Skip to main content Accessibility help

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

  • Katharina E. Fischer (a1), Scott D. Grosse (a2) and Wolf H. Rogowski (a3)


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.



Hide All
1. Anis, AH, Rahman, T, Schechter, MT. Using pharmacoeconomic analysis to make drug insurance coverage decisions. Pharmacoeconomics. 1998;13:119126.
2. Barbieri, M, Hawkins, N, Sculpher, M. Who does the numbers? The role of third-party technology assessment to inform health systems' decision-making about the funding of health technologies. Value Health. 2009;12:193201.
3. Bührlen, B. Innovation in health care: The role of HTA in the introduction of new technologies. [German] Innovation im Gesundheitswesen: Die Rolle von HTA bei der Einführung neuer Technologien. Z Evid Fortbild Qual Gesundh Wesen. 2010;104:703708.
4. Carlsson, P. Health technology assessment and priority setting for health policy in Sweden. Int J Technol Assess Health Care. 2004;20:4454.
5. Daniels, N, Sabin, J. The ethics of accountability in managed care reform. Health Aff (Millwood). 1998;17:5064.
6. Davies, SC, Cronin, E, Gill, M, et al. Screening for sickle cell disease and thalassaemia: A systematic review with supplementary research. Health Technol Assess. 2000;4:i-v, 199.
7. Fischer, KE, Leidl, R, Rogowski, WH. A structured tool to analyse coverage decisions: Development and feasibility test in the field of cancer screening and prevention. Health Policy. 2011;10:290299.
8. Gerhardus, A, Dorendorf, E, Rottingen, JA, Santamera, A. What are the effects of HTA reports on the health system? Evidence from the research literature. In: Velasco, Garrido M, Kristensen, FB, Nielsen, C, Busse, R, eds. health technology assessment and health policy-making in Europe - Current status, challenges and potential. Copenhagen: World Health Organization; 2008:109136.
9. Grosse, SD, Olney, RS, Baily, MA. The cost effectiveness of universal versus selective newborn screening for sickle cell disease in the US and the UK: A critique. Appl Health Econ Health Policy. 2005;4:239247.
10. Grosse, SD, Rogowski, WH, Ross, LF, et al. Population screening for genetic disorders in the 21st century: Evidence, economics, and ethics. Public Health Genomics. 2010;13:106115.
11. Hanney, S, Buxton, M, Green, C, Coulson, D, Raftery, J. An assessment of the impact of the NHS Health Technology Assessment Programme. Health Technol Assess. 2007;11:iii–iv, ix-xi, 1180.
12. Hartz, S, John, J. Public health policy decisions on medical innovations: What role can early economic evaluation play? Health Policy. 2009;89:184192.
13. Health Council of the Netherlands. Neonatal screening. The Hague: Health Council of the Netherlands; 2005.
14. Hutton, J, McGrath, C, Frybourg, JM, et al. Framework for describing and classifying decision-making systems using technology assessment to determine the reimbursement of health technologies (fourth hurdle systems). Int J Technol Assess Health Care. 2006;22:1018.
15. Jonsson, B. Economic evaluation of medical technologies in Sweden. Soc Sci Med. 1997;45:597604.
16. Koopmanschap, MA, Stolk, EA, Koolman, X. Dear policy maker: Have you made up your mind? A discrete choice experiment among policy makers and other health professionals. Int J Technol Assess Health Care. 2010;26:198204.
17. Levy, AR, Mitton, C, Johnston, KM, Harrigan, B, Briggs, AH. International comparison of comparative effectiveness research in five jurisdictions insights for the US. Pharmacoeconomics. 2010;28:813830.
18. Menon, D, Stafinski, T. Role of patient and public participation in health technology assessment and coverage decisions. Expert Rev Pharmacoecon Outcomes Res. 2011;11:7589.
19. OECD. Chapter 4. Decision making and implementation: An analysis of survey results. Health Technologies And Decision Making. Paris: OECD; 2005.
20. Oortwijn, W, Mathijssen, J, Banta, D. The role of health technology assessment on pharmaceutical reimbursement in selected middle-income countries. Health Policy. 2010;95:174184.
21. Packer, C, Simpson, S, Stevens, A. International diffusion of new health technologies: A ten-country analysis of six health technologies. Int J Technol Assess Health Care. 2006;22:419428.
22. Pandor, A, Eastham, J, Beverley, C, Chilcott, J, Paisley, S. Clinical effectiveness and cost-effectiveness of neonatal screening for inborn errors of metabolism using tandem mass spectrometry: A systematic review. Health Technol Assess. 2004;8:iii, 1121.
23. Rogowski, WH, Hartz, SC, John, JH. Clearing up the hazy road from bench to bedside: A framework for integrating the fourth hurdle into translational medicine. BMC Health Serv Res. 2008;8:194.
24. Schwarzer, R, Siebert, U. Methods, procedures, and contextual characteristics of health technology assessment and health policy decision making: Comparison of health technology assessment agencies in Germany, United Kingdom, France, and Sweden. Int J Technol Assess Health Care. 2009;25:305314.
25. Seymour, CA, Thomason, MJ, Chalmers, RA, et al. Newborn screening for inborn errors of metabolism: A systematic review. Health Technol Assess. 1997;1:i-iv, 195.
26. Sundhedsstyrelsen. Biokemisk Screening For Medfodt Sygdom Hos Nyfodte. Copenhagen: Sundhedsstyrelsen; 2008.
27. The International Working Group for HTA Advancement, Neumann, PJ, Drummond, MF, et al. Are key principles for improved health technology assessment supported and used by health technology assessment organizations? Int J Technol Assess Health Care. 2010;26:7178.
28. Velasco, Garrido M, Gerhardus, A, Røttingen, JA, Busse, R. Developing health technology assessment to address health care system needs. Health Policy. 2010;94:196202.
29. Vuorenkoski, L, Toiviainen, H, Hemminki, E. Decision-making in priority setting for medicines–A review of empirical studies. Health Policy. 2008;86:19.
30. Zeuner, D, Ades, AE, Karnon, J, et al. Antenatal and neonatal haemoglobinopathy screening in the UK: Review and economic analysis. Health Technol Assess. 1999;3:i–v, 1186.


Type Description Title
Supplementary Table

Fischer Supplementary Table 1
Supplementary Table 1. Appraisal Aspects - Test Statistics

 Word (91 KB)
91 KB


Altmetric attention score

Full text views

Total number of HTML views: 0
Total number of PDF views: 0 *
Loading metrics...

Abstract views

Total abstract views: 0 *
Loading metrics...

* Views captured on Cambridge Core between <date>. This data will be updated every 24 hours.

Usage data cannot currently be displayed