MDM Policy and Practice最新文献

{"title":"Development and Qualitative Evaluation of a Decision Support Tool for Withdrawal of Biologic Therapy in Nonsystemic Juvenile Idiopathic Arthritis.","authors":"Janine A van Til, Michelle M A Kip, Robert Marinescu-Muster, Karin Groothuis-Oudshoorn, Gillian R Currie, Susanne M Benseler, Joost F Swart, Sebastiaan J Vastert, Nico Wulffraat, Rae S M Yeung, Deborah A Marshall, Maarten J IJzerman","doi":"10.1177/23814683251364199","DOIUrl":"10.1177/23814683251364199","url":null,"abstract":"<p><p><b>Introduction.</b> Limited evidence guides pediatric rheumatologists on when to withdraw biologic therapy in children with juvenile idiopathic arthritis, resulting in wide variation in clinical practice. This study aimed to develop and evaluate a decision support tool (DST) based on expert opinion to support pediatric rheumatologists in making withdrawal decisions. <b>Methods.</b> A literature review, focus groups, interviews, and prior research informed the design of the prototype DST. Evaluation of the DST's face validity, content validity, acceptance, and feasibility was conducted through user testing interviews and a survey among pediatric rheumatologists from the Netherlands and Canada. Findings were summarized using descriptive and qualitative content analyses. <b>Results.</b> The prototype DST requires input on relevant patient, disease, and treatment characteristics. Its primary output is the predicted likelihood of biologic therapy withdrawal. Pediatric rheumatologists can adjust the importance of characteristics and observe the resulting impact on withdrawal likelihood. Eleven pediatric rheumatologists participated in testing. Key themes identified included the need for 1) clear terminology to ensure consistent interpretation of model inputs, 2) concise instructions on how and when to adjust the relative importance of characteristics, and 3) practice rounds to build trust among pediatric rheumatologists in the DST's output. Participants found the DST feasible for clinical use, with its main value in explaining decisions to patients and engaging them in the decision-making process. Suggested future improvements include tracking the outcomes of withdrawal decisions and integrating predictive models based on clinical data. <b>Conclusions.</b> The DST developed in this study was well-received. Its main value lies in helping pediatric rheumatologists explain their decisions to patients and parents. The top priority for further development is integrating scientific evidence on successful withdrawal decisions.</p><p><strong>Highlights: </strong>Decision support tools that provide structure to decisions based on expert opinion can increase transparency and consistency in medical decision making in the absence of clinical evidence.Data from clinical vignette studies that use an experimental design to elicit treatment preferences can be used to predict treatment decision making.A decision support tool to support biologic therapy withdrawal decisions has the most value in explaining the decision to children with nonsystemic juvenile idiopathic arthritis and their parents.</p>","PeriodicalId":36567,"journal":{"name":"MDM Policy and Practice","volume":"10 2","pages":"23814683251364199"},"PeriodicalIF":1.7,"publicationDate":"2025-09-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12480790/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145208005","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Development and Acceptability Testing of a Patient Decision Aid on Levodopa Intestinal Gel for Parkinson Disease.","authors":"Andreanne Tanguay, Caroline Cayer, Isabelle Beaulieu-Boire","doi":"10.1177/23814683251364883","DOIUrl":"10.1177/23814683251364883","url":null,"abstract":"<p><strong>Background: </strong>Parkinson's disease (PD) is a neurodegenerative disease characterized by motor and nonmotor symptoms that worsen over time. In some cases, an advanced treatment may be needed. The use of levodopa-carbidopa intestinal gel (LCIG) is one of these options. However, deciding whether to receive it can be difficult. A patient decision aid (PDA), a tool designed to inform about treatment options, can help and promote patients' participation in decision making.</p><p><strong>Objectives: </strong>This study was conducted to develop a PDA on LCIG and assess its acceptability.