cystic fibrosis 

Digital observational study exploring unmet need and treatment impact in genetic disorder

Flexible patient-centric study demonstrating real-world impact of therapy in late-stage development

  Client success story

Vitaccess has leveraged its innovative research platform to assist a US-based biopharmaceutical company in generating evidence on CF treatment impact in the real world.

  Case study by the numbers

participants recruited over 18 months

57% of participants completed surveys at final 18-month data entry point


consented to contact about future research


    • A US-based biopharmaceutical company approached us about an observational patient- and caregiver-reported outcomes study to complement the phase III clinical trial of their treatment for CF.
    • The client required a flexible study exploring the longitudinal impact of treatment initiation on patients and caregivers in the real world.


    • The study was to describe the impacts of CF on both patients and caregivers before and after treatment initiation, alongside exploring longitudinal trends in cases where patients were already receiving treatment.
    • The study was to deliver a broad patient reach, rapid recruitment, and quick data collection.
    • Study development was to be flexible to incorporate patient cohorts of varying ages at appropriate time-points leading up to market authorization.

  Key deliverables

  • Observational longitudinal patient- and caregiver-reported outcomes study, collecting data on the impact of CF and its treatment in the UK and Ireland.
  • Co-creation with key opinion leaders from each scope country.
  • Ethical approval by relevant review boards.
  • Bespoke data capture over 18-month study period via Vitaccess Real digital platform.
  • Combination of patient and caregiver demographic profiles alongside PRO, caregiver-reported outcome, and single-item measures to comprehensively assess the impact of CF and its treatment.
  • Flexible implementation of a shift in recruitment: initially, adult patients or caregivers alongside patients aged 12–17; subsequently, the addition of caregivers of patients aged 6–11.
  • Presentation of interim analysis at a regional conference with manuscript development in pipeline.

  Client benefit

    • Insights into the impact of treatment initiation in CF to complement phase III clinical trial data.
    • Generation of longitudinal RWE from a broad patient and caregiver population, quickly and at a low cost.
    • Large pool of patients and caregivers providing consent to contact about future research opportunities, thus speeding up recruitment in subsequent studies.
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