MDM policy & practice最新文献

{"title":"Shared Decision Making in Practice and the Perspectives of Health Care Professionals on Video-Recorded Consultations With Patients With Low Health Literacy in the Palliative Phase of Their Disease.","authors":"Ruud T J Roodbeen,&nbsp;Janneke Noordman,&nbsp;Gudule Boland,&nbsp;Sandra van Dulmen","doi":"10.1177/23814683211023472","DOIUrl":"https://doi.org/10.1177/23814683211023472","url":null,"abstract":"<p><p><b>Introduction.</b> An important goal of palliative care is improving the quality of life of patients and their partners/families. To attain this goal, requirements and preferences of patients need to be discussed, preferably through shared decision making (SDM). This enhances patient autonomy and patient-centeredness, requiring active participation by patients. This is demanding for palliative patients, and even more so for patients with limited health literacy (LHL). This study aimed to examine SDM in practice and assess health care professionals' perspectives on their own SDM. <b>Methods.</b> An explanatory sequential mixed methods design was used. Video recordings were gathered cross-sectionally of palliative care consultations with LHL patients (<i>n</i> = 36) conducted by specialized palliative care clinicians and professionals integrating a palliative approach. The consultations were observed for SDM using the OPTION⁵ instrument. Potential determinants of SDM were examined using multilevel analysis. Sequentially, stimulated recall interviews were conducted assessing the perspectives of professionals on their SDM (<i>n</i> = 19). Interviews were examined using deductive thematic content analysis. <b>Results.</b> The average SDM score in practice was moderate, varying greatly between professionals, as shown by the multilevel analysis and by varying degrees of perceived patient involvement in SDM mentioned in the interviews. To improve this, professionals recommended 1) continuously discussing all options with patients, 2) allowing time for patients to talk, and 3) using strategic timing for involving patients in SDM. <b>Discussion.</b> The implementation of SDM for people with LHL in palliative care varies in quality and needs improvement. SDM needs to be enhanced in this care domain because decisions are complex and demanding for LHL patients. Future research is needed that focuses on supporting strategies for comprehensible SDM, best practices, and organizational adaptations.</p>","PeriodicalId":520707,"journal":{"name":"MDM policy & practice","volume":" ","pages":"23814683211023472"},"PeriodicalIF":0.0,"publicationDate":"2021-07-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1177/23814683211023472","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39197145","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":"Patient-Centered Identification of Meaningful Regulatory Endpoints for Medical Devices to Treat Parkinson's Disease.","authors":"Heather L Benz,&nbsp;Brittany Caldwell,&nbsp;John P Ruiz,&nbsp;Anindita Saha,&nbsp;Martin Ho,&nbsp;Stephanie Christopher,&nbsp;Dawn Bardot,&nbsp;Margaret Sheehan,&nbsp;Anne Donnelly,&nbsp;Lauren McLaughlin,&nbsp;Brennan Mange,&nbsp;A Brett Hauber,&nbsp;Katrina Gwinn,&nbsp;William J Heetderks,&nbsp;Murray Sheldon","doi":"10.1177/23814683211021380","DOIUrl":"https://doi.org/10.1177/23814683211021380","url":null,"abstract":"<p><p><b>Introduction.</b> A growing literature has developed on identifying outcomes that matter to patients. This study demonstrates an approach involving patient and regulatory perspectives to identify outcomes that are meaningful in the context of medical devices for Parkinson's disease (PD). <b>Methods.</b> A systematic process was used for specifying relevant regulatory endpoints by synthesizing inputs of various sources and stakeholders. First, a literature review was conducted to identify important benefits, risks, and other considerations for medical devices to treat PD; patient discussion groups (<i>n</i> = 6) were conducted to refine the list of considerations, followed by a survey (<i>n</i> = 29) to prioritize them; and patient and Food and Drug Administration (FDA) reviewers informed specification of the final endpoints. Two FDA clinicians gave clinical and regulatory perspectives at each step. <b>Results.