Presentation (CTI)

OBJECTIVES: Patient retention is challenging in non-site-based longitudinal cohort studies, which often lack the direct oversight typical of site-based or traditional controlled trials. Retention is essential to ensure data integrity, reduce bias and produce robust results. Challenges related to patient retention in rare disease settings include small populations, disease-specific factors and survey fatigue. Guideline recommendations focus on personalised communications (e.g., emails, SMS, phone calls), incentives, and patient association group involvement as strategies for improved retention. The objective is to assess follow-up strategy effectiveness in non-site-based rare disease studies.
METHODS: A review of retention guidelines and best practices was conducted to assess reminder strategies for non-site-based studies. Attention was given to challenges such as survey fatigue, disease-specific symptom burden, and the timing and frequency of follow-ups critical for maintaining cohort integrity. Based on this assessment, retention strategies were developed for our prospective non-site-based studies in rare disease therapeutic areas which customized programmed reminders designed around distinct populations and longitudinal timelines. Retention rates were monitored to evaluate the effectiveness of these approaches.
RESULTS: A structured retention approach was applied across multiple studies. In rare disease studies, recruitment and retention were particularly challenging due to low prevalence and clinical characteristics. Tailored reminders were implemented with consideration for data timepoints, participant burden, disease progression and mortality. For instance, in cohorts of patients with Gaucher disease or narcolepsy, reminders were adapted to fatigue patterns or parallel reminders to caregivers for patients relying on them for study participation. Results demonstrated the importance of automated reminders sensitive to disease characteristics. While some strategies improved retention, varying success levels were achieved from setting up reminders to improve patient retention in certain cohorts.
CONCLUSIONS: Tailored reminder strategies that address population-specific challenges effectively enhance retention in non-site-based longitudinal studies, support better data quality, and underscore the need for adaptable approaches.