Journal of market access & health policy最新文献

{"title":"Methods for Indirect Treatment Comparison: Results from a Systematic Literature Review","authors":"Bérengère Macabeo, Arthur Quenéchdu, Samuel Aballéa, Clément François, Laurent Boyer, Philippe Laramée","doi":"10.3390/jmahp12020006","DOIUrl":"https://doi.org/10.3390/jmahp12020006","url":null,"abstract":"Introduction: Health technology assessment (HTA) agencies express a clear preference for randomized controlled trials when assessing the comparative efficacy of two or more treatments. However, an indirect treatment comparison (ITC) is often necessary where a direct comparison is unavailable or, in some cases, not possible. Numerous ITC techniques are described in the literature. A systematic literature review (SLR) was conducted to identify all the relevant literature on existing ITC techniques, provide a comprehensive description of each technique and evaluate their strengths and limitations from an HTA perspective in order to develop guidance on the most appropriate method to use in different scenarios. Methods: Electronic database searches of Embase and PubMed, as well as grey literature searches, were conducted on 15 November 2021. Eligible articles were peer-reviewed papers that specifically described the methods used for different ITC techniques and were written in English. The review was performed in accordance with the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines. Results: A total of 73 articles were included in the SLR, reporting on seven different ITC techniques. All reported techniques were forms of adjusted ITC. Network meta-analysis (NMA) was the most frequently described technique (in 79.5% of the included articles), followed by matching-adjusted indirect comparison (MAIC) (30.1%), network meta-regression (24.7%), the Bucher method (23.3%), simulated treatment comparison (STC) (21.9%), propensity score matching (4.1%) and inverse probability of treatment weighting (4.1%). The appropriate choice of ITC technique is critical and should be based on the feasibility of a connected network, the evidence of heterogeneity between and within studies, the overall number of relevant studies and the availability of individual patient-level data (IPD). MAIC and STC were found to be common techniques in the case of single-arm studies, which are increasingly being conducted in oncology and rare diseases, whilst the Bucher method and NMA provide suitable options where no IPD is available. Conclusion: ITCs can provide alternative evidence where direct comparative evidence may be missing. ITCs are currently considered by HTA agencies on a case-by-case basis; however, their acceptability remains low. Clearer international consensus and guidance on the methods to use for different ITC techniques is needed to improve the quality of ITCs submitted to HTA agencies. ITC techniques continue to evolve quickly, and more efficient techniques may become available in the future.","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"8 1","pages":"58 - 80"},"PeriodicalIF":0.0,"publicationDate":"2024-04-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140698688","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost-Effectiveness Analysis of Innovative Therapies for Patients with Non-Alcoholic Fatty Liver Disease","authors":"Michał Pochopień, J. Dziedzic, S. Aballéa, Emilie Clay, Iwona Zerda, Mondher Toumi, B. Borissov","doi":"10.3390/jmahp12020005","DOIUrl":"https://doi.org/10.3390/jmahp12020005","url":null,"abstract":"Objective: Currently there are no disease-specific approved therapies for non-alcoholic fatty liver (NAFL) and non-alcoholic steatohepatitis (NASH); however, several treatments are under development. This study aimed to estimate the cost-effectiveness of hypothetical innovative therapies compared with lifestyle intervention alone and combined with pioglitazone, and assess the health economic consequences of their future availability for patients. Methods: A Markov cohort model was developed, considering fourteen disease health states and one absorbing state representing death. Transition probabilities, costs, utilities, and treatment efficacy were based on published data and assumptions. Four treatment strategies were considered, including two existing therapies (lifestyle intervention, small molecule treatment) and two hypothetical interventions (biological and curative therapy). The analysis was performed from the US third-party payer perspective. Results: The curative treatment with the assumed efficacy of 70% of patients cured and assumed price of $500,000 was the only cost-effective option. Although it incurred higher costs (a difference of $188,771 vs. lifestyle intervention and $197,702 vs. small molecule), it generated more QALYs (a difference of 1.58 and 1.38 QALYs, respectively), resulting in an ICER below the willingness-to-pay threshold of $150,000 per QALY. The sensitivity analyses showed that the results were robust to variations in model parameters. Conclusions: This study highlighted