Journal of market access & health policy最新文献

{"title":"Commentary on \"Hydroxychloroquine and azithromycin as a treatment of COVID-19: results of an open label non-randomized clinical trial\" by Gautret et al.","authors":"Mondher Toumi, Samuel Aballea","doi":"10.1080/20016689.2020.1758390","DOIUrl":"10.1080/20016689.2020.1758390","url":null,"abstract":"<p><p>The results of a clinical trial comparing hydroxychloroquine with or without azithromycin to the standard of care for the treatment of COVID-19 were recently published by Philippe Gautret et al. This study provides outstanding results for the combination of hydroxychloroquine and azithromycin over the standard of care, but the evidence was deemed insufficiently robust to warrant a public health decision to widen the use of hydroxychloroquine for the treatment of COVID-19. We provide a scientific critical review of the Gautret et al. publication, put the results in the context of the current knowledge, provide an evaluation of the validity of the results (from a methodologic perspective), and discuss public health implications. The study has a number of limitations, including small sample size, lack of comparability between patients in active treatment and control arms, lack of blinding, use of interim analyses without controlling for the risk of type 1 error, use of analysis in the per-protocol population instead of the intention-to-treat population, and inconsistencies between the study protocol and article. However, none of these observations is of a nature to reverse the conclusions. The study brings useful knowledge consistent with available evidence and clinical practice from China and South Korea, which could have prompted quicker policy decision-making.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"8 1","pages":"1758390"},"PeriodicalIF":0.0,"publicationDate":"2020-05-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/73/5e/ZJMA_8_1758390.PMC7269042.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38042947","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":"Cost-consequence analysis of ¹⁸F-fluciclovine for the staging of recurrent prostate cancer.","authors":"Ivar S Jensen,&nbsp;Joanne Hathway,&nbsp;Philip Cyr,&nbsp;David Gauden,&nbsp;Peter Gardiner","doi":"10.1080/20016689.2020.1749362","DOIUrl":"https://doi.org/10.1080/20016689.2020.1749362","url":null,"abstract":"<p><p><b>Background</b>: Current detection methodologies are often unable to identify the location and extent of recurrent prostate cancer (PCa) leading potentially to 'futile' local therapies in the presence of metastatic disease. The use of ¹⁸ F-fluciclovine PET/CT may lead to better patient management. <b>Objective</b>: The aim of this study was to quantify the economic impact and cost-consequence of using ¹⁸ F-fluciclovine PET/CT in PCa recurrence. <b>Study design</b>: A decision analytic model based on recurrent PCa imaging guidelines. <b>Setting</b>: US hospital. <b>Participants</b>: PCa patients experiencing biochemical recurrence. <b>Intervention</b>: ¹⁸ F-fluciclovine PET/CT was compared to conventional imaging. <b>Main outcome measure</b>: Budget impact, correct diagnoses, futile treatments, and cost-consequence (cost per correct diagnosis) <b>Results</b>: For a hypothetical hospital serving 500,000 individuals, the model showed the use of ¹⁸ F-fluciclovine reduced 'futile' therapies by 19.2%. Re-imaging costs were reduced by 40.2% ($8.2 million); however, when assuming diagnostic and staging costs only, the total costs increased from $31.2 to $34.6 million (10.9%), driven by ¹⁸ F-fluciclovine imaging agent and procedure costs. The cost per 'correct' diagnosis declined $30,673 (46.8%). When including subsequent 5-year patient management, the cost per 'correct' diagnosis declined $410,206 (49.2%). <b>Conclusion</b>: ¹⁸ F-fluciclovine PET/CT imaging may improve the clinical management of men with recurrent PCa with minimal increase in healthcare spending.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"8 1","pages":"1749362"},"PeriodicalIF":0.0,"publicationDate":"2020-04-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/20016689.2020.1749362","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"37878468","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":"Clinical efficacy assessment in severe vernal keratoconjunctivitis: preliminary validation of a new penalties-adjusted corneal fluorescein staining score.","authors":"Andrea Leonardi,&nbsp;Maëva Dupuis-Deniaud,&nbsp;Dominique Bremond-Gignac","doi":"10.1080/20016689.2020.1748492","DOIUrl":"https://doi.org/10.1080/20016689.2020.1748492","url":null,"abstract":"<p><p><b>Introduction and objective</b>: Vernal keratoconjunctivitis (VKC) is a rare allergic eye condition that occurs in children and is characterised by a combination of debilitating symptoms. Repeated use of topical corticosteroid rescue therapy is often necessary in severe forms. This study aims to assess the validity of a new composite endpoint: the penalties-adjusted corneal staining score (PACS-S) proposed as primary endpoint in VEKTIS trial evaluating the efficacy of a new corticosteroid-sparing treatment, VERKAZIA® (ciclosporin 1 mg/ml eye drops), in severe VKC patients. <b>Methodology</b>: This research comprised a systematic literature review to identify efficacy endpoints being proposed in clinical trials for pediatric patients with severe VKC, followed by a remote expert advisory board assessing the validity of the PACS-S. <b>Results</b>: While no agreed or validated endpoint for assessing efficacy in VKC was identified when VEKTIS trial started, the experts' board acknowledged a high face validity of PACS-S as a subjective integrated measure matching the current clinical practice. A fair external validity was considered with regards to VEKTIS trial secondary endpoints. <b>Conclusion</b>: PACS-S appears to be a reliable, valid and clinically meaningful primary endpoint that allows significant improvement over existing endpoints in severe VKC trials. Additional research is needed to validate this endpoint.