Journal of market access & health policy最新文献

{"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":null,"pages":null},"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":null,"pages":null},"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}

{"title":"The impact of amortization of gene therapies funding on the results and conclusions of CEMs and BIMs.","authors":"Hubert Polek,&nbsp;Justyna Janik,&nbsp;Ewelina Paterak,&nbsp;Monique Dabbous,&nbsp;Michał Pochopień,&nbsp;Mondher Toumi","doi":"10.1080/20016689.2023.2232648","DOIUrl":"https://doi.org/10.1080/20016689.2023.2232648","url":null,"abstract":"<p><strong>Background: </strong>Gene replacement therapy (GRT) is a treatment method used to combat or prevent various diseases. Its high one-off cost constitutes a major obstacle for successful market access. This paper aims to assess and discuss the applicability of amortization in models, such as cost-effectiveness models (CEMs) and budget impact models (BIMs) informing HTA recommendations and reimbursement decisions.</p><p><strong>Methods and findings: </strong>A hypothetical CEA and BIA were considered. The objective was to compare the GRT with and without amortization. A straight-line amortization model was used. The CEM and BIM were considered and assessed based on two set of scenarios: considering different amortization duration or different discounting rate. The impact of amortization against the total cost of gene therapy was assessed for all the scenarios. The cost difference between GRT with and without amortization in relation to its total cost was -$58,855, thus amortization does not have a significant impact on the results and conclusions of the cost-effectiveness analysis. For BIM in the base case, amortization had no impact on the results.</p><p><strong>Conclusion: </strong>Amortization has negligible impact on the results of CEM and total BIM and no impact on the conclusions from the model. One exception is the budget impact in case of an amortization period longer than the time horizon of BIM, where a half of the GRT price is moved beyond the model time horizon. Amortization has a distinguishing effect from an accounting perspective, but it does not have any implication for payers.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":null,"pages":null},"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/5a/b7/ZJMA_11_2232648.PMC10334855.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10185493","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":"Budget and health impact of switching eligible patients with atrial fibrillation to lower- dose dabigatran.","authors":"Tanja Fens,&nbsp;Lisa de Jong,&nbsp;Bregt Kappelhoff,&nbsp;Cornelis Boersma,&nbsp;Maarten J Postma","doi":"10.1080/20016689.2023.2247719","DOIUrl":"https://doi.org/10.1080/20016689.2023.2247719","url":null,"abstract":"<p><p><b>Objectives:</b> To assess the comparative budget and health impact of lower-dose dabigatran versus reduced doses of apixaban and rivaroxaban in atrial fibrillation (AF) patients eligible for a lower-/reduced-dose due to individual patient characteristics in the Netherlands. <b>Methods:</b> A budget impact model was developed in accordance with ISPOR guidelines. A 3-year-time horizon was considered, and analyses were conducted from a Dutch healthcare payer's perspective. The model applies published data to local AF-epidemiology, allowing calculations to estimate clinical events (strokes and haemorrhages) and costs. The analyses were based on real-world outcomes from patients with AF receiving a first direct oral anticoagulant (DOAC) prescription for low-dose dabigatran (110 mg) and a reduced dose of apixaban (2.5 mg) or rivaroxaban (15 mg). Two situations of switching treatments from one to another DOAC were modelled: switching from apixaban to dabigatran and from rivaroxaban to dabigatran. Base case results were given as savings per 100 patient-year, per total Dutch population, and events avoided. A univariate sensitivity analysis was conducted to explore the uncertainty around epidemiological and event costs input data. Scenario analyses were performed to estimate the effect of different market shares and potential price reductions due to future patent expiry for the total real-world population from the Netherlands. <b>Results:</b> The 3-years outcomes of switching patients eligible for a lower-/reduced-dose due to individual patient characteristics from apixaban or rivaroxaban to dabigatran resulted in cost savings estimated at €157 or €72 thousand per 100 patient-years, respectively, or €146 million per total Dutch population. Looking into the clinical events, dabigatran reflected the lowest number of mortalities, ischemic strokes, major bleeding, non-major bleeding, and haemorrhagic stroke compared to apixaban and rivaroxaban. The sensitivity analysis consistently reflected cost savings, with the ischeamic stroke events having the biggest impact. Accounting for the Dutch situation, both scenarios showed total savings ranging from €45 to €229 million over 3 years. <b>Conclusions:</b> Switching eligible AF-patients from reduced-dose apixaban or rivaroxaban to lower-dose dabigatran has the potential to reduce healthcare payer's budget expenditures and provide health gains. Cost savings can potentially be further enhanced by market share adjustments and further price reductions.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":null,"pages":null},"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/e1/da/ZJMA_11_2247719.PMC10478629.