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RTI Fellow Don Bailey, Ph.D., makes the case that the current process does not adequately weigh benefits and harms
RESEARCH TRIANGLE PARK, N.C. — A new commentary published in JAMA Pediatrics suggests that the public health community should reconsider how it weighs benefits and harms when determining whether to add a new condition to newborn screening panels.
The piece, authored by RTI Fellow Don Bailey, Ph.D., analyzes the recent vetting process for Krabbe disease, a rare genetic disorder that requires treatment in the first few months of life to prevent death in children with the early infantile form. The Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) considered Krabbe for inclusion in the Recommended Uniform Screening Panel (RUSP), a list of disorders that the U.S. Department of Health and Human Services recommends for states to screen as part of their newborn screening programs.
Ultimately, the committee decided not to add Krabbe to the RUSP, sparking “frustration, dismay and anger” among advocates, Bailey says, and raising important questions as to the tipping point between “do not screen and screen.”
“The purpose of this commentary is to highlight the need to have serious discussions about how net benefit is defined,” said Bailey. “In the Krabbe case, committee members differed on opinions about benefits and harms, and as a result they also differed in their conclusions about net benefit."
In the piece, Bailey suggests that the Krabbe review process is a “harbinger of a future in which most screening decisions will be difficult and contentious.” Preparing for this scenario will require major changes to modernize the national newborn screening system, he says, while also noting that the responsibility and authority to reconsider “net benefit” falls to the ACHDNC.
The commentary includes three recommendations:
- The benefits of early intervention services and access to potentially lifesaving clinical trials made possible by screening are not currently part of the net benefit calculation but should be included.
- The current process weighs potential harm to families in the form of anxiety, stress and uncertainty but should also consider benefits to families, which can translate directly into benefits for the child.
- Strong evidence is required for benefits but not for harms. The same evidence should be required for both.
Bailey concludes that addressing these issues could “positively shape” the future of newborn screening and potentially be a model for other advisory groups.