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"8 1","pages":"1748492"},"PeriodicalIF":0.0,"publicationDate":"2020-04-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/20016689.2020.1748492","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"37878467","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":"Economic evaluation of biosimilars for reimbursement purposes - what, when, how?","authors":"Evelien Moorkens,&nbsp;Hannah Broux,&nbsp;Isabelle Huys,&nbsp;Arnold G Vulto,&nbsp;Steven Simoens","doi":"10.1080/20016689.2020.1739509","DOIUrl":"https://doi.org/10.1080/20016689.2020.1739509","url":null,"abstract":"<p><p><b>Background</b>: Limited previous research and guidelines on the design of economic evaluation for biosimilars have led to unresolved methodological questions on how to assess biosimilars. <b>Objectives</b>: We want to raise awareness of and explore methodological issues for the economic evaluation of biosimilars. <b>Methods</b>: We relied on a literature review, exploratory interviews, and our experiences. <b>Results and Conclusions</b>: In the majority of cases in which reimbursement for a biosimilar is sought, it will not be necessary to conduct an economic evaluation, given that the reference product is already reimbursed and standard of care. If the latter is not the case, a full economic evaluation of the biosimilar versus standard of care is needed. This might also be needed in the case of differences in administration form or adherence (for example, due to a nocebo effect) and to take into account value-added services. The entry of biosimilars and of next-generation biological products should trigger a re-assessment of the entire product class. HTA bodies and reimbursement agencies should provide clear guidance on how to assess the value of a biosimilar in each of these circumstances.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"8 1","pages":"1739509"},"PeriodicalIF":0.0,"publicationDate":"2020-03-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/20016689.2020.1739509","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"37828923","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":"Specialist physician perspectives on non-medical switching of prescription medications.","authors":"Olivia S Costa,&nbsp;Tabassum Salam,&nbsp;Amy Duhig,&nbsp;Aarti A Patel,&nbsp;Ann Cameron,&nbsp;Jennifer Voelker,&nbsp;Brahim Bookhart,&nbsp;Craig I Coleman","doi":"10.1080/20016689.2020.1738637","DOIUrl":"https://doi.org/10.1080/20016689.2020.1738637","url":null,"abstract":"<p><p><b>Introduction</b>: A non-medical switch is a change to a patient's medication regimen for reasons other than lack of clinical response, side-effects or poor adherence. Specialist physicians treat complex patients who may be vulnerable to non-medical switching. <b>Objectives</b>: To evaluate specialist physicians' perceptions regarding the frequency of non-medical switch requests, and the impact on their patients' outcomes and healthcare utilization. <b>Methods</b>: An online survey of randomly sampled physicians spending ≥10% of time providing patient care and having received ≥1 non-medical switch request during the prior 12-months. <b>Results</b>: Among 404 specialist physicians surveyed, non-medical switch requests were reported as very frequent or frequent by 35.0% of oncologists (for injectable cancer agents) and up to 80.3% of endocrinologists (for injectable anti-hyperglycemics). Respondents reported decreased medication effectiveness (25.0% of oncologists to 75.0% of dermatologists) and increased side-effects (32.5% of oncologists to 66.7% of psychiatrists). Most specialists reported very frequent or frequent increases in non-office visits (52.5% of oncologists to 75.3% of endocrinologists) and calls with pharmacies (57.5% of oncologists to 80.5% of rheumatologists) due to non-medical switching. <b>Conclusions</b>: Receipt of non-medical switching requests were common among specialist physicians. Non-medical switching may lead to negative effects on patient care and require increased healthcare utilization.