MDM Policy and Practice最新文献

Background. Assessments of health-related quality of life (HRQoL) are essential in estimating quality-adjusted life-years. It is sometimes not feasible to collect primary HRQoL data, and reliable secondary sources are necessary. Current "off-the-shelf" HRQoL catalogs are based on older diagnosis classifications and include a limited number of diseases. This article aims to provide 1) a Danish EQ-5D-3L-based HRQoL catalog for 199 nationally representative chronic conditions based on ICD-10 codes and 2) a complementary model-based catalog controlling for age, sex, comorbidities, lifestyle, and health risks. Design. A total of 55,616 respondents from 3 national health survey samples were pooled and combined with 7 national registers containing patient-level information on diagnoses, health care activity, and sociodemographics. EQ-5D-3L data were converted to utility scores using the Danish EQ-5D-3L value set to estimate the mean utility for each chronic disease population. Adjusted limited dependent variable mixture models were estimated and used to provide a regression-based catalog of utilities/disutilities. Results. Diseases with the lowest mean EQ-5D score in the Danish population were systemic sclerosis (M34; score = 0.432), fibromyalgia (M797; score = 0.490), rheumatism (M790; score = 0.515), dementia (F00, G30; score = 0.546), posttraumatic stress syndrome (F431; score = 0.557), and systemic atrophies (G10-G14; score = 0.583. Based on the estimated models, the largest estimated disutilities were cystic fibrosis, cerebral palsy, depression, dorsalgia, sclerosis, and fibromyalgia. Lifestyle factors, including perceived stress, loneliness, and body mass index, were also significantly associated with low HRQoL. Conclusions. This study provides a comprehensive nationally representative catalog and a model-based catalog of EQ-5D-3L-based HRQoL scores for Denmark that can be used to describe aspects of disease burden and allocate resources within health care. Additional Stata programs are also provided to facilitate predictions in other populations.

Highlights: A Danish national representative catalog of health-related quality-of-life scores for 199 chronic conditions is presented, which provides population estimates for chronic conditions subgroups that can be used for health economic evaluation.Two separate regression models of EQ-5D-3L utility scores with different sets of control variables are estimated to allow researchers to adjust for differences in the composition of the subgroups and provide a tool that can be used in other settings.Results indicate that health-related quality of life varies across disease groups but is lowest for renal disease, mental and behavioral disorders, benign neoplasms and diseases of the blood, digestive systems, and nervous systems.Health risks and lifestyle factors such as perceived stress, loneliness, and a large body mass index are highly correlated with

Background. To explore preferred and actual involvement in chemotherapy decision making among stage II and III colon cancer (CC) patients by sociodemographic, interpersonal, and intrapersonal communication factors. Methods. Cross-sectional exploratory study collecting self-reported survey data from stage II and III CC patients from 2 cancer centers located in northern Manhattan. Results. Of 88 patients approached, 56 completed the survey. Only 19.3% reported shared involvement in their chemotherapy decisions. We observed significant differences in preferred involvement by gender, with women preferring more physician-controlled decisions. CC patients with higher levels of decisional self-efficacy significantly preferred shared decisions (F = 4.4 [2], P = 0.02). Actual involvement in decisions differed by race (physician controlled 33% for White v. 67% for Other, P < 0.01), age (shared control 18% for ≤55 y, 55% for 55-64 y, and 27% for 65+ y, P = 0.04), and perception of choice (shared control 73% "yes" v. 27% "no,"P = 0.02). Actual or preferred involvement did not differ by stage. Significantly higher levels of medical mistrust (discrimination t = 2.8 [50], P = 0.01; lack of support t = 3.6 [49], P < 0.01), and lower levels of decisional self-efficacy (t = 2.5 [49], P = 0.01) were reported among women. Discussion. Reports of shared involvement around chemotherapy decisions is limited among CC patients. Factors influencing preferred versus actual chemotherapy decision making are complex and may differ; hence, more research is needed to understand and address factors contributing to discordance between preferred and actual involvement in chemotherapy decision making for CC patients.

Highlights: Shared involvement around chemotherapy decisions remains limited for patients diagnosed with colon cancer.Sociodemographic (age, race, gender), interpersonal (medical mistrust), and intrapersonal (decisional self-efficacy, perception of choice) factors that influence preferred involvement in chemotherapy decision making may differ from those influencing actual involvement in chemotherapy decision making.Shared involvement in chemotherapy decisions may look different than currently conceptualized, notably when uncertainty around the benefits exists.

