Appraising the holistic value of Lenvatinib for radio-iodine refractory differentiated thyroid cancer: A multi-country study applying pragmatic MCDA

Abstract

Background: The objective of the study was to reveal through pragmatic MCDA (EVIDEM) the contribution of a broad range of criteria to the value of the orphan drug lenvatinib for radioiodine refractory differentiated thyroid cancer (RR-DTC) in country-specific contexts.

Methods: The study was designed to enable comprehensive appraisal (12 quantitative, 7 qualitative criteria) in the current disease context (watchful waiting, sorafenib) of France, Italy and Spain. Data on the value of lenvatinib was collected from diverse stakeholders during country-specific panels and included: criteria weights (individual and social values); performance scores (judgments on evidence-collected through MCDA systematic review); qualitative impacts of contextual criteria; and verbal and written insights structured by criteria. The value contribution of each criterion was calculated and uncertainty explored.

Results: Comparative effectiveness, Quality of evidence (Spain and Italy) and Disease severity (France) received the greatest weights. Four criteria contributed most to the value of lenvatinib, reflecting its superior Comparative effectiveness (16-22% of value), the severity of RR-DTC (16-22%), significant unmet needs (14-21%) and robust evidence (14-20%). Contributions varied by comparator, country and individuals, highlighting the importance of context and consultation. Results were reproducible at the group level. Impacts of contextual criteria varied across countries reflecting different health systems and cultural backgrounds. The MCDA process promoted sharing stakeholders' knowledge on lenvatinib and insights on context.

Conclusions: The value of lenvatinib was consistently positive across diverse therapeutic contexts. MCDA identified the aspects contributing most to value, revealed rich contextual insights, and helped participants express and explicitly tackle ethical trade-offs inherent to balanced appraisal and decisionmaking.

Keywords: Appraisal; Healthcare decisionmaking; Lenvatinib; Mcda.

Fig. 1

Study design

Fig. 2

Mean (SD) normalised weights assigned to each quantitative criterion by the French (a), Italian (b), Spanish panels (c) using the 5-point direct weight elicitation technique

Fig. 3

Mean (SD) scores for lenvatinib for RR-DTC assigned to each quantitative criterion by the French (a), Italian (b), and Spanish (c) panels versus watchful waiting (1) and sorafenib (2). A constructed, cardinal scoring scale was used, ranging from 0 to 5 for non-comparative and from −5 to 5 for comparative criteria

Fig. 4

Mean value contributions* of each quantitative criterion and overall MCDA value estimates† for lenvatinib for RR-DTC from the French (a), Italian (b), and Spanish (c) panels versus watchful waiting (1) and sorafenib (2). * Value contribution = Normalized weight ×standardized score; †Overall Value Estimate = ∑ Value contribution of all 12 criteria. Error bars show standard deviations across 8 panelists in each country-specific panel

Fig. 5

Impacts of contextual criteria on the appraisal of lenvatinib assigned by panelists in France, Italy and Spain, as percentage of impacts assigned. *Percentage of impacts (positive or negative) of all impacts assigned for a given criterion; Overall impact across criteria = (∑ Positive impacts - ∑ Negative impacts) / ∑ all impacts assigned