</b> Movement symptoms were ranked as most important (ranked 1 or 2 by 72% of participants) and psychological and cognitive symptoms as the next most important (ranked 1 or 2 by 52% of participants). Within movement symptoms, falls, impaired movement, bradykinesia, resting tremor, stiffness, and rigidity were ranked highly. Overall, nine attributes were identified and prioritized as patient-centric for use in clinical trial design and quantitative patient preference studies. These attributes were benefits and risks related to therapeutics for PD as well as other considerations, including time until a medical device is available for patient use. <b>Discussion.</b> This prospective approach identified meaningful and relevant benefits, risks, and other considerations that may be used for clinical trial design and quantitative patient preference studies. Although PD was the focus of this study, the approach can be used to study patient perspectives about other disease or treatment areas.</p>","PeriodicalId":520707,"journal":{"name":"MDM policy & practice","volume":" ","pages":"23814683211021380"},"PeriodicalIF":0.0,"publicationDate":"2021-07-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1177/23814683211021380","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39198144","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":"Real-World Cost-Effectiveness of Bevacizumab With First-Line Combination Chemotherapy in Patients With Metastatic Colorectal Cancer: Population-Based Retrospective Cohort Studies in Three Canadian Provinces.","authors":"Reka E Pataky,&nbsp;Jaclyn Beca,&nbsp;David Tran,&nbsp;Wei Fang Dai,&nbsp;Erind Dvorani,&nbsp;Wanrudee Isaranuwatchai,&nbsp;Stuart Peacock,&nbsp;Riaz Alvi,&nbsp;Winson Y Cheung,&nbsp;Craig C Earle,&nbsp;Scott Gavura,&nbsp;Kelvin K W Chan","doi":"10.1177/23814683211021060","DOIUrl":"https://doi.org/10.1177/23814683211021060","url":null,"abstract":"<p><p><b>Background.</b> Real-world evidence can be a valuable tool when clinical trial data are incomplete or uncertain. Bevacizumab was adopted as first-line therapy for metastatic colorectal cancer (mCRC) based on significant survival improvements in initial clinical trials; however, survival benefit diminished in subsequent analyses. Consequently, there is uncertainty surrounding the cost-effectiveness of bevacizumab therapy achieved in practice. <b>Objective.</b> To assess real-world cost-effectiveness of first-line bevacizumab with irinotecan-based chemotherapy versus irinotecan-based chemotherapy alone for mCRC in British Columbia (BC), Saskatchewan, and Ontario, Canada. <b>Methods.</b> Using provincial cancer registries and linked administrative databases, we identified mCRC patients who initiated publicly funded irinotecan-based chemotherapy, with or without bevacizumab, in 2000 to 2015. We compared bevacizumab-treated patients to historical controls (treated before bevacizumab funding) and contemporaneous controls (receiving chemotherapy without bevacizumab), using inverse-probability-of-treatment weighting with propensity scores to balance baseline covariates. We calculated incremental cost-effectiveness ratios (ICER) using 5-year cost and survival adjusted for censoring, with bootstrapping to characterize uncertainty. We also conducted one-way sensitivity analysis for key drivers of cost-effectiveness. <b>Results.</b> The cohorts included 12,112 (Ontario), 1,161 (Saskatchewan), and 2,977 (BC) patients. Bevacizumab significantly increased treatment costs, with mean ICERs between $78,000 and $84,000/LYG (life-year gained) in the contemporaneous comparisons and $75,000 and $101,000/LYG in the historical comparisons. Reducing the cost of bevacizumab by 50% brought ICERs in all comparisons below $61,000/LYG. <b>Limitations.</b> Residual confounding in observational data may bias results, while the use of original list prices overestimates current bevacizumab cost. <b>Conclusion.</b> The addition of bevacizumab to irinotecan-based chemotherapy extended survival for mCRC patients but at significant cost. At original list prices bevacizumab can only be considered cost-effective with certainty at a willingness-to-pay threshold over $100,000/LYG, but price reductions or discounts have a significant impact on cost-effectiveness.</p>","PeriodicalId":520707,"journal":{"name":"MDM policy & practice","volume":" ","pages":"23814683211021060"},"PeriodicalIF":0.0,"publicationDate":"2021-06-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1177/23814683211021060","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39061322","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":"Improving Shared Decision Making in Latino Men With Prostate Cancer: A Thematic Analysis.","authors":"Joaquin Michel,&nbsp;Jorge Ballon,&nbsp;Sarah E Connor,&nbsp;David C Johnson,&nbsp;Jonathan Bergman,&nbsp;Christopher S Saigal,&nbsp;Mark S Litwin,&nbsp;Dana L Alden","doi":"10.1177/23814683211014180","DOIUrl":"https://doi.org/10.1177/23814683211014180","url":null,"abstract":"<p><p><b>Background.