Funding Decisions for Newborn Screening: A Comparative Review of 22 Decision Processes in Europe

Abstract

Decision-makers need to make choices to improve public health. Population-based newborn screening (NBS) is considered as one strategy to prevent adverse health outcomes and address rare disease patients’ needs. The aim of this study was to describe key characteristics of decisions for funding new NBS programmes in Europe. We analysed past decisions using a conceptual framework. It incorporates indicators that capture the steps of decision processes by health care payers. Based on an internet survey, we compared 22 decisions for which answers among two respondents were validated for each observation. The frequencies of indicators were calculated to elicit key characteristics. All decisions resulted in positive, mostly unrestricted funding. Stakeholder participation was diverse focusing on information provision or voting. Often, decisions were not fully transparent. Assessment of NBS technologies concentrated on expert opinion, literature review and rough cost estimates. Most important appraisal criteria were effectiveness (i.e., health gain from testing for the children being screened), disease severity and availability of treatments. Some common and diverging key characteristics were identified. Although no evidence of explicit healthcare rationing was found, processes may be improved in respect of transparency and scientific rigour of assessment.

Current status of newborn screening worldwide: 2015

“…While each is different, public healthcare systems have defined mechanisms to assess and appraise payment for NBS and most allow for diverse stakeholder participation when considering technology effectiveness, disease severity, and treatment availability. 80 Hearing screening and CCHD screening are usually organized and financed separately from NBS. 81 As in other countries, there has been continuing interest in the storage and use of residual NBS.…”

The Use of Economic Evaluation to Inform Newborn Screening Policy Decisions: The Washington State Experience

“…However, the contrast between the United States and other countries may be more apparent than real. A recent review concluded that the United States is not less likely than Canada to consider economic evaluation in informing health policy decisions, and European countries in general have not used cost‐effectiveness as a criterion for NBS expansions …”

“…A recent review concluded that the United States is not less likely than Canada to consider economic evaluation in informing health policy decisions, 92 and European countries in general have not used cost-effectiveness as a criterion for NBS expansions. 34,35 This article advances the understanding of the use of economic evaluation in US NBS policymaking by documenting and critically evaluating the experience of one state health department. The Washington experience demonstrates that an economic evaluation is feasible, at least for assessing and valuing avoided deaths.…”

“…Although the cost of testing is almost always listed as a criterion, no consensus exists regarding the net balance of costs and benefits . In a sample of 22 European NBS policy decisions, costs were almost always considered, but in only 4 cases were CEAs conducted . Similarly, US NBS policy decisions typically consider the cost of testing but not cost‐effectiveness or cost‐benefit .…”

“…33 In a sample of 22 European NBS policy decisions, costs were almost always considered, but in only 4 cases were CEAs conducted. 34,35 Similarly, US NBS policy decisions typically consider the cost of testing 4,13 but not cost-effectiveness or cost-benefit. 29 The ACHDNC is directed by the US Congress to consider "cost" for a disorder proposed for the RUSP, 36 which can encompass costs incurred by the public health and clinical systems, including short-term follow-up and data systems to track children and assess long-term outcomes.…”