Journal of market access & health policy最新文献

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Bright line or lottery? On significance and value in medical decision making. 亮线还是彩票?论医疗决策的意义与价值。
Journal of market access & health policy Pub Date : 2021-09-20 eCollection Date: 2021-01-01 DOI: 10.1080/20016689.2021.1981574
Jörg Mahlich, Srirangan Dheban
{"title":"Bright line or lottery? On significance and value in medical decision making.","authors":"Jörg Mahlich,&nbsp;Srirangan Dheban","doi":"10.1080/20016689.2021.1981574","DOIUrl":"https://doi.org/10.1080/20016689.2021.1981574","url":null,"abstract":"<p><p>It is widely acknowledged that using p-value thresholds as the basis for making decision on health care spending is not appropriate. In the context of medical decision making, we argue that patient preferences need to be a stronger factor. Depending on attitudes to risk, patients might prefer a medical treatment that performs on average worse than a comparator but offers a small probability of a large gain such as a cure. However, what has been labeled 'value of hope' is not yet fully reflected in the decision-making process of drug approval and health technology assessment (HTA). Therefore, patient risk preferences should be formally incorporated within the decision-making framework for regulatory and reimbursement decisions.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"9 1","pages":"1981574"},"PeriodicalIF":0.0,"publicationDate":"2021-09-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/5b/d5/ZJMA_9_1981574.PMC8462922.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39452113","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}
引用次数: 1
The impact of increasing multitarget stool DNA use among colorectal cancer screeners in a self-insured US employer population. 在自我保险的美国雇主人群中,结肠直肠癌筛查者中增加多靶点粪便DNA使用的影响。
Journal of market access & health policy Pub Date : 2021-09-01 eCollection Date: 2021-01-01 DOI: 10.1080/20016689.2021.1948670
Joanne M Hathway, Lesley-Ann Miller-Wilson, Abhishek Sharma, Ivar S Jensen, Weiyu Yao, Sajjad Raza, Philip D Parks, Milton C Weinstein
{"title":"The impact of increasing multitarget stool DNA use among colorectal cancer screeners in a self-insured US employer population.","authors":"Joanne M Hathway,&nbsp;Lesley-Ann Miller-Wilson,&nbsp;Abhishek Sharma,&nbsp;Ivar S Jensen,&nbsp;Weiyu Yao,&nbsp;Sajjad Raza,&nbsp;Philip D Parks,&nbsp;Milton C Weinstein","doi":"10.1080/20016689.2021.1948670","DOIUrl":"https://doi.org/10.1080/20016689.2021.1948670","url":null,"abstract":"<p><p><b>Background:</b> In the United States (US), colorectal cancer (CRC) is the second leading cause of cancer-related deaths. With the majority of the US population covered by employer-based health plans, employers can play a critical role in increasing CRC screening adherence, which may help avert CRC-related deaths. Therefore, it is important for self-insured employers to consider the impact of appropriate utilization of CRC screening options. <b>Objective:</b> To evaluate the impact of increasing multitarget stool DNA [mt-sDNA (Cologuard®)] use among CRC screeners from the perspective of a US self-insured employer. <b>Methods:</b>A 5-year Markov model was developed to quantify the budget impact of increasing mt-sDNA from 6% to 15% among average-risk screeners using colonoscopy, fecal immunological test, and mt-sDNA. Data on direct medical costs were obtained from published literature, Medicare CPT codes, and the Healthcare cost and Utilization project. Indirect costs included productivity loss due to workplace absenteeism for CRC screening and treatment. <b>Results:</b> With a hypothetical population of 100,000 employees with screeners aged 50-64 years, compared to status quo, increased mt-sDNA utilization resulted in no differences in the numbers of cancers detected and the overall direct and indirect cost savings were ~$214,000 ($0.04 per-employee-per-month) over 5 years. Most of the savings were due to a reduction in the direct medical expenditure related to CRC screening, adverse events, and productivity loss due to colonoscopy screening. Similar results were observed in the model simulation among screeners aged 45-64 years. <b>Conclusion:</b> Increased utilization of mt-sDNA for CRC screening averts direct and indirect medical costs from a self-insured US employer perspective.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"9 1","pages":"1948670"},"PeriodicalIF":0.0,"publicationDate":"2021-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/92/45/ZJMA_9_1948670.PMC8425769.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39409527","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}
