Real-world evidence in achondroplasia: considerations for a standardized data set.

ABSTRACT:

Background

Collection of real-world evidence (RWE) is important in achondroplasia. Development of a prospective, shared, international resource that follows the principles of findability, accessibility, interoperability, and reuse of digital assets, and that captures long-term, high-quality data, would improve understanding of the natural history of achondroplasia, quality of life, and related outcomes.

Methods

The Europe, Middle East, and Africa (EMEA) Achondroplasia Steering Committee comprises a multidisciplinary team of 17 clinical experts and 3 advocacy organization representatives. The committee undertook an exercise to identify essential data elements for a standardized prospective registry to study the natural history of achondroplasia and related outcomes.

Result

SUBMITTER: Alanay Y 

PROVIDER: S-EPMC10294372 | biostudies-literature | 2023 Jun

REPOSITORIES: biostudies-literature