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J. Mark. Access Health Policy, Volume 12, Issue 4 (December 2024) – 10 articles

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12 pages, 250 KiB  
Review
Managing Pharmaceutical Costs in Health Systems: A Review of Affordability, Accessibility and Sustainability Strategies
by Christos Ntais, Michael A. Talias, John Fanourgiakis and Nikolaos Kontodimopoulos
J. Mark. Access Health Policy 2024, 12(4), 403-414; https://doi.org/10.3390/jmahp12040031 - 10 Dec 2024
Viewed by 939
Abstract
Background: This paper reviews cost containment policies to control pharmaceutical expenditure either by regulating the pharmaceutical industry or targeting the demand side. Methods: The method used was the narrative literature review of studies which assessed the effect of pharmaceutical cost containment policies. Results: [...] Read more.
Background: This paper reviews cost containment policies to control pharmaceutical expenditure either by regulating the pharmaceutical industry or targeting the demand side. Methods: The method used was the narrative literature review of studies which assessed the effect of pharmaceutical cost containment policies. Results: Governments worldwide have implemented a great variety of policy measures to manage pharmaceutical expenditure while ensuring fair access to essential medicines. Cost-sharing schemes, value-based pricing, reimbursement, reference pricing, payback mechanisms and the substitution of original drugs with generics and biosimilars are pivotal in these efforts, albeit with differing effectiveness across healthcare systems. Overall, it appears that any gains may be outweighed by the unfavorable effects of policies impacting patients. Although interventions have been created to improve physicians’ prescribing practice, they often achieve very minor benefits and at considerable cost. Policy measures pertaining to the regulation of the supply side must be supported by thorough evaluation in order to ascertain costs and effects and guarantee that unintended consequences are minimized. Conclusions: Policymakers frequently enact numerous laws and regulations to control pharmaceutical expenditure, even if there is limited evidence that they are cost-effective. The most crucial component of any policy’s success, regardless of the one selected, is its evaluation. Further research is needed to develop context-specific guidance that balances cost containment, equity and sustainability. Full article
15 pages, 572 KiB  
Article
Operational Efficiency of Public Hospitals in Greece During the COVID-19 Pandemic: A Comparative Analysis Using DEA and AHP Models
by Athanasios Mitakos and Panagiotis Mpogiatzidis
J. Mark. Access Health Policy 2024, 12(4), 388-402; https://doi.org/10.3390/jmahp12040030 - 10 Dec 2024
Viewed by 707
Abstract
This study evaluates the efficiency of public hospitals in Greece during the COVID-19 epidemic in 2020, using Data Envelopment Analysis (DEA) and the Analytical Hierarchy Process (AHP). Faced with unprecedented pressure from increased demand for medical services, these hospitals had to adapt quickly [...] Read more.
This study evaluates the efficiency of public hospitals in Greece during the COVID-19 epidemic in 2020, using Data Envelopment Analysis (DEA) and the Analytical Hierarchy Process (AHP). Faced with unprecedented pressure from increased demand for medical services, these hospitals had to adapt quickly while playing a crucial role in supporting local economies, similar to the effect of tourism on rural economies. This study reveals that, despite average efficiency scores of 83% for result-oriented models (BCC) and 65% for constant return models (CCR), inefficiencies of scale emerged under the pressures of the pandemic. The AHP, by incorporating qualitative criteria and decision-makers’ preferences, offers a valuable perspective but shows little correlation with DEA’s quantitative results. This research emphasizes the importance of utilizing integrated methods to formulate a more comprehensive assessment, adapted to the complex challenges of the healthcare sector during crisis periods. Full article
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10 pages, 405 KiB  
Review
Gatekeeping or Provider Choice for Sustainable Health Systems? A Literature Review on Their Impact on Efficiency, Access, and Quality of Services
by Christos Ntais, Nikolaos Kontodimopoulos and Michael A. Talias
J. Mark. Access Health Policy 2024, 12(4), 378-387; https://doi.org/10.3390/jmahp12040029 - 6 Dec 2024
Viewed by 759
Abstract
As early as 1978, the World Health Organization set primary healthcare as the basis on which health systems should be built worldwide. However, the health systems of the different countries show considerable variations in terms of the implementation of gatekeeping from primary to [...] Read more.
