Abstract Objective To raise awareness among clinicians and epidemiologists that single-patient (n-of-1) trials are potentially useful for informing personalized treatment decisions for patients with ...chronic conditions. Study Design and Setting We reviewed the clinical and statistical literature on methods and applications of single-patient trials and then critically evaluated the needs for further methodological developments. Results Existing literature reports application of 2,154 single-patient trials in 108 studies for diverse clinical conditions; various recent commentaries advocate for wider application of such trials in clinical decision making. Preliminary evidence from several recent pilot acceptability studies suggests that single-patient trials have the potential for widespread acceptance by patients and clinicians as an effective modality for increasing the therapeutic precision. Bayesian and adaptive statistical methods hold promise for increasing the informational yield of single-patient trials while reducing participant burden, but are not widely used. Personalized applications of single-patient trials can be enhanced through further development and application of methodologies on adaptive trial design, stopping rules, network meta-analysis, washout methods, and methods for communicating trial findings to patients and clinicians. Conclusions Single-patient trials may be poised to emerge as an important part of the methodological armamentarium for comparative effectiveness research and patient-centered outcomes research. By permitting direct estimation of individual treatment effects, they can facilitate finely graded individualized care, enhance therapeutic precision, improve patient outcomes, and reduce costs.
Observational studies demonstrate links between patient-centered communication, quality of life (QOL), and aggressive treatments in advanced cancer, yet few randomized clinical trials (RCTs) of ...communication interventions have been reported.
To determine whether a combined intervention involving oncologists, patients with advanced cancer, and caregivers would promote patient-centered communication, and to estimate intervention effects on shared understanding, patient-physician relationships, QOL, and aggressive treatments in the last 30 days of life.
Cluster RCT at community- and hospital-based cancer clinics in Western New York and Northern California; 38 medical oncologists (mean age 44.6 years; 11 (29%) female) and 265 community-dwelling adult patients with advanced nonhematologic cancer participated (mean age, 64.4 years, 146 55.0% female, 235 89% white; enrolled August 2012 to June 2014; followed for 3 years); 194 patients had participating caregivers.
Oncologists received individualized communication training using standardized patient instructors while patients received question prompt lists and individualized communication coaching to identify issues to address during an upcoming oncologist visit. Both interventions focused on engaging patients in consultations, responding to emotions, informing patients about prognosis and treatment choices, and balanced framing of information. Control participants received no training.
The prespecified primary outcome was a composite measure of patient-centered communication coded from audio recordings of the first oncologist visit following patient coaching (intervention group) or enrollment (control). Secondary outcomes included the patient-physician relationship, shared understanding of prognosis, QOL, and aggressive treatments and hospice use in the last 30 days of life.
Data from 38 oncologists (19 randomized to intervention) and 265 patients (130 intervention) were analyzed. In fully adjusted models, the intervention resulted in clinically and statistically significant improvements in the primary physician-patient communication end point (adjusted intervention effect, 0.34; 95% CI, 0.06-0.62; P = .02). Differences in secondary outcomes were not statistically significant.
A combined intervention that included oncologist communication training and coaching for patients with advanced cancer was effective in improving patient-centered communication but did not affect secondary outcomes.
clinicaltrials.gov Identifier: NCT01485627.
Background: N-of-1 trials (multiple crossover studies conducted in single individuals) may be ideal for determining individual treatment effects and as a tool to estimate heterogeneity of treatment ...effects (HTE) in a population. However, comprehensive data on n-of-1 trial methodology and analysis is lacking. We performed this study to describe n-of-1 trial characteristics, examine treatment changes resulting from n-of-1 trial participation, and to determine if trial reporting is adequate for estimating HTE. Methods: We undertook a systematic review of n-of-1 trials published between 1985 and December 2010. Included trials were those having individual treatment episodes as the unit of randomization and reporting individual-specific treatment effects. We abstracted trial characteristics, treatment change information, and analytic methods. Results: We included 108 trials reporting on 2154 participants. Approximately half (49%) of the trials used a statistical cutoff to determine a superior treatment, whereas the remainder used a graphical comparison (25%) or a clinical significance cutoff (20%). Sixty-seven trials, reporting on 488 people, provided treatment change information: 54% of participants had subsequent treatment decisions consistent with the results of the trial, 8% had decisions inconsistent with trial results, and 38% had ambiguous results. Less than half of the trials (45%) reported adequate information to facilitate the calculation of HTE. Conclusion: N-of-1 trials are a useful tool for enhancing therapeutic precision in a range of conditions and should be conducted more often. To facilitate future meta-analysis, and the estimation of HTE, researchers reporting n-of-1 trial results should clearly describe individual data.