引用次数: 0
Switching from one reference biological to another in stable patients for non-medical reasons: a literature search and brief review. 稳定期患者因非医学原因从一种参考生物制剂转用另一种参考生物制剂:文献检索和简要综述。
Journal of market access & health policy Pub Date : 2021-08-20 eCollection Date: 2021-01-01 DOI: 10.1080/20016689.2021.1964792
Knut Stavem
{"title":"Switching from one reference biological to another in stable patients for non-medical reasons: a literature search and brief review.","authors":"Knut Stavem","doi":"10.1080/20016689.2021.1964792","DOIUrl":"10.1080/20016689.2021.1964792","url":null,"abstract":"<p><p><b>Background:</b> The practice of non-medical switch (NMS) from a reference biological (originator) to a biosimilar is widely accepted in some countries. However, there is little documentation on the impact of NMS from one originator to another originator. <b>Objectives:</b> To assess the consequences for patients of NMS from one biological originator to another, based on existing literature. The focus was on efficacy and cost of treatment with TNF-α-inhibitors in three disease areas. <b>Methods:</b> A literature search was conducted in Ovid (PubMed, EMBASE) and abstracts from meetings in key therapeutic areas, to identify studies reporting efficacy, safety or costs by switching between originator biologics. <b>Results:</b> 167 references were identified and abstracts screened; 36 papers reviewed in full text, and 6 fulfilled the inclusion criteria. Three clinical studies of NMS had very small sample sizes, but suggested that NMS is beneficial. The remaining three studies used administrative data with little clinical information, indicating that NMS was disadvantageous and associated with increased health care utilization and costs. <b>Conclusions:</b> There is very limited documentation on NMS from one originator biological to another, and the literature suffers from methodological limitations. The results are mixed and preclude drawing an overriding conclusion. Future studies, are warranted.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"9 1","pages":"1964792"},"PeriodicalIF":0.0,"publicationDate":"2021-08-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/eb/a5/ZJMA_9_1964792.PMC8381978.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39344177","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}
引用次数: 0
Cost-Effectiveness Analysis of a Prescription Digital Therapeutic for the Treatment of Opioid Use Disorder. 一种治疗阿片类药物使用障碍的处方数字疗法的成本效益分析。
Journal of market access & health policy Pub Date : 2021-08-18 eCollection Date: 2021-01-01 DOI: 10.1080/20016689.2021.1966187
Fulton F Velez, Daniel C Malone
{"title":"Cost-Effectiveness Analysis of a Prescription Digital Therapeutic for the Treatment of Opioid Use Disorder.","authors":"Fulton F Velez,&nbsp;Daniel C Malone","doi":"10.1080/20016689.2021.1966187","DOIUrl":"https://doi.org/10.1080/20016689.2021.1966187","url":null,"abstract":"<p><p>The lack of adequate treatment for many patients with opioid use disorder (OUD) has led to high medical costs ($90B in 2020). An analysis of the cost-effectiveness (cost-utility) of reSET-O, the first and only FDA-approved prescription digital therapeutic (PDT) for the treatment of OUD, is needed to inform value assessments and healthcare decision making. To evaluate the cost-utility of reSET-O in conjunction with treatment-as usual (TAU) compared to TAU alone. A third-party payer-perspective decision analytic model evaluated the cost-effectiveness of reSET-O + TAU relative to TAU (i.e., oral buprenorphine, face-to-face counseling, and contingency management [immediate rewards for negative drug tests logged]) alone over 12 weeks. Clinical effectiveness data (retention in therapy and health state utilities) were obtained from the peer-reviewed literature, while resource utilization and cost data were obtained from a published claims data analyses. Over 12 weeks, the addition of reSET-O to TAU resulted in a gain of 0.003 quality-adjusted life years (QALYs), and $1,014 lower costs, resulting in economic dominance vs. TAU. reSET-O + TAU's was economically dominant (less costly, more effective) vs. TAU alone over 12 weeks, a result that was driven by a reduction in medical costs after initiation of reSET-O observed in a recent real-world claims analysis.