Journal of market access & health policy最新文献

Objectives: 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. Methods: 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. Results: 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. Conclusions: 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.

Introduction: 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. Objective: 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). Methods: Pharmacoeconomic retrospective study, concerning patients who underwent allogeneic HSCT in 2013. Clinical and unit cost data were obtained from medical and administration records. Results: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€ (p > 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 (p < 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 (p < 0.01) and GVHD (p < 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. Conclusion: 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.

Background: The increasing prevalence of obesity imposes a significant cost burden on individuals and societies worldwide.

Objective: 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.

Study design, setting, and participants: 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.

Main outcome measure: The primary outcome was total direct costs (costs of primary and secondary HCRU and prescription medications).

Results: 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.

Conclusion: 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.

Introduction: 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.

Methods: 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.

Results: 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.

Discussion: 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.

Introduction: 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.

Methods: 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.

Results: 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.

Conclusions: Anakinra's introduction, in FMF treatment, provides a financially sustainable option for Italian patients, with savings increasing according to greater use of anakinra.

Background: 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.

Objectives: Identify and summarize FDA approved RPM devices to provide information for the US medical device industry based on previous approvals and the markets' needs.

Methods: 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.

Results: 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).

Conclusion: 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.

Background: Like many developing countries, Colombia faces difficulties in financing health-care services as well as programs for health promotion and health education and there is evidence that its health-care system is underperforming. Objective: To provide evidence-based estimates of potential funding levels and assess the strengths, weaknesses, and viability of innovative funding mechanisms with a focus on treating rare diseases in Colombia. Methods: The strategy involved evidence-based projections of potential funding levels and a qualitative viability assessment using an expert panel. Results: Crowdfunding, corporate donation, and social impact bonds (SIBs) were deemed to be the most viable of numerous potential strategies. Expected funding levels over 10 years for rare diseases in Colombia from crowdfunding, corporate donations, and SIBs were roughly $7,200, $23,000, and $12,400, respectively. Conclusions: Based on the combination of projected funding potential along with expert consensus regarding viability and operability, crowdfunding, corporate donations, and SIBs, especially in combination, have the potential to substantially improve funding for vulnerable patient populations in Colombia.

Purpose: Nowadays, systematic literature reviews (SLRs) and meta-analyses are often placed at the top of the study hierarchy of evidence. The main objective of this paper is to evaluate the trends in SLRs of randomized controlled trials (RCTs) throughout the years. Methods: Medline database was searched, using a highly focused search strategy. Each paper was coded according to a specific ICD-10 code; the number of RCTs included in each evaluated SLR was also retrieved. All SLRs analyzing RCTs were included. Protocols, commentaries, or errata were excluded. No restrictions were applied. Results: A total of 7,465 titles and abstracts were analyzed, from which 6,892 were included for further analyses. There was a gradual increase in the number of annual published SLRs, with a significant increase in published articles during the last several years. Overall, the most frequently analyzed areas were diseases of the circulatory system (n = 750) and endocrine, nutritional, and metabolic diseases (n = 734). The majority of SLRs included between 11 and 50 RCTs each. Conclusions: The recognition of SLRs' usefulness is growing at an increasing speed, which is reflected by the growing number of published studies. The most frequently evaluated diseases are in alignment with leading causes of death and disability worldwide.

Background: Perianal fistula (PF), a complication of Crohn's disease (CD), affects health-related quality of life (QOL).

Objective: To elucidate QOL of health states corresponding to different stages of PF associated with CD in Japan.

Method: This cross-sectional, observational, web-based questionnaire survey assessed eight different health states in patients with CD and PF and individuals without CD (non-patients) from the Medilead Healthcare Panel (MHP) and determined the utility values (QOL scores) in each health state by the time trade-off method. In patients, we determined also the utility value of the current health state associated with CD and the PF. The analysis excluded respondents with logical inconsistencies.

Results: The analysis included 82 patients and 576 non-patients with the same sex and age distribution as the Japanese population. In both groups, mean utility values were higher in remission (patients, 0.78; non-patients, 0.51) than in non-remission states, with lowest values for poor prognosis after proctectomy (patients, 0.13; non-patients, -0.10) and highest values for the state with mild symptoms (patients, 0.60; non-patients, 0.30). In patients, the mean utility value of the current health state was 0.71.

Conclusion: QOL decreases with increasing severity of PF and is lower for good prognosis after proctostomy than for remission.