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  • 1.
    Carlsson, Per
    et al.
    Linköping University, Department of Medical and Health Sciences, Division of Health Care Analysis. Linköping University, Faculty of Health Sciences.
    Hoffman, Mikael
    Linköping University, Department of Medical and Health Sciences, Division of Drug Research. Linköping University, Faculty of Health Sciences. Nätverk för läkemedelsepidemiologi (NEPI).
    Levin, Lars-Åke
    Linköping University, Department of Medical and Health Sciences, Division of Health Care Analysis. Linköping University, Faculty of Health Sciences.
    Sandman, Lars
    Linköping University, Department of Medical and Health Sciences, Division of Health Care Analysis. Linköping University, Faculty of Health Sciences. Högskolan i Borås, Institutionen för vårdvetenskap.
    Wiss, Johanna
    Linköping University, Department of Medical and Health Sciences, Division of Health Care Analysis. Linköping University, Faculty of Health Sciences.
    Prioritering av läkemedel för behandling av patienter med sällsynta sjukdomar2014In: Läkemedel för djur, maskinell dos och sällsynta tillstånd: hantering och prissättning. Slutbetänkande av Läkemedels- och apoteksutredningen, Stockholm: Fritzes, 2014, p. 639-699Chapter in book (Other academic)
    Abstract [sv]

    I vilken utsträckning kan särskilda hänsyn tas vid prioritering av särläkemedel och läkemedel för behandling av sällsynta tillstånd? Bör man i vissa speciella situationer kunna acceptera sämre kostnadseffektivitet och lägre krav på vetenskapligt underlag? Om så, vad är det för villkor/kriterier som då bör vara uppfyllda? Hur stämmer ett sådant undantag med den etiska plattformen för prioriteringar? Finns det andra argument som talar för eller mot en särbehandling av sällsynta tillstånd? Detta är frågor som vi diskuterar i denna rapport som tagits fram på uppdrag av Läkemedels- och apoteksutredningen.

    I rapporten konstaterar vi att särläkemedel är en relativt heterogen grupp av läkemedel och att det finns andra läkemedel med motsvarande egenskaper som inte ansökt eller kunnat ansöka om status som särläkemedel. Ur prioriteringssynpunkt är det därför mer relevant att fokusera på alla typer av läkemedel, som används för sällsynta tillstånd, när vi diskuterar vilka principer för prioriteringar som bör gälla.

    Utifrån litteratur och erfarenheter i andra länder, som formulerat kriterier för en särskild hantering av läkemedel vid sällsynta sjukdomar, drar vi den preliminära slutsatsen att samhället bör kunna betala mer per hälsovinst (kostnad per kvalitetsjusterat levnadsår (QALY)) och acceptera lägre krav på vetenskapligt underlag vid prioritering av läkemedel för behandling av sällsynta sjukdomar om samtliga följande villkor är uppfyllda:

    • att behandlingen har en hög kostnad per hälsovinst som en konsekvens av att den omfattar endast få patienter,
    • att det rör sig om ett tillstånd med mycket stor svårighetsgrad,
    • att det behandlingsalternativ som övervägs på goda grunder ska antas ha en väsentlig effekt,
    • att det inte finns någon alternativ behandling med en väsentlig effekt som förväntas förebygga, bota, fördröja försämring eller lindra det aktuella tillståndet.

    Om dessa kriterier anses uppfyllda kan läkemedlet bedömas i relation till ett förhöjt tröskelvärde för kostnadseffektivitet. Hur högt detta värde kan vara för att anses rimligt tar vi inte ställning till i rapporten, men en viktig ståndpunkt är att det måste finnas en övre gräns för hur hög kostnad per QALY samhället kan acceptera. Detta bör gälla även om ovanstående kriterier är uppfyllda, annars riskerar det uppstå oacceptabla undanträngningseffekter av andra prioriterade åtgärder. Även lägre krav på vetenskapligt underlag skakunna accepteras....

