Journal of market access & health policy最新文献

This study aimed to determine the relative efficacy of bevacizumab gamma (an ophthalmic formulation of bevacizumab) versus alternative interventions relevant to the treatment of neovascular age-related macular degeneration (nAMD) in the United Kingdom (UK) via a systematic literature review (SLR) and network meta-analysis (NMA). An SLR was conducted to identify randomized controlled trials (RCTs) of anti-vascular endothelial growth factor (anti-VEGF) therapies for the treatment of nAMD in adult patients relevant to the UK context. The included anti-VEGF treatments were ranibizumab, aflibercept, faricimab, and bevacizumab gamma. Bayesian NMA models were used to estimate relative efficacy in terms of change from baseline (CFB) in best-corrected visual acuity (BCVA) at 12 months, the proportion of patients gaining 15 or more letters at 12 months, and the proportion of patients losing less than 15 letters at 12 months. Twenty-two relevant RCTs were included in the NMA. At 12 months, all anti-VEGF treatments were similarly efficacious to ranibizumab 0.5 mg every four weeks (Q4W) in terms of CFB in BCVA, the proportion of patients gaining 15 or more letters, and the proportion of patients losing less than 15 letters (except for ranibizumab 0.5 mg every 12 weeks [Q12W] and ranibizumab 0.5 mg pro re nata [PRN]). Bevacizumab gamma provided similar improvements in visual acuity to other anti-VEGF treatments.

There remains a persistent lack of patient-centered evidence on insurance reform and real-world experiences of patients with chronic disease. This study gathered insights around insurance design from chronic disease beneficiaries. This mixed-methods analysis comprised an online survey and virtual focus group sessions (August to December 2023) involving US residents with chronic disease and health insurance. Patients' perspectives on insurance design were explored. Survey data were analyzed descriptively. Key themes were identified from focus group transcripts and direct observations. In total, 146 patients across 15 chronic diseases completed the survey; 29 then participated in focus groups. Although most beneficiaries were satisfied with their health plan, concerns centered on prescription medication affordability due to high deductibles and cost exposure, the disproportionate effects of cost exposure based on income, and the unpredictability of out-of-pocket costs. For some, the financial burden led to financial debt, therapy abandonment, mental health issues, and/or worsening of their condition. Overall, there was broad support for policy solutions to redesign insurance and adjust cost exposure for patients with chronic disease. This research offers valuable patient insights into health insurance design in the US to ensure patients' needs are addressed.

Objective: This confirmatory survey aimed to verify the criterion-related validity and reliability of the final version of the Medical Doctors' Work-Related Quality of Life Questionnaire (WQMD-9), following partial revision of its content. This study also explored the questionnaire's structure and scoring methods. Method: From June to July 2022, the WQMD-9 was administered to 98 MDs selected to match the statistical distribution of MDs in Japan. Criterion-related validity was evaluated using a visual analogue scale (VAS) as the reference standard, and reliability was examined using inter-dimension correlations and Cronbach's α. Results: The correlation coefficient between the VAS score and the simple sum of WQMD-9 dimensions scores was 0.7891, supporting criterion-related validity. Cronbach's α was 0.87, indicating acceptable reliability. Conclusions: The profile-type WQMD-9 consists of nine dimensions-"Workload," "Working time," "Collaboration," "Clinical practice," "Working conditions," "Working environment," "Feelings of fatigue," "Work-life balance," and "Career"-with five levels. In the confirmatory survey population, the WQMD-9 demonstrated criterion-related validity and reliability, suggesting that it can be utilized with simple total scoring approach.