</p><p><strong>Methods: </strong>The International Patient Decision Aid Standards framework was used to develop the PDA. An advisory committee (<i>n</i> = 5) gave feedback on the PDA prototype. Acceptability was evaluated using a cross-sectional descriptive design. A convenience sample of 36 participants (including persons with PD receiving and not receiving LCIG, caregivers, and health care professionals) was used. Acceptability data, sociodemographics, and health literacy were collected using questionnaires and a focus group.</p><p><strong>Results: </strong>Sample characteristics were a mean age of 64.4 y (<i>s</i> = 14 y), university level of education (46.7%), and duration of illness of less than 10 y (80%). The health literacy score was judged as very good ( <math> <mrow> <mover><mrow><mi>x</mi></mrow> <mo>¯</mo></mover> </mrow> </math> = 55.2/70, <i>s</i> = 7.3). Qualitative data analysis allowed for final adjustments to the published PDA version. <b>Conclusions.</b> This study is the first to report the development of a PDA on LCIG and its acceptability testing. Participants found the PDA to be useful and would recommend it. All health care professionals indicated they intended to use it in their practice. More research will be needed to evaluate the PDA's implementation and its effects on users.</p><p><strong>Highlights: </strong>Deciding whether to opt for an advanced Parkinson's disease treatment such as levodopa-carbidopa intestinal gel can be challenging for many patients.A patient decision aid on levodopa-carbidopa intestinal gel for Parkinson's disease persons, caregivers, and health care professionals was developed to support a decision-making process.This article summarizes the steps used for its development and its acceptability testing.</p>","PeriodicalId":36567,"journal":{"name":"MDM Policy and Practice","volume":"10 2","pages":"23814683251364883"},"PeriodicalIF":1.7,"publicationDate":"2025-09-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12423512/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145065236","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Simulation Modeling of Oral Cancer Development with Risk Stratification: How Potential Screening Programs Can Be Evaluated.","authors":"Mutita Siriruchatanon, Emily R Brooks, Alexander R Kerr, Denise M Laronde, Miriam P Rosin, Stella K Kang","doi":"10.1177/23814683251353226","DOIUrl":"10.1177/23814683251353226","url":null,"abstract":"<p><p><b>Background.</b> A barrier to early-stage oral cavity cancer detection is the lack of a defined population and screening regimen satisfying risk-benefit considerations. <b>Methods.</b> We constructed a microsimulation model, Simulation of Cancers of the Oral cavity and Risk Exposures (SCORE), that incorporates risk profiles defined by smoking and alcohol exposure. SCORE simulates the development and progression of oral potentially malignant disorders (OPMD) representing benign, dysplastic, or malignant lesions in the US population starting at age 40 y. OPMD high-risk characteristics of malignant transformation informed a biopsy decision rule. SCORE was calibrated to national cancer registry data. We compared life expectancy in those aged 40 to 60 y with OPMDs, cancer incidence, and cancer-specific deaths across screening strategies with and without the biopsy decision rule, assuming screening every 3 y starting at age 50 y. <b>Results.</b> In US men, all screening strategies reduced cancer incidence and cancer-specific mortality by at least 26% and 20% compared with no screening. Whether with or without a biopsy decision rule, life expectancy among those aged 40 to 60 y with OPMDs was 36.37 ± 0.01 life-years, a gain of 0.03 life-years. However, the use of the biopsy rule improved diagnostic efficiency with 8 biopsies per treatable diagnosis. Screening with or without the biopsy decision rule in high-risk men demonstrated comparable benefit, reducing cancer-specific deaths by 27% and incidence by 20% compared with no screening. Meanwhile, in the non-high-risk subpopulation, applying the biopsy rule avoided the harms of excess procedures, reducing lifetime biopsies by 38% versus biopsy of all OPMDs while preserving reductions in cancer burden. <b>Conclusions.</b> SCORE enables virtual trials of various screening regimens and target populations. Given the time and cost of clinical trials, SCORE may facilitate the evaluation of new technologies and clinical recommendations.