Background. Existing colorectal cancer (CRC) screening models mostly focus on the adenoma pathway of CRC development, overlooking the serrated neoplasia pathway, which might result in overly optimistic screening predictions. In addition, Bayesian inference methods have not been widely used for model calibration. We aimed to develop a CRC screening model accounting for both pathways, calibrate it with approximate Bayesian computation (ABC) methods, and validate it with large CRC screening trials. Methods. A discrete event simulation (DES) of the CRC natural history (DECAS) was constructed using the adenoma and serrated pathways in R software. The model simulates CRC-related events in a specific birth cohort through various natural history states. Calibration took advantage of 74 prevalence data points from the German screening colonoscopy program of 5.2 million average-risk participants using an ABC method. CRC incidence outputs from DECAS were validated with the German national cancer registry data; screening effects were validated using 17-y data from the UK Flexible Sigmoidoscopy Screening sigmoidoscopy trial and a German screening colonoscopy cohort study. Results. The Bayesian calibration rendered 1,000 sets of posterior parameter samples. With the calibrated parameters, the observed age- and sex-specific CRC prevalences from the German registries were within the 95% DECAS-predicted intervals. Regarding screening effects, DECAS predicted a 41% (95% intervals 30%-51%) and 62% (95% intervals 55%-68%) reduction in 17-y cumulative CRC mortality for a single screening sigmoidoscopy and colonoscopy, respectively, falling within 95% confidence intervals reported in the 2 clinical studies used for validation. Conclusions. We presented DECAS, the first Bayesian-calibrated DES model for CRC natural history and screening, accounting for 2 CRC tumorigenesis pathways. The validated model can serve as a valid tool to evaluate the (cost-)effectiveness of CRC screening strategies.

Highlights: This article presents a new discrete event simulation model, DECAS, which models both adenoma-carcinoma and serrated neoplasia pathways for colorectal cancer (CRC) development and CRC screening effects.DECAS is calibrated based on a Bayesian inference method using the data from German screening colonoscopy program, which consists of more than 5 million first-time average-risk participants aged 55 years and older in 2003 to 2014.DECAS is flexible for evaluating various CRC screening strategies and can differentiate screening effects in different parts of the colon.DECAS is validated with large screening sigmoidoscopy and colonoscopy clinical study data and can be further used to evaluate the (cost-)effectiveness of German colorectal cancer screening strategies.

Direct and indirect costs of care influence patients' health choices and the ability to implement those choices. Despite the significant impact of care costs on patients' health and daily lives, patient decision aid (PtDA) and shared decision-making (SDM) guidelines almost never mention a discussion of costs of treatment options as part of minimum standards or quality criteria. Given the growing study of the impact of costs in health decisions and the rising costs of care more broadly, in fall 2021 we organized a symposium at the Society for Medical Decision Making's annual meeting. The focus was on the role of cost information in PtDAs and SDM. Panelists gave an overview of work in this space at this virtual meeting, and attendees engaged in rich discussion with the panelists about the state of the problem as well as ideas and challenges in incorporating cost-related issues into routine care. This article summarizes and extends our discussion based on the literature in this area and calls for action. We recommend that PtDA and SDM guidelines routinely include a discussion of direct and indirect care costs and that researchers measure the frequency, quality, and response to this information.

Purpose. To evaluate patient perceptions of a Web-based decision aid for the treatment of localized prostate cancer. Methods. We assessed patient perceptions of a multicomponent, Web-based decision aid with a preference elicitation/values clarification exercise using adaptive conjoint analysis, the generation of a summary report, and provision of information about localized prostate cancer treatment options. Using a think-aloud approach, we conducted 21 cognitive interviews with prostate cancer patients presented with the decision aid prior to seeing their urologist. Thematic content analysis was used to examine patient perceptions of the tool's components and content prior to engaging in shared decision making with their clinician. Results. Five themes were identified: 1) patients had some negative emotional reactions to the tool, pointing out what they perceived to be unnecessarily negative framing and language used; 2) patients were forced to stop and think about preferences while going through the tool and found this deliberation to be useful; 3) patients were confused by the tool; 4) patients tried to discern the intent of the conjoint analysis questions; and 5) there was a disconnect between patients' negative reactions while using the tool and a contrasting general satisfaction with the final "values profile" created by the tool. Conclusions. Studies are needed to explore the disconnect between patients' expressing negative reactions while going through some components of decision aids but satisfaction with the final output. In particular, we hypothesize that this effect might be explained by cognitive biases such as choice-supportive bias, hindsight bias, and the "IKEA effect." This is one of the first projects to elicit patient reactions while they were completing a decision aid, and we recommend further similar, qualitative postprocess evaluation studies.

