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Uncertainty, evidence and irrecoverable costs: Informing approval, pricing and research decisions for health technologies

Author

Listed:
  • Karl Claxton

    (Centre for Health Economics and Department of Economics and Related Studies, University of York, UK)

  • Stephen Palmer

    (Centre for Health Economics, University of York, UK)

  • Louise Longworth

    (Health Economics Research Group, Brunel University, UK)

  • Laura Bojke

    (Centre for Health Economics, University of York, UK)

  • Susan Griffin

    (Centre for Health Economics, University of York, UK)

  • Claire McKenna

    (Centre for Health Economics, University of York, UK)

  • Marta Soares

    (Centre for Health Economics, University of York, UK)

  • Eldon Spackman

    (Centre for Health Economics, University of York, UK)

  • Jihee Youn

    (Health Economics Research Group, Brunel University, UK)

Abstract

The general issue of balancing the value of evidence about the performance of a technology and the value of access to a technology can be seen as central to a number of policy questions. Establishing the key principles of what assessments are needed, as well as how they should be made, will enable them to be addressed in an explicit and transparent manner. This report presents the key finding from MRC and NHIR funded research which aimed to: i) Establish the key principles of what assessments are needed to inform an only in research (OIR) or Approval with Research (AWR) recommendation. ii) Evaluate previous NICE guidance where OIR or AWR recommendations were made or considered. iii) Evaluate a range of alternative options to establish the criteria, additional information and/or analysis which could be made available to help the assessment needed to inform an OIR or AWR recommendation. iv) Provide a series of final recommendations, with the involvement of key stakeholders, establishing both the key principles and associated criteria that might guide OIR and AWR recommendations, identifying what, if any, additional information or analysis might be included in the Technology Appraisal process and how such recommendations might be more likely to be implemented through publicly funded and sponsored research. The key principles and the assessments and judgments required are discussed in Section 2. The sequence of assessment and judgment is represented as an algorithm, which can also be summarised as a simple set of explicit criteria or a seven point checklist of assessments. The application of the check list of assessment to a series of four case studies in Section 3 can inform considerations of whether such assessments can be made based on existing information and analysis in current NICE appraisal and in what circumstances could additional information and/or analysis be useful. In Section 4, some of the implications that this more explicit assessment of OIR and AWR might have for policy (e.g., NICE guidance and drug pricing), the process of appraisal (e.g., greater involvement of research commissioners) and methods of appraisal (e.g., should additional information, evidence and analysis be required) are drawn together.

Suggested Citation

  • Karl Claxton & Stephen Palmer & Louise Longworth & Laura Bojke & Susan Griffin & Claire McKenna & Marta Soares & Eldon Spackman & Jihee Youn, 2011. "Uncertainty, evidence and irrecoverable costs: Informing approval, pricing and research decisions for health technologies," Working Papers 069cherp, Centre for Health Economics, University of York.
  • Handle: RePEc:chy:respap:69cherp
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    References listed on IDEAS

    as
    1. Palmer, Stephen & Smith, Peter C., 2000. "Incorporating option values into the economic evaluation of health care technologies," Journal of Health Economics, Elsevier, vol. 19(5), pages 755-766, September.
    2. McCabe, C & Claxton, K & Culyer, AJ, 2008. "The NICE Cost-Effectiveness Threshold: What it is and What that Means," MPRA Paper 26466, University Library of Munich, Germany.
    3. Simon Eckermann & Andrew R. Willan, 2008. "The Option Value of Delay in Health Technology Assessment," Medical Decision Making, , vol. 28(3), pages 300-305, May.
    4. Zoe Philips & Karl Claxton & Stephen Palmer, 2008. "The Half-Life of Truth: What Are Appropriate Time Horizons for Research Decisions?," Medical Decision Making, , vol. 28(3), pages 287-299, May.
    5. Claire McKenna & Karl Claxton, 2011. "Addressing Adoption and Research Design Decisions Simultaneously," Medical Decision Making, , vol. 31(6), pages 853-865, November.
    6. Claxton, Karl, 1999. "The irrelevance of inference: a decision-making approach to the stochastic evaluation of health care technologies," Journal of Health Economics, Elsevier, vol. 18(3), pages 341-364, June.
    7. Karl Claxton & Mark Sculpher & Stuart Carroll, 2011. "Value-based pricing for pharmaceuticals: Its role, specification and prospects in a newly devolved NHS," Working Papers 060cherp, Centre for Health Economics, University of York.
    8. Basu, Anirban, 2011. "Economics of individualization in comparative effectiveness research and a basis for a patient-centered health care," Journal of Health Economics, Elsevier, vol. 30(3), pages 549-559, May.
    9. Aaron A. Stinnett & John Mullahy, 1998. "Net Health Benefits: A New Framework for the Analysis of Uncertainty in Cost-Effectiveness Analysis," NBER Technical Working Papers 0227, National Bureau of Economic Research, Inc.
    10. Elisabeth Fenwick & Karl Claxton & Mark Sculpher, 2008. "The Value of Implementation and the Value of Information: Combined and Uneven Development," Medical Decision Making, , vol. 28(1), pages 21-32, January.
    11. Aaron A. Stinnett & John Mullahy, 1998. "Net Health Benefits," Medical Decision Making, , vol. 18(2_suppl), pages 68-80, April.
    12. Karl Claxton & Mark Sculpher & Chris McCabe & Andrew Briggs & Ron Akehurst & Martin Buxton & John Brazier & Tony O'Hagan, 2005. "Probabilistic sensitivity analysis for NICE technology assessment: not an optional extra," Health Economics, John Wiley & Sons, Ltd., vol. 14(4), pages 339-347, April.
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    Cited by:

    1. Louise Longworth & JiHee Youn & Laura Bojke & Stephen Palmer & Susan Griffin & Eldon Spackman & Karl Claxton, 2013. "When Does NICE Recommend the Use of Health Technologies Within a Programme of Evidence Development?," PharmacoEconomics, Springer, vol. 31(2), pages 137-149, February.
    2. Laura Bojke & Andrea Manca & Miqdad Asaria & Ronan Mahon & Shijie Ren & Stephen Palmer, 2017. "How to Appropriately Extrapolate Costs and Utilities in Cost-Effectiveness Analysis," PharmacoEconomics, Springer, vol. 35(8), pages 767-776, August.
    3. Christopher McCabe & Richard Edlin & Peter Hall, 2013. "Navigating Time and Uncertainty in Health Technology Appraisal: Would a Map Help?," PharmacoEconomics, Springer, vol. 31(9), pages 731-737, September.

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