Societal preferences for granting orphan drugs special status in reimbursement decisions

Background: Orphan drugs, for patients with a rare disease, are increasingly available but often do not meet standard cost-effectiveness criteria for reimbursement. Consequently, policymakers regularly face the dilemma whether to relax these criteria for reimbursing orphan drugs. We examined whether—and why—there would be societal support for such differential treatment of orphan drugs. Methods: We conducted a labelled discrete choice experiment in a sample of the adult population (n = 1,172) in the Netherlands. Respondents were presented with ten choices on whether to reimburse an orphan drug given that a non-orphan drug with similar characteristics would not be reimbursed, because it was not cost-effective, and asked to explain their choices. We used random-intercept logit regression models and inductive coding for analysing the quantitative and qualitative data. Results: Of the respondents, 36.4% consistently chose for reimbursing the orphan drug, mostly because “everyone is entitled to live a healthy life and good quality healthcare”, and 17.3% consistently for not reimbursing the orphan drug, mostly because “[this] is unfair to patients with a common disease”. The remaining 46.3% made alternating choices and were more likely to choose for reimbursing orphan drugs when patients were aged between 1 and 70 years, had moderate disease severity, and considerable health gain from treatment. Conclusions: This study finds considerable support but also strong preference heterogeneity amongst members of the public in the Netherlands for differential treatment of orphan drugs in reimbursement decisions, when these drugs do not meet common cost-effectiveness criteria. However, a substantial minority opposes differential treatment, mostly on moral grounds.</p

Challenges with Coverage with Evidence Development Schemes for Medical Devices : A Systematic Review

Objectives: Coverage with evidence development (CED) schemes are particularly relevant for medical devices (MDs), since clinical evidence is often limited at the time of launch and their long-term (cost-) effectiveness heavily depends on how they are adopted into routine clinical practice. The objective of this study was to identify and describe the challenges that payers and manufacturers might face when assessing the desirability of, choosing the research design for, implementing, and evaluating CED schemes for MDs. Methods: A systematic literature review was performed on six databases following PRISMA guidelines. Two independent reviewers assessed the eligibility of studies based on predefined criteria and extracted data from the included articles by using a pre-defined extraction template. The data were synthesised in a narrative review. Results: The systematic search yielded 4,293 articles of which 27 were eligible for inclusion. We identified 20 challenges that are associated with CED schemes for MDs. Five of these challenges relate directly to the characteristics of MDs, and hence are specific to MDs. These challenges concern deciding on whether a CED scheme is required, understanding the relevant uncertainties and risks, identifying meaningful outcomes, defining an adequate duration for a scheme, and market entry of new technologies. Conclusions: Payers and manufacturers of MDs have to address the identified challenges to improve a CED scheme’s chance of success. This can be further improved by public sharing of information about the outcome of applied schemes and way in which stakeholders have addressed the challenges they faced when applying a CED scheme

Societal preferences for granting orphan drugs special status in reimbursement decisions

Background: Orphan drugs, for patients with a rare disease, are increasingly available but often do not meet standard cost-effectiveness criteria for reimbursement. Consequently, policymakers regularly face the dilemma whether to relax these criteria for reimbursing orphan drugs. We examined whether—and why—there would be societal support for such differential treatment of orphan drugs. Methods: We conducted a labelled discrete choice experiment in a sample of the adult population (n = 1,172) in the Netherlands. Respondents were presented with ten choices on whether to reimburse an orphan drug given that a non-orphan drug with similar characteristics would not be reimbursed, because it was not cost-effective, and asked to explain their choices. We used random-intercept logit regression models and inductive coding for analysing the quantitative and qualitative data. Results: Of the respondents, 36.4% consistently chose for reimbursing the orphan drug, mostly because “everyone is entitled to live a healthy life and good quality healthcare”, and 17.3% consistently for not reimbursing the orphan drug, mostly because “[this] is unfair to patients with a common disease”. The remaining 46.3% made alternating choices and were more likely to choose for reimbursing orphan drugs when patients were aged between 1 and 70 years, had moderate disease severity, and considerable health gain from treatment. Conclusions: This study finds considerable support but also strong preference heterogeneity amongst members of the public in the Netherlands for differential treatment of orphan drugs in reimbursement decisions, when these drugs do not meet common cost-effectiveness criteria. However, a substantial minority opposes differential treatment, mostly on moral grounds.</p

Examining the Effect of Depicting a Patient Affected by a Negative Reimbursement Decision in Healthcare on Public Disagreement with the Decision

BackgroundThe availability of increasingly advanced and expensive new health technologies puts considerable pressure on publicly financed healthcare systems. Decisions to not—or no longer—reimburse a health technology from public funding may become inevitable. Nonetheless, policymakers are often pressured to amend or revoke negative reimbursement decisions due to the public disagreement that typically follows such decisions. Public disagreement may be reinforced by the publication of pictures of individual patients in the media. Our aim was to assess the effect of depicting a patient affected by a negative reimbursement decision on public disagreement with the decision.MethodsWe conducted a discrete choice experiment in a representative sample of the public (n = 1008) in the Netherlands and assessed the likelihood of respondents’ disagreement with policymakers’ decision to not reimburse a new pharmaceutical for one of two patient groups. We presented a picture of one of the patients affected by the decision for one patient group and “no picture available” for the other group. The groups were described on the basis of patients’ age, health-related quality of life (HRQOL) and life expectancy (LE) before treatment, and HRQOL and LE gains from treatment. We applied random-intercept logit regression models to analyze the data.ResultsOur results indicate that respondents were more likely to disagree with the negative reimbursement decision when a picture of an affected patient was presented. Consistent with findings from other empirical studies, respondents were also more likely to disagree with the decision when patients were relatively young, had high levels of HRQOL and LE before treatment, and large LE gains from treatment.ConclusionsThis study provides evidence for the effect of depicting individual, affected patients on public disagreement with negative reimbursement decisions in healthcare. Policymakers would do well to be aware of this effect so that they can anticipate it and implement policies to mitigate associated risks

