Journal of market access & health policy最新文献

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.

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.

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: 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.

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.

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 and objectives: Multiple reforms aimed at improving the Chinese population's health have been introduced in recent years, including several designed to improve access to innovative drugs. We sought to review current factors affecting access to innovative drugs in China and to anticipate future trends.

Methods: Targeted reviews of published literature and statistics on the Chinese healthcare system, medical insurance and reimbursement processes were conducted, as well as interviews with five Chinese experts involved in the reimbursement of innovative drugs.

Results: Drug reimbursement in China is becoming increasingly centralized due to the removal of provincial pathways, the establishment of the National Healthcare Security Administration and the implementation of the National Reimbursement Drug List (NRDL), which is now the main route for drug reimbursement in China. There is also an increasing number of other channels via which patients may access innovative treatments, including various types of commercial insurance and special access. Health technology assessment (HTA) and health economic evidence are becoming pivotal elements of the NRDL decision-making process. Alongside the optimization of HTA decision making, innovative risk-sharing agreements are anticipated to be increasingly leveraged in the future to optimize access to highly specialized technologies and encourage innovation while safeguarding limited healthcare funds.

Conclusions: Drug public reimbursement in China continues to align more closely with approaches widely used in Europe in terms of HTA, health economics and pricing. Centralization of decision-making processes for public reimbursement of innovative drugs allows consistency in assessment and access, which optimizes the improvement of the Chinese population's health.

Purpose: Remarkable progress in healthcare technology has recently been made alongside changes in concepts related to drugs and medical devices. It is speculated that this progress benefits not only patients but also healthcare professionals, such as medical doctors. We performed a systematic review of the characteristics of current data assessment tools to measure medical doctors' work-related quality of life (QOL).

Methods: A literature search was conducted through PubMed and Ichushi-Web in 2020. The related search terms used were 'medical doctor,' 'quality of work life,' and 'questionnaire/interview.' Two reviewers independently screened the studies, and the characteristics of the QOL assessment tools used in the identified studies were qualitatively reviewed and summarized.

Results: In total, 5,443 and 760 articles were retrieved from PubMed and Ichushi-Web, respectively, of which 82 studies were included in this review. Sixty-five (79%) studies used structured questionnaires, and 17 (21%) studies used semistructured questionnaires. In terms of the study purpose, the identified studies mainly included four: mental health, the work or labor situation, satisfaction, and QOL. Components used to measure work-related QOL included satisfaction, burnout, QOL, the work environment, stress, mental health, work-life balance, and others. None of the studies used an originally developed QOL questionnaire to assess the work-related benefits of medical doctors.

Conclusion: This systematic review found that there is a lack of studies directly assessing the work-related QOL of medical doctors and a lack of effective data collection tools to assess all work-related QOL components.