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"9 1","pages":"1966187"},"PeriodicalIF":0.0,"publicationDate":"2021-08-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/7e/30/ZJMA_9_1966187.PMC8381930.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39344180","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}
引用次数: 4
Accelerating patient access to oncology medicines with multiple indications in Europe. 加速欧洲患者获得多指征肿瘤药物。
Journal of market access & health policy Pub Date : 2021-08-17 eCollection Date: 2021-01-01 DOI: 10.1080/20016689.2021.1964791
R Lawlor, T Wilsdon, E Darquennes, D Hemelsoet, J Huismans, R Normand, A Roediger
{"title":"Accelerating patient access to oncology medicines with multiple indications in Europe.","authors":"R Lawlor,&nbsp;T Wilsdon,&nbsp;E Darquennes,&nbsp;D Hemelsoet,&nbsp;J Huismans,&nbsp;R Normand,&nbsp;A Roediger","doi":"10.1080/20016689.2021.1964791","DOIUrl":"https://doi.org/10.1080/20016689.2021.1964791","url":null,"abstract":"<p><p><b>Background:</b> In recent years, innovation in oncology has created new challenges for pricing and reimbursement systems. Oncology medicines with multiple indications face a number of access challenges: (1) the number of assessments and administrative burden; (2) aligning price to different values of the same product; (3) managing clinical uncertainty at time of launch; and (4) managing budget uncertainty. These challenges impact a range of stakeholders and can result in delayed patient access to life-saving treatments. Consequently, countries have taken steps to facilitate patient access. <b>Methods:</b> Drawing on the experience across Europe we have reviewed different mechanisms countries have adopted that address these challenges. These include approaches aimed directly at the issue, multi-year-multi-indication (MYMI) agreements (BE, NL), and other approaches to manage access: flexible access agreements for new indications with clinical uncertainty (UK); development of a new agreement for each new indication (IT); and immediate access for new indications and bundled assessments (DE). <b>Results:</b> MYMI agreements are valuable where existing rules mean that every indication faces the same upfront evaluation process that delays patient access. They are also useful in managing budget impact and uncertainty. Other approaches that adopt an indication-specific approach helps manage clinical uncertainty at the time of launch and realise different values for the same product. They can help align price to value, even though indication-based pricing does not exist. Bundled assessments reduce the administrative burden for stakeholders, and the benefits of immediate reimbursement is that patient access is not delayed. <b>Conclusion:</b> The challenges for medicines with multiple indications impact a range of stakeholders and can result in delayed patient access to life-saving treatments. MYMI agreements have created a more pragmatic approach to HTA for medicines with multiple indications to ensure both fast and broad patient access. Continued innovation in oncology will require further innovative approaches in pricing and reimbursement. It is important that policymakers, payers and manufacturers engage in early discussions and are willing to find new solutions to help accelerate patient access to innovative therapies.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"9 1","pages":"1964791"},"PeriodicalIF":0.0,"publicationDate":"2021-08-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/24/57/ZJMA_9_1964791.PMC8381976.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39346609","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}
引用次数: 10
Informing decision makers seeking to improve vaccination programs: case-study Serbia. 向寻求改进疫苗接种规划的决策者提供信息:塞尔维亚案例研究。
Journal of market access & health policy Pub Date : 2021-07-25 eCollection Date: 2021-01-01 DOI: 10.1080/20016689.2021.1938894
