1.
Wiss, Johanna
(författare)
Healthcare Priority Setting and Rare Diseases : What Matters When Reimbursing Orphan Drugs
2017
Doktorsavhandling (övrigt vetenskapligt/konstnärligt) abstract
The rarity of a disease can give rise to challenges that differ from conventional diseases. For example, rarity hampers research and development of new drugs, and patients with severe, rare diseases have limited access to qualified treatments. When drugs are available, clinical evidence has higher uncertainty and the drugs can be very expensive. When setting priorities in the healthcare sector, treatments aimed at patients with rare diseases, so called orphan drugs, have become a source of concern. Orphan drugs seldom show solid evidence of effectiveness or cost-effectiveness. Still, treatments for rare disease patients, available on the European market, has increased rapidly since the adoption of a regulation offering incentives for research and development of orphan drugs. The question arises as to whether the publicly funded health care system should provide such expensive treatments, and if so, to what extent.This doctoral thesis aims to investigate healthcare priority setting and rare diseases in the context of orphan drug reimbursement. Priority setting for orphan drugs is located at the intersection of economic, ethical and psychological perspectives. This intersection is explored by studying the public’s view on the relevance of rarity when setting priorities for orphan drugs, and by examining how orphan drugs are managed when making reimbursement decisions in practice. Papers I and II in this thesis employ quantitative, experimental methods in order to investigate preferences for prioritising rare diseases, and the extent to which psychological factors influence such preferences. Papers III and IV employ qualitative methods to further explore what factors (apart from rarity) influence priority-setting decisions for orphan drugs, as well as how decisions regarding orphan drugs are made in practice in England, France, the Netherlands, Norway and Sweden. Combining quantitative and qualitative methods has provided a more comprehensive understanding of the topic explored in the thesis, and the methods have complemented each other.Paper I shows that there is no general preference for giving higher priority to rare disease patients when allocating resources between rare and common disease patients. However, results show that preferences for treating the rare patients are malleable to a set of psychological factors, in particular “proportion dominance”. Paper II shows that the identifiability of an individual has no, or a negative, influence on the share of respondents choosing to allocate resources to him/her (compared to a nonidentified individual). Paper III confirms that rarity per se is not seen as a factor that should influence priority-setting decisions (i.e. accept a greater willingness to pay for orphan drugs), however, other factors such as disease severity, treatment effect and whether there are treatment alternatives were seen as relevant for consideration. Paper IV explores the challenges with and solutions for orphan drug reimbursement, as perceived by different actors in five European countries. Perceived challenges are related to the components involved when making reimbursement decisions, to the reimbursement system, and to the acceptance of the final decision. Solutions are either specific for orphan drugs, or general measures that can be used for orphan drugs as well as for other drugs.In conclusion, priority setting for orphan drugs is complex and requires particular attention from decision makers. There are many factors to consider when making reimbursement decisions for orphan drugs. The consequences of a decision are potentially severe (both for rare disease patients and for common disease patients, depending on the decision) and psychological factors can potentially influence decisions.
2.
Eckard, Nathalie, et al.
(författare)
Reaching agreement in uncertain circumstances : the practice of evidence-based policy in the case of the Swedish National Guidelines for heart diseases
2017
Ingår i: Evidence and Policy: A Journal of Research, Debate and Practice. - : Informa Healthcare. - 1744-2648. ; :4, s. 687-707
Tidskriftsartikel (refereegranskat) abstract
This paper explores the practice of evidence-based policy in a Swedish healthcare context. The study focused on how policymakers in the specific working group, the Priority-Setting Group (PSG), handled the various forms of evidence and values and their competing rationalities, when producing the Swedish National Guidelines for heart diseases that are based on both clinical and economic evidence and are established to support explicit priority-setting in healthcare. The study contributes to the theoretical and practical debate on evidence-based policy (EBP) by illustrating how the practical tensions of coming to agreement were managed, to a large extent, through deliberation and by creativity.
3.