</p><p><strong>Highlights: </strong>A new oral cancer simulation model with risk factors including degrees of smoking and alcohol exposure, oral lesion features, and sex incorporates more accurate and precise representation of patient risk categories.We evaluated screening strategies for oral potentially malignant disorders with or without risk-stratified biopsy referral in both the general population and subpopulations defined by degrees of smoking and alcohol exposure.Men with a high degree of both smoking and alcohol exposure exhibited a significant reduction in cancer-specific deaths and cancer incidence from screening programs for oral potentially malignant disorders.Screening with risk-stratified biopsy, using a surgical treatment threshold of moderate dysplasia or worse, yielded the greatest efficiency in term of biopsies needed to detect 1 treatable case.</p>","PeriodicalId":36567,"journal":{"name":"MDM Policy and Practice","volume":"10 2","pages":"23814683251353226"},"PeriodicalIF":1.7,"publicationDate":"2025-08-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12368318/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144972624","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Preferences for Attachment Devices for Individuals with Lower-Limb Loss: A Discrete-Choice Study to Inform Regulatory Decisions.","authors":"Leslie Wilson, Matthew Garibaldi, Ruben Vargas, Molly Timmerman","doi":"10.1177/23814683251351044","DOIUrl":"10.1177/23814683251351044","url":null,"abstract":"<p><p><b>Objective.</b> The patient's perspective in shared decision making has expanded to regulatory decision making for medical devices under the Food and Drug Administration's Patient Preference Initiative. <b>Methods.</b> Using choice-based conjoint (CBC) procedures, a discrete-choice experiment measure describing the risks and benefits of osseointegration was designed and used in a preference study among 188 adults with lower-limb loss. Our measure included 8 attributes of 1) risks: chance of infection, complete device failure rate, time without prosthesis, activity limitations, and 2) benefits: avoidance of socket problems, limb perception, improved motion with less fatigue, and chance of limiting daily pain, with 3 to 4 levels each. We used a random, full-profile, balanced-overlap design in which 18 CBC conjoint pairs, sociodemographic, and clinical questions were completed. The analysis included random parameters logit with 1,000 Halton draws and latent class. <b>Results.</b> The least important levels, when compared with their respective attribute baseline level, were for avoiding the highest chance (50%) of serious infection (β = -1.32, <i>P</i> < 0.001), highest chance (40%) of complete device failure (β = -0.96, <i>P</i> < 0.001), and longest (9 mo) time without prosthesis (β = -1.12, <i>P</i> < 0.001). The most preferred levels, when compared with their respective attribute baseline level, were to eliminate daily pain (β = 0.87, <i>P</i> < 0.001; β = 0.62, <i>P</i> < 0.001). The preference for avoiding current osseointegration infection risk (10%) was much lower (β = 0.51, <i>P</i> < 0.001), showing that preferences to avoid the actual infection risk are offset by osseointegrations benefits. Latent class analysis showed 2 distinct classes with some risk averse and some with more balanced preferences. <b>Conclusions.</b> The strongest preferences were seen for attributes avoiding complications; however, individuals demonstrated a willingness to make risk-benefit tradeoffs at current risk levels. These findings can guide future regulatory prosthetic decisions and allow better shared decision making to decrease prosthetic abandonment.</p><p><strong>Highlights: </strong>Importance shown to avoid the actual infection risk of osseointegration can be offset by the individual's importance for the benefits of osseointegration to avoid pain and socket problems and to have rapid device snap on.Individuals also showed they are willing to trade the actual osseointegration device failure rate risks for the likely benefits of osseointegration.Individuals strongly preferred avoiding time without the use of a prosthetic for the time it takes to undergo and recover from the osseointegration procedure, informing the debate for favoring a faster procedure and recovery time if it is safe. However, these preferences were still in the tradeoff range for the benefits of osseointegration.Individuals showed the strongest importance for a potential benefit of","PeriodicalId":36567,"journal":{"name":"MDM Policy and Practice","volume":"10 2","pages":"23814683251351044"},"PeriodicalIF":1.9,"publicationDate":"2025-07-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12231977/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144592529","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Decision Making about Localized Esophageal Cancer Treatment: An Observational Study on Variation in Clinicians' Communication Behavior.","authors":"L F van de Water, G C Scholten, I Henselmans, J Heisterkamp, P M Jeene, F F B M Heesakkers, K J Neelis, B R Klarenbeek, M I van Berge Henegouwen, J W van den Berg, J Buijsen, E D Geijsen, H W M van Laarhoven, E M A Smets","doi":"10.1177/23814683251349473","DOIUrl":"10.1177/23814683251349473","url":null,"abstract":"<p><p><b>Background.