As early as 1978, the World Health Organization set primary healthcare as the basis on which health systems should be built worldwide. However, the health systems of the different countries show considerable variations in terms of the implementation of gatekeeping from primary to secondary healthcare and direct access to specialists and hospital care. This literature review attempts to present the gatekeeping system with references to the UK, Sweden, the Netherlands, and Germany compared to the situation in Greece, where no gatekeeping system exists. Particular emphasis is placed on the impact of gatekeeping on the healthcare system’s efficiency, equity of access, and the quality of the services provided. Evidence on the effects of gatekeeping is conflicting or limited by the low internal validity. Making the right gatekeeping implementation decisions is difficult in the absence of data. High-quality research studies on health outcomes, clinical efficacy, cost-effectiveness, quality of life, healthcare quality, utilisation of healthcare services, the burden in the healthcare system, and the opinions of patients, physicians, and policymakers are all necessary for developing policy. Full article
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9 pages, 237 KiB  
Opinion
Technology Assessment vs. Technology Appraisal—How to Strengthen the Science/Value Dichotomy with EU HTA?
by Sandro Gsteiger, Heiner C. Bucher, James Ryan and Jörg Ruof
J. Mark. Access Health Policy 2024, 12(4), 369-377; https://doi.org/10.3390/jmahp12040028 - 18 Nov 2024
Viewed by 1062
Abstract
Many countries around the world use health technology assessment (HTA) to inform reimbursement and pricing decisions. HTA is often split into two steps, called assessment and appraisal. While the term HTA itself has been defined by international consortia, there is heterogeneity in the [...] Read more.
Many countries around the world use health technology assessment (HTA) to inform reimbursement and pricing decisions. HTA is often split into two steps, called assessment and appraisal. While the term HTA itself has been defined by international consortia, there is heterogeneity in the way different stakeholders use the terms assessment and appraisal. This creates ambiguity regarding which activities are included in technology assessment. With the new EU HTA Regulation, the HTA community should urgently seek to clarify the distinction between assessment and appraisal, as the regulation aims to centralize the clinical part of technology assessment at the European level. Failure to clarify this terminology will put the ambition of the regulation such as increased efficiency and reduction in duplication at risk. In this article, we argue that the distinction between assessment and appraisal should be seen as a science/value dichotomy. We discuss the transition from centralized assessment activities to country-level appraisal, which should culminate in a categorization of the overall added benefit in a local context. Finally, we touch on the important dimension of uncertainty always present in medical decision making. Full article
(This article belongs to the Special Issue European Health Technology Assessment (EU HTA))
27 pages, 749 KiB  
Systematic Review
Economic Evidence on Cost Sharing and Alternative Insurance Designs to Address Moral and Behavioral Hazards in High-Income Health Care Systems: A Systematic Review
by Marlon Graf, James R. Baumgardner, Ulrich Neumann, Iris P. Brewer, Jacquelyn W. Chou and A. Mark Fendrick
J. Mark. Access Health Policy 2024, 12(4), 342-368; https://doi.org/10.3390/jmahp12040027 - 14 Nov 2024
Viewed by 1542
Abstract
In health insurance, “moral hazard” describes the concept that coverage without an out-of-pocket cost to consumers could result in health care utilization beyond economically efficient levels. In response, payers in the United States (US) have designed pharmaceutical benefit plans with significant cost exposure [...] Read more.