Eckard, Nathalie, 1973-
(författare)
The matter of economic evaluations in health policy decision-making : The case of the Swedish national guidelines for heart diseases
2015
Doktorsavhandling (övrigt vetenskapligt/konstnärligt) abstract
Economic evaluations are used to inform decision makers about the efficient allocation of scarce healthcare resources and are generated with the direct intent to support decisions in healthcare. Producing guidelines is a complex process and the inclusion of health-economic aspects in the formulation of the Swedish national guidelines as a basis for the written recommendations (priority gradings), distinguishes them from their European counterparts. Despite the increased use of cost-effectiveness data in decision-making, little is known about the actual use of such data. This thesis covers issues concerning how economic evaluations matter in health policy decision-making. The thesis includes four papers based on the Swedish national guidelines for heart diseases, one of the most prominent examples in Sweden of following the notion of evidence-based policy (EBP), in order to inform explicit priority setting.Both Papers I and II followed a qualitative case study design, based on the same data set. Paper I explored how a specific working group, the Priority Setting Group (PSG), handled the various forms of evidence and values when producing the national guidelines. Two themes were identified in reaching collective agreement in priority gradings; group facilitation activities and avoiding deadlock in the discussion. The work process involved disagreement and negotiation as part of that task. Paper I contributes to the theoretical and practical debate on EBP. Paper II focused on the use of cost-effectiveness data as decision support in the PSG work process. The paper addressed availability of cost-effectiveness data, evidence understanding, interpretation difficulties, and the reliance on evidence. Three themes were identified. The paper contributes to knowledge on how cost-effectiveness evidence was used in actual decision-making. The use of cost-effectiveness evidence was one of many tools employed to avoid deadlock in discussion and to reach a priority grading, when the overall evidence base was weak, in times of uncertainty and on the introduction of new expensive medical technologies.Quantitative research methods were used for both Papers III and IV. Paper III explored how the PSG was presented with cost-effectiveness evidence as decision support and as a basis for their priority gradings. Cost-effectiveness ratios (ICERs) were provided, based on a systematic literature review, as well as how the results may be conveyed and communicated, for the treatment of heart diseases using a cost-effectiveness ranking or league and providing valid information within a limited space, aiding decision makers on the allocation of healthcare resources. The thesis also includes decision support in the form of cost-effectiveness analysis on catheter ablation treatment. Paper IV provides an example of presenting evidence in the form of a decision-analytic model. The modelling approach provides an analytic framework for decision-making, specifically under conditions of uncertainty as in the introduction of new medical technology. Catheter ablation was associated with reduced cost and an incremental gain in quality adjusted life years (QALYs), and was considered a cost-effective treatment strategy compared to the medical treatment strategy in a lifetime perspective.
4.
Jendle, Johan, 1963-, et al.
(författare)
A utility valuation study assessing the impact of postprandial glucose control on quality of life of individuals with type 1 or type 2 diabetes
2018
Ingår i: Journal of Patient-Reported Outcomes. - : Springer Nature. - 2509-8020. ; 2:20
Tidskriftsartikel (refereegranskat) abstract
Background: Consideration of health-related quality of life (HRQOL) in diabetes has been associated with long-term and short-term complications such as hypoglycaemia, but not with short-term glucose control. This study aimed to collect health utilities related to different degrees of poorly controlled postprandial glucose (PPG) and its impact on HRQOL in the UK and in Sweden.Methods: Three health state descriptions were developed based on literature reviews and interviews with people with diabetes and healthcare professionals, characterising mild, moderate and severe impact of postprandial hyperglycaemic symptoms on HRQOL. Time Trade-Off (TTO) interviews with a 10-year trade-off period were conducted with samples of the UK general public and of Swedish people with diabetes. Mean TTO-derived health state values were expressed on a scale from 0 (death) to 1 (full health).Results: One hundred fifty participants from the general population were interviewed in the UK (57% female, mean age 35 years) and 150 participants with diabetes in Sweden (64% female, mean age 51 years, 42% type 1 and 58% type 2 diabetes). The mean TTO-derived health state values were for the UK and Swedish participants: mild impact of poorly PPG control (0.89/0.76); moderate (0.75/0.71); severe (0.56/0.58).Conclusions: Glucose lowering treatments associated with improved control over PPG levels could have important benefits to people with type 1 and type 2 diabetes since findings suggest that increasing severity in postprandial hyperglycaemic symptoms is perceived as having significant negative impact on HRQOL of individuals with type 1 or type 2 diabetes.
