OBJECTIVES : In the context of regulatory authorities’ and payers’ increasing interest in patient-focused decisions, eliciting patient preferences (PPs) to inform healthcare decisions is a growing field of research. This study aimed to investigate how PP studies can support healthcare decision-making.

METHODS : PubMed publications were systematically searched to identify PP studies in healthcare decision-making. Studies conducted in Europe and the US and published from 2014 to 2019 were included. PP studies were classified based on their objective: informing benefit-risk assessment (BRA) or health technology assessment (HTA) decision-making. Key information related to the type of study (quantitative or qualitative), the disease area and the main results of PP elicitation were extracted.

RESULTS : Twenty-two PP studies were identified between 2014 and 2019. The majority of the studies (n=14) were conducted in Europe, while 8 US studies were identified. Fifteen studies (68%) aimed to inform BRA using patients’ benefit-risk tradeoffs, outcomes prioritization, value appreciation of treatment characteristics, and the likelihood of using an approved therapy in specific subgroups. Informing HTA was less common (n=6). The main purpose of these elicitations was evaluating patient willingness-to-pay for treatment and issuing decisions aligned with subgroup preferences, particularly benefit-risk tradeoffs. PP information was mainly assessed through quantitative tools (86%) such as discrete choice experiments (n=16), less frequently through qualitative methods (9%). One study (5%) used both qualitative and quantitative tools. Central nervous system disorders, metabolic disorders, oncology, and autoimmune disorders were the disease areas where PPs were most often elicited (23%, 18%, 14% and 14%, respectively).

CONCLUSIONS : Although the role of preference studies in decision-making is not well established; these studies have the potential to become an important tool for collecting patients’ priorities in a systematic way to better inform regulatory decisions.