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

Abstract

BackgroundThe 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.MethodsThe 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.ConclusionsThe 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.Electronic supplementary materialThe online version of this article (doi:10.1186/s12885-017-3258-9) contains supplementary material, which is available to authorized users.

A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries

“…Researchers in the rare disease area are also looking into the use of MCDA, which has resulted in a list of scientific publications and MCDA model designs, but full consensus on MCDA is still lacking and further research is needed to support implementation in (rare disease) HTA (94,(101)(102)(103)(104)(105)(106)(107)(108)(109)(110)(111)(112)(113)(114)(115)(116).…”

Applicability of the Evidem Multi-Criteria Decision Analysis Framework for Orphan Drugs – Results From a Study in 7 Eurasian Countries.

“…Cultural differences may have an influence, e.g. on field weights attached to patient-reported outcomes (PROs) versus clinical outcomes (36). On the other hand, weights assigned to ëcoreí criteria related to the safety and efficacy of treatment are likely easier to extrapolate across regions.…”

“…Several authors have published weighting results using the EVIDEM model (v3.0) but not with the EVIDEM model that has been adjusted for rare diseases (24,35,36). The results of these 3 studies were based on a single comparative weighting procedure in which points were divided over all 12 EVIDEM criteria at once, regardless of the model structure.…”

“…Wagner et al (36) found the following EVI-DEM criteria to be the highest ranked (among all cri-teria) in France, Italy and Spain (8 stakeholders per country): Comparative effectiveness, Disease Severity, Quality of Evidence and Comparative Safety, Type of Preventive Benefit. Other criteria were more varied between countries, but Comparative Other Costs, PROís and Expert Consensus/CPGís were generally scored low.…”

“…EVIDEM is the model that has been used the most frequently in scientific studies (24,35,36). The focus of these publications has been solely Western Europe, no research has been done concerning the use and applicability of EVIDEM in Eastern European countries.…”