5.
Alwin, Jenny, et al.
(författare)
Utvärdering av försöksverksamhet med service- och signalhundar
2014
Rapport (övrigt vetenskapligt/konstnärligt) abstract
Denna rapport redovisar utvärderingen av en försöksverksamhet med ser-vice- och signalhundar som bedrevs mellan år 2009 och 2014. Utvärderingen inkluderar servicehundar, signalhundar samt alarmerande servicehundar (epilepsihundar och diabeteshundar). Totalt 56 ekipage (förare samt hund) ingick i utvärderingsstudien. Data i studien samlades in före samt efter genomgången service- och signalhundsutbildning. Syftet med utvärderingen är att studera hur certifierade service- och signalhundar påverkar förarnas behov av offentliga stödinsatser och de totala samhällskostnaderna. Dessutom studerades hur service- och signalhundar påverkar förarna med avseende på hälsorelaterad livskvalitet, välbefinnande, självförtroende och fysisk aktivitet samt om användningen av service- och signalhundar är kostnadseffektiv ur ett samhällsperspektiv. Resultat och slutsatserService- och signalhundar minskar i genomsnitt förarnas behov av offentliga stödinsatser med 197 000 kronor (6 procent) under en tioårsperiod.Livskvaliteten för personer med behov av service- och signalhundar är låg jämfört med den allmänna populationen i Sverige. Studien visar på en förbättring i livskvaliteten och välbefinnandet för förare med en certifierad hund.Förarnas grad av fysisk aktivitet ökade med en certifierad hund och majoriteten av dem angav att de ökat sin tid utanför hemmet samt att de även ökat sin tid för att delta i sociala aktiviteter tack vare hunden.Förarnas negativa emotionella upplevelser minskar med en certifierad hund.Förarna beskriver själva den certifierade hunden som ett viktigt verktyg för ökad självständighet och trygghet.Den hälsoekonomiska modellen som analyserar kostnadseffektiviteten visar att ett innehav av en certifierad hund är ett dominant alternativ jämfört med att inte ha en certifierad hund. Detta innebär att kostnaderna ur ett samhällsperspektiv under en tioårsperiod är lägre (-103 000 kronor) samtidigt som effekterna i form av vunna QALY (kvalitetsjusterade levnadsår) är högre (+0,15).Finansieringsanalysen visar att förare som har certifierade hundar sparar resurser åt alla aktörer (stat, kommun och landsting) men får själva ökade utgifter på grund av hunden.Studien baseras på ett lågt antal observationer (56 ekipage). Det i kombination med att det är en stor spridning i resursförbrukningen mellan ekipagen medför att det finns en statistisk osäkerhet i resultaten. Slutsatserna bedöms dock som rimliga eftersom de är samstämmiga.
6.
Andersson, Agneta, et al.
(författare)
Costs of informal care for patients in advanced home care : a population based study
2003
Ingår i: International Journal of Technology Assessment in Health Care. - 0266-4623 .- 1471-6348. ; 19:4, s. 656-663
Tidskriftsartikel (refereegranskat) abstract
Objectives: Several studies have sought to analyze the cost-effectiveness of advanced home care andhome rehabilitation. However, the costs of informal care are rarely included in economic appraisals ofhome care. This study estimates the cost of informal care for patients treated in advanced home careand analyses some patient characteristics that influence informal care costs.Methods: During one week in October 1995, data were collected on all 451 patients in advanced homecare in the Swedish county of O¨ stergo¨ tland. Costs were calculated by using two models: one includingleisure time, and one excluding leisure time. Multiple regression analysis was used to analyze factorsassociated with costs of informal care.Results: Seventy percent of the patients in the study had informal care around the clock during theweek investigated. The patients had, on average, five formal care visits per week, each of which lastedfor almost half an hour. Thus, the cost of informal care constituted a considerable part of the costof advanced home care. When the cost of leisure time was included, the cost of informal care wasestimated at SEK 5,880 per week per patient, or twice as high as total formal caregiver costs. Whenleisure time was excluded, the cost of informal care was estimated at SEK 3,410 per week per patient,which is still 1.2 times higher than formal caregiver costs (estimated at SEK 2,810 per week per patient).Informal care costs were higher among patients who were men, who were younger, who had their ownhousing, and who were diagnosed with cancer.Conclusions: Studies of advanced home care that exclude the cost of informal care substantiallyunderestimate the costs to society, regardless of whether or not the leisure time of the caregiver isincluded in the calculations.
