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Patient preferences for personalized (N-of-1) trials: a conjoint analysis.

ABSTRACT: OBJECTIVE:Despite their promise for increasing treatment precision, Personalized Trials (i.e., N-of-1 trials) have not been widely adopted. We aimed to ascertain patient preferences for Personalized Trials. STUDY DESIGN AND SETTING:We recruited 501 adults with ?2 common chronic conditions from Harris Poll Online. We used Sawtooth Software to generate 45 plausible Personalized Trial designs comprising combinations of eight key attributes (treatment selection, treatment type, clinician involvement, blinding, time commitment, self-monitoring frequency, duration, and cost) at different levels. Conditional logistic regression was used to assess relative importance of different attributes using a random utility maximization model. RESULTS:Overall, participants preferred Personalized Trials with no costs vs. $100 cost (utility difference 1.52 [standard error 0.07], P < 0.001) and with less vs. more time commitment/day (0.16 [0.07], P < 0.015) but did not hold preferences for the other six attributes. In subgroup analyses, participants ?65 years, white, and with income ?$50,000 were more averse to costs than their counterparts (P all <0.05). CONCLUSION:To optimize dissemination, Personalized Trial designers should seek to minimize out-of-pocket costs and time burden of self-monitoring. They should also consider adaptive designs that can accommodate subgroup differences in design preferences.

SUBMITTER: Moise N

PROVIDER: S-EPMC6119511 | biostudies-literature | 2018 Oct

REPOSITORIES: biostudies-literature

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Patient preferences for personalized (N-of-1) trials: a conjoint analysis.

Moise Nathalie N Wood Dallas D Cheung Ying Kuen K YKK Duan Naihua N Onge Tara St TS Duer-Hefele Joan J Pu Tiffany T Davidson Karina W KW Kronish Ian M IM

Journal of clinical epidemiology 20180530

<h4>Objective</h4>Despite their promise for increasing treatment precision, Personalized Trials (i.e., N-of-1 trials) have not been widely adopted. We aimed to ascertain patient preferences for Personalized Trials.<h4>Study design and setting</h4>We recruited 501 adults with ≥2 common chronic conditions from Harris Poll Online. We used Sawtooth Software to generate 45 plausible Personalized Trial designs comprising combinations of eight key attributes (treatment selection, treatment type, clinic ...[more]

PMID: 29859242

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Project description:BackgroundTofacitinib, an oral Janus kinase inhibitor approved for the treatment of rheumatoid arthritis (RA), provides patients with an alternative to subcutaneously or intravenously administered biologic disease-modifying antirheumatic drugs (DMARDs). Little is known about patient preference for novel RA treatments.ObjectiveTo investigate patient preferences for attributes associated with RA treatments.MethodsA choice-based conjoint survey was mailed to 1400 randomly selected commercially insured patients (aged 21-80 years) diagnosed with RA, who were continuously enrolled from May 1, 2012, through April 30, 2013, and had ≥2 medical claims for International Classification of Diseases, Ninth Revision, Clinical Modification diagnosis code 714.0 and no previous biologic DMARD use. Treatment attributes included route of administration; monthly out-of-pocket cost; frequency of administration; ability to reduce daily joint pain and swelling; likelihood of serious adverse events; improvement in the ability to perform daily tasks; and medication burden. Mean attribute importance scores were calculated after adjusting for patient demographics (eg, age, sex, years since diagnosis) using a hierarchical Bayes model. Patient preferences for each treatment attribute were ranked by the importance score. Part-worth utilities (ie, preference scores) were used to perform a conjoint market simulation.ResultsA total of 380 patients (response rate, 27.1%) returned the survey. Their mean age (± standard deviation) was 54.9 (± 9.3) years. Nonrespondents were 2 years younger (mean, 52.9 years; P = .002) but did not differ significantly from respondents in known clinical characteristics. After adjustment for demographic characteristics, mean patients' ranking of treatment attribute importance, in decreasing order, was route of administration, 34.1 (± 15.5); frequency of administration, 16.4 (± 6.8); serious adverse events, 12.0 (± 9.3); cost, 10.1 (± 6.2); medication burden, 9.8 (± 8.2); joint pain reduction, 8.9 (± 3.8); and daily tasks improvement, 8.8 (± 4.7). For the route of administration attribute, the part-worth utility was highest for the oral route. Conjoint simulation results showed that 56.4% of respondents would prefer an oral route of administration.ConclusionBased on this survey completed by 380 patients with RA, commercially insured patients with RA consider the route of administration to be the most important attribute of their RA treatment. In this study, the majority (56.4%) of patients preferred the oral route of administration over other routes. Understanding patient preferences may help to inform provider and payer decisions in treatment selection that may enhance patient adherence to therapy.

