Journal Description
Journal of Market Access & Health Policy
Journal of Market Access & Health Policy
(JMAHP) is an international, peer-reviewed, open access journal that covers all different subdisciplines of ‘market access’ from economic, technical, scientific, sociological, psychological and policy perspectives. The journal is owned by the Market Access Society, and is published quarterly online by MDPI (since Volume 12, Issue 1 - 2024).
- Open Access— free for readers, with article processing charges (APC) paid by authors or their institutions.
- High Visibility: indexed within Scopus, PubMed, PMC, and other databases.
- Rapid Publication: manuscripts are peer-reviewed and a first decision is provided to authors approximately 54.2 days after submission; acceptance to publication is undertaken in 19.6 days (median values for papers published in this journal in the first half of 2024).
- Journal Rank: CiteScore - Q1 (Health Policy)
- Recognition of Reviewers: APC discount vouchers, optional signed peer review, and reviewer names published annually in the journal.
Latest Articles
INEAS’s Cost-Effectiveness Analysis of Vemurafenib: Paving the Way for Value-Based Pricing in Tunisia
J. Mark. Access Health Policy 2024, 12(4), 294-305; https://doi.org/10.3390/jmahp12040023 - 6 Oct 2024
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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
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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.
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Open AccessArticle
A Causal Inference Approach to Mediation Analysis in Vitreomacular Traction: How Much Does Traction Resolution Mediate Functional Outcomes?
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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
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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
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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.
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Open AccessOpinion
The Knowledge and Application of Economics in Healthcare in a High-Income Country Today: The Case of Belgium
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Baudouin Standaert, Désirée Vandenberghe, Mark P. Connolly and Johan Hellings
J. Mark. Access Health Policy 2024, 12(3), 264-279; https://doi.org/10.3390/jmahp12030021 - 4 Sep 2024
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Healthcare is a huge business sector in many countries, focusing on the social function of delivering quality health when people develop illness. The system is essentially financed by public funds based on the solidarity principle. With a large financial outlay, the sector must
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Healthcare is a huge business sector in many countries, focusing on the social function of delivering quality health when people develop illness. The system is essentially financed by public funds based on the solidarity principle. With a large financial outlay, the sector must use economic evaluation methods to achieve better efficiency. The objective of our study was to evaluate and to understand how health economics is used today, taking Belgium as an example of a high-income country. The evaluation started with a historical view of healthcare development and ended with potential projections for its future. A literature review focused on country-specific evaluation reports to identify the health economic methods used, with a search for potential gaps. The first results indicated that Belgium in 2021 devoted 11% of its GDP, 17% of its total tax revenue, and 30% of the national Social Security Fund to health-related activities, totalizing EUR 55.5 billion spending. The main health economic method used was a cost-effectiveness analysis linked to budget impact, assigning reimbursable monetary values to new products becoming available. However, these evaluation methods only impacted at most 20% of the money circulating in healthcare. The remaining 80% was subject to financial regulations (70%) and budgeting (10%), which could use many other techniques of an economic analysis. The evaluation indicated two potentially important changes in health economic use in Belgium. One was an increased focus on budgeting with plans, time frames, and quantified treatment objectives on specific disease problems. Economic models with simulations are very supportive in those settings. The other was the application of constrained optimization methods, which may become the new standard of practice when switching from fee-for-service to pay-per-performance as promoted by value-based healthcare and value-based health management. This economic refocusing to a more constrained approach may help to keep the healthcare system sustainable and affordable in the face of the many future challenges including ageing, climate change, migration, pandemics, logistical limitations, and financial instability.
