Abstract

How to handle orphan drugs for rare diseases is a pressing problem in current health-care. Due to the group size of patients affecting the cost of treatment, they risk being disadvantaged in relation to existing cost-effectiveness thresholds. In an article by Niklas Juth it has been argued that it is irrelevant to take indirectly operative factors like group size into account since such a compensation would risk discounting the use of cost, a relevant factor, altogether. In this article we analyze Juth’s argument and observe that we already do compensate for indirectly operative factors, both outside and within cost-effectiveness evaluations, for formal equality reasons. Based on this we argue that we have reason to set cost-effectiveness thresholds to integrate equity concerns also including formal equality considerations. We find no reason not to compensate for group size to the extent we already compensate for other factors. Moreover, groups size implying a systematic disadvantage also on a global scale, i.e. taking different aspects of the health condition of patients suffering from rare diseases into account, will provide strong reason for why group size is indeed relevant to compensate for (if anything).

Highlights

  • In a market economy where pharmaceuticals are developed and sold for profit, patients suffering from rare diseases risk being systematically disadvantaged in getting access to treatment since they belong to a numerically small group

  • Based on this we argue that we have reason to set cost-effectiveness thresholds to integrate equity concerns including formal equality considerations

  • In systems where treatments of severe diseases are allowed to have a cost in overall health, acceptance of formal equality concerns with the implication of higher cost-effectiveness threshold related to group size will add to this effect

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Summary

Introduction

In a market economy where pharmaceuticals are developed and sold for profit, patients suffering from rare diseases risk being systematically disadvantaged in getting access to treatment since they belong to a numerically small group. Similar sets of norms concerning patient need, cost-effectiveness and formal equality are accepted by a number of health-care systems, and the argument about compensating for the consequences of group size could be generally valid (if sound) [10].

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