Moving beyond the Court of Public Opinion: A Citizens’ Jury Exploring the Public’s Values around Funding Decisions for Ultra-Orphan Drugs

Health system decision-makers need to understand the value of new technology to make “value for money” decisions. Typically, narrow definitions of value are used. This paper reports on a Canadian Citizens’ Jury which was convened to elicit those aspects of value that are important to the public. The criteria used by the public to determine value included those related to the patient, those directly related to caregivers and those directly created for society. Their choices were not binary (e.g., cost vs. health gained), but rather involved multiple factors (e.g., with respect to patient factors: disease severity, health gained with the drug, existence of alternatives, life expectancy, patient age and affordability). Overall, Jurors prioritized funding treatments for ultra-rare disease populations when the treatment offered significant improvements in health and quality of life, and when the pre-treatment health state was considered extremely poor. The prevalence of the disease by itself was not a factor in the choices. Some of the findings differ from previous work, which use survey methods. In our Citizens’ Jury, Jurors were able to become more familiar with the question at hand and were exposed to a broad and balanced collection of viewpoints before and throughout engaging in the exercises. This deliberative approach allows for a more nuanced approach to understanding value.