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Open AccessArticle
Non-Medical Switching or Discontinuation Patterns among Patients with Non-Valvular Atrial Fibrillation Treated with Direct Oral Anticoagulants in the United States: A Claims-Based Analysis
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Michael Ingham, Hela Romdhani, Aarti Patel, Veronica Ashton, Gabrielle Caron-Lapointe, Anabelle Tardif-Samson, Patrick Lefebvre and Marie-Hélène Lafeuille
J. Mark. Access Health Policy 2024, 12(3), 252-263; https://doi.org/10.3390/jmahp12030020 - 2 Sep 2024
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This study assessed direct-acting oral anticoagulant (DOAC) switching/discontinuation patterns in patients with non-valvular atrial fibrillation (NVAF) in 2019, by quarter (Q1–Q4), and associated socioeconomic risk factors. Adults with NVAF initiating stable DOAC treatment (July 2018–December 2018) were selected from Symphony Health Solutions’ Patient
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This study assessed direct-acting oral anticoagulant (DOAC) switching/discontinuation patterns in patients with non-valvular atrial fibrillation (NVAF) in 2019, by quarter (Q1–Q4), and associated socioeconomic risk factors. Adults with NVAF initiating stable DOAC treatment (July 2018–December 2018) were selected from Symphony Health Solutions’ Patient Transactional Datasets (April 2017–January 2021). Switching/discontinuation rates were reported in 2019 Q1–Q4, separately. Non-medical switching/discontinuation (NMSD) was defined as the difference between switching/discontinuation rates in Q1 and mean rates across Q2–Q4. The associations of socioeconomic factors with switching/discontinuation were assessed. Of 46,793 patients (78.7% ≥ 65 years; 52.6% male; 7.7% Black), 18.0% switched/discontinued their initial DOAC in Q1 vs. 8.8% on average in Q2–Q4, corresponding to an NMSD of 9.2%. During the quarter following the switch/discontinuation, more patients who switched/discontinued in Q1 remained untreated (Q1: 77.0%; Q2: 74.3%; Q3: 71.2%) and fewer reinitiated initial DOAC (Q1: 17.6%; Q2: 20.8%; Q3: 24.0%). Factors associated with the risk of switching/discontinuation in Q1 were race, age, gender, insurance type, and household income (all p < 0.05). More patients with NVAF switched/discontinued DOACs in Q1 vs. Q2–Q4, and more of them tended to remain untreated relative to those who switched/discontinued later in the year, suggesting a potential long-term impact of NMSD. Findings on factors associated with switching/discontinuation highlight potential socioeconomic discrepancies in treatment continuity.
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Open AccessArticle
Proposal of a Knowledge Management Model for Complex Systems: Case of the Supervision and Control Subsystem of the Colombian Health System
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Fredy G. Rodríguez-Páez, Diego Cabrera-Moya and Jorge Aurelio Herrera-Cuartas
J. Mark. Access Health Policy 2024, 12(3), 224-251; https://doi.org/10.3390/jmahp12030019 - 21 Aug 2024
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Background: Considering regulatory, supervision, and control health policy, an innovative knowledge management model is proposed for the Colombian health system, which is recognized as a complex system. Methods: A model is constructed through a comparative analysis of various theoretical and conceptual frameworks, and
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Background: Considering regulatory, supervision, and control health policy, an innovative knowledge management model is proposed for the Colombian health system, which is recognized as a complex system. Methods: A model is constructed through a comparative analysis of various theoretical and conceptual frameworks, and an original methodology is proposed based on an analysis of the macroprocesses of the Supervision and Control System (SSC) of the Colombian General Social Security System in Health (SGSSS). After formulating hypotheses and conceptual references, information errors are determined within the different macroprocesses of the SGSSS, including those of governance and the SSC. Results: The risks of generating duplicate, wrong, hidden, or non-existent information arise when the associated regulations need more specificity to be applied in all cases, thus leading to the risk of different interpretations by some actors. In this way, it is possible to hinder the generation of unified information, as there is no clarity as to who is responsible for the generation or creation of certain data. Conclusions: The proposed model is characterized by its flexibility and adaptability, integrating several processes that can be executed simultaneously or cyclically (depending on the system’s needs) and allowing for the generation and feedback of knowledge at different stages, with some processes simultaneously executed to complement each other.
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Open AccessPerspective
Adoption of a Societal Perspective in Economic Evaluations of Musculoskeletal Disorders: A Conceptual Paper
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Francis Fatoye, Tadesse Gebrye, Leo Nherera and Paul Trueman
J. Mark. Access Health Policy 2024, 12(3), 216-223; https://doi.org/10.3390/jmahp12030018 - 12 Aug 2024
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Economic evaluations are used to compare the costs and consequences of healthcare interventions, including those for musculoskeletal (MSK) disorders, which are very common and a major source of morbidity and absence from work. Reimbursement decisions for interventions for MSK disorders by decision-makers rely
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Economic evaluations are used to compare the costs and consequences of healthcare interventions, including those for musculoskeletal (MSK) disorders, which are very common and a major source of morbidity and absence from work. Reimbursement decisions for interventions for MSK disorders by decision-makers rely on the findings of economic evaluations, the design and results of which depend largely on the perspective adopted. Despite methodological advancements in economic evaluations, there are no clear guidelines on the perspective to adopt. This paper explores the adoption of a societal perspective in economic evaluations of MSK disorders. Within health economics evaluations, the most commonly used perspectives include the payer perspective, the healthcare perspective, and the societal perspective. To facilitate optimal resource allocation decisions in order to reduce the significant economic burden of MSK disorders and improve the health outcomes of individuals with these disorders, all costs and benefits associated with interventions for them should be included. Thus, the societal perspective is arguably a preferable option to the others for economic evaluations of interventions for MSK disorders.