</b> Multiple studies have shown that digitally mediated decision aids help prepare patients for medical decision making with their providers. However, few studies have investigated whether decision-support preferences differ between non-English-speaking and English-speaking Latino men with limited literacy. <b>Objective.</b> To identify and compare health information seeking patterns, preferences for information presentation, and interest in digital decision aids in a sample of Southern Californian underserved Latino men with newly diagnosed prostate cancer at a county hospital. <b>Methods.</b> We conducted semistructured, in-depth telephone interviews with 12 Spanish-speaking and 8 English-speaking Latino men using a purposive sampling technique. Following transcription of taped interviews, Spanish interviews were translated. Using a coding protocol developed by the team, two bilingual members jointly analyzed the transcripts for emerging themes. Coder agreement exceeded 80%. Differences were resolved through discussion. <b>Results.</b> Thematic differences between groups with different preferred languages emerged. Most respondents engaged in online health information seeking using cellphones, perceived a paternalistic patient-provider relationship, and expressed willingness to use hypothetical digital decision aids if recommended by their provider. English speakers reported higher digital technology proficiency for health-related searches. They also more frequently indicated family involvement in digital search related to their condition and preferred self-guided, web-based decision aids. In comparison, Spanish speakers reported lower digital technology proficiency and preferred family-involved, coach-guided, paper and visual decision aids. English speakers reported substantially higher levels of formal education. <b>Conclusion.</b> Preferences regarding the use of digital technology to inform prostate cancer treatment decision making among underserved Latino men varied depending on preferred primary language. Effective preparation of underserved Latino men for shared decision making requires consideration of alternative approaches depending on level of education attainment and preferred primary language.</p>","PeriodicalId":520707,"journal":{"name":"MDM policy & practice","volume":" ","pages":"23814683211014180"},"PeriodicalIF":0.0,"publicationDate":"2021-05-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1177/23814683211014180","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39075567","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 Practical Application of a Multiple-Criteria Decision Analysis Framework on Respiratory Inhalers: Is It Always Useful in the MOH Malaysia Medicines Formulary Listing Context?","authors":"Yee Vern Yong,&nbsp;Siti Hajar Mahamad Dom,&nbsp;Nurulmaya Ahmad Sa'ad,&nbsp;Rosliza Lajis,&nbsp;Faridah Aryani Md Yusof,&nbsp;Jamalul Azizi Abdul Rahaman","doi":"10.1177/2381468321994063","DOIUrl":"https://doi.org/10.1177/2381468321994063","url":null,"abstract":"<p><p><b>Objectives.</b> The current health technology assessment used to evaluate respiratory inhalers is associated with limitations that have necessitated the development of an explicit formulary decision-making framework to ensure balance between the accessibility, value, and affordability of medicines. This study aimed to develop a multiple-criteria decision analysis (MCDA) framework, apply the framework to potential and currently listed respiratory inhalers in the Ministry of Health Medicines Formulary (MOHMF), and analyze the impacts of applying the outputs, from the perspective of listing and delisting medicines in the formulary. <b>Methods.</b> The overall methodology of the framework development adhered to the recommendations of the ISPOR MCDA Emerging Good Practices Task Force. The MCDA framework was developed using Microsoft Excel 2010 and involved all relevant stakeholders. The framework was then applied to 27 medicines, based on data gathered from the highest levels of available published evidence, pharmaceutical companies, and professional opinions. The performance scores were analyzed using the additive model. The end values were then deliberated by an expert committee. <b>Results.