Copay accumulator (CA) and copay maximizer (CM) programs in the United States, which prevent manufacturer copay assistance from counting toward deductibles or out-of-pocket (OOP) maximums, are increasingly used, raising concerns about costs and outcomes for patients with major depressive disorder (MDD) or bipolar disorder (BPD) treated with branded atypical antipsychotics (AAPs) and/or antidepressants (ADs). This retrospective claims study used Kythera commercial data (2020-2024) in the United States to identify adults with MDD or BPD who had at least 1 diagnosis and one branded AAP or AD prescription between 2021 and 2023, requiring 12 months' continuous enrollment pre- (2020-2021) and post-index (2023-2024) and at least three months of post-index branded medication use. This retrospective claims study used Kythera commercial data (2020-2024) to identify adults with MDD or BPD who had at least one diagnosis and one branded AAP or AD prescription between 2021 and 2023, requiring 12 months' continuous enrollment pre- and post-index and at least 3 months of post-index branded medication use. Patients were stratified into CA, CM, or standard copay plan (SCP) cohorts, and propensity score matching was used to compare treatment patterns and costs. Both CA and CM groups had significantly higher median OOP costs than SCPs (e.g., $75/$60 vs. $16 for MDD+AAP; p < 0.0001), and higher pharmacy costs among adherent patients. CA patients had poorer adherence and persistence, shorter treatment duration, and higher discontinuation and abandonment rates than SCPs. These findings highlight higher OOP burden and adherence challenges with CA and CM programs, underscoring the need for careful benefit design for US mental health patients.

Background: Stakeholders' perception plays a crucial role in shaping pharmaceutical strategies. Stakeholders are groups interested in pharmaceutical companies' success and outcomes. Stakeholders' perceptions are multifaceted and impact pharmaceutical strategies, from shaping research to enhancing market access, pricing, and corporate reputation. Understanding and actively managing stakeholders' perceptions is vital for pharmaceutical companies to succeed in an increasingly complex and competitive industry. Methods: In this case study, knowledge contributions from stakeholders offered insights and strategies for application in the pharmaceutical sector. Results: Qualitative, exploratory research was conducted, which included the participation of sixteen stakeholders from different countries in Latin America, who responded to a semi-structured interview script, whose data were understood through lexical analysis in the Interface de R pour les Analyses Multimensionnelles de Texts et de Questionnaires (IRaMuTeQ). Conclusions: The results of this study underscore the importance of regulatory knowledge for professionals' support and implementation of international strategies. Regulatory knowledge provides professionals with tools and insights to navigate complex regulatory environments, make informed decisions, and enhance organizational performance in global markets.

Pharmacy technicians (PT) are vital to the efficient and safe operation of hospital pharmacy services, fulfilling a range of technical and clinical responsibilities that directly impact patient care. However, increasing healthcare demands have underscored the importance of adequate staffing levels to sustain service quality and safeguard patient outcomes. This perspective paper explores how appropriate staffing levels for PT in hospital settings are essential and important to support safe, efficient care and a sustainable workforce. It compares evidence-informed staffing models, highlights real-world benchmarks, and proposes governance recommendations to guide policies that strengthen pharmacy services. Recommendations are made to inform clinical governance, suggesting that staffing policies, continuous training, and professional development programs are essential to supporting PT effectiveness and retention. The findings advocate for regulated staffing ratios and governance measures to foster an environment where PTs can deliver high-quality care and uphold safety standards within hospital pharmacies.

Introduction: Market access, pricing, and reimbursement of digital health technologies (DHTs) in Europe are significantly challenged by regulatory fragmentation and various assessment methodologies. Understanding the challenges and priorities of technology developers is essential for developing effective and relevant policy responses. This study explores perceived barriers and developer-driven priorities to inform the development of a harmonized health technology assessment (HTA) framework under the EDiHTA project. Methods: A mixed-methods approach was adopted, including a scoping review to identify key challenges, a survey of 20 DHT developers, and interviews and focus groups with 29 industry representatives from startups to multinational companies across 10 European countries during 2024. Results: Key challenges included a lack of transparency in reimbursement processes, fragmented HTA requirements, and misalignment between traditional evidence models and the agile development of DHTs. Developers highlighted the need to integrate real-world evidence, consider usability and implementation factors, and provide structured, lifecycle-based guidance. Financial barriers and procedural burdens were particularly significant for small and medium-sized enterprises. Conclusions: These findings highlight the need for an HTA framework that reflects the iterative nature of digital development, integrates real-world evidence, and reduces uncertainty for developers. The EDiHTA project aims to respond to these challenges by building a harmonized and flexible approach that aligns with the goals of the European HTA Regulation.