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Open AccessCommunication
Patient Experience and Satisfaction with Orthopedic Services at a Community (Rural) Setting Hospital—How Is It Different from Urban Setting
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Khalid Hasan and Shahin Kayum
J. Mark. Access Health Policy 2024, 12(3), 209-215; https://doi.org/10.3390/jmahp12030017 - 5 Aug 2024
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Patient experience and satisfaction are the keystones in evaluating the effectiveness of clinical care in musculoskeletal medicine. Although all orthopedic settings work on the same principles of providing safe and quality health care, community hospitals represent a unique environment. There may be key
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Patient experience and satisfaction are the keystones in evaluating the effectiveness of clinical care in musculoskeletal medicine. Although all orthopedic settings work on the same principles of providing safe and quality health care, community hospitals represent a unique environment. There may be key differences with regard to patient experience between these settings. Accessibility to care, choices of provider, personalized care, availability of and access to resources, cultural and social variances, and waiting times are a few of the many elements that may impact patient experience and satisfaction. This narrative review aims to explore the core differences in these settings and how they can reflect on patient experience and satisfaction.
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Open AccessArticle
Valuation of Medical Innovation Handling with Uncertainty and Risk
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Mark Nuijten and Stefano Capri
J. Mark. Access Health Policy 2024, 12(3), 199-208; https://doi.org/10.3390/jmahp12030016 - 1 Aug 2024
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Background: The purpose of this paper is to address how to handle uncertainty when performing an economic valuation of a medical innovation R&D project in orphan diseases from the perspective of the investor. Methods: We describe the specific uncertainty related to cash flows
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Background: The purpose of this paper is to address how to handle uncertainty when performing an economic valuation of a medical innovation R&D project in orphan diseases from the perspective of the investor. Methods: We describe the specific uncertainty related to cash flows and the cost of capital for innovation in orphan diseases. The uncertainty in cash flows relates to sales, manufacturing and R&D costs, and probabilities of failure for each phase in the clinical trial program. We consider different net present values (NPVs) and higher standard deviations for orphan drugs compared to non-orphan drugs. Results: Numerical case base examples showed the differences in trade-off by an investor for R&D projects with differences in NPV and level of uncertainty. The investor will transfer the additional uncertainty in cash flows in a higher cost of capital. An alternative approach is the application of an “acceptability curve” based on a probabilistic sensitivity analysis, which displays the cumulative probabilities at a range of different values for the NPV. Finally, we consider uncertainty in the cost of capital itself by applying the Capital Asset Pricing Model (CAPM). Conclusions: In this paper, we described various types of uncertainty and explored various approaches to how to handle uncertainty in the economic valuation of medical innovation in orphan diseases. The bridging of health economics with economic valuation theory in the healthcare market is to our knowledge a novel approach for the valuation of medical innovation by investors.
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Open AccessArticle
Market Access Challenges and Solutions in Cell and Gene Therapy in The Netherlands
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Rimma Velikanova, Sharon Wolters, Hinko S. Hofstra, Maarten J. Postma and Cornelis Boersma
J. Mark. Access Health Policy 2024, 12(3), 181-198; https://doi.org/10.3390/jmahp12030015 - 30 Jul 2024
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With the increasing pipeline of cell and gene therapies (CGTs) and the expected surge in the number of approvals, understanding the market access landscape becomes crucial for timely patient access. This study evaluates the challenges Dutch stakeholders encounter in CGT market access, offering
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With the increasing pipeline of cell and gene therapies (CGTs) and the expected surge in the number of approvals, understanding the market access landscape becomes crucial for timely patient access. This study evaluates the challenges Dutch stakeholders encounter in CGT market access, offering insights for improving time-to-patient access. A traditional literature review was conducted to identify market access challenges and solutions for CGTs. Based on the findings, participants for semi-structured interviews, designed using an interview guide adapted to the Dutch context, were selected to capture diverse perspectives on market access. This review included 124 relevant articles out of 2449, covering several aspects of market access of CGTs. Subsequently, interviews with 16 stakeholders from academia, patient advocacy groups, manufacturers, health insurers, payers, hospital pharmacists, healthcare practitioners, and the Association of Innovative Medicines were conducted. Stakeholders identified challenges and proposed solutions for reimbursement package management, clinical trials, health economics, payment models, and procedural and organisational aspects. Thematic analysis revealed unique country-specific challenges and solutions in the Netherlands. This research provides insights into these challenges and potential solutions, emphasising the need for collaborative efforts among stakeholders to develop practical and multidisciplinary measures to improve the market access landscape for CGTs in the country.
