International Journal of Technology Assessment in Health Care


The role of health technology assessment in coverage decisions on newborn screening

Katharina E. Fischera1, Scott D. Grossea2 and Wolf H. Rogowskia3

a1 Helmholtz Zentrum München, German Research Center for Environmental Health, Institute of Health Economics and Health Care Management

a2 U.S. Centers for Disease Control and Prevention

a3 Helmholtz Zentrum München, German Research Center for Environmental Health and Ludwig–Maximilians–University


Objectives: The role and impact of health technology assessment (HTA) in health policy has been widely discussed. Researchers have started to analyze how decisions on coverage of new technologies are made. Although the involvement of HTA may be an indicator of a well established decision process, this hypothesis requires validation. Also, it is not known whether HTA involvement is associated with other characteristics of decision making like participation or transparency. The primary objective of this study was to develop and test statements on the association between the publication of an HTA and coverage decision making for newborn screening tests in European Union countries.

Methods: Five statements were defined on the relative role of HTA during the steps of decision processes: trigger, participation, publication, assessment, and appraisal. For this purpose, data on twenty-two decision processes in the area of newborn screening across Europe were analyzed, defined as a coverage decision for a given disorder in a specific country. Decision processes were compared by whether the decision was accompanied by the publication of an HTA report. To test differences, nonparametric statistical tests were used.

Results: The decision steps of trigger, participation and publication differed between the HTA and the non-HTA groups. No clear association between HTA and assessment methods in coverage decision making was identified.

Conclusions: It appeared that there is an association between HTA and coverage decision processes that are more explicit, inclusive, and transparent. It is unclear whether HTA is associated with formal evidence reviews and economic evaluations.

(Online publication September 29 2011)


The authors thank Jürgen John for valuable comments on earlier versions of this article, Matthias Hunger and Petra Menn for comments on statistical analysis, and Werner Maier for support with contacting respondents in French. Furthermore, the authors thank Gerard Loeber from the International Society for Neonatal Screening who provided contact to the respondents. All respondents contributed valuable expertise in the survey on decision processes in the area of newborn screening. The authors thank those respondents who preferred to stay anonymous as well as the following who agreed to disclose their names: Tadej Battelino (Slovenia), Matthias Baumgartner (Switzerland), Martina Cornel (The Netherlands), Francois Eyskens (Belgium), Jean Pierre Farriaux (France), David Hougaard (Denmark), Daniela Iorgulescu (Romania), Alastair Kent (England), Milan Macek (Czech Republic), Uta Nennstiel-Ratzel (Germany), Florence Orsini (France), Peter Saugmann-Jensen (Denmark), Toni Torresani (Switzerland), Felix Votava (Czech Republic), Mojca Zerjav-Tansek (Slovenia). Finally, the authors express their gratitude to the very helpful and supportive comments of two anonymous referees. The research leading to these results has received funding from the European Union's Seventh Framework Programme (FP7/2007-2013) under grant agreement No. 223533. The funding source had no role in the study design, data collection, or interpretation of results. The findings and conclusions in this report are those of the authors and do not necessarily represent the official position of the Centers for Disease Control and Prevention.