Cost-effectiveness analysis of neonatal screening of critical congenital heart defects in China

Abstract

Background:Pulse oximetry screening is a highly accurate tool for the early detection of critical congenital heart disease (CCHD) in newborn infants. As the technique is simple, noninvasive, and inexpensive, it has potentially significant benefits for developing countries. The aim of this study is to provide information for future clinical and health policy decisions by assessing the cost-effectiveness of CCHD screening in China.Methods and Findings:We developed a cohort model to evaluate the cost-effectiveness of screening all Chinese newborns annually using 3 possible screening options compared to no intervention: pulse oximetry alone, clinical assessment alone, and pulse oximetry as an adjunct to clinical assessment. We calculated the incremental cost per averted disability-adjusted life years (DALYs) in 2015 international dollars to measure cost-effectiveness. One-way sensitivity analysis and multivariate probabilistic sensitivity analysis were performed to test the robustness of the model. Of the three screening options, we found that clinical assessment is the most cost-effective strategy compared to no intervention with an incremental cost-effectiveness ratio (ICER) of Int$5,728/DALY, while pulse oximetry plus clinical assessment with the highest ICER yielded the best health outcomes. Sensitivity analysis showed that when the treatment rate increased up to 57.5%, pulse oximetry plus clinical assessment showed the best expected values among the three screening options.Conclusion:In China, for neonatal screening for CCHD at the national level, clinical assessment was a very cost-effective preliminary choice and pulse oximetry plus clinical assessment was worth considering for the long term. Improvement in accessibility to treatment is crucial to expand the potential health benefits of screening.

The Methodological Quality and Challenges in Conducting Economic Evaluations of Newborn Screening: A Scoping Review

“…After duplicate removals, 716 articles were considered eligible for screening by title and abstract. After full-text screening, 35 articles were included for the final assessment (see Appendix C for the PRISMA flow diagram) [ 23 , 24 , 25 , 26 , 27 , 28 , 29 , 30 , 31 , 32 , 33 , 34 , 35 , 36 , 37 , 38 , 39 , 40 , 41 , 42 , 43 , 44 , 45 , 46 , 47 , 48 , 49 , 50 , 51 , 52 , 53 , 54 , 55 , 56 , 57 ]. No additional studies were found on the websites of the INAHTA HTA agencies.…”

“…Some researchers explicitly stated that QALYs would have been a preferred measure, but insufficient parameters on the quality of life led to the use of LYs gained [ 32 ]. Some concerns associated with choosing a specific health measure were related to the loss of informative aspects on potential health benefits: for example, in the study by Tobe et al, the choice of “DALYs averted” as the main outcome measure did not allow incorporating information related to other potential health benefits, such as morbidities avoided in the long term when quality weights cannot be collected [ 42 ]; the use of the number of correctly detected cases of hearing loss was preferred over the outcome measures on language and speech development in children, which might have been more informative, in [ 41 ]; and Hatam et al did not include the spill-over effects on patient’s families [ 55 ], even though this could have increased the health benefits of executing the investigated NBS program. Overall, the exclusion of health outcomes was mainly driven by insufficient information and substantial uncertainty on long-term outcomes [ 36 ].…”

“…All authors including sensitivity analyses in their studies specifically stated that this was to account for the uncertainties of the parameters included in the model. In addition to this reason, Tobe et al used sensitivity analysis to accommodate for the geographical and socioeconomic diversity within a target country [ 42 ]. Several EEs also used additional methods to explore other types of uncertainty.…”

“…The authors of the few studies presenting generalizability and transferability issues considered different elements to address these items. While most of the researchers considered the difference in population and health care systems as the main obstacle to generalizability [ 24 , 26 , 28 , 52 , 56 ], they also used sensitivity analysis to enhance the generalizability of the results [ 24 , 52 ] and called for further research to increase their transferability [ 26 , 27 , 30 , 42 ]. Distributional issues mainly considered the limited access to service (geographically and/or logistically) as the “bottleneck” for scaling-up the investigated NBS intervention [ 50 ].…”

Screening for congenital heart defects: diversified strategies in current China

“…In China, the cost-effectiveness of postnatal oximetry screening compared with no intervention varied across regions 36. Under base-case assumptions, clinical assessment is a very cost-effective preliminary choice for neonatal screening of CHD at the national level 37. The intervention is highly cost-effective in the development regions than that in the unprivileged regions.…”