Christophe Sauboin, Jovan Mihajlović, Maarten Jacobus Postma, Regine Geets, Djurdja Antic, Baudouin Standaert
{"title":"Informing decision makers seeking to improve vaccination programs: case-study Serbia.","authors":"Christophe Sauboin,&nbsp;Jovan Mihajlović,&nbsp;Maarten Jacobus Postma,&nbsp;Regine Geets,&nbsp;Djurdja Antic,&nbsp;Baudouin Standaert","doi":"10.1080/20016689.2021.1938894","DOIUrl":"https://doi.org/10.1080/20016689.2021.1938894","url":null,"abstract":"<p><p><b>Background:</b>The optimisation of vaccine policies before their implementation is beholden upon public health decision makers, seeking to maximise population health. In this case study in Serbia, the childhood vaccines under consideration included pneumococcal conjugate vaccination (PCV), rotavirus (RV) vaccination and varicella zoster virus (VZV) vaccination. <b>Objective:</b> The objective of this study is to define the optimal order of introduction of vaccines to minimise deaths, quality adjusted life years (QALYs) lost, or hospitalisation days, under budget and vaccine coverage constraints. <b>Methods:</b> A constrained optimisation model was developed including a static multi-cohort decision-tree model for the three infectious diseases. Budget and vaccine coverage were constrained, and to rank the vaccines, the optimal solution to the linear programming problem was based upon the ratio of the outcome (deaths, QALYs or hospitalisation days) per unit of budget. A probabilistic decision analysis Monte Carlo simulation technique was used to test the robustness of the rankings. <b>Results:</b> PCV was the vaccine ranked first to minimise deaths, VZV vaccination for QALY loss minimisation and RV vaccination for hospitalisation day reduction. Sensitivity analysis demonstrated the most robust ranking was that for PCV minimizing deaths. <b>Conclusion:</b> Constrained optimisation modelling, whilst considering all potential interventions currently, provided a comprehensive and rational approach to decision making.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"9 1","pages":"1938894"},"PeriodicalIF":0.0,"publicationDate":"2021-07-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/20016689.2021.1938894","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39292079","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}
引用次数: 2
The effects of market concentration on health care price and quality in hospital markets in Ibadan, Nigeria. 市场集中度对尼日利亚伊巴丹医院市场医疗保健价格和质量的影响。
Journal of market access & health policy Pub Date : 2021-06-22 DOI: 10.1080/20016689.2021.1938895
Bosede Olanike Awoyemi, Olanrewaju Olaniyan
{"title":"The effects of market concentration on health care price and quality in hospital markets in Ibadan, Nigeria.","authors":"Bosede Olanike Awoyemi,&nbsp;Olanrewaju Olaniyan","doi":"10.1080/20016689.2021.1938895","DOIUrl":"https://doi.org/10.1080/20016689.2021.1938895","url":null,"abstract":"<p><p>Evidence about the Nigerian health indicators show that the quality of health care in Nigeria is low and inflation of health care prices also persists. Theoretically, by observing the market concentration, inferences can be drawn as to how hospitals conduct themselves, which allows the evaluation of the market performance. Therefore, the effects of market concentration on the health care price and quality were examined. Market concentration was measured by Herfindahl Hirschman Index (HHI) and four hospital concentration ratios (CR<sub>4</sub>). The values of HHI were disaggregated into the less and more concentrated markets. Quality of health care was measured by the staff-nurse-patient ratio. Ordinary Least Square (OLS) was used to estimate the effects of market concentration on price and quality of health care. The price of health care was found to be 13.4% lower in the less concentrated markets than in the more concentrated market. Income significantly and positively influenced health care prices by 17.8%. Also, a low HHI lead to 33.4% increase in Staff-nurse Patient Ratio (SPR) indicating that the quality of health care was higher in less concentrated markets as hospitals increased the treatment intensity via staff-nurse patient ratio. A less concentrated market is linked with higher health care quality and lower health care prices. Therefore, a strategy that will reduce market concentration so as to enhance consumer welfare in terms of price and quality is recommended.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"9 1","pages":"1938895"},"PeriodicalIF":0.0,"publicationDate":"2021-06-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/20016689.2021.1938895","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39149466","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}
