University of Michigan researchers going to use a $2.5 million grant from AHRQ to study how long-term health outcomes and cost-effectiveness applies to newborn screening. The project team will include investigators from a wide range of disciplines who have expertise in evidence synthesis, policy analysis for newborn screening programs, and have clinical expertise.
The study aims to provide comparative effectiveness evidence and cost-effectiveness results for newborn screening to look at medical disorders like Krabbe, Pompe, and Phenylketonuria.
• Krabbe disease is a degenerative disorder that affects the nervous system
• Pompe disease is an inherited disorder that disables the heart and skeletal muscles
• Phenylketonuria (PKU) is an inherited disorder that can cause intellectual and developmental disabilities if not treated.
There are costs and potential harms associated with doing screenings, follow-ups, and treatments therefore, it is important to recognize both the benefits and costs involved in screening newborns. The study’s results could impact lawmakers and their recommendations for the tests given to newborns in terms of potentially harmful disorders. According to study leader Dr. Lisa A.Prosser, PhD, “New technology has made it easier and less expensive to screen newborns for additional disorders.”
Prosser is a core faculty member of the University of Michigan’s Child Health Evaluation and Research (CHEAR) Unit that analyzes the healthcare system in terms of how to finance care for children. Since its inception, the CHEAR Unit has been awarded over $40 million in research grants from federal, state, and foundation sources.
The five year multidisciplinary study will use computer modeling to simulate clinical trials for newborn screening programs. By combining information from primary data collection, systematic reviews, published and unpublished data, and expert opinions; study investigators will be able to project long-term health and economic outcomes for conditions that can be detected by newborn screenings.
The project will also include an advisory committee to include representatives from state and federal agencies, clinical experts, and patient advocacy groups to insure policy relevance and provide recommendations for disseminating research findings.
