University of Michigan Researchers to Study Health Outcomes and Economics of Newborn Screening

Researchers will use computer modeling and patient surveys to study health and economic outcomes for screening of Krabbe disease, Pompe disease, and phenylketonuria

ANN ARBOR, Mich., March 1, 2012 /PRNewswire-USNewswire/ – University of Michigan researchers will use a $2.5 million grant from the Agency for Healthcare Research and Quality to study long-term health outcomes and cost-effectiveness of newborn screening.

This study, led by Dr. Lisa A. Prosser, Ph.D., M.S., could impact lawmakers' recommendations on panels of tests given to newborns for potentially harmful disorders.   

The five-year, multi-disciplinary study will use computer modeling to simulate clinical trials of newborn screening programs. The study aims to provide comparative effectiveness evidence and cost-effectiveness results for newborn screening for three rare, but potentially disabling or fatal, conditions:

• Krabbe disease, a degenerative disorder that affects the nervous system
• Pompe disease, an inherited disorder that disables the heart and skeletal muscles
• Phenylketonuria (PKU), an inherited disorder that can cause intellectual and developmental disabilities if not treated

"New technology has made it easier and less expensive to screen newborns for additional disorders," says Prosser, the principal investigator on the project, associate professor of pediatrics and a core faculty member of the CHEAR Unit.

"However, there are costs and potential harms associated with screening follow-up and treatment. It's important to recognize both the benefits and costs of newborn screening programs. Decisions about new conditions for mandatory screening should also reflect public opinion for public health priorities," says Prosser, who is also an associate professor of health management and policy at the U-M School of Public Health.

The study will also conduct a survey of public values for newborn screening programs. This information will be of direct interest to state and national policy makers when considering new conditions for recommended newborn screening panels.

By combining information from primary data collection, systematic reviews, published and unpublished data, and expert opinion, study investigators will project long-term health and economic outcomes for conditions that can be detected by newborn screening.

The project team will include investigators from a wide range of disciplines who have expertise in evidence synthesis, policy analysis of newborn screening programs, and clinical expertise. The project will also include an advisory committee that includes representatives from state and federal agencies, clinical experts, and patient advocacy groups to insure policy relevance and provide recommendations for disseminating research findings.

Prosser's research often focuses on comparative and cost-effectiveness of children's health interventions. She is a member of the external evidence review group for the Advisory Committee on Heritable Disorders in Newborns and Children. She also holds adjunct faculty appointments at Harvard Medical School and the Harvard School of Public Health.

Co-investigators on this project include Beth Tarini, M.D., M.S. (University of Michigan), Wen Ye, Ph.D. (University of Michigan), Alex Kemper, M.D., M.P.H. (Duke University), James Perrin, M.D. (Massachusetts General Hospital), and Janice Bach, M.S. C.G.C. (Michigan Department of Community Health).

About the U-M CHEAR Unit: Founded in 1998, U-M's Child Health Evaluation and Research (CHEAR) Unit is a national leader in the analysis of the American health care system and the organization and financing of 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 CHEAR Unit comprises core faculty from the Schools of Medicine, Social Work, Dentistry, Pharmacy, Nursing, Public Health, Business and Law. Multidisciplinary teams provide the structure for research of community, state and national child health policies, practices, and programs. There are more than 30 faculty affiliated with CHEAR.

SOURCE University of Michigan Health System