The Social Value of Orphan Medicinal Products

Now online available: First public presentation and discussion of the European Social Preference Measurement (ESPM) project at the Multi-Stakeholder Symposium on improving patient access to rare disease therapies, organized by “Rare Diseases Europe” (EURORDIS) in Brussels, February 24, 2016.

Some of the health care interventions for patients with rare and ultra-rare disorders rank among the most expensive ones worldwide, with costs per patient treated occasionally exceeding 100,000€ per year. Needless to say, these interventions often do not meet conventional benchmarks for cost effectiveness. Whenever health care policy-makers and Health Technology Assessment (HTA) agencies rely on the logic of cost effectiveness to inform judgments about “value for money”, this situation turns into an obstacle to reimbursement and, therefore, patient access to treatment.

According the current majority view among health economists, “value” should be determined on grounds of individual (“selfish”) preferences for health states, which may be measured by means of maximum individual willingness-to-pay or – more narrowly – be approximated by estimating quality-adjusted life years (QALYs). Neither approach captures non-selfish (or “social”) preferences, which – for example – have been shown to include a concern for the worst off (for example, cancer patients) and a wish to share scarce resources fairly (for example, with patients with numerous comorbidities) – as opposed to relatively less concern for minor or self-limiting health problems (for example, tattoo removals or common colds). Of particular interest in the present context, implementation of the conventional approach must have the potential to disenfranchise many patients with rare and ultra-rare disorders from any chance to effective medical treatment.

The objective of the break-out session was to facilitate a debate about the moral relevance and the limitations of social value judgments for a meaningful economic evaluation of health care interventions, with particular emphasis on rare and ultra-rare disorders. The far-reaching implications for economic evaluation principles were discussed. Against this background, the need to produce more robust empirical evidence on social preferences was highlighted. The European Social Preferences Study (EPMS) project was presented as a potentially important step forward.

Also see Recent Scientific Presentations.