  • 2.
    Carlsson, Per
    et al.
    Linköping University, Department of Medical and Health Sciences, Health Technology Assessment and Health Economics. Linköping University, Faculty of Health Sciences.
    Hoffmann, Mikael
    Linköping University, Department of Medical and Health Sciences, Clinical Pharmacology. Linköping University, Faculty of Health Sciences.
    Levin, Lars-Åke
    Linköping University, Department of Medical and Health Sciences, Health Technology Assessment and Health Economics. Linköping University, Faculty of Health Sciences.
    Sandman, Lars
    Högskolan i Borås.
    Wiss, Johanna
    Linköping University, Department of Medical and Health Sciences, Health Technology Assessment and Health Economics. Linköping University, Faculty of Health Sciences.
    Prioritering och finansiering av läkemedel för behandling av patienter med sällsynta sjukdomar: Reviderad version2012Report (Other academic)
    Abstract [en]

    An addendum to the terms of reference for the inquiry on certain issues regarding pricing, accessibility, and market conditions in the pharmaceutical and pharmacy sector (Dir. 2011:82) calls for an analysis of the need for special solutions in decisions on subsidising orphan drugs. An orphan drug is a drug that fulfils certain conditions and is thereby covered by special stimulus measures prior to approval by agencies that evaluate medical products. Orphan drugs also have the possibility, but not the right, to hold sole rights in the marketplace for 10 years.

    The report includes an analysis of the ethics platform and the Pharmaceutical Benefits Act, which serve as the Dental and Pharmaceutical Benefits Agency’s base for making decisions on subsidies. One conclusion drawn is that the current ethics platform, along with the modification of the cost-effectiveness principle that appears in the Pharmaceutical Benefits Act, offers the option to determine that society is prepared to pay more per health benefit for drugs targeted at very rare and severe diseases/conditions.

    The human dignity principle indicates that irrelevant group affiliation or group characteristics should not affect equality in a patient group’s opportunities and outcomes regarding health. Whether a patient group is small or large is an irrelevant group characteristic, and the size of the group or the rarity of the condition should not affect the group’s opportunities for treatment, or the possibility to achieve an equitable health outcome compared to other larger groups or groups with more common diseases. However, the consequence of higher costs for intervention that can result from rarity could be grounds for special treatment.

    The needs and solidarity principle indicates that all citizens should be given equal opportunities to achieve good health and that we should achieve health outcomes that are as equitable as possible for citizens. If we allow the development costs for orphan drugs to influence the possibility to access these drugs, then the citizens are not given equal opportunities to achieve good health. Hence, we draw the conclusion that people with rare and severe conditions should be given opportunities to achieve good health that are equal to those given to people with common and serious conditions.

    The cost-effectiveness principle. When lower cost effectiveness is primarily due to high costs connected with the size of the patient group, i.e. the market size for a drug, and not poor effects from the intervention, there is just cause, on the grounds of equity, to try to influence the cost aspects of the drug and in some cases accept a lower level of cost effectiveness. The cost can be influenced, for example, by pricing based on special agreements with the company marketing the drug.

    There is value in knowing that we live in a compassionate society that cares for and attempts to help people that find themselves in life-threatening or other difficult situations. This value stems partly from the assertion that knowledge creates greater security and trust in the community (based on self interest since we are all at risk of contracting a severe condition) and partly from a more altruistic motivation stemming from the assertion that we are affected positively by knowing that we live in a compassionate society. This is a factor that should also be considered in cost-effectiveness analyses. The size of this value is uncertain.

    Orphan drugs are a relatively heterogeneous group, and there are other drugs with corresponding characteristics that have not applied for status as orphan drugs. From a standpoint of priority setting, it is more relevant to focus instead on drugs that are used for rare and severe diseases. Drugs for very rare and severe conditions, particularly when no other treatment is available, should therefore be processed in a special manner regardless of whether or not they are classified as an orphan drug. To assure equitable access, transparent decisions, and appropriate care it is preferable to use a model involving national decision- making for such drugs. Three alternative principles for decisions and financing can be discussed: 1) the state as the principal authority, 2) collaboration between the state and the county councils, and 3) collaboration among the county councils.

  • 3.
    Carlsson, Per
    et al.
    Linköping University, Department of Medical and Health Sciences, Division of Health Care Analysis. Linköping University, Faculty of Health Sciences.
    Hoffmann, Mikael
    Nätverk för läkemedelsepidemiologi, NEPI.
    Levin, Lars-Åke
    Linköping University, Department of Medical and Health Sciences, Division of Health Care Analysis. Linköping University, Faculty of Health Sciences.
    Sandman, Lars
    Linköping University, Department of Medical and Health Sciences, Division of Health Care Analysis. Linköping University, Faculty of Health Sciences.
    Wiss, Johanna
    Linköping University, Department of Medical and Health Sciences, Division of Health Care Analysis. Linköping University, Faculty of Health Sciences.
    Prioritering och finansiering av läkemedel för behandling av patienter med sällsynta sjukdomar: Reviderad version2015Report (Other academic)
    Abstract [en]

