Preferences and willingness to pay for personalized nutrition interventions:Discrete choice experiments in Europe and the United States

This study gives insight into what intervention-related factors are crucial for using personalized nutrition (PN) interventions, as well as what the general population is willing to pay for PN. This was done by focusing on two different types of PN (i.e., PN advice and personalized meals) in two discrete choice experiments (DCEs). The DCEs were conducted in four European countries and the United States, including at least 500 respondents per country aged 18–65 years. Panel mixed multinomial logit models were used to evaluate the preferences. Results show that for both types of PN in all countries, the total expenditure on nutrition was the most crucial factor when choosing a PN intervention. The participation rate for specific hypothetical scenario's varied but was considered high overall (maximum 81 % for ‘PN advice’ and 87 % for ‘personalized meals’ in Spain). Moreover, highest willingness to pay estimates were found for six kilograms of weight loss. For example, Polish respondents were willing to spend an extra 25.78 euros per week for ‘personalized meals’ for a 4-month period to lose six kilograms. Our models showed preference heterogeneity between, but also within, the different countries. In conclusion, this study showed that people seem willing to pay for and participate in PN interventions. Since PN interventions may improve health outcomes, policymakers should consider subsidizing some of the costs, financially incentivizing PN interventions or introducing commitment lotteries to encourage uptake. More research is needed to study heterogeneity in preferences.</p

The cost-effectiveness of dapagliflozin compared to DPP-4 inhibitors in the treatment of type 2 diabetes mellitus in the Netherlands

Aim: When glycaemic control for people with type 2 diabetes is not achieved with metformin and sulfonylurea alone, adding another oral anti-diabetes drug, such as a sodium–glucose co-transporter 2 (SGLT2) or dipeptidyl peptidase-4 (DPP-4) inhibitor, is an alternative to starting insulin. The aim of this study is to determine the cost-effectiveness of dapagliflozin (an SGLT2 inhibitor) compared with DPP-4 inhibitors when added to metformin and sulfonylurea in people with type 2 diabetes in the Netherlands. Methods: A cost–utility analysis is performed using the Cardiff diabetes model, a fixed-time increment stochastic simulation model informed by ‘United Kingdom Prospective Diabetes Study 68’ risk equations. The base-case analysis uses a 40-year time horizon, a Dutch societal perspective and differential discounting (4% for costs, 1.5% for effects). Inputs are obtained from the literature and Dutch price lists. Univariate and probabilistic sensitivity analysis are performed. Results: Dapagliflozin is dominant compared with DPP-4 inhibitors, resulting in a €990 cost saving and a 0.28 quality-adjusted life year gain over 40 years. Cost savings are associated mainly with treatment costs and a reduced incidence of micro- and macrovascular complications, among others nephropathy, myocardial infarction and stroke. Results are robust to changes in input parameters. Conclusions: Dapagliflozin is a cost-saving alternative to DPP-4 inhibitors when added to metformin and sulfonylurea. The incidence of micro- and macrovascular complications is lower for people treated with dapagliflozin. Uncertainty around this outcome is low

PCN1 COST ANALYSIS OF HLA-IDENTICAL SIBLING AND VOLUNTARY UNRELATED ALLOGENEIC BONE MARROW AND PERIPHERAL BLOOD STEM CELL TRANSPLANTATION IN ADULTS WITH ACUTE MYELOCYTIC LEUKAEMIA OR ACUTE LYMPHOBLASTIC LEUKAEMIA

