 ‘Mapping’ Health State Utility Values from Non-preference-Based Measures: A Systematic Literature Review in Rare DiseasesMichela Meregaglia (),
Amanda Whittal,
Elena Nicod and
Michael Drummond
PharmacoEconomics, 2020, vol. 38, issue 6, No 3, 557-574 Abstract: Abstract Background The use of patient-reported outcome measures (PROMs) to monitor the effects of disease and treatment on patient symptomatology and daily life is increasing in rare diseases (RDs) (i.e. those affecting less than one in 2000 people); however, these instruments seldom yield health state utility values (HSUVs) for cost-utility analyses. In such a context, ‘mapping’ allows HSUVs to be obtained by establishing a statistical relationship between a ‘source’ (e.g. a disease-specific PROM) and a ‘target’ preference-based measure [e.g. the EuroQol-5 Dimension (EQ-5D) tool]. Objective This study aimed to systematically review all published studies using ‘mapping’ to derive HSUVs from non–preference-based measures in RDs, and identify any critical issues related to the main features of RDs, which are characterised by small, heterogeneous, and geographically dispersed patient populations. Methods The following databases were searched during the first half of 2019 without time, study design, or language restrictions: MEDLINE (via PubMed), the School of Health and Related Research Health Utility Database (ScHARRHUD), and the Health Economics Research Centre (HERC) database of mapping studies (version 7.0). The keywords combined terms related to ‘mapping’ with Orphanet’s list of RD indications (e.g. ‘acromegaly’) in addition to ‘rare’ and ‘orphan’. ‘Very rare’ diseases (i.e. those with fewer than 1000 cases or families documented in the medical literature) were excluded from the searches. A predefined, pilot-tested extraction template (in Excel®) was used to collect structured information from the studies. Results Two groups of studies were identified in the review. The first group (n = 19) developed novel mapping algorithms in 13 different RDs. As a target measure, the majority used EQ-5D, and the others used the Short-Form Six-Dimension (SF-6D) and 15D; most studies adopted ordinary least squares (OLS) regression. The second group of studies (n = 9) applied previously published algorithms in non-RDs to comparable RDs, mainly in the field of cancer. The critical issues relating to ‘mapping’ in RDs included the availability of very few studies, the relatively high number of cancer studies, and the absence of research in paediatric RDs. Moreover, the reviewed studies recruited small samples, showed a limited overlap between RD-specific and generic PROMs, and highlighted the presence of cultural and linguistic factors influencing results in multi-country studies. Lastly, the application of existing algorithms developed in non-RDs tended to produce inaccuracies at the bottom of the EQ-5D scale, due to the greater severity of RDs. Conclusions More research is encouraged to develop algorithms for a broader spectrum of RDs (including those affecting young children), improve mapping study quality, test the generalisability of algorithms developed in non-RDs (e.g. HIV) to rare variants or evolutions of the same condition (e.g. AIDS wasting syndrome), and verify the robustness of results when mapped HSUVs are used in cost-utility models. Date: 2020 Downloads: (external link) Related works: Export reference: BibTeX RIS (EndNote, ProCite, RefMan) HTML/Text Persistent link: https://EconPapers.repec.org/RePEc:spr:pharme:v:38:y:2020:i:6:d:10.1007_s40273-020-00897-4 Ordering information: This journal article can be ordered from DOI: 10.1007/s40273-020-00897-4 Access Statistics for this article PharmacoEconomics is currently edited by Timothy Wrightson and Christopher I. Carswell More articles in PharmacoEconomics from Springer |
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