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Open AccessArticle
Comparative Efficacy of Finerenone versus Canagliflozin in Patients with Chronic Kidney Disease and Type 2 Diabetes: A Matching-Adjusted Indirect Comparison
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David Cherney, Kerstin Folkerts, Paul Mernagh, Mateusz Nikodem, Joerg Pawlitschko, Peter Rossing and Neil Hawkins
J. Mark. Access Health Policy 2024, 12(3), 169-180; https://doi.org/10.3390/jmahp12030014 - 25 Jul 2024
Cited by 1
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This study aimed to close an evidence gap concerning the relative efficacy of finerenone versus SGLT2is in patients with chronic kidney disease (CKD) and type 2 diabetes (T2D). Canagliflozin was selected as a proxy for the SGLT2i class. Patient-level data of two randomized
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This study aimed to close an evidence gap concerning the relative efficacy of finerenone versus SGLT2is in patients with chronic kidney disease (CKD) and type 2 diabetes (T2D). Canagliflozin was selected as a proxy for the SGLT2i class. Patient-level data of two randomized controlled trials (RCTs) of finerenone (FIDELIO-DKD and FIGARO-DKD) were used alongside aggregated data from CREDENCE, an RCT of canagliflozin. To account for meaningful between-study heterogeneity between each finerenone trial and CREDENCE, a matching-adjusted indirect comparison of a range of efficacy outcomes was undertaken for each finerenone study versus CREDENCE. These results were meta-analyzed, enabling the estimation of the relative effects of finerenone against canagliflozin. For the cardiorenal composite endpoint, the hazard ratio (HR) comparing finerenone to canagliflozin was 1.07 (95% CI: 0.83 to 1.36). The corresponding HRs for all-cause mortality, end-stage kidney disease and cardiovascular death were 0.99 (95% CI: 0.73 to 1.34), 1.03 (95% CI: 0.68 to 1.55) and 0.94 (95% CI: 0.64 to 1.37), respectively. The absence of statistically significant differences was consistent throughout the main analysis and a range of sensitivity analyses. Based on this study, using a large sample of data and adjusted for meaningful differences between the baseline characteristics of the included RCTs, there was no statistically significant evidence indicating a difference in the efficacy of finerenone compared to canagliflozin in the treatment of CKD in patients with T2D.
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Open AccessArticle
Expanding the Availability of Scalp Cooling to All Patients at Risk of Chemotherapy-Induced Alopecia
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Portia Lagmay-Fuentes, Andrea Smith, Shannon Krus, Laurie Lewis, Asma Latif, Tiffany Gagliardo and Manpreet Kohli
J. Mark. Access Health Policy 2024, 12(3), 158-168; https://doi.org/10.3390/jmahp12030013 - 10 Jul 2024
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Alopecia is an undesirable side effect of cancer chemotherapy. The mitigation of alopecia is a desirable adjunct treatment for patients with cancer. FDA-cleared scalp cooling (SC) devices have been successfully used to prevent or reduce chemotherapy-induced alopecia (CIA). This paper provides an understanding
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Alopecia is an undesirable side effect of cancer chemotherapy. The mitigation of alopecia is a desirable adjunct treatment for patients with cancer. FDA-cleared scalp cooling (SC) devices have been successfully used to prevent or reduce chemotherapy-induced alopecia (CIA). This paper provides an understanding of the implementation and value of the new Insurance-Based Billing Model used in the USA for SC and its benefits compared with the original self-pay model. This improved compensation change will result in all patients in need, including underserved and disadvantaged populations, receiving equitable healthcare by allowing access to this valuable supportive care technology.
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Open AccessArticle
Opportunities and Challenges in Cross-Country Collaboration: Insights from the Beneluxa Initiative
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Zilke Claessens, Michiel Lammens, Liese Barbier and Isabelle Huys
J. Mark. Access Health Policy 2024, 12(3), 144-157; https://doi.org/10.3390/jmahp12030012 - 9 Jul 2024
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National pricing and reimbursement agencies face growing challenges with complex health technologies, prompting European policy advancements. Beneluxa is a cross-country collaboration involving Belgium, the Netherlands, Luxemburg, Austria, and Ireland that aims to address sustainable access to medicines. In view of the soon-to-be-implemented EU
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National pricing and reimbursement agencies face growing challenges with complex health technologies, prompting European policy advancements. Beneluxa is a cross-country collaboration involving Belgium, the Netherlands, Luxemburg, Austria, and Ireland that aims to address sustainable access to medicines. In view of the soon-to-be-implemented EU HTA Regulation, insights and experiences from stakeholders with Beneluxa cross-country collaboration could provide possible transferable learnings. Therefore, this research aims to (i) identify the opportunities and challenges faced by Beneluxa, (ii) gather insights from stakeholders, namely (possible) applicants and policymakers, within and beyond Beneluxa on the initiative and broader cross-country collaboration principles, and (iii) transfer these insights into learnings and recommendations in anticipation of the full implementation of the new HTA Regulation. Fifteen semi-structured interviews were conducted with industry and European HTA/policy stakeholders. The principal challenges discussed by stakeholders encompass hesitancy from the industry toward Beneluxa assessments, which were attributed to procedural and timeline uncertainties, legislative framework ambiguity, and challenges in terms of industry’s internal organization. Another challenge highlighted is the resource-intensive nature of the procedure due to diverse approaches among member states. In addition, industry stakeholders mentioned limited communication and procedural complexity. Despite challenges, both stakeholder groups recognized important opportunities for cross-country collaboration. Transferable insights for future cross-country collaboration include transparent communication, clear legislative embedding, internal industry restructuring to facilitate joint HTAs, and member state support for conducting collaborative assessments. The study underscores diverging views among stakeholders on cross-country collaboration’s potential to support HTA and the market access of complex health technologies. While acknowledging benefits, there still are challenges, including industry hesitancy, emphasizing the need for transparent communication and clear guidance in the evolving EU HTA landscape.
