Dean Suhr, President and co-founder of the MLD Foundation, discusses newborn screening updates and plans for gaining recommended uniform screening panel (RUSP) approval.
As noted by Mr. Suhr, the recent termination of the Advisory Committee on Heritable Disorders in Newborns and Children by the Secretary of Health and Human Services has created some uncertainty in advocacy groups trying to get new genetic conditions on newborn screening panels. As Mr. Suhr explains, the metachromatic leukodystrophy (MLD) community, whose RUSP nomination was supposed to be up for voting this May, has had to switch strategies.
Contrary to the termination of the Advisory Committee, however, newborn screening is not going away. The oversight and services for newborn screening has been moved to the Agency for a Healthy America Maternal and Child Health bureau. While plans for a new advisory committee are not clear, Mr. Suhr stresses the importance of rebuilding a RUSP review committee.
The community remains hopeful that the removal of the Advisory Committee will lead to the rebuilding of anew and improved newborn screening advisory committee. By addressing previous issues and concerns, increasing the patient voice, and streamlining processes, this may be possible. Advocacy groups have plans to work in this direction, starting with a meeting to find solutions.
As for the MLD community, the MLD Foundation plans on bypassing the committee vote phase altogether and going straight to the Secretary of Health and Human Services with their information package. The Duchenne community is looking at a similar path, all in hopes of getting these two conditions on the newborn screening panel.
For more information on advocacy work in the rare disease space, visit https://checkrare.com/rare-disease-advocacy-2/