7.
Aronsson, Mattias, et al.
(författare)
Cost-effectiveness of endovascular thrombectomy in patients with acute ischemic stroke.
2016
Ingår i: Neurology. - : American Academy of neurology. - 0028-3878 .- 1526-632X. ; 86:11, s. 1053-9
Tidskriftsartikel (refereegranskat) abstract
Objective:To evaluate the cost-effectiveness of adding endovascular thrombectomy to standard care in patients with acute ischemic stroke.Methods:The cost-effectiveness analysis of endovascular thrombectomy in patients with acute ischemic stroke was based on a decision-analytic Markov model. Primary outcomes from ESCAPE, Extending the Time for Thrombolysis in Emergency Neurological Deficits-Intra-Arterial (EXTEND-IA), Multicenter Randomized Clinical Trial of Endovascular Treatment for Acute Ischemic Stroke in the Netherlands (MR CLEAN), Endovascular Revascularization With Solitaire Device Versus Best Medical Therapy in Anterior Circulation Stroke Within 8 Hours (REVASCAT), and Solitaire with the Intention for Thrombectomy as Primary Endovascular Treatment for Acute Ischemic Stroke (SWIFT PRIME) along with data from published studies and registries were used in this analysis. We used a health care payer perspective and a lifelong time horizon to estimate costs and effects.Results:The model showed that adding thrombectomy with stent retrievers to guideline-based care (including IV thrombolysis) resulted in a gain of 0.40 life-years and 0.99 quality-adjusted life-years along with a cost savings of approximately $221 per patient. The sensitivity analysis showed that the results were not sensitive to changes in uncertain parameters or assumptions.Conclusions:Adding endovascular treatment to standard care resulted in substantial clinical benefits at low costs. The results were consistent throughout irrespective of whether data from ESCAPE, EXTEND-IA, MR CLEAN, REVASCAT, or SWIFT PRIME were used in this model.
8.
Aronsson, Mattias, et al.
(författare)
Cost-effectiveness of high-sensitivity faecal immunochemical test and colonoscopy screening for colorectal cancer
2017
Ingår i: British Journal of Surgery. - : WILEY. - 0007-1323 .- 1365-2168. ; 104:8, s. 1078-1086
Tidskriftsartikel (refereegranskat) abstract
Background: Colorectal cancer screening can decrease morbidity and mortality. However, there are widespread differences in the implementation of programmes and choice of strategy. The primary objective of this study was to estimate lifelong costs and health outcomes of two of the currently most preferred methods of screening for colorectal cancer: colonoscopy and sensitive faecal immunochemical test (FIT). Methods: A cost-effectiveness analysis of colorectal cancer screening in a Swedish population was performed using a decision analysis model, based on the design of the Screening of Swedish Colons (SCREESCO) study, and data from the published literature and registries. Lifelong cost and effects of colonoscopy once, colonoscopy every 10 years, FIT twice, FIT biennially and no screening were estimated using simulations. Results: For 1000 individuals invited to screening, it was estimated that screening once with colonoscopy yielded 49 more quality-adjusted life-years (QALYs) and a cost saving of (sic)64 800 compared with no screening. Similarly, screening twice with FIT gave 26 more QALYs and a cost saving of (sic)17 600. When the colonoscopic screening was repeated every tenth year, 7 additional QALYs were gained at a cost of (sic)189 400 compared with a single colonoscopy. The additional gain with biennial FIT screening was 25 QALYs at a cost of (sic)154 300 compared with two FITs. Conclusion: All screening strategies were cost-effective compared with no screening. Repeated and single screening strategies with colonoscopy were more cost-effective than FIT when lifelong effects and costs were considered. However, other factors such as patient acceptability of the test and availability of human resources also have to be taken into account.
