Universal Newborn Cystic Fibrosis Screening Pays Off

NORWICH, England, April 6 -- Routine screening of newborns for cystic fibrosis is cost effective, found researchers here.

The cost of therapy for children eventually diagnosed clinically with cystic fibrosis was 60% to 400% more than for those diagnosed by newborn screening, said Erika J. Sims, Ph.D., of the University of East Anglia, and colleagues.

These treatment savings alone would offset 60% to 73% of screening program costs in a conservative estimate, they wrote in the April 7 issue of The Lancet.

Previous studies have tied early treatment as a result of screening by age two months to better growth, less need for long-term therapies, and even better cognitive development.

In 2004, the CDC in Atlanta recommended that states add the test to their existing panel of newborn screening tests. At least 27 states have done so already with another four to seven likely to begin soon. In England, where the cost-effectiveness was evaluated, CF screening of newborns is not routine.

"If clear clinical benefit does not always persuade governments to implement screening, cost benefits might," the investigators wrote. "The costs of screening are an important part of such decision making."

The study included children ages one to nine years old with CF identified by screening within two months of birth (184) or after clinical presentation without screening (950).

The researchers estimated yearly costs of long-term and nebulized therapies and intravenous antibiotics based on data in the United Kingdom Cystic Fibrosis Database from 2000 to 2002. The cost estimates for CF screening included staff, overheads, and consumables used in adding the test to Scotland's established newborn screening program for phenylketonurea and congenital hypothyroidism.

Among the findings, Dr. Sims and colleagues reported:

• For mixed CF genotype patients, clinical diagnosis was associated with 400% higher median treatment costs than with screening (,442 versus , P<0.0001).
• For homozygous genotype patients, the difference was smaller but still significant (median ,516 versus ,090, P=0.0001).
• Adjusting for Pseudomonas aeruginosa infection status, clinically diagnosed patients received significantly more treatment than those identified by screening (heterozygous ,126 more, P=0.02, homozygous ,739, P=0.009).

Overall, an estimated 34% to 121% of newborn screening costs would be offset by lower treatment costs. The estimated annual screening cost would be ,971,551 for the entire United Kingdom whereas the median cost saving for treatment would be ,001,326 with a mean of ,601,744.

Using a more conservative model that took into account infants who present clinically prior to screening or who have a rare genotype not picked up by the screening assay, the mean cost saving would still have been ,791,198 to ,156,940 (60.2% to 72.6% offset).

Furthermore, the estimated cost savings in 2002 alone would have offset 100% of the estimated costs of implementing the program.

Adding indirect costs from time invested in care by parents and caregivers and days of school missed would likely have further increased the cost saving, the researchers said.

However, they acknowledged there is substantial variation in cost of the screening assay used. In their study, the 31-DNA mutation screen cost .44, which was double the .66 reported for the single-DNA mutation test used by Wisconsin, for instance.

The study is among the first to look at cost-effectiveness rather than cost-comparisons between methods, according to an editorial by Bridget Wilcken, M.B., Ch.B., and Kevin Gaskin, M.D., both of the Children's Hospital at Westmead and University of Sydney in Australia.

In their editorial, Drs. Wilcken and Gaskin cautioned that other treatment costs, such as those for inpatient care, were not included in the analysis because they are not captured in the database used.

Nonetheless, "Sims' findings probably apply widely outside the United Kingdom," they noted.

The economic, clinical, and social evidence weighs "unreservedly" in favor of international adoption of universal newborn screening programs for CF, the researchers concluded.

"Even if cost savings do not offset the total costs of a screening program, improvements in health resulting from the program justify implementation, if the ratio of costs and benefits are within an acceptable margin," Dr. Sims and colleagues wrote.