BackgroundThe multiplicity of issues, including uncertainty and ethical dilemmas, and policies involved in appraising interventions for rare diseases suggests that multicriteria decision analysis (MCDA) based on a holistic definition of value is uniquely suited for this purpose. The objective of this study was to analyze and further develop a comprehensive MCDA framework (EVIDEM) to address rare disease issues and policies, while maintaining its applicability across disease areas.MethodsSpecific issues and policies for rare diseases were identified through literature review. Ethical and methodological foundations of the EVIDEM framework v3.0 were systematically analyzed from the perspective of these issues, and policies and modifications of the framework were performed accordingly to ensure their integration.ResultsAnalysis showed that the framework integrates ethical dilemmas and issues inherent to appraising interventions for rare diseases but required further integration of specific aspects. Modification thus included the addition of subcriteria to further differentiate disease severity, disease-specific treatment outcomes, and economic consequences of interventions for rare diseases. Scoring scales were further developed to include negative scales for all comparative criteria. A methodology was established to incorporate context-specific population priorities and policies, such as those for rare diseases, into the quantitative part of the framework. This design allows making more explicit trade-offs between competing ethical positions of fairness (prioritization of those who are worst off), the goal of benefiting as many people as possible, the imperative to help, and wise use of knowledge and resources. It also allows addressing variability in institutional policies regarding prioritization of specific disease areas, in addition to existing uncertainty analysis available from EVIDEM.ConclusionThe adapted framework measures value in its widest sense, while being responsive to rare disease issues and policies. It provides an operationalizable platform to integrate values, competing ethical dilemmas, and uncertainty in appraising healthcare interventions.Electronic supplementary materialThe online version of this article (doi:10.1007/s40273-015-0340-5) contains supplementary material, which is available to authorized users.

Resource utilization in advanced NET in the United States is significant overall and data suggests progression has an impact on resource utilization regardless of NET tumor site.

Neuroendocrine tumours (NET) are often diagnosed at an advanced stage when the prognosis is poor for patients, who often experience diminished quality of life (QoL). As new treatments for NET become available, it is important to characterise the associated outcomes, costs and QoL. A comprehensive search was performed to systematically review available data in advanced NET regarding cost of illness/resource utilisation, economic studies/health technology assessment and QoL. Four rounds of sequential review narrowed the search results to 22 relevant studies. Most focused on surgical procedures and diagnostic tools and contained limited information on the costs and consequences of medical therapies. Multiple tools are used to assess health-related QoL in NET, but few analyses have been conducted to assess the comparative impact of available treatment alternatives on QoL. Limitations include English language and the focus on advanced NET; ongoing terminology and classification changes prevented pooled statistical analyses. This systematic review suggests a lack of comparative economic and outcomes data associated with NET treatments. Further research on disease costs, resource utilisation and QoL for patients with advanced NET is warranted.

Overall, the available literature suggests that although SBS-IF affects a relatively small population, the clinical and humanistic burden is significant, and there is an unmet need for effective therapeutic options that target the underlying problem of inadequate absorptive capacity of the remaining intestine. Consequently, many patients with SBS-IF remain dependent on long-term PN/IV support, adding to the burden imposed by the underlying disorder.

Results suggest that sunitinib-refractory metastatic renal cell carcinoma patients treated with everolimus may experience significantly improved OS outcomes compared to those treated with sorafenib. However, because this is not a randomized controlled trial, the results should be interpreted as those from an observational study.

Advanced NET is associated with significant resource use across subtypes and countries, and resource utilization is likely to increase on disease progression. There remains an unmet need for therapeutic options after disease progression.

A477value-based pricing, which reflects disease burden, therapeutic innovation, and social values, is suggested. This study attempts to measure the importance of costeffectiveness and other values for a new anticancer drug. Methods: Through literature reviews and experts' advices, eight health insurance benefit criteria were selected: disease severity, size of population affected by disease, pediatric medicine, alternative drugs, innovativeness, clinical benefit, cost-effectiveness, and budget impact. Preference for the criteria was investigated by using Discrete Choice Experiments(DCE), Analytic Hierarchy Process (AHP), swing weighting (SWING), and direct point allocation (DIRECT). The survey was conducted in three hundred general population through face to face interview. Respondents were selected using stratified random sampling by age, sex and region. The conditional logistic regression for DCE was conducted with STATA ver.12. Results: In the preference investigation using DCE, people preferentially considered disease severity (OR: 1.837, 95% CI: 1.673 to 2.017), alternative drugs (OR: 1.556, 95% CI: 1.458 to 1.661), and size of population affected by disease (OR: 1.408, 95% CI: 1.285 to 1.543). According to the results by using AHP, respondents considered clinical benefit to be the most important, followed by cost-effectiveness and disease severity as the main evaluation items. As estimated by SWING and DIRECT, clinical benefit was also evaluated as the most important item. There was no difference in the first to third priority evaluation items between SWING and DIRECT. ConClusions: The priorities derived from all methodologies show that clinical benefit and disease severity were more important than cost-effectiveness in general terms. In the situation where decision-making is mostly centered on cost-effectiveness, our results may be seen as the social demand that clinical benefit and the influence of applicable disease should be reflected appropriately in the insurance coverage.

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