9.
Aronsson, Mattias, 1989-, et al.
(författare)
Designing an optimal screening program for unknown atrial fibrillation : a cost-effectiveness analysis.
2017
Ingår i: Europace. - Oxford : Oxford University Press. - 1099-5129 .- 1532-2092. ; 19:10, s. 1650-1656
Tidskriftsartikel (refereegranskat) abstract
Aims: The primary objective of this study was to use computer simulations to suggest an optimal age for initiation of screening for unknown atrial fibrillation and to evaluate if repeated screening will add value.Methods and results: In the absence of relevant clinical studies, this analysis was based on a simulation model. More than two billion different designs of screening programs for unknown atrial fibrillation were simulated and analysed. Data from the published scientific literature and registries were used to construct the model and estimate lifelong effects and costs. Costs and effects generated by 2 147 483 648 different screening designs were calculated and compared. Program designs that implied worse clinical outcome and were less cost-effective compared to other programs were excluded from the analysis. Seven program designs were identified, and considered to be cost effective depending on what the health-care decision makers are ready to pay for gaining a quality-adjusted life-year (QALY). Screening at the age of 75 implied the lowest cost per gained QALY (€4 800/QALY).Conclusion: In conclusion, examining the results of more than two billion simulated screening program designs for unknown atrial fibrillation, seven designs were deemed cost-effective depending on how much we are prepared to pay for gaining QALYs. Our results showed that repeated screening for atrial fibrillation implied additional health benefits to a reasonable cost compared to one-off screening.
10.
Aronsson, Mattias, 1989-
(författare)
Health Economic Evaluations of Screening Programs - Applications and Method Improvements
2017
Doktorsavhandling (övrigt vetenskapligt/konstnärligt) abstract
Screening to detect diseases early is attractive as it can improve the prognosis and decrease costs, but it is often a problematic concept and there are several pitfalls. Many healthy individuals have to be investigated to avoid a disease in a few, which results in a dilemma because to save a few, many are exposed to a procedure that could potentially harm them. Other examples of problems associated with screening are latent diseases and over-treating. The question of optimal design of a screening program is another source of uncertainty for decision-makers, as a screening program may potentially be implemented in very different ways. This highlights the need for structured analyses that weigh benefits against the harms and costs that occur as consequences of the screening.The aim of this thesis is, therefore, to explore, develop and implement methods for health economic evaluations of screening programs. This is done to identify problems and suggest solutions to improve future evaluations and in extension policy making.This aim was analysed using decision analytic cost-effectiveness analyses constructed as Markov models. These are well-suited for this task given the sequential management approach where all relevant data are unlikely to come from a single source of evidence. The input data were in this thesis obtained from the published literature and were complemented with data from Swedish registries and the included case studies. The case studies were two different types of screening programs; a program of screening for unknown atrial fibrillation and a program to detect colorectal cancer early. Further, the implementation of treatment with thrombectomy and novel oral anticoagulants were used to illustrate how factors outside the screening program itself have an impact on the evaluations.As shown by the result of the performed analyses, the major contribution of this thesis was that it provided a simple and systematic approach for the economic evaluation of multiple screening designs to identify an optimal design.In both the included case studies, the screening was considered costeffective in detecting the disease; unknown atrial fibrillation and colorectal cancer, respectively. Further, the optimal way to implement these screening programs is dependent on the threshold value for cost-effectiveness in the health care sector and the characteristics of the investigated cohort. This is because it is possible to gain increasingly more health benefits by changing the design of the screening program, but that the change in design also results in higher marginal costs. Additionally, changes in the screening setting were shown to be important as they affect the cost-effectiveness of the screening. This implies that flexible modelling with continuously updated models are necessary for an optimal resource allocation.