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Open AccessArticle
The Role of Medical Societies and the Relevance of Clinical Perspective in the Evolving EU HTA Process: Insights Generated at the 2023 Fall Convention and Survey of the European Access Academy
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Elaine Julian, Oriol Solà-Morales, Maria João Garcia, Francine Brinkhuis, Mira Pavlovic, Carlos Martín-Saborido, Robin Doeswijk, Rosa Giuliani, Anne Willemsen, Wim Goettsch, Bernhard Wörmann, Urania Dafni, Heiner C. Bucher, Begoña Pérez-Valderrama, Renato Bernardini, Fabrizio Gianfrate, Carin A. Uyl-de Groot and Jörg Ruof
J. Mark. Access Health Policy 2024, 12(3), 128-143; https://doi.org/10.3390/jmahp12030011 - 22 Jun 2024
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Background: This work aimed to determine the role and action points for the involvement of medical societies in the European Health Technology Assessment (EU HTA) Methods: An online pre-convention survey was developed addressing four areas related to the EU HTA: (i) medical societies’
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Background: This work aimed to determine the role and action points for the involvement of medical societies in the European Health Technology Assessment (EU HTA) Methods: An online pre-convention survey was developed addressing four areas related to the EU HTA: (i) medical societies’ role; (ii) role of clinical guidelines; (iii) interface with the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS); and (iv) approaching ‘best-available evidence’ (BAE). A descriptive analysis of questionnaire outcomes was conducted to inform the European Access Academy (EAA) Fall Convention 2023. Within the working groups (WGs), action points were identified and prioritised. Results: A total of 57 experts from 15 countries responded to the survey. The WGs were attended by (i) 11, (ii) 10, (iii) 12, and (iv) 12 experts, respectively, representing a variety of national backgrounds and stakeholder profiles. The most relevant action points identified were as follows: (i) incorporation of clinical context into population, intervention, comparator, outcomes (PICO) schemes, (ii) timely provision of up-to-date therapeutic guidelines, (iii) ensuring the inclusion of MCBS insights into the EU HTA process, and (iv) considering randomized controlled trials (RCTs) as the gold standard and leveraging regulatory insights if development programs only include single-arm trials. Conclusions: The involvement of medical societies is a critical success factor for the EU HTA. The identified key action points foster the involvement of patient associations and medical societies.
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Open AccessCommunication
Future Possible Changes in Medically Underserved Areas in Japan: A Geographic Information System-Based Simulation Study
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Akihisa Nakamura, Eiji Satoh, Tatsuya Suzuki, Soichi Koike and Kazuhiko Kotani
J. Mark. Access Health Policy 2024, 12(2), 118-127; https://doi.org/10.3390/jmahp12020010 - 3 Jun 2024
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Background: A decrease in populations could affect healthcare access and systems, particularly in medically underserved areas (MUAs) where depopulation is becoming more prevalent. This study aimed to simulate the future population and land areas of MUAs in Japan. Methods: This study covered 380,948
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Background: A decrease in populations could affect healthcare access and systems, particularly in medically underserved areas (MUAs) where depopulation is becoming more prevalent. This study aimed to simulate the future population and land areas of MUAs in Japan. Methods: This study covered 380,948 1 km meshes, 87,942 clinics, and 8354 hospitals throughout Japan as of 2020. The areas outside a 4 km radius of medical institutions were considered as MUAs, based on the measure of areas in the current Japanese Medical Care Act. Based on the population estimate for a 1 km mesh, the population of mesh numbers of MUAs was predicted for every 10 years from 2020 to 2050 using geographic information system analysis. If the population within a 4 km radius from a medical institution fell below 1000, the institution was operationally assumed to be closed. Results: The number of MUAs was predicted to decrease from 964,310 (0.77% of the total Japanese population) in 2020 to 763,410 (0.75%) by 2050. By 2050, 48,105 meshes (13% of the total meshes in Japan) were predicted to be new MUAs, indicating a 31% increase in MUAs from 2020 to 2050. By 2050, 1601 medical institutions were tentatively estimated to be in close proximity. Conclusions: In Japan, the population of MUAs will decrease, while the land area of MUAs will increase. Such changes may reform rural healthcare policy and systems.