the potential benefits of therapies aimed at curing a disease rather than stopping its progression. Nonetheless, each of the analyzed therapies could be cost-effective compared with lifestyle intervention at a relatively high price.","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"95 4","pages":"35 - 57"},"PeriodicalIF":0.0,"publicationDate":"2024-04-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140751664","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Publisher's Note: A New Chapter for the <i>Journal of Market Access and Health Policy</i> (<i>JMAHP</i>)-Continued Publication by MDPI.","authors":"Clàudia Aunós","doi":"10.3390/jmahp12010001","DOIUrl":"10.3390/jmahp12010001","url":null,"abstract":"","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"12 1","pages":"1"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10832262/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140041095","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":"Discounting health gain: a different view.","authors":"Baudouin Standaert, Olivier Ethgen","doi":"10.1080/20016689.2023.2275350","DOIUrl":"10.1080/20016689.2023.2275350","url":null,"abstract":"<p><p>At least since the Age of Enlightenment, good health has been a tenet for society. Healthy societies could learn better, work harder, improve their wealth, and live longer. Today societies focus on life expectancy, as we value long and healthy lives. As illustrated by the provision of COVID-19 vaccines first for the elderly, societies value life-saving actions. Paradoxically, health economic assessments conventionally devalue long-lasting health through the practice of discounting health benefits along with costs. However, health, with its intrinsic and instrumental characteristics, is not synonymous with money cash, a tradeable asset that devalues with time. If improving healthy life expectancy is a societal ambition, it seems counter-intuitive to value future health less as a result of an artificial mathematical construct when evaluating economically new medical interventions. In this paper, we investigate the application of discounting health in healthcare and consider paradoxical findings, especially in relation to disease prevention with vaccination. We argue that there is no economically sustainable argument to discount health gains, except for the benefit of the payer with a goal of spending less on life-saving products. If that is the objective for discounting health, there are other means to achieve the same goal in a more transparent and simpler way. From the long-term perspective of healthcare development, not discounting health gains would encourage research that values long-term effects. This in turn has the potential to benefit the investor, the payer, and the patient/consumer, improving the situation from multiple perspectives.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"11 1","pages":"2275350"},"PeriodicalIF":0.0,"publicationDate":"2023-11-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10627044/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"71489702","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":"Observational study on the therapeutic management and economic burden of adult patients with moderate to severe plaque psoriasis in France – the POP study","authors":"A. P. Villani, N. Quiles Tsimaratos, A. Crochard, A. Gherardi, A. Panes, A. Schmidt, M. Hueber Kollen, I. Borget","doi":"10.1080/20016689.2023.2270293","DOIUrl":"https://doi.org/10.1080/20016689.2023.2270293","url":null,"abstract":"Background: Data on the therapeutic management and healthcare cost of moderate to severe psoriasis in France are scarce.","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"338 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135320862","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Inappropriate dosing of direct oral anticoagulants: findings from a clinical vignette study and physician survey.","authors":"Ahmet Fuat, Emmanuel Ako, David Hargroves, Douglas Holden, Amrit Caleyachetty, Matthew Carter, James Harris, Carol Roberts, Nnanyelu Nzeakor, Burcu Vardar, Helen Williams","doi":"10.1080/20016689.2023.2267327","DOIUrl":"10.1080/20016689.2023.2267327","url":null,"abstract":"<p><strong>Objective: </strong>Direct oral anticoagulants (DOACs) are first-line therapy for stroke prevention for 1.4 million atrial fibrillation (AF) patients in the UK. However, the rates of DOAC dosing below evidence-based recommendations are estimated between 9% and 22%. This study explores specific patient and physician factors associated with prescribing inappropriate DOAC underdoses.</p><p><strong>Methods: </strong>DOAC-prescribing physicians within the UK completed both a clinical vignette survey, which contained 12 hypothetical patient profiles designed to replicate DOAC prescribing scenarios, and a physician survey to capture sociodemographic, clinical experience, and prescriber-related beliefs and motivations related to DOAC prescribing. Eight patient factors based on a literature search and an expert consultation process were varied within the vignettes. Associations between the prescribers' dosing choices and patient factors were explored via multilevel logistic regression. The analysis is focused on the most frequently selected DOACs, apixaban and rivaroxaban, both of which have different dosing guidelines.