitive and physical limitations
  • Secure data capture aligned with FDA patient experience data guidance
  • A cost-effective model suitable for smaller biotechs

What ExtendMed delivered

  • A tailored Health Expert Connect™ environment supporting separate patient and caregiver forums
  • Integrated online surveys using validated instruments
  • Scheduling and management for flexible one-on-one interviews
  • Secure, compliant data collection suitable for regulatory submission
  • A moderated, asynchronous structure allowing participation across disease phases
  • Affordable access for smaller biotechs through ExtendMed’s inclusive pricing model

The challenge

Imbue Partners faced a critical mission: capture the lived experiences of patients and caregivers with CSF1R/ALSP to support an FDA submission for a first-of-its-kind therapy. The disease’s rapid cognitive decline and emotional intensity made traditional research impossible.

They needed a method to:

  • Reach dispersed families with varied technological abilities
  • Gather validated clinical and qualitative data across multiple modalities
  • Comply with FDA’s “Voice of the Patient” documentation standards
  • Enable honest, unfiltered input from caregivers and patients alike

ExtendMed’s solution

ExtendMed’s Health Expert Connect™ platform was configured for a three-part research model:

  1. Asynchronous Discussion Forums: Separate spaces for patients and caregivers encouraged open sharing and minimized emotional bias.
  2. Validated Surveys: Clinical assessments provided quantifiable, directional data aligned with trial metrics.
  3. Flexible Interviews: Short sessions accommodated participants’ cognitive and emotional needs.
This asynchronous model met participants where they were—geographically, physically, and emotionally—while capturing rich, actionable insights. Even non-technical caregivers were supported through personalized onboarding, ensuring no voice went unheard.

The results

  • FDA-ready data: Comprehensive documentation of disease burden, supporting expedited review
  • Methodological innovation: A replicable model for patient experience research in rare diseases
  • Community impact: Participants reported feeling seen, connected, and supported
  • Expanded access: Smaller biotechs gained a cost-effective path to conduct sophisticated research
  • Ongoing replication: Imbue and ExtendMed now plan to repeat the model across additional therapeutic areas

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