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Open AccessArticle
Real-World Evidence to Reinforce Clinical Trial Evidence in Health Technology Assessment: A Critical Review of Real-World Evidence Requirements from Seven Countries and Recommendations to Improve Acceptance
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Katia Thokagevistk, Céline Coppo, Laetitia Rey, Amanda Carelli, Veronica Díez, Sarah Vaselenak, Liana Oliveira, Ajay Patel, Emilia Sicari, Teresa Ramos, Susanne Schach, Erika Schirghuber, Alex Simpson, Remy Choquet and Katell Le Lay
J. Mark. Access Health Policy 2024, 12(2), 105-117; https://doi.org/10.3390/jmahp12020009 - 20 May 2024
Abstract
Background: Real-world evidence (RWE) can reinforce clinical trial evidence in health technology assessment (HTA). Objectives: Review HTA bodies’ (HTAbs) requirements for RWE, real uses, and acceptance across seven countries (Brazil, Canada, France, Germany, Italy, Spain, and the United Kingdom) and outline recommendations that
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Background: Real-world evidence (RWE) can reinforce clinical trial evidence in health technology assessment (HTA). Objectives: Review HTA bodies’ (HTAbs) requirements for RWE, real uses, and acceptance across seven countries (Brazil, Canada, France, Germany, Italy, Spain, and the United Kingdom) and outline recommendations that may improve acceptance of RWE in efficacy/effectiveness assessments and appraisals processes. Methods: RWE requirements were summarized based on HTAbs’ guidelines. Acceptance by HTAbs was evaluated based on industry experience and case studies. Results: As of June 2022, RWE methodological guidelines were in place in three of the seven countries. HTAbs typically requested analyses based on local data sources, but the preferred study design and data sources differed. HTAbs had individual submission, assessment, and appraisal processes; some allowed early meetings for the protocol and/or results validation, though few involved external experts or medical societies to provide input to assessment and appraisal. The extent of submission, assessment, and appraisal requirements did not necessarily reflect the degree of acceptance. Conclusion: All the countries reviewed face common challenges regarding the use of RWE. Our proposals address the need to facilitate collaboration and communication with industry and regulatory agencies and the need for specific guidelines describing RWE design and criteria of acceptance throughout the assessment and appraisal processes.
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Open AccessOpinion
EU HTA Regulation and Joint Clinical Assessment—Threat or Opportunity?
by
Volker Schuster
J. Mark. Access Health Policy 2024, 12(2), 100-104; https://doi.org/10.3390/jmahp12020008 - 13 May 2024
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The vision of a unified European HTA is by no means a new endeavor. At its core are the publicly declared ambitions to harmonize assessments of clinical data within the EU and avoid the duplication of efforts. Not surprisingly, these ambitions are publicly
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The vision of a unified European HTA is by no means a new endeavor. At its core are the publicly declared ambitions to harmonize assessments of clinical data within the EU and avoid the duplication of efforts. Not surprisingly, these ambitions are publicly announced to be motivating the new 2022 EU HTA regulation. However, industry experts typically see more of a risk for additional bureaucracy resulting in delays, further scrutiny, and one additional EU (clinical) dossier to submit on top of all national HTA dossiers, which could be considered a duplication of effort and therefore counterproductive. Regardless of how the details of the process will be defined and how the entire process will work in practice, we can be sure that EU officials will refer to the EU HTA and Joint Clinical Assessment (JCA) in particular as a learning system. The purpose of this article is to take a closer look at the new EU HTA regulation and analyze threats and opportunities for manufacturers and what the resulting opportunities and threats will be at the affiliate level throughout the EU.
