1.
Wiss, Johanna
(author)
Healthcare Priority Setting and Rare Diseases : What Matters When Reimbursing Orphan Drugs
2017
Doctoral thesis (other academic/artistic) 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.
Wiss, Johanna, 1984-, et al.
(author)
Prioritizing Rare Diseases: Psychological Effects Influencing Medical Decision Making
2017
In: Medical decision making. - : Sage Publications. - 0272-989X .- 1552-681X.
Journal article (peer-reviewed) abstract
Background. Measuring societal preferences for rarity has been proposed to determine whether paying pre- mium prices for orphan drugs is acceptable. Objective. To investigate societal preferences for rarity and how psychological factors affect such preferences. Method. A postal survey containing resource allocation dilemmas involving patients with a rare disease and patients with a common disease, equal in severity, was sent out to a randomly selected sample of the population in Sweden (return rate 42.3%, n = 1270). Results. Overall, we found no evidence of a general preference for prioritizing treat- ment of patients with rare disease patients over those with common diseases. When treatment costs were equal, most respondents (42.7%) were indifferent between the choice options. Preferences for prioritizing patients with common diseases over those with rare diseases were more frequently displayed (33.3% v. 23.9%). This tendency was, as expected, amplified when the rare disease was costlier to treat. The share of respondents choosing to treat patients with rare diseases increased when present- ing the patients in need of treatment in relative rather than absolute terms (proportion dominance). Surprisingly, identifiability did not increase preferences for rarity. Instead, identifying the patient with a rare disease made respondents more willing to prioritize the patients with common diseases. Respondents’ levels of education were significantly associated with choice—the lower the level of education, the more likely they were to choose the rare option. Conclusions. We find no support for the existence of a general preference for rarity when setting health care priorities. Psychological effects, especially proportion dominance, are likely to play an important role when pre- ferences for rarity are expressed.
3.
Andersson, Agneta, et al.
(author)
Costs of informal care for patients in advanced home care : a population based study
2003
In: International Journal of Technology Assessment in Health Care. - 0266-4623 .- 1471-6348. ; 19:4, s. 656-663
Journal article (peer-reviewed) 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.
4.
Lundqvist, Martina, 1986-, et al.
(author)
Effects of eating breakfast and school breakfast programmes on children and adolescents : a systematic review
2018
Reports (other academic/artistic) abstract
Background: Breakfast is often described as the most important meal of the day. The regularity of wholesome, daily meal patterns comprised of healthy foods is important for the physical and mental development of children and adolescents. One way to make sure that children and adolescents eat breakfast on a regular basis is to serve it at school. Several published reviews have examined the effects of eating breakfast and studied effects of school breakfast programmes on children and adolescents. Informed decisions of whether to promote eating breakfast or to introduce a school breakfast programme require a broader perspective.The aim was to conduct a systematic review of scientific publications that study the effects potentially relevant for economic evaluations of eating breakfast or implementing school breakfast programmes for children and adolescents.Method: In the systematic literature review, studies were identified by searching the electronic databases PubMed, CINAHL, Web of Science and PsycINFO from January 2000 through October 2017. The following inclusion criteria guided the selection of the identified studies: published articles from peer-reviewed journals with full text in English, studies collecting primary data, quantitative studies, studies performed in countries comparable to Sweden in terms of access and quality of nutrients, economic and social conditions and publications studying at least one of the topics; academic achievement, quality of life and wellbeing, risk factors/morbidity or cognitive performance.Results: Twenty-six studies on eating breakfast and eleven studies on school breakfast programmes fulfilled the inclusion criteria and were judged to be of at least moderate quality were included in the analysis. The results of the review of studies on eating breakfast showed positive and conclusive effects on cognitive performance, academic achievement, quality of life, well-being and risk factors/morbidity. This indicates that eating breakfast is important. The results of the studies on school breakfast varied. Minor positive effects were seen on cognitive performance and academic achievement. Most studies showed no effects and all studies suffered from different methodological weaknesses.Conclusions: The overall assessment of the studies on eating breakfast indicated positive effects. The studies on school breakfast programmes showed minor positive effects, but the majority of the studies showed no effects and all studies suffered from different methodological weaknesses. In order to assess the cost-effectiveness of eating breakfast and of school breakfast programmes we need to know more about how the identified affects influence the societal cost and the individuals gain in quality-adjusted life years both in the short and the long term.