</b> A total of eight main criteria and seven subcriteria were determined by the stakeholders. The economic criterion was weighted at 30%. Among the noneconomic criteria, \"patient suitability\" was weighted the highest. Based on the MCDA outputs, the expert committee recommended one potential medicine (out of three; 33%) be added to the MOHMF and one existing medicine (out of 24; 4%) be removed/delisted from the MOHMF. The other existing medicines remained unchanged. <b>Conclusions.</b> Although this framework was useful for deciding to add new medicines to the formulary, it appears to be less functional and impactful for the removal/delisting existing medicines from the MOHMF. The generalizability of this conclusion to other formulations remains to be confirmed.</p>","PeriodicalId":520707,"journal":{"name":"MDM policy & practice","volume":" ","pages":"2381468321994063"},"PeriodicalIF":0.0,"publicationDate":"2021-03-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1177/2381468321994063","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38794587","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":"How Risky Is That Risk Sharing Agreement? Mean-Variance Tradeoffs and Unintended Consequences of Six Common Risk Sharing Agreements.","authors":"Gregory S Zaric","doi":"10.1177/2381468321990404","DOIUrl":"https://doi.org/10.1177/2381468321990404","url":null,"abstract":"<p><p><b>Background.</b> Pharmaceutical risk sharing agreements (RSAs) are commonly used to manage uncertainties in costs and/or clinical benefits when new drugs are added to a formulary. However, existing mathematical models of RSAs ignore the impact of RSAs on clinical and financial risk. <b>Methods.</b> We develop a model in which the number of patients, total drug consumption per patient, and incremental health benefits per patient are uncertain at the time of the introduction of a new drug. We use the model to evaluate the impact of six common RSAs on total drug costs and total net monetary benefit (NMB). <b>Results.</b> We show that, relative to not having an RSA in place, each RSA reduces expected total drug costs and increases expected total NMB. Each RSA also improves two measures of risk by reducing the probability that total drug costs exceed any threshold and reducing the probability of obtaining negative NMB. However, the effects on variance in both NMB and total drug costs are mixed. In some cases, relative to not having an RSA in place, implementing an RSA can increase variability in total drug costs or total NMB. We also show that, for some RSAs, when their parameters are adjusted so that they have the same impact on expected total drug cost, they can be rank-ordered in terms of their impact on variance in drug costs. <b>Conclusions.</b> Although all RSAs reduce expected total drug costs and increase expected total NMB, some RSAs may actually have the undesirable effect of increasing risk. Payers and formulary managers should be aware of these mean-variance tradeoffs and the potentially unintended results of RSAs when designing and negotiating RSAs.</p>","PeriodicalId":520707,"journal":{"name":"MDM policy & practice","volume":" ","pages":"2381468321990404"},"PeriodicalIF":0.0,"publicationDate":"2021-02-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1177/2381468321990404","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"25404685","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":"Promoting Reflection on Medical Maximizing-Minimizing Preferences May Create Undesired Effects on Decisions About Low-Benefit and High-Benefit Care.","authors":"Brian J Zikmund-Fisher,&nbsp;Victoria A Shaffer,&nbsp;Laura D Scherer","doi":"10.1177/2381468320987498","DOIUrl":"https://doi.org/10.1177/2381468320987498","url":null,"abstract":"<p><strong>Background: </strong>Medical maximizing-minimizing (MM) preferences predict a variety of medical decisions. We tested whether informing people about their MM preferences and asking them to reflect on the pros and cons of that preference would improve medical decisions when clear clinical recommendations exist.</p><p><strong>Methods: </strong>We surveyed 1219 US adults age 40+ that were sampled to ensure a 50%/50% distribution of medical maximizers versus minimizers. Participants either received no MM feedback (Control) or received feedback about their MM type and instructions to reflect on how that MM type can be helpful in some circumstances and problematic in others (Reflection). All participants then completed five hypothetical decision scenarios regarding low-value care services (e.g., head computed tomography scan for mild concussion) and three about high-value care (e.g., flu vaccination).