MDM Policy and Practice最新文献

Background. Chimeric antigen receptor (CAR) T-cell therapies are approved as second-line (2L) or later therapy for diffuse large B-cell lymphoma (DLBCL). Recently, bispecific T-cell antibodies (BsAbs) have been approved as third-line (3L) treatments. The cost-effectiveness of different treatment sequences is unknown. This study aims to evaluate the cost-effectiveness of axicabtagene ciloleucel (axi-cel) compared with other treatment options for 2L DLBCL, from a US health care perspective at a cost-effectiveness threshold of $150,000 per quality-adjusted life-year (QALY). Design. This economic evaluation used a discrete event simulation decision. Model inputs were derived from 8 clinical trials and the published literature. Simulated patients received 2L axi-cel followed by 3L treatments, which were compared with treatment sequences of 2L intended autologous stem cell transplant (ASCT), polatuzumab vedotin with bendamustine and rituximab (Pola-BR), tafasitamab with lenalidomide (tafa-len), or rituximab with gemcitabine and oxaliplatin (R-GemOx), all of which were followed by 3L treatments (salvage chemotherapy, BsAbs, or axi-cel). In addition, axi-cel was compared directly with glofitamab and epcoritamab in 3L. Costs and QALYs, discounted at 3.0%, were used to derive incremental cost-effectiveness ratios (ICERs) and net monetary benefits (NMBs). Results. In the 2L base case, axi-cel was cost-effective compared with intended ASCT (ICER $145,004/QALY), which was cost-effective compared with R-GemOx (ICER $9,495/QALY). Axi-cel maximized NMB at $150,000 and $200,000/QALY thresholds, whereas intended ASCT maximized NMB at $100,000/QALY. In 3L-focused comparisons with epcoritamab and glofitamab, axi-cel was dominant and cost-effective (ICER $122,224/QALY), respectively. Axi-cel maximized NMB at $150,000 and $200,000/QALY thresholds, whereas glofitamab maximized NMB at $100,000/QALY. Conclusions. The findings of the study suggest that although other treatments were cost-effective at lower thresholds, axi-cel is a cost-effective treatment option in 2L/3L settings in the United States.

Highlights: This study investigated whether axicabtagene ciloleucel (axi-cel) is cost-effective in second-line (2L) and third-line (3L) treatment sequences in the current relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL) treatment paradigm.Using a novel treatment sequencing model, axi-cel was found to be cost-effective in both 2L treatment sequences and in direct comparisons with 3L bispecific T-cell antibodies.These findings suggest that axi-cel is a cost-effective treatment for R/R DLBCL regardless of treatment line positioning.

Background. Hepatocellular carcinoma (HCC) treatment decisions are becoming increasingly complex as new treatment options emerge. Improved understanding of tradeoffs and patient preferences in treatment decisions will enhance patient-provider discussions, improve treatment development, and inform HCC treatment guidelines. We performed a qualitative study involving patients with HCC and medical providers to assess the role of patient preferences in HCC treatment choices. Methods. Patient participants included those with HCC seen within a single tertiary care center. Provider participants involved physicians and advanced practice providers who cared for patients with HCC from a single center. Baseline and posttreatment patient interviews were conducted by trained qualitative research experts, informed by semi-structured interview guides, and analyzed using thematic analysis with pilot-tested codebooks. Summaries included a narrative description of the themes and subthemes that emerged related to each code, and illustrative quotes were used to highlight each theme. Results. The baseline interview involved 30 patients with HCC (22 of whom participated in follow-up interviews) and 10 providers who cared for patients with HCC. Patients identified factors considered when making treatment decisions included provider confidence and experience, patient prior cancer experiences, other health issues, and faith. Providers primarily discussed the role of Barcelona Clinic Liver Cancer stage, liver function, performance status, and eligibility of liver transplantation in making treatment recommendations. There was general agreement among providers that there is a need to better understand the role of patient values to improve care for HCC. Limitations. Qualitative interviews were limited to patients and providers from a single center. Conclusions. This qualitative study provided information on the variety of values considered by both patients and providers in HCC treatment decisions and the importance of considering tradeoffs of efficacy, toxicity, and inconvenience/costs.