    To what extent can special consideration be given towards prioritising orphan drugs and drugs for treating rare conditions? In certain special situations should we accept lower cost effectiveness and lower standards for scientific evidence? If so, what are the conditions/criteria that should be fulfilled? How would such exceptions fit in with the ethical platform for priority setting? Are there other arguments that speak for or against special handling of rare conditions? These are questions we discuss in this report commissioned by the Committee on Pharmaceuticals and Pharmacies.

    In this report we describe orphan drugs as a relatively heterogeneous group of pharmaceuticals, and discuss that other drugs with similar characteristics have not applied for, or were able to apply for, status as orphan drugs. Hence, from a priority setting perspective it is more relevant to focus on all types of drugs used for rare conditions when we discuss the principles that should apply when setting priorities.

    Based on literature and experiences from other countries that have formulated criteria for special handling of drugs for rare diseases, we have drawn the preliminary conclusion that society should be able to pay more per health gain (cost per quality-adjusted life year [QALY]) and accept lower standards for scientific evidence when prioritising drugs for treating rare diseases if all of the following conditions are met:

    • that treatment has a high cost per health gain as a consequence treating only a few patients
    • that it involves a health condition with a very high level of severity
    • that the treatment option being considered is assumed, based on firm grounds, to have a substantial effect
    • that no alternative treatment having a substantial effect is available that can be expected to prevent, cure, delay, or ameliorate the health condition in question.

    If these criteria are met, the drug can be evaluated in relation to an elevated threshold value for cost effectiveness. In this report we do not address how high this value should be to be considered reasonable, but an important point is that there must be an upper limit regarding how high of a cost per QALY society can accept. This should apply even if the above criteria are met to avoid the risk of unacceptable displacement of other prioritised interventions. Even lower standards for scientific evidence could be acceptable.

    These preliminary conclusions were tested against the Swedish ethical platform and from a perspective of welfare economics.

    Our conclusions from the ethical analysis are:

    The human dignity principle allows for special handling of rare conditions that can generate high treatment costs. Hence, our first criterion is acceptable based on the ethical platform. When a higher threshold value (cost per health gain achieved) for treating rare conditions is accepted, these patients are given greater equity in access to health compared to other patients (assuming that the other criteria are also met). However, this does not automatically mean that all rare conditions generating high treatment costs should receive special handling.

    According to the needs and solidarity principle society should strive, to the extent possible, to give all citizens equal opportunities for care; but also enable them to achieve a level of health that is as equitable as possible. The latter assumes that we primarily prioritise those who are furthest from such an equitable level, i.e. those with the greatest needs. To be able to influence the opportunities and outcomes regarding health, it is essential that the interventions used can actually help people approach more equitable outcomes, i.e. that these interventions have a substantial effect on health.

    The cost-effectiveness principle states that a reasonable relationship between costs and effects should be pursued in choosing between different interventions or areas of activity. Very severe conditions should take precedence over minor conditions, even if the interventions, in contrast to the former, are associated with relatively higher costs per health gain. When the above criteria are met in treating a rare condition it only means that the treatment can be subject to evaluation of whether a higher threshold value can be accepted. Hence, this does not automatically mean that the treatment should be offered or that a drug, for example, should be included among the pharmaceutical benefits. Even when these criteria are met, and hence there is reason for special handling of the treatment, decisions makers in this context should decide what constitutes a reasonable relationship between costs and effects, i.e. what is an acceptable threshold value in a particular decision-making situation.

    In conclusion, we believe that the current ethical platform along with the modification of the cost effectiveness principle, which appears in more recent legislation, offers the opportunity to give special consideration when prioritising drugs targeted at rare and very severe conditions. For a drug to be considered for special handling, it should have a substantial effect on the condition in question. There is a difference between treatments that have poor cost effectiveness due to minor effects and those that have poor cost effectiveness due to high costs. The difference arises since the effect size of the treatment influences its potential to contribute to an outcome in line with the health and quality of life of the rest of the population, where a greater effect size approaches this in a better way. Our interpretation is that if there are two interventions with the same cost effectiveness ratio and the same severity level, but where one has greater effects on the condition than the other, it is the one with the greatest effects that should be given priority.

