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Healthcare Priority Setting and Rare Diseases: What Matters When Reimbursing Orphan Drugs
Linköping University, Department of Medical and Health Sciences, Division of Health Care Analysis. Linköping University, Faculty of Medicine and Health Sciences.ORCID iD: 0000-0001-7620-8607
2017 (English)Doctoral 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.

Place, publisher, year, edition, pages
Linköping: Linköping University Electronic Press, 2017. , 89 p.
Series
Linköping University Medical Dissertations, ISSN 0345-0082 ; 1571
National Category
Health Care Service and Management, Health Policy and Services and Health Economy Medical Ethics Family Medicine Nursing Political Science (excluding Public Administration Studies and Globalization Studies)
Identifiers
URN: urn:nbn:se:liu:diva-136820DOI: 10.3384/diss.diva-136820ISBN: 9789176855478 (print)OAI: oai:DiVA.org:liu-136820DiVA: diva2:1091650
Public defence
2017-06-01, Berzeliussalen, Universitetssjukhuset, Linköping, 10:00 (English)
Opponent
Supervisors
Available from: 2017-04-27 Created: 2017-04-27 Last updated: 2017-05-09Bibliographically approved
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, Letter (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
Keyword
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: 2017-06-27Bibliographically 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, 492-502 p.Article 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. 

Keyword
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: 2017-04-27Bibliographically approved

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