Project description:We used conjoint analysis-a method that assesses complex decision making-to quantify patients' choices when selecting an osteoporosis therapy. While 60% of people prioritized medication efficacy when deciding among treatments, the remaining 40% highly valued factors other than efficacy, suggesting the need for personalized shared decision-making tools.IntroductionIn this study, we aimed to examine patient decision-making surrounding osteoporosis medications using conjoint analysis.MethodsWe enrolled osteoporosis patients at an academic medical center to complete an online conjoint exercise which calculated each patient's relative importance score of 6 osteoporosis medication attributes (higher = greater relative importance in decision-making). We used latent class analysis to identify distinct segments of patients with similar choice patterns and then used logistic regression to determine if demographics and osteoporosis disease features were associated with latent class assignment.ResultsOverall, 304 participants completed the survey. The rank order of medication attributes by importance score was the following: efficacy at preventing hip fractures (accounted for 31.0% of decision making), mode of administration (17.5%); risk of serious side effects (16.6%); dose frequency (13.9%); efficacy at preventing spine fractures (12.5%); risk of non-serious side effects (8.4%). We found that 60.9% of the cohort prioritized medication efficacy as their top factor when selecting among the therapies. Being a college graduate, having stronger beliefs on the necessity of using medications for osteoporosis, and never having used osteoporosis medicines were the only factors associated with prioritizing medication efficacy for fracture prevention over the other factors in the decision-making process.ConclusionsWhile about 60% of patients prioritized efficacy when selecting an osteoporosis therapy, the remaining 40% valued other factors more highly. Furthermore, individual patient characteristics and clinical factors did not reliably predict patient decision making, suggesting that development and implementation of shared decision-making tools is warranted.

Project description:IntroductionTo identify patient preference drivers related to the management of wet age-related macular degeneration (wet AMD).MethodsIn this cross-sectional study, a self-explicated 'conjoint analysis' survey was administered online to eligible patients with wet AMD (receiving anti-vascular endothelial growth factor [VEGF] treatment for at least 12 months) from the USA, Canada, UK, France, Spain, Germany, Italy, Japan, Taiwan, and Australia. The survey consisted of six domains with 21 attributes, which were selected on the basis of a literature review, social media listening, and tele-interviews/discussions with patients, clinical experts, and patient groups. Utility and relative importance scores were generated for each attribute and utility difference significance testing was performed using 'unequal variances t tests'. The Patient Activation Measure (PAM-13) questionnaire was administered to assess patients' knowledge, skill, and confidence in self-management.ResultsA total of 466 patients (mean age, 68 years; women, 54%; binocular wet AMD, 28%) with an average anti-VEGF treatment duration of 3.9 years completed the survey. The most important preference domains were 'treatment effects on vision' (non-significant) and 'vision-related symptom burdens' (p < 0.001), followed by 'treatment risk' (p < 0.05), 'impact on daily activities' (p < 0.05), 'burden of clinic/hospital visits' (p < 0.001), and 'impact on psychological well-being'. The five most important attributes in order of importance were clarity of vision, treatment effect on symptoms, quality of vision, time to treatment effect, and time to re-administration. The two most important attributes globally were also in the top three attributes across countries. The majority of participants in the study were level 3 or level 4 of the PAM-13 questionnaire.ConclusionsThis study identified the most important disease and treatment attributes to patients using patient-centred methods. The data showed the degree of harmonization of preferences across geographies and that participants actively adopt behaviours required for improved treatment outcomes. The identified preference drivers may inform future clinical development.