    We also studied whether there are any arguments from a socioeconomic efficiency standpoint that speak in favour of or against special handling of rare conditions. This has been done based on theoretical and empirical literature on the subject and preliminary data from a Swedish study. Some studies suggest there is a social value: first in the knowledge that people receive care regardless of the situation, which creates greater security and confidence in society based on self interest since everyone in society is at risk for severe conditions; and second, based on more altruistic motivation where people in general can be influenced in a positive way knowing that they live in a compassionate society.

    However, purely empirical studies do not offer any evidence that people in general think that treatment of rare diseases should receive special handling, everything else being equal. In other words, no empirical studies show that rarity per se is a relevant criterion in priority setting – this was also the conclusion of our previous ethical argument in this context (see Carlsson et al 2012). Preliminary data from an ongoing Swedish study support this conclusion to some extent. When complementing the analysis in the same study with attitude questions and focus group interviews, a more detailed picture emerges – namely, support can be found for special handling if the condition is rare, has a high level of severity, and is the only treatment option.

    After deciding that special consideration can be given in prioritising drugs for severe and rare conditions, based on the ethical platform and from a welfare economy perspective, positions must be taken on a series of practical considerations in designing procedures for priority setting, namely:

    • Based on the goal to reduce differences and guarantee care on equal terms for rare conditions, it would be preferable to use a model involving national decision making. The same principles for prioritising drugs for rare and severe conditions should apply regardless of how the drug reaches the patient, i.e. regardless of whether it involves prescribed or requisitioned drugs. Whether one or more bodies set priorities is an organisational issue that we do not address in this report.
    • From a priority setting standpoint there should be no difference between orphan drugs and other drugs used for rare conditions. When it comes to decisions on whether orphan drugs in general should be included under the pharmaceutical benefits system, they should be assessed and approved by the Dental and Pharmaceutical Benefits Agency in usual fashion, while drugs targeted at conditions that are both very rare and very severe may need special consideration and handled according to a modified procedure. This should include the cost per health gain and dealing with uncertainty of the scientific evidence, and should apply whether or not the drug has orphan status.
    • In some situations where the scientific evidence is uncertain, problems arise in deciding on general use of expensive drugs for severe diseases on a group level. In this context it seems reasonable to initially allow some exceptions in the evidence for cost effectiveness related to severe diseases when, for various reasons, practical factors stand in the way of producing such evidence. However, a conflict can arise in both accepting a higher willingness to pay and accepting greater uncertainty in the scientific evidence. In such cases it is particularly important to pursue adequate follow-up and try to reduce uncertainty as quickly as possible and reassess the decision.
    • Various principles are available to define patient groups with very severe and rare conditions. Groups may be defined based on the prevalence of the condition in the population or on the number of patients with a given condition that the drug in question will be used to treat (in other words, the number of patients who are potential candidates for treatment). The latter method appears to be advantageous when the particular problem we address here relates to the fact that few patients are candidates for treatment, not how usual/unusual the condition/disease is. In the report we do not take a definitive position on what constitutes a suitable limit. This needs further study. However, we believe that such a limit should be set much lower than the usual definition for orphan drugs. In Sweden’s case, a reasonable starting point for discussion could be that the expected estimated size of the patient group should not exceed 200 patients (1 in 50 000 inhabitants) during 5 years. Experience from England, for instance, supports this definition.
    • One way to arrive at a reasonable threshold for cost effectiveness in different situations involving rare conditions is to designate some entity to make decisions transparently. Such decisions could then be discussed broadly and possibly be subject to reassessment. By comparing with other situations in health care and society where decisions are made on interventions for severe conditions and small patient groups, the development of accepted practice can be accelerated. Even comparisons with decisions in other countries can give an indication of reasonable threshold values in various situations.
  • 4.
    Gustavsson, Erik
    et al.
    Linköping University, Department of Medical and Health Sciences, Division of Health and Society. Linköping University, Department of Medical and Health Sciences, Division of Health Care Analysis. Linköping University, Faculty of Health Sciences.
    Sandman, Lars
    Linköping University, Department of Medical and Health Sciences, Division of Health Care Analysis. Linköping University, Faculty of Health Sciences. Högskolan i Borås, Institutionen för vårdvetenskap.
    Wiss, Johanna
    Linköping University, Department of Medical and Health Sciences, Division of Health Care Analysis. Linköping University, Faculty of Health Sciences.
    Rättviseteorier och prioriteringar2013In: Att välja rättvist: om prioriteringar i hälso- och sjukvården / [ed] Per Carlsson och Susanne Waldau, Lund: Studentlitteratur, 2013, 1, p. 49-64Chapter in book (Other academic)
    Abstract [sv]

