부산대학교 도서관

Value in drugs do not always lies simply in them curing diseases: they may also be used to prolong life, improve its quality, and relieve patients' families. How much these elements matter should weigh on reimbursement decisions. Rare diseases often lack strong clinical evidence; hence, overlooking other elements that society values may lead to suboptimal decisions. We examine the preferences for treatments of non‐oncological rare diseases among 16 experts in Poland with PAPRIKA—a choice‐based elicitation method. Among seven attributes, clinical effect was found to be the most important, followed by returning the patient's family to normal functioning. Whether a treatment is used in children or any alternative treatment is available were the least important attributes. The respondents have a non‐linear, diminishing preference for clinical effects, and an almost linear (negative) preference for cost. We develop a new method of sensitivity analysis that accounts for the non‐uniqueness of part‐worth utilities and the possibility of respondents answering inattentively to some questions, and we find our results to be robust. When looking at heterogeneity, three types of respondents deviating from mean‐preferences emerge, in terms of what particular factors they place emphasis on: (a) cost and effectiveness, (b) to whom technology is offered (prognosis and availability of alternative), (c) the impact on family, safety, and caring for children. The part‐worth utilities identified can be used to support multiple criteria decision‐making in Poland. [ABSTRACT FROM AUTHOR]