Economic Evidence for Decision Making in Pediatric Populations

Abstract

When a child is affected by serious or chronic illness, the illness and associated treatment process not only has significant impact on the child, but also on the child’s caregiver and other family members. Therefore, it is important to recognize and consider the effect of the illness and the corresponding support needed for the child as well as the child’s family. This dissertation provides economic evidence for three issues in the decision-making process of interventions and policies that would potentially support both the pediatric population and their families. The first chapter evaluated the long-term cost-effectiveness of newborn screening (NBS) and high costs treatment of spinal muscular atrophy (SMA) in the U.S. Recently available treatments for SMA, such as nusinersen (drug) and onasemnogene abeparvovec-xioi (gene therapy), carry high costs which raises the question of whether screening for SMA will be a policy that is considered economically favorable. A state-transition model was developed to compare strategies: (1) NBS and gene therapy, and (2) NBS and drug treatment, and (3) clinical identification and drug treatment. When incorporating the costs and health outcomes from both individuals with SMA and their caregiver, the results showed that when compared with clinical identification strategies, NBS with gene therapy, onasemnogene abeparvovec-xioi, had an incremental cost-effectiveness ratio of $103,669/quality-adjusted life-years (QALY), which is considered favorable under conventional willingness-to-pay thresholds, while NBS with drug treatment, nusinersen, did not. The second chapter estimated the budget impact of NBS and treatment for SMA from the perspective of the Medicaid/Children’s Health Insurance Program (CHIP) program, as they are most likely to support the high-cost treatments of SMA. Using Michigan as an example, a budget impact analysis was carried out using a developed state-transition model and a spreadsheet model. Compared with clinical identification and drug treatment (nusinersen), the budget impact on the Medicaid/CHIP program for the strategy of NBS with gene therapy (onasemnogene abeparvovec-xioi), started at an estimated of $12 million in the first year and decreased to $7 million in the fifth year. Over a five-year period, the budget impact is projected to be $48 million, with a potential range between $21 million to nearly $100 million using alternative assumptions. The third chapter explored what are the most important items when measuring family spillover effects for children with complex chronic conditions (CCC) from an economic perspective, with the goal of developing a core outcome set for measuring family spillover effects for children with complex chronic illness. This study followed the methodology developed by the COMET (Core Outcome Measures in Effectiveness Trials) Initiative, and conducted a literature review, best-worst scaling survey, and stakeholder meetings. The best-worst scaling pilot survey of 30 respondents showed that, overall, items related to quality of life and informal caregiving time were chosen most frequently as having relatively higher impact on families of children with CCC, while items related to costs were chosen less frequently. The items that were chosen most frequently as most impact include: “Quit jobs or did not pursue a job in order to care for the child”, followed by “Caregivers’ quality of life” and “Family member’s quality of life”.