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BackgroundIncorporating patient preference (PP) information into decision-making has become increasingly important to many stakeholders. However, there is little guidance on which patient preference assessment methods, including preference exploration (qualitative) and elicitation (quantitative) methods, are most suitable for decision-making at different stages in the medical product lifecycle (MPLC). This study aimed to use an empirical approach to assess which attributes of PP assessment methods are most important, and to identify which methods are most suitable, for decision-makers’ needs during different stages in the MPLC.MethodsA four-step cumulative approach was taken: 1) Identify important criteria to appraise methods through a Q-methodology exercise, 2) Determine numerical weights to ascertain the relative importance of each criterion through an analytical hierarchy process, 3) Assess the performance of 33 PP methods by applying these weights, consulting international health preference research experts and review of literature, and 4) Compare and rank the methods within taxonomy groups reflecting their similar techniques to identify the most promising methods.ResultsThe Q-methodology exercise was completed by 54 stakeholders with PP study experience, and the analytical hierarchy process was completed by 85 stakeholders with PP study experience. Additionally, 17 health preference research experts were consulted to assess the performance of the PP methods. Thirteen promising preference exploration and elicitation methods were identified as likely to meet decision-makers’ needs. Additionally, eight other methods that decision-makers might consider were identified, although they appeared appropriate only for some stages of the MPLC.ConclusionsThis transparent, weighted approach to the comparison of methods supports decision-makers and researchers in selecting PP methods most appropriate for a given application.

Background Patients, now generally well informed through dedicated websites and support organizations, are beginning to look askance at clinical experimentation. We conducted a survey investigation to verify whether women with endometriosis would still accept to participate in a randomized controlled trial (RCT) on treatment for pelvic pain. Methods A total of 500 patients consecutively self-referring to an academic outpatient endometriosis clinic, were asked to compile two questionnaires focused on hypothetical comparisons between a new drug and a standard drug, and between medical and surgical treatment, for endometriosis-associated pelvic pain. The main outcome measure was the percentage of patients willing to participate in a theoretical RCT. Results A total of 239 (48 %) women would decline participation in a comparative study on a new drug and a standard drug, as 204 (41 %) would prefer the former medication, and 35 (7 %) the latter. Fifty women (10 %) would participate in a RCT, but only 24 (5 %) would accept blinding. The most frequently chosen option was the patient preference trial (211; 42 %). No significant differences were observed in demographic and clinical characteristics between the 50 women who would accept and the 450 who would decline to be enrolled in a RCT. A total of 229 women (46 %) would decline participation in a comparative study on medical versus surgical treatment, as 186 (37 %) would prefer pharmacological therapy and 43 (9 %) a surgical procedure. Only 11 (2 %) women would participate in such a RCT. More than half of the women (260; 52 %) selected the patient preference trial. No significant variations in distributions of answers were observed between women who did or did not undergo a previous surgical procedure. Conclusion Only a small minority of the women included in our study sample would accept randomization, and even less so blinding. Patient preference appears to play a central role when planning interventional trials on endometriosis-associated pelvic pain. Adequately designed observational analytic studies could be considered when recruitment in a RCT appears cumbersome.