Highlights: Hepatocellular carcinoma (HCC) treatment decisions are often complex and may become increasingly so as new treatment options emerge.Improved understanding of tradeoffs and patient preferences in treatment decisions will enhance patient-provider discussions, facilitate patient-centered trials to develop new treatments, and inform HCC treatment guidelines.This qualitative study of patients and providers provided information on the values considered in HCC treatment decisions and the importance of considering the tradeoffs of efficacy, toxicity, and inconvenience/costs.These insights can be used to develop preference elicitation tools, perform large-scale preference elicitation surveys, and systematically assess and incorporate patient preferences into treatment decisions.

Economic evaluations that incorporate value-of-information analysis frequently conclude that the greatest information value relates to replicating short-term clinical trials. This study builds on recent guidance relating to extrapolation in economic evaluation by assessing the impact of alternative approaches to representing the uncertainty around unobserved/extrapolated data with respect to incremental outcomes and value of information. When the uncertainty over unobserved and observed data is considered distinct but correlated (i.e., has a joint distribution), it is demonstrated that the value to replicating short-term clinical studies is lessened and that further studies relating to the unobserved periods likely provide more value.

Highlights: Current practice in economic evaluation often involves the inappropriate specification of uncertainty with respect to unobserved data.Appropriate specification of uncertainty will lead to more pertinent recommendations over future clinical studies.

Background. Implementing a lung cancer screening (LCS) program with low-dose computed tomography (LDCT) is complex, requiring health care organizations to consider several steps along the screening continuum from eligibility assessment to recommended follow-up testing adherence. The evidence to support LDCT screening implementation remains unclear. Purpose. To summarize interventions facilitating LCS initiation, adoption, or improvement within health care organizations. Data Sources. Librarian-assisted literature reviews identified published studies between January 1, 2011, and December 31, 2023, using CINAHL, Cochrane Library, Embase, Ovid Medline, PsycINFO, and Scopus. Study Selection. Published interventions focusing on any step in the LCS process before lung cancer diagnosis, including risk/eligibility assessment, shared decision making (SDM), and annual screening or diagnostic testing. Data Abstraction. We used a title/abstract review process, full-text review, and risk-of-bias assessments. We characterized studies by design, unit of observation, participant sociodemographic characteristics, primary outcome, and step in the LCS process. DistillerSR and Covidence were used for data management. Data Synthesis. We identified 64 study-eligible published articles, including 19 randomized and 45 nonrandomized studies. SDM interventions were most frequently studied (n = 20) followed by initial LCS uptake (n = 12). Most studies (n = 33) evaluated educational interventions, typically in one-on-one settings. Studies assessed at either low or moderate/some risk of bias reported statistically significant findings in the domains of improved knowledge (n = 7) and other aspects of decision making (n = 8), such as perceived risk or decisional conflict. Findings regarding LCS uptake were more variable. Limitations. The review includes only English-language studies published prior to 2024. The risk of bias was high among 5 of the randomized clinical trials and serious among 27 of the quasi-experimental design studies. Conclusions. LCS intervention strategies have focused on SDM and initial LCS uptake, leaving gaps in knowledge about how to support risk and eligibility assessment, adherence to annual screening, or diagnostic testing. Expanding interventions beyond those that are education focused and with single-level targets would expand the LDCT screening implementation evidence base.

Highlights: Most lung cancer screening (LCS) interventions evaluated to date have been educational in nature and focused primarily on shared decision making or the initial uptake of screening, with some interventions demonstrating statistically significant improvements in patient knowledge and initial LCS order/uptake.A critical gap in knowledge remains regarding how to effectively support LCS eligibility assessment as well as adherence to annual screening and

Background. It is unclear how to optimize the user interface and user experience of cancer screening artificial intelligence (AI) tools for clinical decision-making in primary care. Methods. We developed an electronic survey for US primary care clinicians to assess 1) general attitudes toward AI in cancer screening and 2) preferences for various aspects of AI model deployment in the context of colorectal, breast, and lung cancer screening. We descriptively analyzed the responses. Results. Ninety-nine surveys met criteria for analysis out of 733 potential respondents (response rate 14%). Ninety (>90%) somewhat or strongly agreed that their medical education did not provide adequate AI training. A plurality (52%, 39%, and 37% for colon, breast, and lung cancers, respectively) preferred that AI tools recommend the interval to the next screening as compared with the 5-y probability of future cancer diagnosis, a binary recommendation of "screen now," or identification of suspicious imaging findings. In terms of workflow, respondents preferred generating a flag in the electronic health record to communicate an AI prediction versus an interactive smartphone application or the delegation of findings to another healthcare professional. No majority preference emerged for an explainability method for breast cancer screening. Limitations. The sample was primarily obtained from a single health care system in the Northeast. Conclusions. Providers indicated that AI models can be most helpful in cancer screening by providing prescriptive outputs, such as recommended intervals until next screening, and by integrating with the electronic health record. Implications. A preliminary framework for AI model development in cancer screening may help ensure effective integration into clinical workflow. These findings can better inform how healthcare systems govern and receive reimbursement for services that use AI.