Highlights: We explored perceptions of a decision aid with education about localized prostate cancer treatment and preference elicitation using adaptive conjoint analysis.Patients found the tool useful but were also confused by it, tried to discern the intent of the questions, and expressed negative emotional reactions.In particular, there was a disconnect between patients' negative reactions while using the tool and general satisfaction with the final values profile generated by the tool, which is an area for future research.

Objectives: Pneumococcal vaccine recommendations have become increasingly complex. This study aims to understand how national immunization technical advisory groups (NITAGs) and health technology assessment (HTA) agencies of 5 European countries and the United States formed their pneumococcal vaccine recommendations, by providing reviewed evidence and key drivers for new recommendations.

Methods: Centers for Disease Control and Prevention, European Centre for Disease Prevention and Control, and National Health Authorities Web sites were screened to capture the evolution of pneumococcal recommendations. A narrative review was conducted on NITAGs and HTA bodies' Web sites. Assessments of pneumococcal vaccines published from 2009 to 2022 were included.

Results: Thirty-four records were identified including 21 assessments for risk groups, 17 for elderly, and 12 for children. Burden of disease and vaccine characteristics were almost systematically reviewed during assessments. All 6 countries recommended the use of higher-valent pneumococcal vaccine (PCV; i.e., PCV10 and PCV13) in childhood vaccination programs, given their broader serotype coverage and their comparable profile to PCV7. PCV13 was progressively added to the vaccine schedule (in addition to polysaccharide vaccine) in at least the high-risk group, given the high burden in this population and expected additional benefits of PCV13. For the elderly, unlike the United States, European countries issued negative recommendation for PCV13 routine use because of substantial herd effects from childhood vaccination program making PCV13 likely not cost-effective.

Conclusions: This research provides an overview of decision-making processes for higher-valent PCVs recommendations and could be of interest to anticipate the place of next generation of PCVs in the vaccination landscape.

Highlights: By describing evidence-based criteria for decision making, this study emphasizes the framework analysis of NITAGs and HTA bodies when assessing pneumococcal vaccines and demonstrates that variation exists between countries and also according to population evaluated.While the burden of disease and immunogenicity/efficacy data were almost systematically reviewed by national stakeholders, economic assessments were reported to a lesser extent but played a major role in the limited use of PCV13 in the adult population.

Background. Personal autonomy in lung cancer screening is advocated internationally, but health systems diverge in their approach, mandating either shared decision making (with a health care professional) or individual decision making. Studies of other cancer screening programs have found that individual preferences for the level of involvement in screening decisions vary across different sociodemographic groups and that aligning approaches with individual preferences has the potential to improve uptake. Method. For the first time, we examined preferences for decision control among a cohort of UK-based high-risk lung cancer screening candidates (N = 727). We used descriptive statistics to report the distribution of preferences and chi-square tests to examine associations between decision preferences and sociodemographic variables. Results. Most (69.7%) preferred to be involved in the decision with varying degrees of input from a health care professional. Few (10.2%) wanted to make the decision alone. Preferences were also associated with educational attainment. Conclusion. These findings suggest one-size-fits-all approaches may be inadequate in meeting diverse preferences, particularly those placing sole onus on the individual.

Highlights: Preferences for involvement in decision making about lung cancer screening are heterogeneous among high-risk individuals in the United Kingdom and vary by educational attainment.Further work is needed to understand how policy makers might implement hybrid approaches to accommodate individual preferences and optimize lung cancer screening program outcomes.

Purpose: Our aim was to understand the decision making of patients in hospital who wanted cardiopulmonary resuscitation despite low probability of benefit.

Methods: We included patients admitted to general medical wards who had a low chance of surviving in-hospital cardiopulmonary resuscitation (CPR) and had an order in the chart to administer CPR. We developed an interview guide to explore participants' decision-making process, sources of information, and emotions associated with this decision.

Results: We developed 3 themes from the data. 1) "Life is worth living . . . for now": Participants describe their enjoyment of life and desire to carry on in their current state. 2) "Making sense of CPR outcomes": Participants saw CPR outcomes as binary, either they live, or they die; deciding not to receive CPR means choosing death. Participants were optimistic they would survive CPR and cited personal experience and TV as information sources. 3) "Decision process": Participants did not engage in shared decision making. Instead, they were asked a binary yes/no question with no reflection on their values or discussion about harms or benefits.