In health insurance, “moral hazard” describes the concept that coverage without an out-of-pocket cost to consumers could result in health care utilization beyond economically efficient levels. In response, payers in the United States (US) have designed pharmaceutical benefit plans with significant cost exposure (e.g., co-pays, co-insurance, or deductibles). While substantial evidence links patient cost exposure to reduced drug spending, it remains unclear to what degree this translates into greater efficiency or an indiscriminate drop in overall consumption also reducing needed utilization. We conducted a systematic literature review to understand whether commonly implemented utilization management (UM) strategies and insurance designs with a behavioral or value-based (BID/VBID) component have been explored as tools to mitigate moral hazard and to assess how cost-sharing policies and innovative insurance designs impact consumer spending. Eligible studies compared conventional cost-exposure policies to BID/VBID, including tiered cost-sharing and other UM strategies. We found that broad implementation of patient cost exposure is not well supported by empirical evidence assessing efficiency—defined as the use of clinically appropriate services with value at or above the marginal cost of health care utilization in the contemporary US setting. As a result, payers and policy makers alike ought to explore insurance alternatives that more closely align health care consumption incentives to value of care. Full article
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16 pages, 2205 KiB  
Article
Clinical Benefits of Sustained Oral Nirmatrelvir/Ritonavir Use for the Outpatient Treatment of COVID-19: Findings from the Taiwanese Health Authority Perspective Using a Decision Tree Modeling Approach
by Matthew Sussman, Jennifer Benner, Tendai Mugwagwa, Jackie Lee, Sheng-Tzu Hung, Ya-Min Yang and Yixi Chen
J. Mark. Access Health Policy 2024, 12(4), 326-341; https://doi.org/10.3390/jmahp12040026 - 12 Nov 2024
Viewed by 951
Abstract
Despite the observed clinical benefits of nirmatrelvir/ritonavir (NMV/r), it is uncertain whether Taiwan will continue covering NMV/r for high-risk individuals with mild-to-moderate coronavirus disease 2019 (COVID-19). This analysis assessed the impact of sustained utilization of NMV/r on COVID-19-associated healthcare resource utilization (HCRU) and [...] Read more.
Despite the observed clinical benefits of nirmatrelvir/ritonavir (NMV/r), it is uncertain whether Taiwan will continue covering NMV/r for high-risk individuals with mild-to-moderate coronavirus disease 2019 (COVID-19). This analysis assessed the impact of sustained utilization of NMV/r on COVID-19-associated healthcare resource utilization (HCRU) and mortality from the Taiwanese health authority perspective (THAP). A decision tree model estimated the incremental number of clinical events associated with NMV/r utilization over a 30-day period. Model results compared (1) a base case using current rates of NMV/r from the THAP, and (2) a hypothetical scenario assuming the current supply of NMV/r is not extended in Taiwan. NMV/r utilization rates included 80% and 0% in the base case and hypothetical scenario, respectively. Outcomes included the number of hospitalizations involving a general ward (GW) stay, intensive care unit (ICU) stay, and mechanical ventilation (MV) use, as well as the number of bed days, symptom days, and hospitalization deaths. Based on epidemiologic data, 150,255 patients with COVID-19 were eligible for treatment from the THAP. In the hypothetical scenario, HCRU increased by 175% compared to the base case, including increases in hospitalizations involving GW, ICU, and MV use (differences: 2067; 623; 591, respectively), bed days (difference: 51,521), symptom days (difference: 51,714), and deaths (difference: 480). Findings indicate that sustained utilization of NMV/r from the THAP reduces the clinical burden of mild-to-moderate COVID-19 through the reduced incidence of COVID-19-related HCRU and deaths. Full article
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9 pages, 231 KiB  
Communication
Developer and Partnership Differences in COVID-19 and Other Infections: Insights from DNA Vaccines
by Ryo Okuyama
J. Mark. Access Health Policy 2024, 12(4), 317-325; https://doi.org/10.3390/jmahp12040025 - 29 Oct 2024
Viewed by 1030
Abstract
Historically, vaccine development has been heavily supported by government and public institutions. On the other hand, private biopharmaceutical companies have played a significant role in the development of innovative new therapies using novel pharmaceutical technologies. COVID-19 vaccines using new vaccine technologies, such as [...] Read more.