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10195018","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 analysis of allogeneic hematopoietic stem cell transplantation in the Bone Marrow Transplant Center of Tunisia.","authors":"Leila Achour,&nbsp;Chema Drira,&nbsp;Mohamed Zied Sboui,&nbsp;Ikram Fazaa,&nbsp;Mohamed Ali Soussi,&nbsp;Senda Hammami,&nbsp;Tarek Ben Othman,&nbsp;Myriam Razgallah Khrouf","doi":"10.1080/20016689.2023.2236851","DOIUrl":"https://doi.org/10.1080/20016689.2023.2236851","url":null,"abstract":"<p><p><b>Introduction:</b> New procedures and diagnostic tests in hematopoietic stem cell transplantation (HSCT) are associated with a significant increase in costs. The last cost estimate of allogeneic HSCT done in Tunisia was in 1996 and concerned only direct medical costs. Therefore, an updated cost analysis is needed. <b>Objective:</b> Analysis of direct costs during the first-year post-allogeneic HSCT in two groups of patients: Bone Marrow Transplant (Allo-BMT) and Peripheral Blood Stem Cell Transplant (Allo-PBSCT) and identification of factors leading to interindividual variations in costs in order to compare these costs with the budget allocated by the payer (CNAM). <b>Methods:</b> Pharmacoeconomic retrospective study, concerning patients who underwent allogeneic HSCT in 2013. Clinical and unit cost data were obtained from medical and administration records. <b>Results:</b>This study showed that the average direct cost of allogeneic HSCT in the population during the first year reached 56 638€. The average cost of Allo-BMT was 63 612€, and Allo-PBSCT was 45 966€ (<i>p</i> > 0.05). The initial hospitalization counted for 88% of total direct cost with an average cost of 41 441€ in Allo-BMT and 24 672€ in Allo-PBSCT (<i>p</i> < 0.05). Direct medical costs represented more than 70% of total direct costs, drugs, and laboratory tests occupied the largest share. Antifungals, antitumors, and antiviral drugs were the most expensive pharmaceutical classes with a mean cost, respectively, of 4 526€; 3 737€ and 3 268€. Some clinical criteria were significantly related to total direct costs like length of aplasia (<i>p</i> < 0.01) and GVHD (<i>p</i> < 0.05). However, the type of blood disease, its risk, length of mucositis, and the treatment protocol have no effect on the costs for all allogeneic patients. <b>Conclusion:</b> Our results showed that the costs of Allo HSCT have exceeded by far the budget allocated by the CNAM to the center, since the 90s to this day. That's why the total reimbursement mechanism should be revised.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":null,"pages":null},"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/cc/54/ZJMA_11_2236851.PMC10416733.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10547139","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The association between body mass index groups and metabolic comorbidities with healthcare and medication costs: a nationwide biobank and registry study in Finland.","authors":"Aino Vesikansa,&nbsp;Juha Mehtälä,&nbsp;Katja Mutanen,&nbsp;Annamari Lundqvist,&nbsp;Tiina Laatikainen,&nbsp;Tero Ylisaukko-Oja,&nbsp;Tero Saukkonen,&nbsp;Kirsi H Pietiläinen","doi":"10.1080/20016689.2023.2166313","DOIUrl":"https://doi.org/10.1080/20016689.2023.2166313","url":null,"abstract":"<p><strong>Background: </strong>The increasing prevalence of obesity imposes a significant cost burden on individuals and societies worldwide.</p><p><strong>Objective: </strong>In this nationally representative study, the association between body mass index (BMI) groups and the number of metabolic comorbidities (MetC) with total direct costs was investigated in the Finnish population.</p><p><strong>Study design, setting, and participants: </strong>The study cohort included 5,587 adults with BMI ≥18.5 kg/m² who participated in the cross-sectional FinHealth 2017 health examination survey conducted by the Finnish Institute for Health and Welfare. Data on healthcare resource utilization (HCRU) and drug purchases were collected from national healthcare and drug registers.</p><p><strong>Main outcome measure: </strong>The primary outcome was total direct costs (costs of primary and secondary HCRU and prescription medications).</p><p><strong>Results: </strong>Class I (BMI 30.0-34.9 kg/m²) and class II - III (BMI ≥35.0 kg/m²) obesity were associated with 43% and 40% higher age- and sex-adjusted direct costs, respectively, compared with normal weight, mainly driven by a steeply increased comorbidity in the higher BMI groups. In all BMI groups combined, individuals with ≥2 MetCs comprised 39% of the total study population and 60% of the total costs.</p><p><strong>Conclusion: </strong>To manage the cost burden of obesity, treatment should be given equal consideration as other chronic diseases, and BMIs ≥30.0 kg/m² should be considered in treatment decisions.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":null,"pages":null},"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/c7/d5/ZJMA_11_2166313.PMC9858397.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10582071","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":"Reply to: <i>comment on increased reliance on physician assistants: an access-quality tradeoff?