</p><p><strong>Results: </strong>There were no significant differences between the Control and Reflection groups in five of eight scenarios. In three scenarios (two low-benefit and one high-benefit), we observed small effects in the nonhypothesized direction for the MM subgroup least likely to follow the recommendation (e.g., maximizers in the Reflection group were more likely to request low-benefit care).</p><p><strong>Conclusions: </strong>Asking people to reflect on their MM preferences may be a counterproductive strategy for optimizing patient decision making around quality of care.</p>","PeriodicalId":520707,"journal":{"name":"MDM policy & practice","volume":" ","pages":"2381468320987498"},"PeriodicalIF":0.0,"publicationDate":"2021-01-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1177/2381468320987498","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"25383219","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 Validation of Three Regional Microsimulation Models for Predicting Colorectal Cancer Screening Benefits in Europe.","authors":"Andrea Gini,&nbsp;Maaike Buskermolen,&nbsp;Carlo Senore,&nbsp;Ahti Anttila,&nbsp;Dominika Novak Mlakar,&nbsp;Piret Veerus,&nbsp;Marcell Csanádi,&nbsp;Erik E L Jansen,&nbsp;Nadine Zielonke,&nbsp;Sirpa Heinävaara,&nbsp;György Széles,&nbsp;Nereo Segnan,&nbsp;Harry J de Koning,&nbsp;Iris Lansdorp-Vogelaar","doi":"10.1177/2381468320984974","DOIUrl":"https://doi.org/10.1177/2381468320984974","url":null,"abstract":"<p><p><b>Background.</b> Validated microsimulation models have been shown to be useful tools in providing support for colorectal cancer (CRC) screening decisions. Aiming to assist European countries in reducing CRC mortality, we developed and validated three regional models for evaluating CRC screening in Europe. <b>Methods.</b> Microsimulation Screening Analysis-Colon (MISCAN-Colon) model versions for Italy, Slovenia, and Finland were quantified using data from different national institutions. These models were validated against the best available evidence for the effectiveness of screening from their region (when available): the Screening for COlon REctum (SCORE) trial and the Florentine fecal immunochemical test (FIT) screening study for Italy; the Norwegian Colorectal Cancer Prevention (NORCCAP) trial and the guaiac fecal occult blood test (gFOBT) Finnish population-based study for Finland. When published evidence was not available (Slovenia), the model was validated using cancer registry data. <b>Results.</b> Our three models reproduced age-specific CRC incidence rates and stage distributions in the prescreening period. Moreover, the Italian and Finnish models replicated CRC mortality reductions (reasonably) well against the best available evidence. CRC mortality reductions were predicted slightly larger than those observed (except for the Florentine FIT study), but consistently within the corresponding 95% confidence intervals. <b>Conclusions.</b> Our findings corroborate the MISCAN-Colon reliability in supporting decision making on CRC screening. Furthermore, our study provides the model structure for an additional tool (EU-TOPIA CRC evaluation tool: http://miscan.eu-topia.org) that aims to help policymakers and researchers monitoring or improving CRC screening in Europe.</p>","PeriodicalId":520707,"journal":{"name":"MDM policy & practice","volume":" ","pages":"2381468320984974"},"PeriodicalIF":0.0,"publicationDate":"2021-01-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1177/2381468320984974","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"25383221","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":"Pharmacologic, Dietary, and Psychological Treatments for Irritable Bowel Syndrome With Constipation: Cost Utility Analysis.","authors":"Eric D Shah,&nbsp;Jessica K Salwen-Deremer,&nbsp;Peter R Gibson,&nbsp;Jane G Muir,&nbsp;Shanti Eswaran,&nbsp;William D Chey","doi":"10.1177/2381468320978417","DOIUrl":"https://doi.org/10.1177/2381468320978417","url":null,"abstract":"<p><p><b>Introduction.</b> Irritable bowel syndrome (IBS) is the most common gastroenterology referral and one of the most common gastrointestinal complaints in primary care. We performed a cost-utility analysis of the most common treatments available in general practice for IBS with constipation (IBS-C), the most expensive IBS subtype. <b>Methods.