Designing an accessible, financially viable healthcare system is a key challenge for society. The value-based healthcare (VBHC) strategic model aims to simultaneously improve the quality of healthcare and the efficiency of health systems. The aim of this research was to describe the perceptions of different stakeholders in the Portuguese health industry about the creation of value and the understanding of VBHC as a competitive advantage. A qualitative study was conducted using the inductive method of Braun and Clarke, designed according to the COREQ criteria. Based on the results of the literature review, a semi-structured script for an interview was created, consisting of eight questions. The initial interview script was based on a thorough narrative literature review and tested with two professionals with practical experience in VBHC. The final version of the semi-structured interview guide consisted of eight open-ended questions. The questions were designed to elicit in-depth, reflective responses, and their neutrality was reviewed to avoid leading language that might introduce bias. As the interviews progressed, minor iterative changes were made to include participant-suggested additions, always maintaining alignment with the research objectives. This iterative process was essential to capture the nuanced perspectives of stakeholders and conformed to COREQ standards for qualitative research. A total of 15 stakeholders in VBHC were interviewed. The interviews were transcribed and coded, and 605 codes were created, divided into subthemes and themes. VBHC implementation faces several challenges, requiring a collaborative effort by the stakeholders involved, to achieve a comprehensive vision of value and appropriate multi-stakeholder alignment. The implementation of VBHC can confer a sustainable competitive advantage, and its adoption as a strategic model will be inevitable in the future.

Background: Expected utility has been deployed in order to predict health behaviour in health economic evaluation. However, only limited variance in health behaviour is explained by this construct. This limited explained variance is often attributed to the dubious foundational postulates underlying the construct (e.g., absolute rationality, complete information, fixed preferences). Due to these limitations it has been hypothesized that substituting or complementing expected utility with experienced utility may enhance predictions of health behaviour. As this hypothesis has not yet been subjected to empirical scrutiny, this study examines if deployment of experienced utility or expected utility and experienced utility combined enhances predictions of health behaviour relative to expected utility separately.

Methods: Online questionnaires were distributed across a panel of Dutch citizens (N = 2550). The questionnaire includes items and scales on sample characteristics, expected utility, experienced utility and health behaviour. Data analysis was conducted by employing descriptive, reliability, validity and model statistics.

Results: Experienced utility has a significant direct effect on health behaviour that is stronger than expected utility. Experienced utility also explains more variance in health behaviour than expected utility. Expected utility and experienced utility combined have a significant direct and indirect effect on health behaviour that is stronger than each type of utility separately. Expected utility and experienced utility combined also explain more variance in health behaviour than each type of utility separately.

Conclusions: Deploying experienced utility separately or in combination with expected utility in health economic evaluation seems pertinent as it has considerable impact on health behaviour and may provide health economists with an even sturdier foundation for conducting health economic evaluation.

Gene therapies that induce the body to produce therapeutic anti-vascular endothelial growth factor (anti-VEGF) proteins are an emerging topic related to neovascular age-related macular degeneration (nAMD). Continuous delivery of anti-VEGF protein directly to the target tissue offers the possibility of lifelong efficacy without the need for repeated and frequent eye injections. This novel approach could revolutionize patient management through optimizing clinical outcomes while simplifying service delivery. However, such gene therapies are anticipated to face unique challenges related to patients' access and health technology assessment (HTA), and their integration into real-world eyecare practices. This article presents key elements raised at the European Access Academy (EAA) Fall convention (held in Rome in October 2024) regarding anticipated HTA challenges for gene therapies in nAMD. The important role of HTA and policymakers in ensuring that emerging gene therapies are accessible to all eligible patients is also highlighted. This article mainly focuses on the need for a fit-for-purpose EU HTA framework to address the widely varying utilization of standard of care in nAMD clinical practice, and to incorporate considerations about the long-term durability of gene therapies in nAMD. The importance of integrating real-world evidence (RWE) into the EU HTA framework is also discussed.