Item does not contain fulltextAllogeneic stem cell transplantation (SCT) is one of the most expensive medical procedures. However, only a few studies to date have addressed the costs of HLA-identical sibling transplantation and only one study has reported costs of unrelated transplantation. No recent cost analysis with a proper follow-up period and donor identification expenses is available on related or voluntary matched unrelated donor (MUD) SCT for adult AML or ALL. Therefore, we calculated direct medical (hospital) costs based on 97 adults who underwent HLA-identical sibling bone marrow transplantation (BMT) or peripheral blood stem cell transplantation (PBSCT), and patients who received a graft from a MUD between 1994 and 1999. The average costs per transplanted patient were Euro 98,334 (BMT), Euro 151,754 (MUD), and Euro 98,977 (PBSCT), including donor identification expenses, 2 years follow-up and costs of patients who were not transplanted after they had been planned to receive an allograft. The majority of these costs was generated during the hospitalisation for graft infusion. For MUD transplants, nearly one-third of these costs was spent on the search for a suitable donor. For patients who were alive after 2 years, cumulative expenses were calculated to be Euro 103,509 (BMT), Euro 173,587 (MUD), and Euro 105,906 (PBSCT)

A health technology assessment of personalized nutrition interventions using the EUnetHTA HTA Core Model

Objectives Poor nutrition links to chronic diseases, emphasizing the need for optimized diets. The EU-funded project PREVENTOMICS, introduced personalized nutrition to address this. This study aims to perform a health technology assessment (HTA) comparing personalized nutrition interventions developed through this project, with non-personalized nutrition interventions (control) for people with normal weight, overweight, or obesity. The goal is to support decisions about further development and implementation of personalized nutrition. Methods The PREVENTOMICS interventions were evaluated using the European Network for HTA Core Model, which includes a methodological framework that encompasses different domains for value assessment. Information was gathered via [1] different statistical analyses and modeling studies, [2] questions asked of project partners and, [3] other (un)published materials. Results Clinical trials of PREVENTOMICS interventions demonstrated different body mass index changes compared to control; differences ranged from -0.80 to 0.20 kg/m2. Long-term outcome predictions showed generally improved health outcomes for the interventions; some appeared cost-effective (e.g., interventions in UK). Ethical concerns around health inequality and the lack of specific legal regulations for personalized nutrition interventions were identified. Choice modeling studies indicated openness to personalized nutrition interventions; decisions were primarily affected by intervention's price. Conclusions PREVENTOMICS clinical trials have shown promising effectiveness with no major safety concerns, although uncertainties about effectiveness exist due to small samples (n=60-264) and short follow-ups (10-16 weeks). Larger, longer trials are needed for robust evidence before implementation could be considered. Among other considerations, developers should explore financing options and collaborate with policymakers to prevent exclusion of specific groups due to information shortages.</p

CHANGING NURSING CARE TIME AS AN EFFECT OF CHANGED CHARACTERISTICS OF THE DIALYSIS POPULATION

Background: The population of dialysis patients is ageing. Dialysis nurses are confronted with geriatric patients with multiple comorbidities. Nurses are confronted with an increasing burden of care. Objectives: The present study focused on the question of whether, over time, the increasing age and comorbidities of the haemodialysis population increased nursing care time. Furthermore, we studied potential changes in the predictors of the required nursing time. Design: Observational study. Participants: A total of 980 dialysis patients from 12 dialysis centres were included. Measurements: Nurses filled out the classification tool for each patient and completed a form for reporting patient characteristics for groups of relevant haemodialysis patients at baseline and after 1 and four years. Changes in patient and dialysis characteristics were analysed, as well as the estimated nursing care time needed. Results: An increase in the nursing time needed for dialysis was largely due to decreased mobility, closing of the vascular access and a greater need for psychosocial attention and was most strongly present in incident dialysis patients. The time needed for dialysis decreased as patient participation increased and vascular access changed from catheters to fistulae. Over the four-year period, the average overall needed nursing care time per haemodialysis session did not change. Conclusions: Our study shows that the average nursing time needed per patient did not change in the four-year observation period. However, more time is required for incident patients; thus, if a centre has high patient turnover, more nursing care time is needed

Applying a cost-based pricing model for innovative cancer treatments subject to indication expansion:A case study for pembrolizumab and daratumumab

