Journal of market access & health policy最新文献

In the process of determining whether a health technology should be covered by healthcare systems, patients and their representatives were initially excluded from both evaluations and decision-making. In Europe, direct dialogue between patient organisations and regulatory authorities-particularly in the pharmaceutical sector-began in the early 1990s. It was only decades later, as the high cost of medicines created new challenges, that authorities recognised the necessity of engaging with patients. Patients' contributions to the assessment of a health technology begin with discussions about the need for the technology in question. Initially, these discussions involve the developer, and later-after research and development-regulators, HTA assessors, and payers. Given that multiple technologies may be under development, patients and their organisations often prioritise those that generate the most interest within the patient community. They can then share their perspectives with evaluators during the horizon-scanning phase. Another key contribution is the role patients play in guiding clinical research by participating in scientific advice. Finally, during the assessment and appraisal stages, various methods are used to gather their views.

The regulation of new medicine prices must balance financial sustainability with equitable access to innovation. Value-Based Pricing (VBP) strategies seek to align drug prices with their clinical and societal impact. The Pharmaceutical Innovativeness Index (PII) is a transparent and reproducible tool proposed to assess the degree of innovativeness of new medicines, with potential to support pricing decisions within economic regulation frameworks. An exploratory qualitative study was conducted through a focus group study was conducted with experts in health economics and pharmaceutical regulation to evaluate the applicability of the PII and to discuss key domains relevant to the assessment of pharmaceutical innovation. Responses were collected anonymously using an interactive digital platform and analyzed through inductive thematic content analysis. Based on these findings, the research team developed a conceptual pricing model integrating the PII with additional value-based criteria. Two hypothetical case studies were created to explore its practical feasibility. Participants identified Added Therapeutic Value (ATV) and Unmet Therapeutic Need (UTN) as the most relevant domains, while Methodological Quality (MQ) and Study Design (SD) were also recognized as essential to ensure rigor and reduce bias. The PII scores showed strong alignment with expert judgment in the illustrative case studies. The proposed model incorporates international best practices-such as the efficiency frontier approach-and additional dimensions including safety and incremental innovation. Overall, the PII demonstrated potential to enhance transparency, consistency, and regulatory efficiency in drug pricing decisions in Brazil. However, it should currently be regarded as an exploratory framework requiring further empirical validation and regulatory adaptation before implementation.

Hard-to-heal wounds are a major burden to healthcare systems. Electrical stimulation therapy (EST) is known to improve clinical outcomes, but cost-effectiveness analysis is lacking. The aim was to explore the cost-effectiveness of EST with standard of care (SoC) versus SoC alone. A systematic review and meta-analysis of randomised controlled studies (RCTs) were conducted. Fourteen RCTs were identified, representing 783 patients. EST + SoC, versus SoC alone, significantly increased the proportion of wounds healed (odds ratio [OR] 2.46 [95% CI, 1.75-3.46], p < 0.0001) and significantly decreased the mean time to healing (-2.67 weeks (95% CI, 1.49-3.84, p < 0.00001). A cost-effectiveness model was developed based on these findings and on the usage and cost of the EST device used in the largest included RCT. Weekly costs of community wound care were taken from published estimates and inflated to 2024 levels, reflecting costs in the UK. In a hypothetical cohort of 100 patients treated over 12 weeks, EST + SoC was estimated to save over GBP 38,000 overall, reduce nursing visits by 385 and lead to 154 more ulcer-free weeks. In conclusion, EST used in the community is a cost-effective addition to SoC with the ability to improve outcomes and reduce human and financial burden of hard-to-heal wounds.

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.