Examining the Effect of Depicting a Patient Affected by a Negative Reimbursement Decision in Healthcare on Public Disagreement with the Decision

BackgroundThe availability of increasingly advanced and expensive new health technologies puts considerable pressure on publicly financed healthcare systems. Decisions to not—or no longer—reimburse a health technology from public funding may become inevitable. Nonetheless, policymakers are often pressured to amend or revoke negative reimbursement decisions due to the public disagreement that typically follows such decisions. Public disagreement may be reinforced by the publication of pictures of individual patients in the media. Our aim was to assess the effect of depicting a patient affected by a negative reimbursement decision on public disagreement with the decision.MethodsWe conducted a discrete choice experiment in a representative sample of the public (n = 1008) in the Netherlands and assessed the likelihood of respondents’ disagreement with policymakers’ decision to not reimburse a new pharmaceutical for one of two patient groups. We presented a picture of one of the patients affected by the decision for one patient group and “no picture available” for the other group. The groups were described on the basis of patients’ age, health-related quality of life (HRQOL) and life expectancy (LE) before treatment, and HRQOL and LE gains from treatment. We applied random-intercept logit regression models to analyze the data.ResultsOur results indicate that respondents were more likely to disagree with the negative reimbursement decision when a picture of an affected patient was presented. Consistent with findings from other empirical studies, respondents were also more likely to disagree with the decision when patients were relatively young, had high levels of HRQOL and LE before treatment, and large LE gains from treatment.ConclusionsThis study provides evidence for the effect of depicting individual, affected patients on public disagreement with negative reimbursement decisions in healthcare. Policymakers would do well to be aware of this effect so that they can anticipate it and implement policies to mitigate associated risks

Challenges with coverage with evidence development schemes for medical devices: A systematic review

Objectives: Coverage with evidence development (CED) schemes are particularly relevant for medical devices (MDs), since clinical evidence is often limited at the time of launch and their long-term (cost-) effectiveness heavily depends on how they are adopted into routine clinical practice. The objective of this study was to identify and describe the challenges that payers and manufacturers might face when assessing the desirability of, choosing the research design for, implementing, and evaluating CED schemes for MDs. Methods: A systematic literature review was performed on six databases following PRISMA guidelines. Two independent reviewers assessed the eligibility of studies based on predefined criteria and extracted data from the included articles by using a pre-defined extraction template. The data were synthesised in a narrative review. Results: The systematic search yielded 4293 articles of which 27 were eligible for inclusion. We identified 20 challenges that are associated with CED schemes for MDs. Five of these challenges relate directly to the characteristics of MDs, and hence are specific to MDs. These challenges concern deciding on whether a CED scheme is required, understanding the relevant uncertainties and risks, identifying meaningful outcomes, defining an adequate duration for a scheme, and market entry of new technologies. Conclusions: Payers and manufacturers of MDs have to address the identified challenges to improve a CED scheme's chance of success. This can be further improved by public sharing of information about the outcome of applied schemes and way in which stakeholders have addressed the challenges they faced when applying a CED scheme

Think of the Children: A Discussion of the Rationale for and Implications of the Perspective Used for EQ-5D-Y Health State Valuation

_Objectives:_ The recently published EQ-5D-Y valuation protocol prescribes the general public values EQ-5D-Y health states for a 10-year-old child. This child perspective differ

Willingness to Pay for Health-Related Quality of Life Gains in Relation to Disease Severity and the Age of Patients

_Objectives:_ Decision-making frameworks that draw on economic evaluations increasingly use equity weights to facilitate a more equitable and fair allocation of healthcare resources. These weights can be attached to health gains or reflected in the monetary threshold against which the incremental cost-effectiveness ratios of (new) health technologies are evaluated. Currently applied weights are based on different definitions of disease severity and do not account for age-related preferences in society. However, age has been shown to be an important equity-relevant characteristic. This study examines the willingness to pay (WTP) for health-related quality of life (QOL) gains in relation to the disease severity and age of patients, and the outcome of the disease. _Methods:_ We obtained WTP estimates by applying contingent-valuation tasks in a representative sample of the public in The Netherlands (n = 2023). We applied random-effects generalized least squares regression models to estimate the effect of patients’ disease severity and age, size of QOL gains, disease outcome (full recovery/death 1 year after falling ill), and respondent characteristics on the WTP. _Results:_ Respondents’WTP was higher for more severely ill and younger patients and for larger-sized QOL gains, but lower for patients who died. However, the relations were nonlinear and context dependent. Respondents with a lower age, who were male, had a higher household income, and a higher QOL stated a higher WTP for QOL gains. _Conclusions:_ Our results suggest that—if the aim is to align resource-allocation decisions in healthcare with societal preferences—currently applied equity weights do not suffice

Looking back and moving forward

The 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