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- Arna S Desser, research fellow1,
- Dorte Gyrd-Hansen, professor2,
- Jan Abel Olsen, professor3,
- Sverre Grepperud, professor1,
- Ivar Sønbø Kristiansen, professor12
- 1Department of Health Management and Health Economics, University of Oslo, PO Box 1089, Blindern, N-0317 Oslo, Norway
- 2Institute of Public Health, University of Southern Denmark, Odense, Denmark
- 3Department of Community Medicine, University of Tromsø, Norway
- Correspondence to: A S Desser
- Accepted 12 July 2010
Objective To determine whether a general societal preference for prioritising treatment of rare diseases over common ones exists and could provide a justification for accepting higher cost effectiveness thresholds for orphan drugs.
Design Cross sectional survey using a web based questionnaire.
Participants Random sample of 1547 Norwegians aged 40-67.
Main outcome measure Choice between funding treatment for a rare disease versus a common disease and how funds should be allocated if it were not possible to treat all patients, for each of two scenarios: identical treatment costs per patient and higher costs for the rare disease. Respondents rated five statements concerning attitudes to equity on a five point Likert scale (5=completely agree).
Results For the equal cost scenario, 11.2% (9.6% to 12.8%) of respondents favoured treating the rare disease, 24.9% (21.7% to 26.0%) the common disease, and 64.9% (62.6% to 67.3%) were indifferent. When the rare disease was four times more costly to treat, the results were, respectively, 7.4% (6.1% to 8.7%), 45.3% (42.8% to 47.8%), and 47.3% (44.8% to 49.8%). Rankings for attitude on a Likert scale indicated strong support for the statements “rare disease patients should have the right to treatment even if more expensive” (mean score 4.5, SD 0.86) and “resources should be used to provide the greatest possible health benefits” (3.9, 1.23).
Conclusions Despite strong general support for statements expressing a desire for equal treatment rights for patients with rare diseases, there was little evidence that a societal preference for rarity exists if treatment of patients with rare diseases is at the expense of treatment of those with common diseases.
We thank Arvid Heiberg for providing information on several rare diseases, Erik Nord for discussions on the design of the survey, and the two reviewers for their comments during the review process.
Contributors: ASD designed the study, analysed the data, and wrote the paper. DGH, JAO, SG, and ISK contributed to the study design and interpretation of results. ISK conceived the study. All authors approved the final version of the paper. ASD is the guarantor.
Funding: This research was supported by grants from the Norwegian Research Council and the health economics research programme, HERO, at the University of Oslo.
Competing interests: All authors have completed the Unified Competing Interest form at www.icmje.org/coi_disclosure.pdf (available on request from the corresponding author) and all authors want to declare (1) Financial support for the submitted work from the Norwegian Research Council and the Health Economics Research Programme (HERO) at the University of Oslo. ISK (2) has received gifts, travel funds, honorariums, consultancy fees or salary from a wide range of public institutions, not for profit organisations or for profit organisations that may have an interest in orphan drug issues. The other authors declare no interests under (2), and all authors declare that they have (3) No spouses, partners, or children with relationships with commercial entities that might have an interest in the submitted work; (4) No non-financial interests that may be relevant to the submitted work.
Ethical approval: Not required.
Data sharing: Survey data are available from the corresponding author on request.
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