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"8 1","pages":"1738637"},"PeriodicalIF":0.0,"publicationDate":"2020-03-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/20016689.2020.1738637","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"37828922","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":"Understanding the global measurement of willingness to pay in health.","authors":"Jean A McDougall,&nbsp;Wesley E Furnback,&nbsp;Bruce C M Wang,&nbsp;Jörg Mahlich","doi":"10.1080/20016689.2020.1717030","DOIUrl":"https://doi.org/10.1080/20016689.2020.1717030","url":null,"abstract":"<p><p><b>Objective</b>: To understand the different methodologies used to elicit willingness to pay for health and the value of a statistical life year through surveys. <b>Methodology</b>: A systematic review of the literature was undertaken to identify studies using surveys to estimate either willingness to pay for health or the value of a statistical life year. Each study was reviewed and the study setting, sample size, sample description, survey administration (online or face to face), survey methodology, and results were extracted. The results of the studies were then compared to any published national guidelines of cost-effectiveness thresholds to determine their accuracy. <b>Results</b>: Eighteen studies were included in the review with 15 classified as willingness to pay and 3 value of a statistical life. The included studies covered Asia (n = 6), Europe (n = 4), the Middle East (n = 1), and North America (n = 5), with one study taking a global perspective. There were substantial differences in both the methodologies and the estimates of both willingness to pay and value of a statistical life between the different studies. <b>Conclusion</b>: Different methods used to elicit willingness to pay and the value of a statistical life year resulted in a wide range of estimates.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"8 1","pages":"1717030"},"PeriodicalIF":0.0,"publicationDate":"2020-02-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/20016689.2020.1717030","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"37726675","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":"Family caregiving in dementia and its impact on quality of life and economic burden in Japan-web based survey.","authors":"Ataru Igarashi,&nbsp;Ayako Fukuda,&nbsp;Lida Teng,&nbsp;Fan-Fan Ma,&nbsp;Julie Dorey,&nbsp;Yoshie Onishi","doi":"10.1080/20016689.2020.1720068","DOIUrl":"https://doi.org/10.1080/20016689.2020.1720068","url":null,"abstract":"<p><p><b>Background</b>: Dementia has become a growing health-care problem in the rapidly ageing Japanese population. This study assesses the impact of dementia on quality of life, economic burden, and productivity loss. <b>Objective</b>: The objective of this study was to assess the impact of dementia on the Quality of Life (QoL), economic burden, and productivity loss among families living with dementia. <b>Methods</b>: An online survey was conducted among families who lived with relatives with dementia. Demographic data and information about health condition and costs of long-term care and treatment were collected. Participants were asked to answer the EuroQol (EQ-5D-5L) questionnaire, Zarit Burden Interview (ZARIT-8), and Work Productivity and Activity Impairment Questionnaire (WPAI). Multivariate analyses were conducted to assess factors associated with burden by families living with dementia. <b>Results</b>: Six hundred and thirty-five participants completed the survey. Of these participants, 50.5% were primary caregivers. Overall, 78.7% of dementia patients suffered from Alzheimer, and 43.9% needed long-term care. Compared to non-primary caregivers, primary caregivers had lower health utility scores (0.896 vs 0.873; p = 0.02), higher burden of caregiving (ZARIT-8: 21.1 vs 24.5; p < 0.0001), and higher overall work impairment (40.2% vs 20.8%; p < 0.0001), absenteeism (15.3% vs 5.7%; p < 0.0001), and presenteeism-related impairment (33.2% vs 17.3%; p < 0.0001). <b>Conclusion</b>: Families living with dementia caring for a person with dementia experience increased burden. Health policies related to dementia need to be considered not only for patients, but also for their families living with dementia to improve their QoL.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"8 1","pages":"1720068"},"PeriodicalIF":0.0,"publicationDate":"2020-02-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/20016689.2020.1720068","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"37702682","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 to assess for the full economic value of vaccines? From past to present, drawing lessons for the future.","authors":"Baudouin Standaert,&nbsp;Christophe Sauboin,&nbsp;Rodrigo DeAntonio,&nbsp;Alen Marijam,&nbsp;Jorge Gomez,&nbsp;Lijoy Varghese,&nbsp;Sharon Zhang","doi":"10.1080/20016689.2020.1719588","DOIUrl":"https://doi.org/10.1080/20016689.2020.1719588","url":null,"abstract":"<p><p><b>Background:</b>Cost-effectiveness analysis (CEA) is the economic analysis method most commonly applied today in the context of replacing one treatment with a new one in a developed healthcare system to improve efficiency. CEA is often requested by local healthcare decision-makers to grant reimbursement. New preventative interventions, such as new vaccines, may however have much wider benefits inside and outside healthcare, when compared with treatment. These additional benefits include externalities on indirect clinical impact, reallocation of specific healthcare resources, improved quality of care, better productivity, better disease control, better fiscal revenues, and others. But these effects are sometimes difficult to integrate into a meaningful CEA result. They may appear as specific benefits for specific stakeholders, other than the stakeholders in healthcare. <b>Objective:</b> Based on a historical view about the application of economic assessments for vaccines our objective has been to make the inventory of who was/is interested in knowing the economic value of vaccines, in what those different stakeholders are likely to see the benefit from their perspective and how  were/are we able to measure those benefits