</i>","authors":"Bhavneet Walia,&nbsp;Harshdeep Banga,&nbsp;David Larsen","doi":"10.1080/20016689.2023.2232192","DOIUrl":"https://doi.org/10.1080/20016689.2023.2232192","url":null,"abstract":"We thank Cawley, Hooker, and Nicholson [1] for engagement with our work [Walia et al. [2]], and herein provide a response. Their comment continues discussion of a vitally important topic in health care: the optimal role and scope of Medical Doctors (MDs) and Physician Assistants (PAs) in healthcare systems. This continued, fact-based discussion has potential to improve healthcare quality for patients at each level of healthcare. Cawley, Hooker, and Nicholson [1] find fault with our understanding of Brock et al. [3], which we cite in our original study. In characterizing Brock et al.’s work, we accepted the abstract, which states, ‘Diagnosis-related malpractice allegations varied by provider type, with physicians having significantly fewer reports (31.9%) than PAs (52.8%) or NPs (40.6%) over the observation period.’ After publication of our article, we were made aware by Cawley, Hooker, and Nicholson that, contrary to this abstract summary, we needed to calculate for ourselves the relative frequency of diagnosis-related malpractice allegations from data presented in Table 4 of Brock et al. It appears that a main implication in the body of Brock et al. opposes the wording of the abstract in finding that malpractice allegations were lower among PAs than physicians. We encourage authors to seek a revision of Brock et al. to clarify their findings. So what does this mean for our findings? The results from Brock et al. are discordant with the results from Yawn and Wollan [4], which we have also cited in favor of our suggestion that increasing PAs will lead to an access-quality tradeoff. Another study by Lozada et al. [5] finds that the average sampled Nurse Practitioner or Physician Assistant overprescribes opioids at more than twice the rate of the average sampled MD, where prescription is, of course, a primary treatment dimension of care that follows diagnosis. When contextualizing the original results of Brock et al. and the comments of Cawley, Hooker, and Nicholson, it is further important to note that some US states (e.g., Arizona) treat surpervising physicians as liable for PA malpractice. The US National Practitioner Data Bank data upon which Brock et al. rely reports medical malpractice payer incidence data and is therefore subject to bias (e.g., whenever a PA commits malpractice but the supervising physician is liable). This potentially substantial source of bias was not noted nor considered in the research design of Brock et al. and, further, was not noted by Cawley, Hooker, and Nicholson. A more appropriate research design for Brock et al. would have been to separate states according to whether supervising physicians are liable (payers) for PA medical malpractice prior to analysis. Further research on malpractice allegations is apparently needed, however. We encourage interested parties, including PA advocacy groups, to fund objective scientists to conduct such research. We wish to emphasize that the presence of a tradeoff between PAs and MD","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":null,"pages":null},"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/b0/1d/ZJMA_11_2232192.PMC10324457.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10564373","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":"Projecting direct medical costs and productivity benefits of improving access to advanced therapy for rheumatoid arthritis: a projection modelling study.","authors":"Chee Yoong Foo,&nbsp;Nurul Azwani Nadia Mansor,&nbsp;Shereen Suyin Ch'ng,&nbsp;Mollyza Mohd Zain","doi":"10.1080/20016689.2023.2173117","DOIUrl":"https://doi.org/10.1080/20016689.2023.2173117","url":null,"abstract":"<p><strong>Introduction: </strong>To ensure the sustainability of the AT access improvement, it is important that health system stakeholders have timely, analyzed information accessible for reference and decision-making support. In this study, we projected the direct costs required as well as the expected direct medical cost-offset and productivity benefits resulting from improving the disease control.</p><p><strong>Methods: </strong>We implemented a deterministic, prevalence-based mathematical model to project the annual cost of rheumatoid arthritis (RA) management within the public healthcare system in Malaysia. We also calculated the annual productivity loss due to uncontrolled RA in monetary value. Using the projection model, we compared the projected costs of the status quo scenario vs. several scenarios of improved advanced therapy (AT) access over a 5-year period.</p><p><strong>Results: </strong>We projected that between 10,765 and 11,024 RA patients in Malaysia over the period of 2020-2024 will need access to AT due to treatment failure with conventional synthetic disease modifying antirheumatic drugs (DMARDs). The projected net total medical cost under the status quo scenario were 163.5 million annually on average (approximately MYR 15,000 per patient per year). Cost related to health service utilization represented the heaviest component, amounting to 71.8% followed by drug cost (24.7%). Under the access improvement scenarios, drug cost constituted a higher proportion of the total medical, ranging from 25.6% to 30.4%. In contrast, the cost of health service utilization shown a reverse pattern (reducing to between 66.3% and 70.1%). Productivity costs were also expected to reduce as AT access improved leading to better outcomes. Treatment shifts to targeted synthetic DMARDs in anticipation of price adjustment appeared to have a cost saving advantage to the health system if all other parameters remain unchanged.