</b> We developed a decision analytic model evaluating guideline-recommended and Food and Drug Administration-approved drugs, supplements, and dietary/psychological interventions. Model inputs were derived from \"global symptom improvement\" outcomes in systematic reviews of clinical trials. Costs were derived from national datasets. Analysis was performed with a 1-year time horizon from patient and payer perspectives. We analyzed a prototypical managed-care health plan with no cost-sharing to the patient. <b>Results.</b> From a payer perspective, global IBS treatments (including low FODMAP, cognitive behavioral therapy [CBT], neuromodulators), which are not specific to the IBS-C bowel subtype were less expensive than on-label prescription drug treatments. From a patient perspective, on-label prescription drug treatment with linaclotide was the least expensive treatment strategy. Drug prices and costs to manage untreated IBS-C were most important determinants of payer treatment preferences. Effects of treatment on missed work-days and need for repeated appointments to complete treatment were the most important determinants of treatment preference to patients. <b>Discussion.</b> Due mostly to prescription drug prices, neuromodulators, low FODMAP, and CBT appear cost-effective compared to on-label drug treatments from a payer perspective in cost-utility analysis. These findings may explain common treatment barriers in clinical practice.</p>","PeriodicalId":520707,"journal":{"name":"MDM policy & practice","volume":" ","pages":"2381468320978417"},"PeriodicalIF":0.0,"publicationDate":"2021-01-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1177/2381468320978417","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"25316668","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":"Parkinson's Patients' Tolerance for Risk and Willingness to Wait for Potential Benefits of Novel Neurostimulation Devices: A Patient-Centered Threshold Technique Study.","authors":"Brett Hauber,&nbsp;Brennan Mange,&nbsp;Mo Zhou,&nbsp;Shomesh Chaudhuri,&nbsp;Heather L Benz,&nbsp;Brittany Caldwell,&nbsp;John P Ruiz,&nbsp;Anindita Saha,&nbsp;Martin Ho,&nbsp;Stephanie Christopher,&nbsp;Dawn Bardot,&nbsp;Margaret Sheehan,&nbsp;Anne Donnelly,&nbsp;Lauren McLaughlin,&nbsp;Katrina Gwinn,&nbsp;Andrew Lo,&nbsp;Murray Sheldon","doi":"10.1177/2381468320978407","DOIUrl":"https://doi.org/10.1177/2381468320978407","url":null,"abstract":"<p><p><b>Background.</b> Parkinson's disease (PD) is neurodegenerative, causing motor, cognitive, psychological, somatic, and autonomic symptoms. Understanding PD patients' preferences for novel neurostimulation devices may help ensure that devices are delivered in a timely manner with the appropriate level of evidence. Our objective was to elicit preferences and willingness-to-wait for novel neurostimulation devices among PD patients to inform a model of optimal trial design. <b>Methods.</b> We developed and administered a survey to PD patients to quantify the maximum levels of risks that patients would accept to achieve potential benefits of a neurostimulation device. Threshold technique was used to quantify patients' risk thresholds for new or worsening depression or anxiety, brain bleed, or death in exchange for improvements in \"on-time,\" motor symptoms, pain, cognition, and pill burden. The survey elicited patients' willingness to wait to receive treatment benefit. Patients were recruited through Fox Insight, an online PD observational study. <b>Results.</b> A total of 2740 patients were included and a majority were White (94.6%) and had a 4-year college degree (69.8%). Risk thresholds increased as benefits increased. Threshold for depression or anxiety was substantially higher than threshold for brain bleed or death. Patient age, ambulation, and prior neurostimulation experience influenced risk tolerance. Patients were willing to wait an average of 4 to 13 years for devices that provide different levels of benefit. <b>Conclusions.</b> PD patients are willing to accept substantial risks to improve symptoms. Preferences are heterogeneous and depend on treatment benefit and patient characteristics. The results of this study may be useful in informing review of device applications and other regulatory decisions and will be input into a model of optimal trial design for neurostimulation devices.</p>","PeriodicalId":520707,"journal":{"name":"MDM policy & practice","volume":" ","pages":"2381468320978407"},"PeriodicalIF":0.0,"publicationDate":"2021-01-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1177/2381468320978407","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"25316666","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}