Historically, vaccine development has been heavily supported by government and public institutions. On the other hand, private biopharmaceutical companies have played a significant role in the development of innovative new therapies using novel pharmaceutical technologies. COVID-19 vaccines using new vaccine technologies, such as mRNA and adenoviral vectors, were rapidly developed by emerging biopharmaceutical companies in collaboration with large corporations and public organizations. This underscores the crucial role of emerging biopharma and public–private partnerships in advancing new vaccine technologies. While these innovations have been suggested as models for future vaccines, their applicability to other infectious diseases requires careful assessment. This study investigated the characteristics of the developers and partnerships in the development of DNA vaccines as a next-generation vaccine platform. The analysis revealed that while emerging biopharmaceutical companies and private–private and private–public partnerships were crucial during the COVID-19 pandemic, public organizations and public–public collaborations primarily led to the clinical development of vaccines for other diseases. Strategies for vaccine development using new vaccine technologies should be tailored to the specific characteristics of each disease. Full article
11 pages, 700 KiB  
Review
Adapting Efficiency Analysis in Health Systems: A Scoping Review of Data Envelopment Analysis Applications During the COVID-19 Pandemic
by Athanasios Mitakos and Panagiotis Mpogiatzidis
J. Mark. Access Health Policy 2024, 12(4), 306-316; https://doi.org/10.3390/jmahp12040024 - 22 Oct 2024
Viewed by 1452
Abstract
Objective: To synthesize the current evidence base concerning the application of Data Envelopment Analysis (DEA) in healthcare efficiency during the COVID-19 pandemic using a scoping review of 13 primary studies. Methods: We consulted databases including Web of Science (WoS) and Scopus, as well [...] Read more.
Objective: To synthesize the current evidence base concerning the application of Data Envelopment Analysis (DEA) in healthcare efficiency during the COVID-19 pandemic using a scoping review of 13 primary studies. Methods: We consulted databases including Web of Science (WoS) and Scopus, as well as manual search entries up to September 2022. Included studies were primary applications of DEA for assessing healthcare efficiency during the COVID-19 pandemic. Key findings derived from thematic analysis of repeating pattern observations were extracted and tabulated for further synthesis, taking into consideration the variations in DMU definitions, the inclusion of undesirable outputs, the influence of external factors, and the infusion of advanced technologies in DEA. Results: The review observed a diverse application of DMUs, ranging from healthcare supply chains to entire national health systems. There was an evident shift towards incorporating undesirable outputs, such as mortality rates, in the DEA models amidst the pandemic. The influence of external and non-discretionary factors became more pronounced in DEA applications, highlighting the interconnected nature of global health challenges. Notably, several studies integrated advanced computational methods, including machine learning, into traditional DEA, paving the way for enhanced analytical capabilities. Conclusions: DEA, as an efficiency analysis tool, has exhibited adaptability and evolution in its application in the context of the COVID-19 healthcare crisis. By recognizing the multifaceted challenges posed by the pandemic, DEA applications have grown more comprehensive, integrating broader societal and health outcomes. This review provides pivotal insights that can inform policy and healthcare strategies, underscoring the importance of dynamic and comprehensive efficiency analysis methodologies during global health emergencies. Full article
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12 pages, 1708 KiB  
Article
INEAS’s Cost-Effectiveness Analysis of Vemurafenib: Paving the Way for Value-Based Pricing in Tunisia
by Mouna Jameleddine, Nabil Harzallah, Hela Grati, Marie Christine Odabachian Jebali, Jaafar Chemli, Sebastián García Martí, Natalie Soto, Andrés Pichon-Riviere and Chokri Hamouda
J. Mark. Access Health Policy 2024, 12(4), 294-305; https://doi.org/10.3390/jmahp12040023 - 6 Oct 2024
Viewed by 942
Abstract
The Tunisian Health Technology Assessment (HTA) body, INEAS, conducted a cost-effectiveness analysis (CEA) of vemurafenib in the treatment of locally advanced or metastatic BRAF V600-mutated melanoma. The objective of this analysis was to enable the use of value-based pricing as a new approach [...] Read more.
