OBJECTIVES: Severe hypercholesterolemia is a major cause of death from coronary heart disease. Statins have been the cornerstone of lipid therapy for the last two decades. New adjunctive drug therapies (PSCK9-inhibitors) passed authorization processes in EU and US and have been launched recently. The aim of the empirical study was to document patients’ preferences with regard to the adjuvant drug therapy of apheresis-treated patients with severe familial hypercholesterolemia.

METHODS: A systematic literature search was conducted to identify patient-relevant outcomes. Within N=10 semi-structured interviews the decision model was generated. Seven patient relevant characteristics were identified and described by three levels. For the discrete choice experiment (DCE), an experimental design (7*3) was generated using Ngene-Software. The design consisted of 96 choices, which were divided into 8 blocks. The survey was conducted between November 2015 to April 2016 with computer-assisted personal interviews.

RESULTS: N=348 apheresis-treated patients participated (64.9% male). The preference analysis (random parameter logit estimation) showed that patients had clear preferences for all attributes included and that the efficacy criterion "reduction of LDL-C level in blood" (Level Difference/LD: 2.808) was the most significant characteristic in the treatment of hypercholesterolemia. The second rank was taken by "risk of myopathy" (LD: 1.239). In third place was "frequency of apheresis" (LD: 0.821) followed by "risk of cognitive impairment" (LD: 0.554), "number of additional injections" (LD: 0.526) and "risk of hypotension" (LD: 0.341). Within the random parameter logit estimation all coefficients proved to be significant at the level of p≤0.01.

CONCLUSIONS: This study ascertained the essential decision criteria for an optimal therapy from the patients’ perspective. It seems that clinical efficacy is dominant from the patients' perspective. Hence “reduction of lipoprotein (LDL-C) in blood” is ranked highest above the patient relevant mode of administration (apheresis frequency, additional injections) and side effects characteristics.