Highlights: Clinicians do not feel their undergraduate or graduate medical education has properly prepared them to engage with AI in patient care.We provide a preliminary framework for deploying AI models in primary care-based cancer screening.This framework may have implications for health system governance and provider reimbursement in the age of AI.

Background. Chronic hepatitis B (CHB) is a lifelong disease requiring long-term or indefinite therapy, resulting in substantial economic burden. Thus, careful consideration must be used in the selection of therapies. Aim. This analysis assessed the cost-effectiveness of tenofovir alafenamide (TAF) compared with tenofovir disoproxil fumarate (TDF) and entecavir (ETV) from the perspective of the Taiwan National Health Insurance Administration Healthcare payer for the management of CHB over a lifetime horizon. Methods. An individual patient simulation model assessed the impact of treatment on CHB infection for liver- and safety-related outcomes. Patients could achieve spontaneous or treatment-induced responses, experience a reactivation of the disease, develop long-term liver complications, or experience treatment-related renal or bone complications. Patient population profiles were based on clinical trial and real-world data. Data on clinical parameters (safety, mortality, resistance risk, and flare), health utilities, and costs were sourced from the published literature. Results. TAF was associated with fewer liver disease events and fewer cases of bone and renal complications per 100 person-years. TAF also had higher eAg and sAg seroconversion compared with TDF and ETV. As compared with both treatments, TAF was both more effective and more costly, resulting in incremental cost-effectiveness ratios of USD 3,348 and USD 3,940 per quality-adjusted life-year gained versus TDF and ETV, respectively. Conclusion. TAF leads to better health outcomes at acceptable incremental costs compared with the most commonly used therapies in the management of CHB, thus making it a cost-effective option for the treatment of CHB in Taiwan.

Highlights: The cost-effectiveness of tenofovir alafenamide (TAF) versus tenofovir disoproxil fumarate (TDF) and entecavir (ETV) was assessed in patients with chronic hepatitis B in Taiwan.TAF was associated with fewer liver disease events, fewer cases of bone and renal complications, and higher eAG and sAG seroconversion compared with TDF and ETV; TAF was found to be cost-effective compared with both treatments.

Background. Breast cancer screening via mammography for women younger than 50 y sparks controversy due to balancing benefits and risks. In Israel, specific criteria govern early screening initiation, yet global studies reveal low adherence to guidelines for this demographic. Objectives. This study aims to report on young women's referrals for screening mammography in Israel, assess adherence to guidelines, and identify factors influencing guideline adherence. Design, Setting, and Participants. A cross-sectional study analyzed referral letters for screening mammography issued to women aged 18 to 49 y from March 2019 to February 2020 in 2 districts of Israel's largest health care provider. Exclusions included women with a history of breast cancer or BRCA mutations. Of 9,960 letters, 1,287 were randomly selected for adherence assessment, with 13% of nonadherent cases further reviewed. Main Outcomes and Measures. Primary outcomes included categorizing referrals as adherent or nonadherent to guidelines. Additional measures explored correlations between adherence and patient characteristics (e.g., age, comorbidities) and the referring physician's specialty. Results. A total of 999 referral letters were included in the statistical analysis. Referrals spanned all ages but skewed toward women older than 40 y. Of the referrals, 45% (452) came from general surgeons and 32% (327) from family physicians. Twenty-four percent (303) of referrals were blank, and 1% (4) involved risk-benefit discussions. In total, 109 (10.9%) of the referrals strictly adhered to guidelines; under a lenient approach, 30.6% (307) adhered. General surgeons adhered more frequently than gynecologists did (32.8% [109] v. 14.9% [11], P = 0.014). Conclusions and Relevance. Despite official guidelines, many physicians in Israel did not follow recommendations for breast cancer screening in women younger than 50 y, highlighting a gap between evidence-based medicine and clinical practice.

