FLOWER Study Virtual Long-Term Data Collection on Rare Diseases Across the United States

What this Trial Is About

Researchers are conducting FLOWER, a fully virtual, nationwide observational study to collect and analyze clinical data from people with rare diseases. This study aims to gather detailed, standardized medical information in real time from patients with conditions affecting fewer than 200,000 individuals nationwide, including diseases like Alpha-Thalassemia, Amyloidosis, Duchenne Muscular Dystrophy, and others. The study is designed to overcome traditional research challenges by reducing burdens on patients and healthcare providers. Participants contribute by electronically consenting and allowing access to their medical records from various sources, including hospitals, clinics, pharmacies, and genetic testing vendors. Data collected include electronic medical records, imaging files, molecular and genetic information, and patient or caregiver-reported outcomes. The study staff standardize and code this information using recognized medical ontologies to create a comprehensive, longitudinal dataset without requiring data entry from treating physicians. During the study, researchers monitor several outcomes over five years, including overall survival, medication safety, developmental milestones, functional and motor abilities, and symptom changes. The study involves no direct interventions, focusing instead on gathering and analyzing real-world data. Participation is entirely remote and ongoing, with data collected continuously to provide a detailed picture of rare diseases natural history and progression.

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases