The Committee received feedback from families and clinicians that the process of nominating a condition for the RUSP is difficult and burdensome, and that it does not consider some factors important to families. After thoughtful deliberation, the Committee decided to update the nomination and evidence review processes.
Consistent with previous practice, the Committee decided to pause on accepting new condition nominations for six months (from December 2023 to May 2024). Pausing nominations provides a uniform standard path for all subsequent nominations to follow, instead of some nominations following one process while other nominations follow a different process.
During the six-month pause, HRSA staff and the Committee Chair will remain available to provide technical assistance to potential nominators on core elements that are needed for nominations, such as the need for published data on the newborn screening test, confirmation test, short- and long-term follow-up, treatment, and utility of identification pre-symptomatically versus through clinical ascertainment.
For updates on the Committee’s progress and how to participate, please be sure to check the meeting summaries for the next two meetings taking place in January and May 2024.
Previously submitted nominations that are in evidence review will follow the current process (Krabbe disease and Duchenne Muscular Dystrophy). Nominations submitted after the six-month pause will follow the new process.