The Tunisian Health Technology Assessment (HTA) body, INEAS, conducted a cost-effectiveness analysis (CEA) of vemurafenib in the treatment of locally advanced or metastatic BRAF V600-mutated melanoma. The objective of this analysis was to enable the use of value-based pricing as a new approach to price negotiation. This study was part of a broader HTA report that was prepared in response to a joint request from the regulatory authorities and the CNAM, Tunisia’s compulsory insurance scheme. Our analysis was based on a probabilistic Markov cohort model that calculated the costs and quality-adjusted life years (QALY) associated with vemurafenib compared to the standard of care from a public payer perspective. The CEA indicated that vemurafenib provides a gain of 0.38 life years (1.78 vs. 1.4) for an incremental cost of USD 101,106.62 from the perspective of the main public payer (CNAM). This study revealed an incremental cost-effectiveness ratio (ICER) of 163,311.40 USD/QALY and 163,911.46 USD/QALY, respectively, from the CNAM and public health facilities’ perspectives. Vemurafenib cannot be considered cost-effective in terms of what has normally been considered a reasonable willingness to pay (WTP) in Tunisia. A significant price reduction would be necessary to bring the incremental cost-effectiveness ratio to an acceptable level. Full article
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14 pages, 625 KiB  
Article
A Causal Inference Approach to Mediation Analysis in Vitreomacular Traction: How Much Does Traction Resolution Mediate Functional Outcomes?
by Benedicte Lescrauwaet, Stijn Vansteelandt, Timothy L. Jackson, SriniVas R. Sadda and Luc Duchateau
J. Mark. Access Health Policy 2024, 12(4), 280-293; https://doi.org/10.3390/jmahp12040022 - 30 Sep 2024
Viewed by 723
Abstract
Modern mediation analysis techniques supplement the primary intention-to-treat analysis with the aim to shed light onto the treatment mechanism. We investigate to what extent the anatomic marker vitreomacular adhesion resolution (VMAR) mediates vision benefits, comparing ocriplasmin vs. a sham regimen. A causal mediation [...] Read more.
Modern mediation analysis techniques supplement the primary intention-to-treat analysis with the aim to shed light onto the treatment mechanism. We investigate to what extent the anatomic marker vitreomacular adhesion resolution (VMAR) mediates vision benefits, comparing ocriplasmin vs. a sham regimen. A causal mediation analysis is applied to randomized trial data including 218 participants with vitreomacular traction. Logistic regression models are used to estimate the total treatment effect (TTE) on binary outcomes. Outcomes, assessed at month 24, included visual acuity improvement (VA-I): ≥2-line increase in VA; visual function questionnaire improvement (VFQ-I): ≥5-point increase in the 25-item visual function questionnaire composite score (VFQ-25cs); visual function improvement (VF-I): defined as either a VA-I or a clinically meaningful improvement in the VFQ-25cs. Quantity of interest is the breakdown of TTE into an indirect (through VMAR) and direct effect to estimate the extent to which the TTE is transmitted through the mediating variable (VMAR) vs. other pathways. Causal effects are expressed as risk differences. Indirect effects for VFQ-I, VA-I, and VF-I are 5.7%, 11.8%, and 5.2%, respectively, representing the increase in the probability of a vision improvement if VMAR status were changed for each participant to the extent that it is affected by ocriplasmin. The direct effects are 8.3%, 12.1%, and 24.1% respectively, capturing the effect of treatment on the probability of a vision improvement if ocriplasmin left each participant’s VMAR status unchanged. The relative treatment effect of ocriplasmin on the functional outcome VA-I is to a large extent the result of its effect on VMAR, while an improvement in the patient-reported outcomes VFQ-I or VF-I was only partially mediated by VMAR. Full article
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