BACKGROUND: Expanding the indication of already approved immuno-oncology drugs presents treatment opportunities for patients but also strains healthcare systems. Cost-based pricing models are discussed as a possibility for cost containment. This study focuses on two drugs, pembrolizumab (Keytruda) and daratumumab (Darzalex), to explore the potential effect of indication broadening on the estimated price when using the cost-based pricing (CBP) model proposed by Uyl-de Groot and Löwenberg (2018). METHODS: The model was used to calculate cumulative yearly prices, cumulative prices per indication, and non-cumulative indication-based prices using inputs such as research and development (R&amp;D) costs, manufacturing costs, eligible patient population, and a profit margin. A deterministic stepwise analysis and scenario analysis were conducted to examine how sensitive the estimated price is to the different input assumptions. RESULTS: The yearly cumulative cost-based prices (CBPs) ranged from €52 to €885 for pembrolizumab per vial and €823 to €31,941 for daratumumab per vial. Prices were higher in initial years or indications due to smaller patient populations, decreased over time or after additional indications. Sensitivity analysis showed that the number of eligible patients had the most significant impact on the estimated price. In the scenario analysis the profit margin contributed most to a higher CBPs for both drugs. Lower estimates resulted from assumed lower R&amp;D costs. DISCUSSION: The estimated CBPs are consistently lower than Dutch list prices for pembrolizumab (€2,861), mainly resulting from larger patient populations in registered indications. However, daratumumab's list prices fall within the range of modeled CBPs depending on the year or indication (€4,766). Both CBPs decrease over time or with additional indications. The number of eligible patients and initial R&amp;D costs have the most significant influence on the CBPs. These findings contribute to the ongoing discussions on pharmaceutical pricing, especially concerning cancer drugs with expanding indications.</p

International Society for Pharmacoeconomics and Outcomes Research Comments on the American Society of Clinical Oncology Value Framework

As members of the International Society for Pharmacoeconomics and Outcomes Research, we read with great interest the new American Society of Clinical Oncology (ASCO) conceptual framework to assess the value of cancer treatment options.1 We applaud the Value in Cancer Care Task Force for proposing a conceptual framework to support clinicians and patients in assessing the value of new cancer treatments. We acknowledge the challenges facing clinician–patient decision making, particularly concerning cancer treatments. Like ASCO, we recognize that the cost of treatments is increasingly being placed on patients through cost sharing and that engaging patients as part of making individual treatment decisions is of high importance. The ASCO framework highlights the growing tension among patients, insurance companies, and product manufacturers in a dynamic health care environment. In that light, the framework deserves a field test, and we look forward to seeing the outcome of that experience. We also appreciate the opportunity to offer comments and suggestions on the ASCO framework at this early stage, and our membership stands ready to support ASCO in future enhancements

Second-line treatment for acute graft-versus-host disease with mesenchymal stromal cells. a decision model

Objective: No standard second-line treatment exists for acute graft-versus-host disease steroid-refractory (SR-aGvHD), and long-term outcomes remain poor. Mesenchymal stromal cells (MSCs) have been evaluated as treatment, but no disease model (DM) exists that integrates and extrapolates currently available evidence. The aim of this study was to develop such a DM to describe the natural history of SR-aGvHD and to predict long-term outcomes. Method: The DM was developed in collaboration with experts in haematology-oncology. Subsequently, a model simulation was run. Input parameters for transition and survival estimates were informed by published data of clinical trials on MSC treatment for SR-aGvHD. Parametric distributions were used to estimate long-term survival rates after MSCs. Results: The newly developed DM is a cohort model that consists of eight health states. For the model simulation, we obtained data on 327 patients from 14 published phase II trials. Due to limited evidence, DM structure was simplified and several assumptions had to be made. Median overall survival was 3.2 years for complete response and 0.5 years for no complete response. Conclusion: The DM provides a comprehensive overview on the second-line treatment pathway for aGvHD and enables long-term predictions that can be used to perform a cost-effectiveness analysis comparing any treatment for SR-aGvHD