Background: Patients vary in their willingness and ability to actively participate in medical consultations. Because more active patient participation contributes to improved health outcomes and ...quality of care, it is important to understand factors affecting the way patients communicate with healthcare providers. Objectives: The objectives of this study were to examine the extent to which patient participation in medical interactions is influenced by 1) the patient's personal characteristics (age, gender, education, ethnicity); 2) the physician's communication style (eg, use of partnership-building and supportive talk); and 3) the clinical setting (eg, the health condition, medical specialty). Research Design and Subjects: The authors conducted a post hoc cross-sectional analysis of 279 physician-patient interactions from 3 clinical sites: 1) primary care patients in Sacramento, California, 2) patients with systemic lupus erythematosus (SLE) from the San Francisco Bay area, and 3) patients with lung cancer from a VA hospital in Texas. Main Outcome Measures: The outcome measures included the degree to which patients asked questions, were assertive, and expressed concerns and the degree to which physicians used partnership-building and supportive talk (praise, reassurance, empathy) in their consultations. Results: The majority of active participation behaviors were patientinitiated (84%) rather than prompted by physician partnershipbuilding or supportive talk. Patients who were more active participants received more facilitative communication from physicians, were more educated, and were more likely to be white than of another ethnicity. Women more willingly expressed negative feelings and concerns. There was considerable variability in patient participation across the 3 clinical settings. Female physicians were more likely to use supportive talk than males, and physicians generally used less supportive talk with nonwhite compared with white patients. Conclusions: Patient participation in medical encounters depends on a complex interplay of personal, physician, and contextual factors. Although more educated and white patients tended to be more active participants than their counterparts, the strongest predictors of patient participation were situation-specific, namely the clinical setting and the physician's communicative style. Physicians could more effectively facilitate patient involvement by more frequently using partnership-building and supportive communication. Future research should investigate how the nuances of individual clinical settings (eg, the health condition, time allotted for the visit) impose constraints or opportunities for more effective patient involvement in care.
The goal of patient-centered communication (PCC) is to help practitioners provide care that is concordant with the patient's values, needs and preferences, and that allows patients to provide input ...and participate actively in decisions regarding their health and health care. PCC is widely endorsed as a central component of high-quality health care, but it is unclear what it is and how to measure it. PCC includes four communication domains: the patient's perspective, the psychosocial context, shared understanding, and sharing power and responsibility. Problems in measuring PCC include lack of theoretical and conceptual clarity, unexamined assumptions, lack of adequate control for patient characteristics and social contexts, modest correlations between survey and observational measures, and overlap of PCC with other constructs. We outline problems in operationalizing PCC, choosing tools for assessing PCC, choosing data sources, identifying mediators of PCC, and clarifying outcomes of PCC. We propose nine areas for improvement: (1) developing theory-based operational definitions of PCC; (2) clarifying what is being measured; (3) accounting for the communication behaviors of each individual in the encounter as well as interactions among them; (4) accounting for context; (5) validating of instruments; (6) interpreting patient ratings of their physicians; (7) doing longitudinal studies; (8) examining pathways and mediators of links between PCC and outcomes; and (9) dealing with the complexity of the construct of PCC. We discuss the use of observational and survey measures, multi-method and mixed-method research, and standardized patients. The increasing influence of the PCC literature to guide medical education, licensure of clinicians, and assessments of quality provides a strong rationale for further clarification of these measurement issues.
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