    Hälso- och sjukvårdens resurser räcker inte till alla behov och önskemål från patienter och medborgare, vilket gör att personal och beslutsfattare hamnar i svåra situationer. Hur ska vi veta att det är rätt patienter som tvingas stå tillbaka? Att välja rättvist tar upp centrala begrepp och etiska principer kring prioriteringar. Boken beskriver även metoder för att göra prioriteringar på ett systematiskt sätt och erfarenheter av såväl nationella som internationella prioriteringar.

    Prioriteringsbeslut fattas på alla nivåer och kan gälla fördelning av resurser till olika verksamheter, behandlingsbeslut av enskilda patienter eller investeringar i nya medicinska metoder. Boken ger förslag på hur beslutsunderlagen kan förbättras. Här ges anvisningar om hur man mäter behov och nytta hos patientgrupper och i befolkningen, hur kostnadseffektivitet beräknas och hur man skapar ett kunskapsunderlag. På så sätt får läsaren inte bara ta del av prioriteringarnas teori utan även av deras praktik, inte minst genom konkreta exempel på hur öppna prioriteringar i dag tillämpas i svensk hälso- och sjukvård.

    Boken är avsedd för dig som arbetar kliniskt eller planerar att arbeta med prioriteringar – nationellt, i landsting eller i kommuner. Boken ger även en värdefull inblick för dig som vill lära mer om hur vårdens svåra val ska kunna hanteras i framtiden.

  • 5.
    Gustavsson, Erik
    et al.
    Linköping University, Department of Medical and Health Sciences, Division of Health Care Analysis. Linköping University, Faculty of Health Sciences. Linköping University, Department of Medical and Health Sciences, Division of Health and Society. Linköping University, Faculty of Arts and Sciences.
    Wiss, Johanna
    Linköping University, Department of Medical and Health Sciences, Division of Health Care Analysis. Linköping University, Faculty of Health Sciences.
    Multikriterieanalyser vid prioriteringar inom hälso- och sjukvården: kriterier och analysmetoder2013Report (Other academic)
    Abstract [en]

    The first part of this report draws attention to the vast amount of different criteria for policymakers to consider in priority setting decisions. There are several similarities between criteria that have been used in decision making in different countries and regions. However, it is interesting to highlight an important difference between the Swedish ethical platform and other countries and regions. The difference lies in the lexical ordering between the principles in the Swedish platform. This implies that one should take into account the principle of human dignity before the principle of need and solidarity and that the same relation should apply between the principle of need and solidarity and the principle of cost effectiveness. This strict hierarchy does not exist between principles in other countries. Similarities between different ethical platforms are especially found in relation to the principle of need or severity of the disease of which both play an important role in all countries and regions. Cost- effectiveness is another criterion that appears in all platforms that we have included in this report. Furthermore, it should be mentioned that the effect of an intervention plays an important role in all of the studies and reports that we have included. In several cases, there is a special focus on the prioritization of interventions with a proven efficacy. The above mentioned criteria are the most commonly present in reports on priority setting in health care but there are many other criteria which may be relevant to consider. It should also be noted that there are great similarities between the various reports on what should not constitute a basis for prioritization. For example, many countries and regions are reluctant to include chronological age as one criterion in their official reports.

    The second part of the report presents the methods of multi-criteria analysis; an umbrella term for various methods that uses a formalized process and ranks different alternatives on how they perform on a number of selected criteria. The different steps of a general multi-criteria analysis are presented: (1) identification of the object (2) construction of the model (3) evaluation of results and (4) the development of an action plan. A performance matrix, which is a standard tool for multi-criteria, is illustrated. In the next step, a selection of a number of various methods is presented, namely: direct analysis of the performance matrix, ordinal methods, the even swap-method, multi-attribute utility theory (MAUT), and the analytic hierarchy process (AHP). A number of applications of multi-criteria analysis have been performed in the area of health care decision making and the advantages and disadvantages of applying these methods in this area are highlighted.