</p><p><strong>Discussion: </strong>Improving AT access for RA patients towards the aspirational target appeared to be feasible given the current health budget in Malaysia. Broader socio-economic consequences of productivity and income loss should be included as an important part of the policy consideration. The financial implication of different AT utilization mixes and the anticipated price adjustment will likely result in some cost saving to the health system.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":null,"pages":null},"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/8a/4a/ZJMA_11_2173117.PMC9930832.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10768566","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":"Artificial intelligence and remote patient monitoring in US healthcare market: a literature review.","authors":"Ayushmaan Dubey,&nbsp;Anuj Tiwari","doi":"10.1080/20016689.2023.2205618","DOIUrl":"https://doi.org/10.1080/20016689.2023.2205618","url":null,"abstract":"<p><strong>Background: </strong>Artificial intelligence (AI) enables remote patient monitoring (RPM) which reduces costs by triaging patients to optimize hospitalization and avoid complications. The FDA regulates AI in medical devices and aims to ensure patient safety, effectiveness, and transparent AI solutions.</p><p><strong>Objectives: </strong>Identify and summarize FDA approved RPM devices to provide information for the US medical device industry based on previous approvals and the markets' needs.</p><p><strong>Methods: </strong>We searched publicly available databases on FDA-approved RPM devices. Selection criteria were established to classify a solution as AI. Technical information was analyzed on pre-identified 16 parameters for the qualified solutions.</p><p><strong>Results: </strong>A total of 47 RPM devices were reviewed, among which 12.8% were classified as a De Novo product and the remaining devices fell under the 510(K) FDA category. The cardiovascular (74%) AI RPM solutions dominated the US market, followed by ECG-based arrhythmia detection algorithms (59.4%), and Hemodynamics and Vital Sign monitoring algorithms (21.9%). The trend observed in the FDA rejected devices was their inability to be classified into clinically relevant categories (Criteria 2 and 3).</p><p><strong>Conclusion: </strong>The market needs more innovative RPM solutions under the De Novo category, as there are very few. The transparency is low on the technical aspect of AI algorithms. The market needs AI algorithms that can effectively classify patients rather than merely improve device functionality.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":null,"pages":null},"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/cf/96/ZJMA_11_2205618.PMC10158563.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9485155","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":"Budget impact analysis of anakinra in the treatment of familial Mediterranean fever in Italy.","authors":"A Aiello,&nbsp;E E Mariano,&nbsp;M Prada,&nbsp;L Cioni,&nbsp;C Teruzzi,&nbsp;R Manna","doi":"10.1080/20016689.2023.2176091","DOIUrl":"https://doi.org/10.1080/20016689.2023.2176091","url":null,"abstract":"<p><strong>Introduction: </strong>Familial Mediterranean Fever (FMF) is a hereditary autoinflammatory disease that significantly reduces occupational productivity and quality-of-life in affected patients. Italy has an estimated FMF prevalence of 1 in 60,000 people. While colchicine is the primary treatment for FMF, biologics are administered to intolerant and non-responder patients. Anakinra and canakinumab are the only biologics approved and reimbursed for FMF in Italy. Both medicines have demonstrated efficacy in FMF patients yet differ in treatment costs. This study aimed to perform a budget impact analysis (BIA) following anakinra's reimbursement for FMF treatment, considering pharmaceutical costs from the Italian National Healthcare Service (NHS) perspective.</p><p><strong>Methods: </strong>A 'Reference scenario' (all patients treated with canakinumab) was compared to an 'Alternative scenario', with increased anakinra market shares. The target population was estimated based on the Italian population, epidemiological and market research data. Drugs costs were estimated based on Summary of Product Characteristics and net ex-factory prices. Sensitivity analyses were implemented to test results' robustness.</p><p><strong>Results: </strong>The base case analysis showed an overall cumulative expenditure of €30,586,628 for 'Reference scenario' and € 16,465,548 for 'Alternative scenario'. A cumulative savings of €14,121,080 (46.2%) was calculated over 3 years as a result of the reimbursement and increasing uptake of anakinra. The sensitivity analyses, even considering a discount of 50% for canakinumab, confirmed the base case results.</p><p><strong>Conclusions: </strong>Anakinra's introduction, in FMF treatment, provides a financially sustainable option for Italian patients, with savings increasing according to greater use of anakinra.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":null,"pages":null},"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/ff/f4/ZJMA_11_2176091.PMC9930828.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10768568","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}