引用次数: 1
Economic evaluation of betibeglogene autotemcel (Beti-cel) gene addition therapy in transfusion-dependent β-thalassemia. β-地中海贫血输血依赖性β-地中海贫血β-基因自体细胞(β-细胞)基因添加治疗的经济评价。
Journal of market access & health policy Pub Date : 2021-06-07 DOI: 10.1080/20016689.2021.1922028
Anuraag R Kansal, Odette S Reifsnider, Sarah B Brand, Neil Hawkins, Anna Coughlan, Shujun Li, Lael Cragin, Clark Paramore, Andrew C Dietz, J Jaime Caro
{"title":"Economic evaluation of betibeglogene autotemcel (Beti-cel) gene addition therapy in transfusion-dependent β-thalassemia.","authors":"Anuraag R Kansal,&nbsp;Odette S Reifsnider,&nbsp;Sarah B Brand,&nbsp;Neil Hawkins,&nbsp;Anna Coughlan,&nbsp;Shujun Li,&nbsp;Lael Cragin,&nbsp;Clark Paramore,&nbsp;Andrew C Dietz,&nbsp;J Jaime Caro","doi":"10.1080/20016689.2021.1922028","DOIUrl":"https://doi.org/10.1080/20016689.2021.1922028","url":null,"abstract":"<p><p><b>Background</b>: Standard of care (SoC) for transfusion-dependent β-thalassemia (TDT) requires lifelong, regular blood transfusions as well as chelation to reduce iron accumulation. <b>Objective</b>: This study investigates the cost-effectiveness of betibeglogene autotemcel ('beti-cel'; LentiGlobin for β-thalassemia) one-time, gene addition therapy compared to lifelong SoC for TDT. <b>Study design</b>: Microsimulation model simulated the lifetime course of TDT based on a causal sequence in which transfusion requirements determine tissue iron levels, which in turn determine risk of iron overload complications that increase mortality. Clinical trial data informed beti-cel clinical parameters; effects of SoC on iron levels came from real-world studies; iron overload complication rates and mortality were based on published literature. <b>Setting</b>: USA; commercial payer perspective <b>Participants</b>: TDT patients age 2-50 <b>Interventions</b>: Beti-cel is compared to SoC. <b>Main outcome measure</b>: Incremental cost-effectiveness ratio (ICER) utilizing quality-adjusted life-years (QALYs) <b>Results</b>: The model predicts beti-cel adds 3.8 discounted life years (LYs) or 6.9 QALYs versus SoC. Discounted lifetime costs were $2.28 M for beti-cel ($572,107 if excluding beti-cel cost) and $2.04 M for SoC, with a resulting ICER of $34,833 per QALY gained. <b>Conclusion</b>: Beti-cel is cost-effective for TDT patients compared to SoC. This is due to longer survival and cost offset of lifelong SoC.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"9 1","pages":"1922028"},"PeriodicalIF":0.0,"publicationDate":"2021-06-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/20016689.2021.1922028","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39110997","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}
引用次数: 9
Modeling long-term health and economic implications of new treatment strategies for Parkinson's disease: an individual patient simulation study. 模拟帕金森病新治疗策略的长期健康和经济影响:个体患者模拟研究。
Journal of market access & health policy Pub Date : 2021-06-03 DOI: 10.1080/20016689.2021.1922163
Conor Chandler, Henri Folse, Peter Gal, Ameya Chavan, Irina Proskorovsky, Conrado Franco-Villalobos, Yunyang Yang, Alex Ward
{"title":"Modeling long-term health and economic implications of new treatment strategies for Parkinson's disease: an individual patient simulation study.","authors":"Conor Chandler,&nbsp;Henri Folse,&nbsp;Peter Gal,&nbsp;Ameya Chavan,&nbsp;Irina Proskorovsky,&nbsp;Conrado Franco-Villalobos,&nbsp;Yunyang Yang,&nbsp;Alex Ward","doi":"10.1080/20016689.2021.1922163","DOIUrl":"https://doi.org/10.1080/20016689.2021.1922163","url":null,"abstract":"<p><p><b>Background</b>: Simulation modeling facilitates the estimation of long-term health and economic outcomes to inform healthcare decision-making. <b>Objective</b>: To develop a framework to simulate progression of Parkinson's disease (PD), capturing