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Untangling the Complexity of Funding Recommendations: A Comparative Analysis of Health Technology Assessment Outcomes in Four European Countries

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Abstract

Objectives

Health Technology Assessment (HTA) agencies produce recommendations that guide public funding of pharmaceuticals, based on various criteria. We explored factors that may contribute to explaining differences in coverage decisions by the National Institute for Health and Care Excellence (NICE) in England and Wales, the Scottish Medicines Consortium (SMC), the Dutch College voor Zorgverzekeringen (CVZ), and the French Haute Autorité de Santé (HAS).

Methods

A dataset of 977 HTA decisions made in 2004–2009 was created. A three-category outcome variable was used (decision to ‘recommend’, ‘restrict’ or ‘not recommend’ a technology). Multivariate analyses explored impacts of clinical, economic, process and socio-economic variables in their decision making.

Results

Relative to the CVZ and adjusting for a range of confounders, technologies were more likely to be recommended by NICE and HAS, and restricted or not-recommended by the SMC. Recommendation was significantly associated (p ≤ 0.10) with several variables: strength of clinical evidence (number of trials, use of active comparator-arm, demonstration of clinical superiority) orphan status and indication for cancer. Simultaneous assessment of multiple rather than single pharmaceuticals was associated with increased probability of restriction.

Conclusions

In this European multi-HTA study, appraisal outcomes differed significantly across HTA bodies. A range of evidence and non-evidence factors were associated with HTA decisions, confirming the value of comprehensive, multivariate analyses. Nevertheless, a large proportion of variance in HTA decisions remained unexplained, suggesting that greater transparency of decision making is needed, along with associated further research.

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Notes

  1. While prices of 1A listed technologies are pre-defined based on already existing reference technologies, it is the manufacturer that submits a proposal with a price in the anticipated range according to the therapeutic differentiation of the product, and it is not the CVZ who asks for a price reduction to meet the reference price rule as a result of the appraisal. In addition, both 1A and 1B result in similar coverage from the patient perspective. This is why we decided to consider both 1A and 1B listings recommendations by the CVZ.

  2. It should be noted that since 2005, CVZ has increasingly used cost-effectiveness evidence in its decision making, in particularly for technologies evaluated for 1B listing and specific instances. However, because of the fact that in the sample of CVZ decisions between 2004 and 2009, only 11 % of technologies (<30) were supported by reported ICER data, it was not included in base-case model 2.

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Acknowledgments

The authors would like to thank Michael Drummond and Alistair McGuire for their valuable comments on the methods used and interpretation of analyses that were performed.

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Authors and Affiliations

  1. London School of Economics and Political Science, Houghton Street, London, WC2A 2AE, UK

    Karin H. Cerri, Martin Knapp & Jose-Luis Fernandez

  2. Janssen Pharmaceutica NV, Beerse, Belgium

    Karin H. Cerri

Authors
  1. Karin H. Cerri
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  2. Martin Knapp
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Correspondence to Karin H. Cerri.

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Funding

No funding was received for the conduct or publication of this study.

Conflicts of interest

KC was an employee of Bristol-Myers Squibb during the time this research was conducted. MK has received funding from NICE for PSSRU’s part in the NICE Collaborating Centre for Social Care. JLF has no conflicts of interest to declare.

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Cerri, K.H., Knapp, M. & Fernandez, JL. Untangling the Complexity of Funding Recommendations: A Comparative Analysis of Health Technology Assessment Outcomes in Four European Countries. Pharm Med 29, 341–359 (2015). https://doi.org/10.1007/s40290-015-0112-8

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