</p><p><strong>Results: </strong>In all, 336 prescribers (69% male; 233/336) completed the survey, mostly general physicians (GPs) (45%) or cardiology specialists (36%) with a mean of 17.9 years' experience. Most prescribers (73%; 244/336) inappropriately underdosed at least once; rates between GPs and specialists were nearly identical. Patient factors most strongly associated with apixaban inappropriate underdosing included a history of major bleeding and falls. For rivaroxaban, these were major bleeding and severe frailty. Only 32% (106/335) of prescribers reported DOAC dosing guidelines as the sole influence on their prescribing behaviour. Among prescribers who did not inappropriately underdose, greater prescribing confidence was aligned to increased perception of inappropriate underdose risk.</p><p><strong>Conclusions: </strong>Overall, patient factors such as major bleeding and severe frailty were found to be associated with inappropriate underdosing of apixaban and rivaroxaban. Furthermore, prescribers who were more confident in DOAC prescribing, and were more worried about the risk of stroke, were significantly less likely to inappropriately underdose. These findings suggest that all prescribers, regardless of speciality, may benefit from education and training to raise awareness of the risks associated with inappropriate DOAC underdosing.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"11 1","pages":"2267327"},"PeriodicalIF":0.0,"publicationDate":"2023-10-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10634268/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"89720980","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":"Women and children’s real-world economic and drug indicators from 2015 to 2019","authors":"Jun Zou, Che Zhang, Guohua Jia, Wei Lu","doi":"10.1080/20016689.2023.2270297","DOIUrl":"https://doi.org/10.1080/20016689.2023.2270297","url":null,"abstract":"ABSTRACT Objectives: To obtain real-world data on rational drug use, pharmacoeconomic regarding women’s and children’s health and the benefits of Hainan free trade port (HFTP) health policies, we retrospectively investigated drug indicators, prescribing trend, and economic data. Method: We retrospectively gathered the data from the database of the hospital information system and the quality indicators of pharmacy; we compared the monthly indicators from 2015 to 2019. Results: In 2017, the HFTP maternal mortality ratio (MMR) was 24.46. In 2019, the HFTP infant mortality rate (‰) was 4.15, and the under-five mortality rate (‰) was 6.19. A total of 1,922,798 prescriptions included in the analysis, the defined daily dose of 2015–2019 ranged from 46.59 to 32.34. In 2019, the proportions of antibiotics prescribed in outpatient, emergency, and inpatient care were 14.19%, 16.68%, and 46.26%, respectively. The injection prescription percentage ranged from 13.08% to 8.08%. The proportion of medicine income to total hospital income of 2015–2019 ranged from 26.66% to 25.31%. Conclusion: According to the analysis of women’s and children’s real-world drug data, economic investment and strict quality control of antimicrobial stewardship programs can lead to the rational use of drugs.","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"2 3","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-10-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"134910413","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Ivabradine in patients with heart failure: a systematic literature review.","authors":"Zeba M Khan, Jean Baptiste Briere, Elzbieta Olewinska, Fatma Khrouf, Mateusz Nikodem","doi":"10.1080/20016689.2023.2262073","DOIUrl":"10.1080/20016689.2023.2262073","url":null,"abstract":"<p><p><b>Background:</b> Heart failure is a chronic disease linked with significant morbidity and mortality, and uncontrolled resting heart rate is a risk factor for adverse outcomes. This systematic literature review aimed to assess the efficacy, safety, and patient-reported outcomes (PROs) of ivabradine in patients with heart failure (HF) with reduced ejection fraction (HFrEF) in randomized controlled trials (RCTs) and observational studies. <b>Methods:</b> We searched electronic databases from their inception to July 2021 to include studies that reported on efficacy, safety, or PROs of ivabradine in patients with HFrEF. <b>Results:</b> Of 1947 records screened, 51 RCTs and 6 observational studies were identified. Ivabradine on top of background therapy demonstrated a significant reduction in composite outcomes including hospitalization for HF or cardiovascular death. In addition, observational studies suggested that ivabradine was associated with a significant reduction in mortality. Across all studies, ivabradine use on top of background therapy was associated with greater reductions in heart rate, improved EF, and improved health-related quality of life (QoL) and comparable risk of total adverse events compared to those treated with background therapy alone. <b>Conclusions:</b> Ivabradine on top of background therapy is beneficial for heart rate, hospitalization risk for HF, mortality, EF, and patients' QoL. Moreover, these benefits were achieved with no significant increase in the overall risk of total adverse events.