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Open AccessReview
Conceptualisation and Role of Market Access in Pharmaceutical Industry: A Scoping Review
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Clara Fatoye, Gillian Yeowell, Eula Miller, Isaac Odeyemi and Chidozie Mbada
J. Mark. Access Health Policy 2024, 12(2), 81-99; https://doi.org/10.3390/jmahp12020007 - 1 May 2024
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Background: Understanding the concept and dynamic process of the evolution of professional identity and roles of market access (MA) in the pharmaceutical industry (pharma) is critical to personal, interpersonal, and professional levels of development and impact. Objective: The aim was to carry out
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Background: Understanding the concept and dynamic process of the evolution of professional identity and roles of market access (MA) in the pharmaceutical industry (pharma) is critical to personal, interpersonal, and professional levels of development and impact. Objective: The aim was to carry out a scoping review of the conceptualisation of MA within pharma. Data Sources: BioMed Central, WorldCat.org, and Directory of Open Access Journals were searched from 2003 to 2023. Study Selection: All articles on concepts or definitions and other surrogate terms on MA in pharma were selected. Data Extraction: Keywords generated from an initial cursory literature search on MA in pharma were used in conjunction with AND/OR as search terms. Using the data charting method, key findings were mapped and summarised descriptively. inductive analysis was performed, allowing codes/themes that are relevant to the concept to emerge. Data Synthesis: Arskey and O’Malley’s six-stage framework and the PRISMA extension for scoping reviews extension checklist were used as the review and reporting templates. The databases search yielded 222 results. Following title and abstract screening, a total of 146 papers were screened, and 127 of them were excluded. Full-text review was conducted for 19 papers that were deemed by two reviewers to meet the eligibility criteria. One of the authors arbitrated on disputed papers for inclusion. Only 14 of the included papers were found to meet the criteria for the final analysis. Five conceptual dimensions of MA in pharma were identified as “right products”, “right patient”, “right price”, “right point” (time), and “right place” (setting). Conclusions: Market access in pharma is a process that commences with the development and availability of the right products that are proven to be efficacious and disease/condition-specific (including medications, medical devices, and vaccines); specifically produced for the right patients or end users who will maximise best clinical outcomes and economic value; delivered at the right point in a timely, sustained, and efficient manner, given at the right price (commercially viable or reimbursed price that represents good value); and conducted within the economic, policy, societal, and technological contexts, with the overarching goal of achieving the best patient outcomes and ensuring product profitability.
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Open AccessSystematic Review
Methods for Indirect Treatment Comparison: Results from a Systematic Literature Review
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Bérengère Macabeo, Arthur Quenéchdu, Samuel Aballéa, Clément François, Laurent Boyer and Philippe Laramée
J. Mark. Access Health Policy 2024, 12(2), 58-80; https://doi.org/10.3390/jmahp12020006 - 16 Apr 2024
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Introduction: Health technology assessment (HTA) agencies express a clear preference for randomized controlled trials when assessing the comparative efficacy of two or more treatments. However, an indirect treatment comparison (ITC) is often necessary where a direct comparison is unavailable or, in some cases,
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Introduction: Health technology assessment (HTA) agencies express a clear preference for randomized controlled trials when assessing the comparative efficacy of two or more treatments. However, an indirect treatment comparison (ITC) is often necessary where a direct comparison is unavailable or, in some cases, not possible. Numerous ITC techniques are described in the literature. A systematic literature review (SLR) was conducted to identify all the relevant literature on existing ITC techniques, provide a comprehensive description of each technique and evaluate their strengths and limitations from an HTA perspective in order to develop guidance on the most appropriate method to use in different scenarios. Methods: Electronic database searches of Embase and PubMed, as well as grey literature searches, were conducted on 15 November 2021. Eligible articles were peer-reviewed papers that specifically described the methods used for different ITC techniques and were written in English. The review was performed in accordance with the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines. Results: A total of 73 articles were included in the SLR, reporting on seven different ITC techniques. All reported techniques were forms of adjusted ITC. Network meta-analysis (NMA) was the most frequently described technique (in 79.5% of the included articles), followed by matching-adjusted indirect comparison (MAIC) (30.1%), network meta-regression (24.7%), the Bucher method (23.3%), simulated treatment comparison (STC) (21.9%), propensity score matching (4.1%) and inverse probability of treatment weighting (4.1%). The appropriate choice of ITC technique is critical and should be based on the feasibility of a connected network, the evidence of heterogeneity between and within studies, the overall number of relevant studies and the availability of individual patient-level data (IPD). MAIC and STC were found to be common techniques in the case of single-arm studies, which are increasingly being conducted in oncology and rare diseases, whilst the Bucher method and NMA provide suitable options where no IPD is available. Conclusion: ITCs can provide alternative evidence where direct comparative evidence may be missing. ITCs are currently considered by HTA agencies on a case-by-case basis; however, their acceptability remains low. Clearer international consensus and guidance on the methods to use for different ITC techniques is needed to improve the quality of ITCs submitted to HTA agencies. ITC techniques continue to evolve quickly, and more efficient techniques may become available in the future.