</b> For localized esophageal cancer, more than 1 curative treatment option is available. As these different options are associated with substantially different treatment outcomes, decision making can be complex. Moreover, treatment decision making for a patient involves multiple health care providers (HCPs) from different disciplines over time, who might have their own role and perspective on the decision-making process. This study aims to describe how HCPs communicate during treatment decision consultations with patients with localized esophageal cancer. <b>Methods.</b> Audio recordings of 20 preintervention scripted standardized patient assessments (SPAs) from the SOURCE trial were used. Using 2 highly similar cases, acted by a simulated patient, considerably reduced variation at patient level. Audio recordings were content coded using open coding and rated on the degree of patient involvement in decision making using the OPTION-12. <b>Results.</b> Radiation and surgical HCPs discussed 1 to 4 different treatment options, from a total of 5 different options observed over all consultations. They discussed 0 to 11 different side effects and complications, from a total of 28. While some HCPs explicitly presented a choice, many used various implicit forms of suggesting a choice and either implicitly or explicitly marked their own preferences for treatment. Consultations showed a mean OPTION-12 score of 40.11 (range 0-100). <b>Conclusions.</b> This study shows extensive practice variation in how and to what extent standardized patients with localized esophageal cancer were involved in decision making and in the number and type of treatment options and pros and cons that were presented to them. <b>Implications.</b> The findings suggest a need for mutual alignment within oncologic HCPs treating patients with esophageal cancer.</p><p><strong>Highlights: </strong>Practice variation was found in how and to what extent health care providers involved standardized patients with localized esophageal cancer in decision making.Health care providers varied in the number and type of treatment options and pros and cons they presented.These findings suggest a need for multidisciplinary alignment.</p>","PeriodicalId":36567,"journal":{"name":"MDM Policy and Practice","volume":"10 1","pages":"23814683251349473"},"PeriodicalIF":1.9,"publicationDate":"2025-06-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12214366/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144555223","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Health Economic Evaluation of Routine Hepatocellular Carcinoma Surveillance for People with Compensated Cirrhosis to Support Australian Clinical Guidelines.","authors":"Joachim Worthington, Emily He, Michael Caruana, Stephen Wade, Barbara de Graaff, Anh Le Tuan Nguyen, Jacob George, Karen Canfell, Eleonora Feletto","doi":"10.1177/23814683251344962","DOIUrl":"10.1177/23814683251344962","url":null,"abstract":"<p><p><b>Background.</b> Liver cancer is the only cancer in Australia with rising incidence and mortality rates, despite the potential for early detection through surveillance of high-risk individuals. Hepatocellular carcinoma (HCC), the most common form of primary liver cancer, has curative treatment options available if detected early. Six-monthly HCC surveillance is recommended for people with liver cirrhosis and was proposed for inclusion in the 2023 Cancer Council Australia <i>Clinical Practice Guidelines for Hepatocellular Carcinoma Surveillance for People at High Risk in Australia</i>. To evaluate the proposed 2023 guideline recommendation, we developed Policy1-Liver, a novel mathematical model of liver disease, HCC, and surveillance. We then assessed the health and economic implications of 6-monthly HCC surveillance in Australia via ultrasound, with or without alpha-fetoprotein. <b>Methods.