  • 6.
    Wiss, Johanna
    Linköping University, Department of Medical and Health Sciences, Division of Health Care Analysis. Linköping University, Faculty of Medicine and Health Sciences.
    Healthcare Priority Setting and Rare Diseases: What Matters When Reimbursing Orphan Drugs2017Doctoral thesis, comprehensive summary (Other academic)
    Abstract [en]

    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.

    List of papers
    1. Prioritizing Rare Diseases: Psychological Effects Influencing Medical Decision Making
    Open this publication in new window or tab >>Prioritizing Rare Diseases: Psychological Effects Influencing Medical Decision Making
    2017 (English)In: Medical decision making, ISSN 0272-989X, E-ISSN 1552-681XArticle in journal (Refereed) Published
    Abstract [en]

    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.  

    Place, publisher, year, edition, pages
    Sage Publications, 2017
    Keywords
    orphan drugs; rare diseases; priority setting; societal preferences; survey; resource allocation; proportion dominance; identifiability.
    National Category
    Economics Health Care Service and Management, Health Policy and Services and Health Economy Psychology (excluding Applied Psychology)
    Identifiers
    urn:nbn:se:liu:diva-134647 (URN)10.1177/0272989X17691744 (DOI)000403060600008 ()
    Funder
    Ragnar Söderbergs stiftelseMarianne and Marcus Wallenberg Foundation
    Note

    Funding agencies: Swedish Association of the Pharmaceutical Industry (LIF); Ragnar Soderberg Foundation

    Available from: 2017-02-21 Created: 2017-02-21 Last updated: 2018-04-09Bibliographically approved
    2. The influence of identifiability and singularity in moral decision making
    Open this publication in new window or tab >>The influence of identifiability and singularity in moral decision making
    Show others...
    2015 (English)In: Judgment and decision making, ISSN 1930-2975, E-ISSN 1930-2975, Vol. 10, no 5, p. 492-502Article in journal (Refereed) Published
    Abstract [en]

    There is an increased willingness to help identified individuals rather than non-identified, and the effect of identifiability is mainly present when a single individual rather than a group is presented. However, identifiability and singularity effects have thus far not been manipulated orthogonally. The present research uses a joint evaluation approach to examine the relative contribution of identifiability and singularity in moral decision-making reflecting conflicting values between deontology and consequentialism. As in trolley dilemmas subjects could either choose to stay with the default option, i.e., giving a potentially life-saving vaccine to a single child, or to actively choose to deny the single child the vaccine in favor of five other children. Identifiability of the single child and the group of children was varied between-subjects in a 2x2 factorial design. In total 1,232 subjects from Sweden and the United States participated in three separate experiments. Across all treatments, in all three experiments, 32.6% of the subjects chose to stay with the deontological default option instead of actively choosing to maximize benefits. Results show that identifiability does not always have a positive effect on decisions in allocation dilemmas. For single targets, identifiability had a negative or no effect in two out of three experiments, while for the group of targets identifiability had a more stable positive effect on subjects’ willingness to allocate vaccines. When the effect of identifiability was negative, process data showed that this effect was mediated by emotional reactance. Hence, the results show that the influence of identifiability is more complex than it has been previously portrayed in the literature on charitable giving. 

    Keywords
    identifiable victim effect, singularity effect, resource allocation, trolley dilemma, moral judgment, decision making, charitable giving
    National Category
    Applied Psychology
    Identifiers
    urn:nbn:se:liu:diva-122128 (URN)000362067700009 ()
    Note

    Funding text: Ragnar Soderberg Foundation; U.S. National Science Foundation [SES-1227729, SES-1427414]

    Available from: 2015-10-19 Created: 2015-10-19 Last updated: 2017-12-01Bibliographically approved
  • 7.
    Wiss, Johanna
    et al.
    Linköping University, Department of Medical and Health Sciences, Division of Health Care Analysis. Linköping University, Faculty of Medicine and Health Sciences.
    Andersson, David
    Linköping University, Department of Management and Engineering, Economics. Linköping University, Faculty of Arts and Sciences.
    Slovic, Paul
    Decis Res, Honolulu, HI USA; Univ Oregon, Eugene, OR 97403 USA.
    Västfjäll, Daniel
    Linköping University, Department of Behavioural Sciences and Learning, Psychology. Linköping University, Faculty of Arts and Sciences. Decis Res, Honolulu, HI USA.
    Tinghög, Gustav
    Linköping University, Department of Management and Engineering, Economics. Linköping University, Faculty of Arts and Sciences.
    The influence of identifiability and singularity in moral decision making2015In: Judgment and decision making, ISSN 1930-2975, E-ISSN 1930-2975, Vol. 10, no 5, p. 492-502Article in journal (Refereed)
    Abstract [en]