Project description:IntroductionUnderstanding patient preferences for characteristics of treatments facilitates patient participation and doctor-patient communication and enhances patient-centered care. Patient participation is especially important for urethral stricture disease, which has no definitive treatment guidelines favoring either endoscopic incision or open reconstruction, making patient preference an important factor in treatment choice. However, to date, there have been no studies assessing factors that patients value when choosing a treatment option.MethodsWe employ choice-based conjoint analysis to assess patient preferences in the trade-offs of treatment attributes for urethral stricture disease. Male patients undergoing treatment or follow-up examination for urethral stricture disease were recruited through a University Medical Practice. We included 169 patients in the analysis. Six attributes of both risk and benefit were examined: treatment type, success rate, number of future procedures, post-treatment catheter duration, recovery time, and copayment amount.ResultsThe treatment success rate was by far the most important attribute. Relative to a 25% success rate (OR = 1) an 85% success rate (OR = 26.72, p<.01) increased patient preference by approximately 27 times. Furthermore, patients are willing to pay a $10,000 copayment to double the success rate from 25% to 50%. Patients demonstrated a strong aversion to time with a urinary catheter. Catheter duration for 1 week or less (OR = .67, p<.01) reduced patient preference by about 1.5 times when compared to requiring no catheter. We also found that patients place low importance on both how invasive the treatment seems and low copayment amounts but are willing to pay $10,000 copayment for an open reconstruction surgery compared with an endoscopic incision procedure.ConclusionThe findings highlight the importance of shared and detailed physician/patient discussions of all the risk and benefits of each treatment choice and suggest that conjoint analysis may be helpful as a decision aid to guide discussions with individual patients deciding on a treatment.

Project description:OBJECTIVE:To describe individual patient preferences for Personalised Trials and to identify factors and conditions associated with patient preferences. DESIGN:Each participant was presented with 18 conjoint questions via an online survey. Each question provided two choices of Personalised Trials that were defined by up to eight attributes, including treatment types, clinician involvement, study logistics and trial burden on a patient. SETTING:Online survey of adults with at least two common chronic conditions in the USA. PARTICIPANTS:A nationally representative sample of 501 individuals were recruited from the Chronic Illness Panel by Harris Poll Online. Participants were recruited from several sources, including emails, social media and telephone recruitment of the target population. MAIN OUTCOME MEASURES:The choice of Personalised Trial design that the participant preferred with each conjoint question. RESULTS:There was large variability in participants' preferences for the design of Personalised Trials. On average, they preferred certain attributes, such as a short time commitment and no cost. Notably, a population-level analysis correctly predicted 62% of the conjoint responses. An empirical Bayesian analysis of the conjoint data, which supported the estimation of individual-level preferences, improved the accuracy to 86%. Based on estimates of individual-level preferences, patients with chronic pain preferred a long study duration (p?0.001). Asthma patients were less averse to participation burden in terms of data-collection frequency than patients with other conditions (p=0.002). Patients with hypertension were more cost-sensitive (p<0.001). CONCLUSION:These analyses provide a framework for elucidating individual-level preferences when implementing novel patient-centred interventions. The data showed that patient preference in Personalised Trials is highly variable, suggesting that individual differences must be accounted for when marketing Personalised Trials. These results have implications for advancing precise interventions in Personalised Trials by indicating when rigorous scientific principles, such as frequent monitoring, is feasible in a substantial subset of patients.

Dataset Information

Patient preferences for personalized (N-of-1) trials: a conjoint analysis.

Publications

Patient preferences for personalized (N-of-1) trials: a conjoint analysis.

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