and to report them well. <b>Results:</b> The historical view disclosed a limited interest in the economic assessment of vaccines at start, more than 50 years ago, that was comparable to the assessment of looking for more efficiency in new industries through optimization exercises. Today, we are exposed to a very rich panoply of different stakeholders (n= 16). They have their specific interest in many different facets of the vaccine benefit of which some are well known in the conventional economic analysis (n=9), but most outcomes are hidden and not enough evaluated and reported (n=26). Meanwhile we discovered that many different methods of evaluation have been explored to facilitate the measurement and reporting of the benefits (n=18). <b>Conclusion:</b> Our recommendation for future economic evaluations of new vaccines is therefore to find the right combination among the three entities of stakeholder type selection, outcome measure of interest for each stakeholder, and the right method to apply. We present at the end examples that illustrate how successful this approach can be.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"8 1","pages":"1719588"},"PeriodicalIF":0.0,"publicationDate":"2020-01-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/20016689.2020.1719588","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"37702681","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":"Outcomes-based reimbursement for gene therapies in practice: the experience of recently launched CAR-T cell therapies in major European countries.","authors":"Jesper Jørgensen, Eve Hanna, Panos Kefalas","doi":"10.1080/20016689.2020.1715536","DOIUrl":"10.1080/20016689.2020.1715536","url":null,"abstract":"<p><p><b>Background</b>: The experience of Kymriah® and Yescarta® provides real-world examples of how health-care systems approach and manage the reimbursement of one-off, high-cost, cell, and gene therapies, and the decision uncertainty and affordability challenges they present. <b>Objective</b>: To provide an overview of the reimbursement schemes used for Kymriah® and Yescarta® in France, Germany, Italy, Spain, and the UK (EU5) as per the final quarter of 2019; to identify challenges and derive learnings for future product launches. Methodology: Secondary research, complemented by primary research with key market access stakeholders. <b>Findings</b>: Kymriah® and Yescarta® have relatively uniform list prices across the EU5, and are reimbursed according to their marketing authorisations. In France and the UK, reimbursement is on the condition of collecting additional data (at the cohort level) and subject to future reassessments; elsewhere, rebates (Germany) or staged payments (Italy and Spain) are linked to individual patient outcomes. <b>Conclusions</b>: The experience of Kymriah® and Yescarta® shows an increased appetite for outcomes-based reimbursement (OBR) in the EU5, with notably novel approaches applied in Italy and Spain (outcomes-based staged payments). Thus, real-world evidence (RWE) has become an increasingly powerful lever for demonstrating the value of health benefits in the clinical setting.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"8 1","pages":"1715536"},"PeriodicalIF":0.0,"publicationDate":"2020-01-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/97/00/ZJMA_8_1715536.PMC7006635.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"37664905","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":"Tendering and biosimilars: what role for value-added services?","authors":"Steven Simoens,&nbsp;Raymond Cheung","doi":"10.1080/20016689.2019.1705120","DOIUrl":"https://doi.org/10.1080/20016689.2019.1705120","url":null,"abstract":"<p><p><b>Background</b>: Access to biologic medicines (including biosimilars) across Europe is largely governed by a process of tendering conducted by health authorities. Over-reliance on treatment costs in awarding tenders has the potential to hinder competition and undermine the long-term sustainability of biosimilars. <b>Objective</b>: To assess the extent and impact of consideration of 'value-added services' (VAS) in tendering for biosimilars, we conducted a narrative review of published literature. <b>Results</b>: Findings from survey-based publications indicated that tendering practices for biosimilars are widely used, with cost being the main determinant of success and little detail being available on other criteria where these apply. Criteria (of therapeutic and technical interest) beyond price were included in one tendering specification for infliximab (originator and biosimilars), while a separate tender for the same product included VAS in the form of therapeutic drug monitoring, measurement of antibodies and calprotectin. <b>Conclusions</b>: Published evidence concerning inclusion of VAS in tendering for biosimilars is lacking. Development and implementation of standardized criteria and methods of assessment for tenders may avoid manufacturers facing segmented markets, encourage competition and the longer-term sustainability of biosimilars, and realize the healthcare system and patient benefits these treatments can bring.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"8 1","pages":"1705120"},"PeriodicalIF":0.0,"publicationDate":"2019-12-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/20016689.2019.1705120","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"37596314","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}