</b> Policy1-Liver was calibrated to existing data sources on liver disease, HCC, and health care costs in Australia. We assessed the impact of 6-monthly routine HCC surveillance with ultrasound with or without alpha-fetoprotein testing as well as a range of other sensitivity analyses and alternative scenarios such as varying surveillance adherence and intervals to assess potential future modifications to surveillance. <b>Results.</b> We estimated that 6-monthly HCC surveillance, with or without alpha-fetoprotein, can increase early-stage diagnoses to up to 81% and reduce HCC mortality by 22% in people with cirrhosis. We estimate an incremental cost-effectiveness ratio of $28,423 per quality-adjusted life-year for 6-monthly surveillance with ultrasound alone compared with no surveillance. <b>Conclusions.</b> These findings support guideline-recommended 6-monthly HCC surveillance with ultrasound, affirming its health benefits and cost-effectiveness, and demonstrate the potential to improve cost-effectiveness by refining surveillance intervals and improving early-stage HCC survival. Supporting implementation of the surveillance guidelines will play a key role in improving HCC mortality rates in Australia.</p><p><strong>Highlights: </strong>Routine surveillance can improve the likelihood of early-stage detection of liver cancer, improving survival.Our modeling found that routine HCC surveillance with ultrasound would be cost-effective for people with liver cirrhosis in Australia.These findings can inform guidelines and investment in liver cancer control for high-risk patients.</p>","PeriodicalId":36567,"journal":{"name":"MDM Policy and Practice","volume":"10 1","pages":"23814683251344962"},"PeriodicalIF":1.7,"publicationDate":"2025-06-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12202943/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144530088","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Evaluation of a Decision Support Intervention for Adolescents and Young Adults Newly Diagnosed with Cancer: A Pilot Randomized Trial.","authors":"Lamia P Barakat, Shannon N Hammer, Yansong Wen, Ashley Anil, Lisa A Schwartz, Anne Reilly, Rochelle Bagatell, Marilyn M Schapira, Yimei Li, Janet A Deatrick","doi":"10.1177/23814683251344624","DOIUrl":"10.1177/23814683251344624","url":null,"abstract":"&lt;p&gt;&lt;p&gt;&lt;b&gt;Background.&lt;/b&gt; Limited involvement in treatment-related decision making can affect adolescent and young adult (AYA) cancer outcomes and well-being. Information on developmentally consistent approaches to enhance involvement in and experiences with decision making is lacking. In a pilot randomized trial, we evaluated the feasibility, acceptability, and usability of a Web-based decision support intervention (DECIDES) for treatment-related decisions, with/without coach support. &lt;b&gt;Methods.&lt;/b&gt; Newly diagnosed/relapsed AYA (15-24 y old) and caregivers were randomized to usual care (&lt;i&gt;n&lt;/i&gt; = 11), DECIDES (&lt;i&gt;n&lt;/i&gt; = 21), or DECIDES+ (&lt;i&gt;n&lt;/i&gt; = 21 with a coach). Feedback on DECIDES was obtained in interviews with AYA and caregivers (DECIDES, DECIDES+) and oncology clinicians (&lt;i&gt;n&lt;/i&gt; = 13). Feasibility, acceptability, and usability are described. Quantitative data were integrated with qualitative data. Mean differences (MDs) between DECIDES groups and usual care at 8 wk after randomization are presented for knowledge, decision-making involvement, and decision processes. &lt;b&gt;Results.&lt;/b&gt; High retention was achieved. High acceptability and good-excellent usability of DECIDES were reported; qualitative data were congruent with these ratings. AYA and caregivers with a coach demonstrated higher engagement with DECIDES. Based on the MDs, as compared with usual care, AYA (DECIDES and DECIDES+) reported higher shared decision making (MD = 12.58, 11.93), higher decision-making involvement (MD = 19.31, 9.78), and lower decision regret (MD = -13.19, -16.55), respectively, and caregivers (DECIDES, DECIDES+) reported higher shared decision making and AYA decision-making involvement. Minimal changes to knowledge were observed. &lt;b&gt;Conclusions.&lt;/b&gt; DECIDES is feasible, acceptable, and usable for AYA treatment-related decision making with possible increases in AYA involvement. Findings suggest that decision support interventions warrant further evaluation for AYA across the treatment trajectory and for a range of treatment-related decisions. Delivery closer to the initial diagnosis and inclusion of a coach for engagement may be advantageous. This study is registered at ClinicalTrials.gov (NCT ID No. NCT06191679).