Limitations: The probability of successful CPR in our sample is unknown. Findings may be different in a population who is imminently dying but still requesting CPR.

Conclusions: Participants chose CPR because they perceived life as worth living and CPR as a chance worth taking. Participants did not want to be left in a severely debilitated state but did not have accurate information about this risk.

Implications: Decision making about CPR in-hospital can be improved if it is grounded in accurate risk understanding and the patient's values and wishes.

Background. Antimicrobial resistance (AMR) is a global public health threat. The wider implications of AMR, such as the impact of antibiotic resistance (ABR) on surgical procedures, are yet to be quantified. The objective of this study was to produce a conceptual modeling framework to provide a basis for estimating the current and potential future consequences of ABR for surgical procedures in England. Design. A framework was developed using literature-based evidence and structured expert elicitation. This was applied to populations undergoing emergency repair of the neck of the femur and elective colorectal resection surgery. Results. The framework captures the implications of increasing ABR by allowing for higher rates of surgical site infection (SSI) as the effectiveness of antibiotic prophylaxis wanes and worsened outcomes following SSIs to reflect reduced antibiotic treatment effectiveness. The expert elicitation highlights the uncertainty in quantifying the impact of ABR, reflected in the results. A hypothetical SSI rate increase of 14% in a person undergoing emergency repair of the femur could increase costs by 39% (-2% to 108% credible interval [CI]) and decrease quality-adjusted life-years by 11% (0.4% to 62% CI) over 15 y. Conclusions. The modeling framework is a starting point for addressing the implication of ABR on the outcomes and costs of surgeries. Due to clinical uncertainty highlighted in the expert elicitation process, the numerical outputs of the case studies should not be focused on but rather the framework itself, illustration of the evidence gaps, the benefit of expert elicitation in quantifying parameters with limited data, and the potential magnitude of the impact of ABR on surgical procedures. Implications. The framework can be used to support research surrounding the health and cost burden of ABR in England.

Highlights: The modeling framework is a starting point for assessing the health and cost impacts of antibiotic resistance on surgeries in England.Formulating a framework and synthesizing evidence to parameterize data gaps provides targets for future research.Once data gaps are addressed, this modeling framework can be used to feed into overall estimates of the health and cost burden of antibiotic resistance and evaluate control policies.

Background and Objectives. Risk-tolerance measures from patient-preference studies typically focus on individual adverse events. We recently introduced an approach that extends maximum acceptable risk (MAR) calculations to simultaneous maximum acceptable risk thresholds (SMART) for multiple treatment-related risks. We extend these methods to include the computation and display of confidence intervals and apply the approach to 3 published discrete-choice experiments to evaluate its utility to inform regulatory decisions. Methods. We generate MAR estimates and SMART curves and compare them with trial-based benefit-risk profiles of select treatments for depression, psoriasis, and thyroid cancer. Results. In the depression study, SMART curves with 70% to 95% confidence intervals portray which combinations of 2 adverse events would be considered acceptable. In the psoriasis example, the asymmetric confidence intervals for the SMART curve indicate that relying on independent MARs versus SMART curves when there are nonlinear preferences can lead to decisions that could expose patients to greater risks than they would accept. The thyroid cancer application shows an example in which the clinical incidence of each of 3 adverse events is lower than the single-event MARs for the expected treatment benefit, yet the collective risk profile surpasses acceptable levels when considered jointly. Limitations. Nonrandom sample of studies. Conclusions. When evaluating conventional MARs in which the observed incidences are near the estimated MARs or where preferences demonstrate diminishing marginal disutility of risk, conventional MAR estimates will overstate risk acceptance, which could lead to misinformed decisions, potentially placing patients at greater risk of adverse events than they would accept. Implications. The SMART method, herein extended to include confidence intervals, provides a reproducible, transparent evidence-based approach to enable decision makers to use data from discrete-choice experiments to account for multiple adverse events.

Highlights: Estimates of maximum acceptable risk (MAR) for a defined treatment benefit can be useful to inform regulatory decisions; however, the conventional metric considers one adverse event at a time.This article applies a new approach known as SMART (simultaneous maximum acceptable risk thresholds) that accounts for multiple adverse events to 3 published discrete-choice experiments.Findings reveal that conventional MARs could lead decision makers to accept a treatment based on individual risks that would not be acceptable if multiple risks are considered simultaneously.