THE EVALUATION OF ECONOMIC METHODS TO ASSESS THE SOCIAL VALUE OF MEDICAL INTERVENTIONS FOR ULTRA-RARE DISORDERS (URDS)

Author(s)

Schlander M1, Garattini S2, Holm S3, Kolominsky-Rabas PL4, Nord E5, Persson U6, Postma MJ7, Richardson J8, Simoens S9, de Sola-Morales O10, Tolley K11, Toumi M12
1Institute for Innovation & Valuation in Health Care (InnoVal-HC), Wiesbaden, Germany, 2Mario Negri Institute for Pharmacological Research, Milano, Italy, 3University of Manchester, Manchester, UK, 4University of Erlangen, Erlangen, Germany, 5Norwegian Institute of Public Health, Oslo, Norway, 6The Swedish Institute for Health Economics (IHE), Lund, Sweden, 7University of Groningen, Groningen, The Netherlands, 8Monash University, Clayton, Victoria, Australia, 9KU Leuven, Leuven, Belgium, 10Sabirmedical, Barcelona, Spain, 11Tolley Health Economics Ltd., Buxton, Derbyshire, UK, 12University Claude Bernard Lyon 1, Lyon, France

Objectives:  To develop a set of criteria to critically appraise the strengths and weaknesses of health economic methods for the systematic valuation of interventions for ultra-rare disorders (URDs). Methods:  An international group of clinical and health economic experts met in conjunction with the Annual European ISPOR Congresses in Berlin/Germany and Dublin/Ireland, November 2012 and 2013, to deliberate and agree on a set of criteria to assess the potential of the various methods, which have been used or proposed to estimate the social value of medical interventions for URDs. Results:  The group identified a broad set of potential criteria, which may be grouped according to the following dimensions:  theoretical foundations (normative premises, i.e., links to moral and economic theories, including - but not limited to – nonutilitarian consequentialist and deontological reasoning, definition and treatment of core concepts of economic thinking such as opportunity costs and efficiency), empirical underpinnings (social preferences related to attributes of the health condition or of the person afflicted with it), and pragmatic aspects (feasibility of implementation and potential for bias and misuse).  For each of the dimensions, a set of criteria has been agreed upon, which in turn will need further scrutiny and justification.  Conclusions:  Previously, a need had been identified for modifications or alternatives to the conventional logic of cost effectiveness applying benchmarks for the maximum allowable cost per quality-adjusted life year (QALY).  We propose a framework for the systematic assessment how well different evaluation approaches reflect prevalent social norms and value judgments.  As a next step, the framework shall be applied on multi-criteria decision analysis methods and social cost value analysis, either using the person trade-off (PTO) or the relative social willingness-to-pay (RS-WTP) instrument.

Conference/Value in Health Info

2014-11, ISPOR Europe 2014, Amsterdam, The Netherlands

Value in Health, Vol. 17, No. 7 (November 2014)

Code

CP1

Topic

Health Policy & Regulatory

Disease

Rare and Orphan Diseases

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