학술논문

Development of a Prospective Real‐World Data Clinical Registry of Children and Adolescents With Migraine.
Document Type
Article
Source
Headache: The Journal of Head & Face Pain. Feb2020, Vol. 60 Issue 2, p405-415. 11p. 2 Diagrams, 2 Charts.
Subject
*MIGRAINE prevention
*BENCHMARKING (Management)
*DATABASE design
*DATABASE management
*REPORTING of diseases
*DRUG labeling
*DRUG laws
*INFORMATION display systems
*LONGITUDINAL method
*CASE studies
*MEDICAL cooperation
*MEDICAL protocols
*MEDICAL technology
*MENTORING
*HEALTH outcome assessment
*PUBLIC health surveillance
*RESEARCH
*PATIENT participation
*GOVERNMENT regulation
*SOFTWARE architecture
*CLINICAL trial registries
*DATA quality
*ACQUISITION of data
*MOBILE apps
*STAKEHOLDER analysis
*ADOLESCENCE
*CHILDREN
Language
ISSN
0017-8748
Abstract
Objective: To develop a multicenter, multistakeholder, prospective clinical registry of children and adolescents with migraine to support the collection of real‐world data of sufficient quality to support regulatory submissions and provide site‐based infrastructure support for future clinical trials. Background: As new migraine treatments come to market, pediatric efficacy and safety trials of these agents are needed. A clinical registry is an ideal regulatory strategy to provide both real‐world data and site infrastructure to execute these trials. Design: Multicenter, multistakeholder, prospective real‐world data clinical registry of children and adolescents, 4‐17 years of age, diagnosed with migraine with or without aura. Participants will be followed for up to 12 months at 3‐month intervals, with interval recording of clinical data at study sites and self‐reported data via mobile health application, as well as biobanking. We developed electronic case report forms that incorporated routinely collected clinical data with National Institute of Neurological Disorders and Stroke Headache Common Data Elements (Version 2.0). All data are captured in a 21 CFR Part 11 – compliant electronic data capture system – augmented by a real‐time, web‐based, and customizable data visualization platform. We engaged vendors to provide ancillary biobanking, patient data entry, and data visualization services. Results: We used an iterative and highly collaborative multistakeholder approach to design and implement a streamlined registry protocol with input from all participating US sites. At each design and implementation step, we received input from therapeutic area experts, the US Food and Drug Administration (FDA), the National Institutes of Health, patient and parent advocates, health technology partners, drug developers, and site‐based clinical investigators. The registry is governed by a multistakeholder steering committee with representation from sites, industry partners, patient advocates, and a member from the FDA (non‐voting with respect to steering committee matters). The multistakeholder and site‐driven approach to registry design and execution was highly efficient and resulted in the first patient enrolled within 6 months of concept development. Conclusions: By ensuring regulatory compliant implementation of the registry, we created both a source of real‐world data and a multisite platform for the conduct of future clinical trials that can be submitted to regulatory authorities to support inclusion of pediatric data in approved drug labeling. A highly collaborative approach with broad stakeholder engagement at all stages of the registry development was a key to our operational success. [ABSTRACT FROM AUTHOR]