a b s t r a c tThe increasing demand for healthcare and the resulting pressure on available budgets render priority setting inevitable. If societies aim to improve health and distribute health(care) fairly, equity-efficiency trade-offs are necessary. In the Netherlands, proportional shortfall (PS) was introduced to quantify necessity of care, allowing a direct equity-efficiency trade-off. This study describes the history and application of PS in the Netherlands and examines the theoretical and empirical support for PS as well as its current role in healthcare decision making. We reviewed the international literature on PS from 2001 onwards, along with publicly accessible meeting reports from the Dutch appraisal committee, Adviescommissie Pakket (ACP), from 2013 to 2016. Our results indicate that there is support for the decision model in which necessity is quantified and incremental cost-effectiveness ratios are evaluated against associated monetary reference values. The model enables a uniform framework for priority setting across all healthcare sectors. Although consensus about the application of PS has not yet been reached and alternative ways to quantify necessity were found in ACP reports, PS has increasingly been applied in decision making since 2015. However, empirical support for PS is limited and it may insufficiently reflect societal preferences regarding age and reducing lifetime-health inequalities. Hence, further investigation into refining PS-or exploration of another approach-appears warranted for operationalising the equity-efficiency trade-off.

BackgroundIn the context of priority setting, a differential cost-effectiveness threshold can be used to reflect a higher societal willingness to pay for quality-adjusted life-year gains in the worse off. However, uncertainty in the estimate of severity can lead to problems when evaluating the outcomes of cost-effectiveness analyses.ObjectivesThis study standardizes the assessment of severity, integrates its uncertainty with the uncertainty in cost-effectiveness results and provides decision makers with a new estimate: the severity-adjusted probability of being cost effective.MethodsSeverity is expressed in proportional and absolute shortfall and estimated using life tables and country-specific EQ-5D values. We use the three severity-based cost-effectiveness thresholds (€20.000, €50.000 and €80.000, per QALY) adopted in The Netherlands. We exemplify procedures of integrating uncertainty with a stylized example of a hypothetical oncology treatment.ResultsApplying our methods, taking into account the uncertainty in the cost-effectiveness results and in the estimation of severity identifies the likelihood of an intervention being cost effective when there is uncertainty about the appropriate severity-based cost-effectiveness threshold.ConclusionsHigher willingness-to-pay thresholds for severe diseases are implemented in countries to reflect societal concerns for an equitable distribution of resources. However, the estimates of severity are uncertain, patient populations are heterogeneous, and this can be accounted for with the severity-adjusted probability of being cost effective proposed in this study. The application to the Netherlands suggests that not adopting the new method could result in incorrect decisions in the reimbursement of new health technologies.

Background: Priority setting in healthcare can be guided by both efficiency and equity principles. The latter principle is often explicated in terms of disease severity and, for example, defined as absolute or proportional shortfall. These severity operationalizations do not explicitly consider patients' age, even though age may be inextricably related to severity and an equity-relevant characteristic.Objective: This study examines the relative strength of societal preferences for severity and age for informing allocation decisions in healthcare.Methods: We elicited preferences for severity and age in a representative sample of the public in The Netherlands (N = 1025) by applying choice tasks and person-trade-off tasks in a design in which severity levels and ages varied both separately and simultaneously between patient groups. We calculated person trade-off ratios and, in addition, applied ordinary least squares regression models to aid interpretation of the ratios when both severity and age varied.Results: Respondents attached a higher weight (median of ratios: 2.46-3.50) to reimbursing treatment for relatively more severely ill and younger patients when preferences for both were elicited separately. When preferences were elicited simultaneously, respondents attached a higher weight (median of ratios: 1.98 and 2.42) to reimbursing treatment for relatively younger patients, irrespective of patients' severity levels. Ratios varied depending on severity level and age and were generally higher when the difference in severity and age was larger between groups. Conclusions:Our results suggest that severity operationalizations and equity weights based on severity alone may not align with societal preferences. Adjusting decision-making frameworks to reflect age-related societal preferences should be considered.

Objectives Medical devices are potentially good candidates for coverage with evidence development (CED) schemes, as clinical data at market entry are often sparse and (cost-)effectiveness depends on real-world use. The objective of this research was to explore the diffusion of CED schemes for devices in Europe, and the factors that favour or hamper their utilization. Methods We conducted structured interviews with 25 decision-makers from 22 European countries to explore the characteristics of existing CED programmes for devices, and how decision makers perceived 13 pre-identified challenges associated with initiating and operating CED schemes for devices. We also collected data on individual schemes that were either initiated or still ongoing in the last 5 years. Results We identified seven countries with CED programmes for devices and 78 ongoing schemes. The characteristics of CED programmes varied across countries, including eligibility criteria, roles and responsibilities of stakeholders, funding arrangements, and type of decisions being contemplated at the outset of each scheme. We observed a high variability in how decision makers perceived CED-related challenges possibly reflecting country-specific arrangements and different experiences with CED. One general finding across all countries was that relatively little attention was paid to the evaluation of schemes, both during and at their completion. Conclusions CED programmes for devices with different characteristics exist in Europe. Decision-makers’ perceptions differ on the challenges associated with these schemes. More exchange of knowledge and experience will help decision makers anticipate the likely challenges in CED schemes for devices, and to learn from good practices existing elsewhere.

Objectives EQ-5D-Y-3L health states are valued by adults taking the perspective of a 10-year-old child. Compared to valuation of adult EQ-5D instruments, this entails two changes to the perspective: (i) child health states are valued instead of adult health states and: (ii) health states are valued for someone else instead of for oneself. Although earlier work has shown that these combined changes yield different values for child and adult health states that are otherwise equal, it currently remains unclear why. Hence, we aimed to disentangle the effects of both changes. Methods A sample of 205 students (mean age: 19.48) was surveyed. Each respondent completed visual analogue scale (VAS) and time trade-off (TTO) tasks for five EQ-5D-Y-3L states, using four randomly ordered perspectives: (i) self-adult (themselves), (ii) other-adult (someone their age), (iii) self-child (themselves as a 10-year-old), (iv) other-child (a child of 10 years old). We compared how each perspective impacted outcomes, precision and quality of EQ-5D-Y-3L valuation. Results Overall, differences between perspectives were consistent, with their direction being dependent on the health states and respondents. For VAS, the effect on outcomes of valuation depended on severity, but variance was higher in valuation with child perspectives. For TTO, we observed that EQ-5D-Y-3L states valued on behalf of others (i.e., children or adults) received higher valuations, but lower variances. Conclusion The use of a different perspective appears to yield systematic differences in EQ-5D-Y-3L valuation, with considerable heterogeneity between health states and respondents. This may explain mixed findings in earlier work.