The primary goal of this project is to identify, document, and disseminate the top patient-centered comparative clinical effectiveness research (CER) topics and research priorities of the achondroplasia community, while strengthening meaningful community engagement in research. It brings together adults with achondroplasia, parents of children with achondroplasia (including parents with and without achondroplasia), clinicians, and researchers. This approach is designed to ensure diverse representation of a broad range of community and professional priorities through facilitated conversations.

This project is crucial because perceptions about quality of life and care priorities of individuals with achondroplasia are often informed by research involving a relatively small subset of participants enrolled in pharmaceutical trials in which the primary endpoint is increased height. Quality of life research is typically conducted with and funded by the pharmaceutical industry, which may bias the research and distort broader community experiences and perspectives. This project intentionally includes individuals with varied experiences, such as those who have pursued pharmaceutical interventions as well as those who have pursued medical, social, and community-based supports and services. In doing so, the project seeks to support a more comprehensive, balanced, and inclusive research agenda.