Development

A domain-level approach was selected to begin populating the Rare Disease COA Resource. The first iteration of the Rare Disease COA Resource focuses on the assessment of daily function in pediatric, non-oncologic populations. This overarching domain was broken down into the daily function subdomains of self-care, gross motor function, fine motor function, and communication. Other subdomains will be added over time.

Inclusion of COAs in the Resource

A Rare Disease COA Resource Development Subcommittee was formed to select COAs for inclusion in the Rare Disease COA Resource. Members of this subcommittee included representatives from sponsor firms with expertise in the domains of interest, FDA colleagues, academic partners with clinical expertise, and COA development experts. Advisory Panels were convened to review the results of gap analyses and selected COAs. Panel membership for the daily function subdomains of self-care, gross motor function, and fine motor function consisted of an Occupational Therapist, Physical Therapist, Caregiver Representative, and for Communication/Language an Occupational Therapist, Speech and Language Pathologist, and Caregiver Representative.

A consensus process between each Advisory Panel and the Rare Disease COA Resource Development Subcommittee determined the final COAs to include in the Rare Disease COA Resource.

FDA funding disclosure

Specific funding for the establishment of the Rare Disease COA Consortium was provided by FDA grant U01FD006882.