Cost-Effectiveness and Social Values in Health Care Priority Setting: Normative Reasons and Public Deliberation

Abstract

Paper 1 discusses the question of whether health policy should treat drugs for rare diseases (“orphan drugs”) differently from drugs for common diseases. Due to the small number of potential patients, orphan drugs are less likely to be developed and tend to be expensive when they come to market. To promote development of and access to these treatments, many countries have instituted policies that give special consideration to orphan drugs in a variety of ways. This paper asks whether disease prevalence is a morally relevant characteristic that warrants such special treatment. After examining reasons given in the literature for distinguishing between rare and common diseases, I argue that it is not. It is the severity of orphan diseases that drives our judgments that important claims are being overlooked when orphan treatments are unavailable, not their prevalence. Unlike prevalence, severity is an appropriate consideration for priority setting. Therefore, policies aiming to treat all claims equally should make prevalence irrelevant rather than making it the basis of differential treatment. Paper 2 is a qualitative study of public values on the question of how the severity of a condition and its prevalence should affect priority setting. As part of two citizens’ juries in Alberta, Canada, participants engaged in a deliberative exercise designed to elicit trade-offs between helping small groups with severe conditions and larger groups with less severe conditions. A thematic analysis of transcripts of the deliberations indicates that the public would support funding high-cost drugs to meet the needs of a few when the interventions for rare conditions are life-saving; extend life enough to give hope of future improvement; and relieve otherwise intractable symptoms, especially pain. Considerations of whether a treatment manages symptoms or alters the underlying condition take low priority. These findings can inform Canada’s current drive to establish a national orphan drug policy. Paper 3 examines the use of cost-effectiveness thresholds in the British National Health System (NHS). A recent report states that the NHS’ cost-effectiveness threshold should be lowered from £30,000 to £12,936, to reflect the opportunity cost of a quality-adjusted life-year (QALY) rather than a measure of societal willingness to pay for a QALY. This paper argues that if the threshold is to be determined by opportunity costs, then the understanding of opportunity cost needs to be broadened to include not only QALYs foregone, but also effects on equity, financial protection, and other social values. Even if a broader notion of opportunity cost is considered, deriving the threshold from these costs raises a question of justification: opportunity costs are determined by political factors and facts about the health care system that are unrelated to patients’ claims on the NHS, and may seem arbitrary from their point of view.