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Open AccessArticle
Cost-Effectiveness Analysis of Innovative Therapies for Patients with Non-Alcoholic Fatty Liver Disease
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Michal Pochopien, Jakub Wladyslaw Dziedzic, Samuel Aballea, Emilie Clay, Iwona Zerda, Mondher Toumi and Borislav Borissov
J. Mark. Access Health Policy 2024, 12(2), 35-57; https://doi.org/10.3390/jmahp12020005 - 2 Apr 2024
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Objective: Currently there are no disease-specific approved therapies for non-alcoholic fatty liver (NAFL) and non-alcoholic steatohepatitis (NASH); however, several treatments are under development. This study aimed to estimate the cost-effectiveness of hypothetical innovative therapies compared with lifestyle intervention alone and combined with pioglitazone,
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Objective: Currently there are no disease-specific approved therapies for non-alcoholic fatty liver (NAFL) and non-alcoholic steatohepatitis (NASH); however, several treatments are under development. This study aimed to estimate the cost-effectiveness of hypothetical innovative therapies compared with lifestyle intervention alone and combined with pioglitazone, and assess the health economic consequences of their future availability for patients. Methods: A Markov cohort model was developed, considering fourteen disease health states and one absorbing state representing death. Transition probabilities, costs, utilities, and treatment efficacy were based on published data and assumptions. Four treatment strategies were considered, including two existing therapies (lifestyle intervention, small molecule treatment) and two hypothetical interventions (biological and curative therapy). The analysis was performed from the US third-party payer perspective. Results: The curative treatment with the assumed efficacy of 70% of patients cured and assumed price of $500,000 was the only cost-effective option. Although it incurred higher costs (a difference of $188,771 vs. lifestyle intervention and $197,702 vs. small molecule), it generated more QALYs (a difference of 1.58 and 1.38 QALYs, respectively), resulting in an ICER below the willingness-to-pay threshold of $150,000 per QALY. The sensitivity analyses showed that the results were robust to variations in model parameters. Conclusions: This study highlighted the potential benefits of therapies aimed at curing a disease rather than stopping its progression. Nonetheless, each of the analyzed therapies could be cost-effective compared with lifestyle intervention at a relatively high price.
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Open AccessArticle
An Inclusive Civil Society Dialogue for Successful Implementation of the EU HTA Regulation: Call to Action to Ensure Appropriate Involvement of Stakeholders and Collaborators
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Thomas Desmet, Elaine Julian, Walter Van Dyck, Isabelle Huys, Steven Simoens, Rosa Giuliani, Mondher Toumi, Christian Dierks, Juliana Dierks, Antonella Cardone, Francois Houÿez, Mira Pavlovic, Michael Berntgen, Peter Mol, Anja Schiel, Wim Goettsch, Fabrizio Gianfrate, Stefano Capri, James Ryan, Pierre Ducournau, Oriol Solà-Morales and Jörg Ruofadd
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J. Mark. Access Health Policy 2024, 12(1), 21-34; https://doi.org/10.3390/jmahp12010004 - 14 Mar 2024
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Objectives: Stakeholder involvement has long been considered a success factor for a joint European health technology assessment (HTA) process, and its relevance is now anchored in the EU HTA Regulation’s (EU HTAR) legislative wording. Therefore, we aimed to explore the roles, challenges, and
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Objectives: Stakeholder involvement has long been considered a success factor for a joint European health technology assessment (HTA) process, and its relevance is now anchored in the EU HTA Regulation’s (EU HTAR) legislative wording. Therefore, we aimed to explore the roles, challenges, and most important activities to increase the level of involvement per stakeholder group. Methods: At the 2022 Fall Convention of the European Access Academy (EAA), working groups addressed the involvement of patients, clinicians, regulators, health technology developers (HTD), and national HTA bodies and payers within the EU HTA process. Each working group revisited the pre-convention survey results, determined key role characteristics for each stakeholder, and agreed on the most important activities to fulfill the role profile. Finally, the activities suggested per group were prioritized by plenary group. Results: The prioritized actions for patients included training and capacity building, the establishment of a patient involvement committee, and the establishment of a patient unit at the EC secretariat. For clinicians, it included alignment on evidence assessment from a clinical vs. HTA point of view, capacity building, and standardization of processes. The most important actions for regulators are to develop joint regulatory-HTA guidance documents, align processes and interfaces under the regulation, and share discussions on post-licensing evidence generation. HTDs prioritized scientific advice capacity and the review of the scoping process, and further development of the scope of the assessment report fact checks. The top three actions for national HTA bodies and payers included clarification on the early HTD dialogue process, political support and commitment, and clarification on financial support. Conclusions: Addressing the activities identified as the most important for stakeholders/collaborators in the EU HTA process (e.g., in the implementation of the EU HTA Stakeholder Network and of the guidance documents developed by the EUnetHTA 21 consortium) will be key to starting an “inclusive civil society dialogue”, as suggested by the European Commission’s Pharmaceutical Strategy.
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Guest Editors: Jörg Ruof, Mondher ToumiDeadline: 31 December 2024