motor and non-motor symptoms, clinical outcomes, and associated costs over a lifetime. <b>Methods</b>: A patient-level simulation was implemented accounting for individual variability and interrelated changes in common disease progression scales. Predictive equations were developed to model progression for newly diagnosed patients and were combined with additional sources to inform long-term progression. Analyses compared a hypothetical disease-modifying therapy (DMT) with a standard of care to explore the drivers of cost-effectiveness. <b>Results</b>: The equations captured the dependence between the various measures, leveraging prior values and rates of change to obtain realistic predictions. The simulation was built upon several interrelated equations, validated by comparison with observed values for the Movement Disorder Society Unified PD Rating Scale (MDS-UPDRS) and UPDRS subscales over time. In a case study, disease progression rates, patient utilities, and direct non-medical costs were drivers of cost-effectiveness. <b>Conclusions</b>: The developed equations supported the simulation of early PD. This model can support conducting simulations to inform internal decision-making, trial design, and strategic planning early in the development of new DMTs entering clinical trials.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"9 1","pages":"1922163"},"PeriodicalIF":0.0,"publicationDate":"2021-06-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/20016689.2021.1922163","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39089623","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}
引用次数: 2
Impact of health technology assessment on prescribing patterns of inhaled fixed-dose combination triple therapy in chronic obstructive pulmonary disease. 卫生技术评价对慢性阻塞性肺疾病吸入式固定剂量联合三联疗法处方模式的影响。
Journal of market access & health policy Pub Date : 2021-06-02 DOI: 10.1080/20016689.2021.1929757
Jennifer Cook, Chloe Bloom, Jen Lewis, Zoe Marjenberg, Jaime Hernando Platz, Sue Langham
{"title":"Impact of health technology assessment on prescribing patterns of inhaled fixed-dose combination triple therapy in chronic obstructive pulmonary disease.","authors":"Jennifer Cook,&nbsp;Chloe Bloom,&nbsp;Jen Lewis,&nbsp;Zoe Marjenberg,&nbsp;Jaime Hernando Platz,&nbsp;Sue Langham","doi":"10.1080/20016689.2021.1929757","DOIUrl":"https://doi.org/10.1080/20016689.2021.1929757","url":null,"abstract":"<p><p><b>Background</b>: Evidence suggests that triple therapy for patients with chronic obstructive pulmonary disease (COPD) is being used in a broader range of patients than recommended by guidelines, which may have health and cost implications. <b>Objective</b>: To explore the relationship between national health technology assessment (HTA) agency appraisals and market penetration of two fixed-dose combination (FDC) triple therapies. <b>Study design</b>: HTAs from Q3 2017 to Q1 2020 from 10 countries were evaluated. <b>Intervention</b>: Glycopyrronium bromide/formoterol fumarate/beclomethasone (Trimbow®) and umeclidinium/vilanterol/fluticasone furoate (Trelegy™ Ellipta®). <b>Main outcome measure</b>: HTA restrictions and prescribing rates (days of therapy). <b>Results</b>: Seven countries (70%) imposed restrictions on use including prescription only for patients stable on free-combination triple therapy or not controlled on dual therapy, requirement of a specialist prescription or therapeutic plan, prescription only for patients with severe COPD, and use as second-line therapy or later. In general, countries that have imposed restrictions on the use of FDC triple therapies have seen a lower than average uptake. <b>Conclusion</b>: Payer guidance on prescribing FDC triple therapy may potentially support more appropriate prescribing in line with clinical guidelines. It is important for payers to consider which restrictions would ensure the most efficient use of scarce resources.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"9 1","pages":"1929757"},"PeriodicalIF":0.0,"publicationDate":"2021-06-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/20016689.2021.1929757","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39091054","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}
引用次数: 1
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