A pilot program funded by the state of California showed that precision medicine for critically ill babies enrolled in California’s Medicaid program reduced their suffering and yielded better health outcomes, while decreasing the cost of their healthcare, saving the Golden State $2.5 million.
The pilot program, Project Baby Bear, was launched in 2018. Project Baby Bear used rapid Whole Genome Sequencing (rWGS) provided by Rady Children’s Institute for Genomic Medicine (RCIGM) to analyze the genetic code of 178 infants whose families are enrolled in the California Medical Assistance Program (Medi-Cal).
The genomic testing provided doctors and families with diagnostic information that enabled them to make medical decisions that led to shorter hospital stays, fewer invasive procedures, and targeted personalized care.
RCIGM is within Rady Children’s Hospital-San Diego, the nation’s sixth largest children’s hospital and one of five hospitals with California Children’s Services accredited regional (Level IV) neonatal and pediatric intensive care units (ICUs) that participated in the pilot program statewide.
The other four hospitals were: CHOC Children’s—Children’s Hospital of Orange County in Orange, CA; University of California, Davis Children’s Hospital in Sacramento, CA; University of California, San Francisco Benioff Children’s Hospital in Oakland, CA; and Valley Children’s Hospital in Madera, CA.
“Project Baby Bear has demonstrated that rapid sequencing in newborns save lives and is actually cost effective, and has shown how next generation sequencing has revolutionized the process of making complex diagnoses in pediatric medicine in a matter of days,” said Katherine A. Rauen, MD, PhD, chief of genomic medicine at UC Davis Children’s Hospital and co-lead of the UC Davis site for Project Baby Bear.
The 178 babies enrolled across all five sites nearly doubled the state-mandated genome sequencing requirement of 100 babies in the pilot program’s appropriation of $2 million under California’s 2018-19 state budget.
According to a Final Report submitted to the California Department of Health Care Services and released yesterday by Rady Children’s, Project Baby Bear provided diagnoses for 76 babies (43%) of those tested with rWGS. The testing led to a change in the management of 55 babies (31%) that resulted in fewer hospital days, fewer procedures or new therapies.
A total 35 rare conditions that occur in less than 1 in 1 million births were diagnosed. Project Baby Bear helped doctors identify the exact cause of those rare genetic diseases in an average of three days, rather than the four to six weeks offered by standard genetic testing.
“Project Baby Bear significantly shortened the time needed to accurately diagnose and optimally treat these critically ill children,” Stephen Kingsmore, MD, DSc, President and CEO of RCIGM, said in a statement.
rWGS permitted doctors to discharge babies sooner—513 fewer hospital days—and reduce the number of procedures that may have been performed in the absence of a precise diagnosis, Rady reported. As a result, California saved $2.5 million in healthcare spending.
Among the avoided procedures, the report stated, were 11 major surgeries—including a major reconstructive surgery on the upper airway and a bowel surgery—and 16 fewer diagnostic tests including open muscle, liver and other biopsies that are performed under general anesthesia.
“To successfully provide rapid precision medicine for California’s most vulnerable children, the program depended on the participation of multidisciplinary teams integrated within each of the five hospital systems. These included medical doctors, genetic counselors, nurses, laboratory personnel, IT personnel and supportive staff,” the report stated. “It was this coordinated system of care coupled with a rapid test turnaround time that led to the project’s dramatic success.”
Project Baby Bear enjoyed in-kind contributions totaling more than $400,000 in the form of programmatic support from Rady Children’s and RCIGM. That allowed 90% of the $2 million budgeted for the pilot program to go directly to supporting the care and management of critically ill babies, Rady said in the report.