&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Highlights: &lt;/strong&gt;Adolescents and young adults with newly diagnosed cancer, and their caregivers, endorsed that decision support is important for their understanding of cancer and treatment and for enhanced communication with their health care teams.Implementation of a decision support intervention shortly after cancer diagnosis is acceptable, feasible, and usable for adolescents and young adults and their caregivers.Oncology clinicians confirm the usability of decision support interventions for their adolescent and young adult patients at diagnosis.Access to a decision support intervention at diagnosis may improve the involvement of adolescents and young adults in their treatment-related decision making and decision processes.Fut","PeriodicalId":36567,"journal":{"name":"MDM Policy and Practice","volume":"10 1","pages":"23814683251344624"},"PeriodicalIF":1.9,"publicationDate":"2025-06-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12202930/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144530089","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Cost-Effectiveness Analysis of Diffuse Large B-Cell Lymphoma Treatment Pathways in the United States.","authors":"Anik R Patel, Bradley Kievit, Ken Hasegawa, Markqayne Ray, Rishika Sharma, Sarahmaria Hofmann, Rob Blissett, Frederick L Locke","doi":"10.1177/23814683251345780","DOIUrl":"10.1177/23814683251345780","url":null,"abstract":"<p><p><b>Background.</b> 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). <b>Design.</b> 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). <b>Results.</b> 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. <b>Conclusions.</b> 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.</p><p><strong>Highlights: </strong>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.</p>","PeriodicalId":36567,"journal":{"name":"MDM Policy and Practice","volume":"10 1","pages":"23814683251345780"},"PeriodicalIF":1.9,"publicationDate":"2025-06-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12198509/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144508693","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Exploring the Role of Patient Preferences in Hepatocellular Carcinoma Treatment Decisions: A Qualitative Study.","authors":"Andrew M Moon, Daniel Richardson, Gabriel V Lupu, Donna M Evon, Hanna K Sanoff, Jessica Carda-Auten, Randall Teal, Myra Waheed, Ethan Basch, David M Mauro, Ted K Yanagihara, David A Gerber, Neil D Shah, Oren K Fix, Hersh Shroff, Tammy Triglianos, Jonathan D Sorah, Jingquan Jia, Ashwin Somasundaram, Lynne I Wagner, Michael D Kappelman, Matthew Schooler, Julia R Phillips, Hiwot A Ekuban, Ariel E Sanderford, A Sidney Barritt","doi":"10.1177/23814683251340055","DOIUrl":"10.1177/23814683251340055","url":null,"abstract":"<p><p><b>Background.</b> 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. <b>Methods.</b> 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. <b>Results.</b> 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. <b>Limitations.</b> Qualitative interviews were limited to patients and providers from a single center. <b>Conclusions.</b> 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.</p><p><strong>Highlights: </strong>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.</p>","PeriodicalId":36567,"journal":{"name":"MDM Policy and Practice","volume":"10 1","pages":"23814683251340055"},"PeriodicalIF":1.9,"publicationDate":"2025-05-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12123149/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144200333","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Impact of Alternative Specifications of Uncertainty Relating to Extrapolation in Decision Models.","authors":"Doug Coyle, Kathryn Coyle","doi":"10.1177/23814683251340058","DOIUrl":"10.1177/23814683251340058","url":null,"abstract":"<p><p>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.</p><p><strong>Highlights: </strong>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.</p>","PeriodicalId":36567,"journal":{"name":"MDM Policy and Practice","volume":"10 1","pages":"23814683251340058"},"PeriodicalIF":1.9,"publicationDate":"2025-05-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12123150/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144200334","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}