    There is an increased willingness to help identified individuals rather than non-identified, and the effect of identifiability is mainly present when a single individual rather than a group is presented. However, identifiability and singularity effects have thus far not been manipulated orthogonally. The present research uses a joint evaluation approach to examine the relative contribution of identifiability and singularity in moral decision-making reflecting conflicting values between deontology and consequentialism. As in trolley dilemmas subjects could either choose to stay with the default option, i.e., giving a potentially life-saving vaccine to a single child, or to actively choose to deny the single child the vaccine in favor of five other children. Identifiability of the single child and the group of children was varied between-subjects in a 2x2 factorial design. In total 1,232 subjects from Sweden and the United States participated in three separate experiments. Across all treatments, in all three experiments, 32.6% of the subjects chose to stay with the deontological default option instead of actively choosing to maximize benefits. Results show that identifiability does not always have a positive effect on decisions in allocation dilemmas. For single targets, identifiability had a negative or no effect in two out of three experiments, while for the group of targets identifiability had a more stable positive effect on subjects’ willingness to allocate vaccines. When the effect of identifiability was negative, process data showed that this effect was mediated by emotional reactance. Hence, the results show that the influence of identifiability is more complex than it has been previously portrayed in the literature on charitable giving. 

  • 8.
    Wiss, Johanna
    et al.
    Linköping University, Department of Medical and Health Sciences, Division of Health Care Analysis. Linköping University, Faculty of Medicine and Health Sciences.
    Carlsson, Per
    Linköping University, Department of Medical and Health Sciences, Division of Health Care Analysis. Linköping University, Faculty of Medicine and Health Sciences.
    Prioritering av särläkemedel: en internationell utblick2016Report (Other academic)
    Abstract [sv]

    Denna rapport presenterar en översikt över hur fem europeiska länder hanterar subventionsbeslut av särläkemedel, dvs. läkemedel som används för att behandla patienter med svåra och sällsynta sjukdomar. Hälsoekonomiska utvärderingar visar att särläkemedel sällan är kostnadseffektiva när man utgår från traditionella gränsvärden avseende kostnad för vunnen hälsa. En ökning av antalet särläkemedel på den europeiska marknaden har bidragit till livliga diskussioner i Sverige och i andra länder om hur dessa ska hanteras inom ramen för rådande läkemedelssubventionssystem. Olika länder har hanterat frågan om dyra behandlingar riktade till patienter med sällsynta sjukdomar på olika sätt och utifrån ett svenskt perspektiv är det därför intressant att observera och inspireras av hur andra europeiska länder har hanterat frågan om särläkemedel.Syftet med denna rapport är: (1) Att beskriva prioriteringsprocessen av särläkemedel i Sverige, Nederländerna, Norge, Frankrike och England. Fokus ligger på de beslutskriterier som ligger till grund för beslut samt på hur prioriteringsprocessen ser ut; (2) Att redovisa lösningar och arbetssätt från respektive land som bidrag till den fortsatta diskussionen om prioritering och finansiering av särläkemedel i Sverige.

  • 9.
    Wiss, Johanna
    et al.
    Linköping University, Department of Medical and Health Sciences, Division of Health Care Analysis. Linköping University, Faculty of Medicine and Health Sciences.
    Levin, Lars-Åke
    Linköping University, Department of Medical and Health Sciences, Division of Health Care Analysis. Linköping University, Faculty of Medicine and Health Sciences.
    David, Andersson
    Linköping University, Department of Management and Engineering, Economics. Linköping University, Faculty of Arts and Sciences.
    Tinghög, Gustav
    Linköping University, Department of Management and Engineering, Economics. Linköping University, Faculty of Arts and Sciences. Linköping University, Department of Medical and Health Sciences, Division of Health Care Analysis. Linköping University, Faculty of Medicine and Health Sciences.
    Prioritizing Rare Diseases: Psychological Effects Influencing Medical Decision Making2017In: Medical decision making, ISSN 0272-989X, E-ISSN 1552-681XArticle in journal (Refereed)
    Abstract [en]

    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.  

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