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"11 1","pages":"2262073"},"PeriodicalIF":0.0,"publicationDate":"2023-10-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/ae/f7/ZJMA_11_2262073.PMC10552613.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41179739","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":"Rule of Prevention: a potential framework to evaluate preventive interventions for rare diseases.","authors":"Eddie Gibson, Daniel A Ollendorf, Steven Simoens, David E Bloom, Federico Martinón-Torres, David Salisbury, Johan Louis Severens, Mondher Toumi, Daniel Molnar, Kinga Meszaros, Woo-Yun Sohn, Najida Begum","doi":"10.1080/20016689.2023.2239557","DOIUrl":"10.1080/20016689.2023.2239557","url":null,"abstract":"<p><p><b>Background:</b> The benefits of preventive interventions lack comprehensive evaluation in standard health technology assessments (HTA), particularly for rare and transmissible diseases. <b>Objective:</b> To identify possible considerations for future HTA using analogies between the treatment and prevention of rare diseases. <b>Study design:</b> An Expert panel meeting assessed whether one HTA assessment framework can be applied to assess both rare disease treatments and preventive interventions. Experts also evaluated the range of value elements currently included in HTAs and their applicability to rare, transmissible, and/or preventable diseases. <b>Results:</b> A broad range of value should be considered when assessing rare, transmissible disease prevention. Although standard HTA can be applied to transmissible diseases, the risk of local outbreaks and the need for large-scale prevention programs suggest a modified assessment framework, capable of incorporating prevention-specific value elements in HTAs. A 'Rule of Prevention' framework was proposed to allow broader value considerations anchored to severity, equity, and prevention benefits in decision-making for preventive interventions for rare transmissible diseases. <b>Conclusion:</b> The proposed prevention framework introduces an explicit initial approach to consistently assess rare transmissible diseases, and to incorporate the broader value of preventive interventions compared with treatment.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"11 1","pages":"2239557"},"PeriodicalIF":0.0,"publicationDate":"2023-08-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/4c/06/ZJMA_11_2239557.PMC10424616.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10250976","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":"Rapid literature review: definition and methodology.","authors":"Beata Smela,&nbsp;Mondher Toumi,&nbsp;Karolina Świerk,&nbsp;Clement Francois,&nbsp;Małgorzata Biernikiewicz,&nbsp;Emilie Clay,&nbsp;Laurent Boyer","doi":"10.1080/20016689.2023.2241234","DOIUrl":"https://doi.org/10.1080/20016689.2023.2241234","url":null,"abstract":"<p><p><b>Introduction:</b> A rapid literature review (RLR) is an alternative to systematic literature review (SLR) that can speed up the analysis of newly published data. The objective was to identify and summarize available information regarding different approaches to defining RLR and the methodology applied to the conduct of such reviews. <b>Methods:</b> The Medline and EMBASE databases, as well as the grey literature, were searched using the set of keywords and their combination related to the targeted and rapid review, as well as design, approach, and methodology. Of the 3,898 records retrieved, 12 articles were included. <b>Results:</b> Specific definition of RLRs has only been developed in 2021. In terms of methodology, the RLR should be completed within shorter timeframes using simplified procedures in comparison to SLRs, while maintaining a similar level of transparency and minimizing bias. Inherent components of the RLR process should be a clear research question, search protocol, simplified process of study selection, data extraction, and quality assurance. <b>Conclusions:</b> There is a lack of consensus on the formal definition of the RLR and the best approaches to perform it. The evidence-based supporting methods are evolving, and more work is needed to define the most robust approaches.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"11 1","pages":"2241234"},"PeriodicalIF":0.0,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/e6/58/ZJMA_11_2241234.PMC10392303.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10196195","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}