Highlights: Question Are screening mammography referrals, given to women younger than 50 y of age, adherent to current guidelines? Findings In this cross-sectional study of a randomly selected sample of 1,287 referral letters, given to women aged 18 to 50 y, only 10.9% were adherent with the guidelines when examined with a strict approach and 30.6% with a forgiving approach. Adherence significantly correlated with the field of the referring physician. Meaning Despite known risks of screening mammography, women younger than 50 y are commonly referred to such screening in a deviation from current guidelines.

Background. The format in which probabilities are presented influences comprehension and interpretation. Purpose. To develop comprehensive evidence-based guidance about how to communicate probabilities in health and to identify strengths and weaknesses in the literature. This article presents methods for the review of probability communication and is accompanied by several results articles. Data Sources. MEDLINE, Embase, CINAHL, the Cochrane Library, PsycINFO, ERIC, ACM Digital Library; hand search of 4 journals. Study Selection. Two reviewers conducted screening to identify experimental and quasi-experimental research that compared 2 or more formats for presenting quantitative health information to patients or lay audiences. Data Extraction. In our conceptual framework, people make sense of a stimulus (data in a data presentation format) by performing cognitive tasks, resulting in perceptual, affective, cognitive, or behavioral responses measured as 1 of 14 distinct outcomes. The study team developed custom instruments to extract concepts, conduct risk-of-bias evaluation, and evaluate individual findings for credibility. Data Synthesis. Findings were grouped into tables by task and outcome for evidence synthesis. Limitations. Reviewer error could have led to missing relevant studies despite having 2 independent reviewers screening each article. The granular data extraction and syntheses slowed the work and may have made it less replicable. Credibility was evaluated by only 2 experts. Conclusions. After reviewing 26,793 titles and abstracts, we identified 316 articles about probability communication. Data extraction produced 1,119 individual findings, which were grouped into 37 evidence tables, each containing evidence on up to 10 data presentation format comparisons. The Making Numbers Meaningful project required novel methods for classifying and synthesizing research, which reveal patterns of strength and weakness in the probability communication literature.

Highlights: The Making Numbers Meaningful project conducted a comprehensive systematic review of experimental and quasi-experimental research that compared 2 or more formats for presenting quantitative health information to patients or other lay audiences. The current article focuses on probability information.Based on a conceptual taxonomy, we reviewed studies based on the cognitive tasks required of participants, assessing 14 distinct possible outcomes.Our review identified 316 articles involving probability communications that generated 1,119 distinct research findings, each of which was reviewed by multiple experts for credibility.The overall pattern of findings highlights which probability communication questions have been well researched and which have not. For example, there has been far more research on communicating single probabilities than on communicating more complex information such as

Background. To develop guidance on the effect of data presentation format on communication of health probabilities, the Making Numbers Meaningful project undertook a systematic review. Purpose. This article, one in a series, covers evidence about "difference tasks," in which a reader examines a stimulus to evaluate differences between probabilities, such as the effect of a risk factor or therapy on the chance of a disease. This article covers the effect of format on 4 outcomes: 1) identifying a probability difference (identification) or recalling it (recall), 2) identifying the largest or smallest of a set of probability differences (contrast outcome), 3) placing a probability difference into a category such as "elevated" or "below average" (categorization outcome), and 4) performing computations (computation outcome). Data Sources. MEDLINE, Embase, CINAHL, the Cochrane Library, PsycINFO, ERIC, ACM Digital Library; hand search of 4 journals. Finding Selection. Pairwise screening to identify experimental/quasi-experimental research comparing 2 or more formats for quantitative health information. This article reports on 53 findings derived from 35 unique studies reported in 32 papers. Data Extraction. Pairwise extraction of information on stimulus (data in a data presentation format), cognitive task, and perceptual, affective, cognitive, or behavioral outcomes. Data Synthesis. Most evidence involving outcomes of difference-level cognitive tasks was weak or insufficient. Evidence was strong that 1) computations involving differences are easier with rates per 10ⁿ than with percentages or 1 in X rates and 2) adding graphics to numbers makes it easier to perform difference-level computations. Limitations. A granular level of evidence syntheses leads to narrow guidance rather than broad statements. Conclusions. Although many studies examined differences between probabilities, few were comparable enough to generate strong evidence.

Highlights: Most evidence about the effect of format on ability to evaluate differences in probabilities was weak or insufficient because of too few comparable studies.Strong evidence showed that computations relevant to differences in probabilities are easier with rates per 10ⁿ than with 1 in X rates.Adding graphics to probabilities helps readers compute differences between probabilities.