Journal of Market Access & Health Policy

10 pages, 3602 KiB

Open AccessCommunication

Future Possible Changes in Medically Underserved Areas in Japan: A Geographic Information System-Based Simulation Study

by 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

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 [...] Read more.

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. Full article

► Show Figures

Figure 1

13 pages, 311 KiB

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

by 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 [...] Read more.

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. Full article

5 pages, 422 KiB

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

Abstract

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 [...] Read more.

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. Full article

► Show Figures

Figure 1

19 pages, 488 KiB

Open AccessReview

Conceptualisation and Role of Market Access in Pharmaceutical Industry: A Sco** Review

by 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

Abstract

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 [...] Read more.

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 sco** 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 sco** 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. Full article

► Show Figures

Figure 1

23 pages, 1482 KiB

Open AccessSystematic Review

Methods for Indirect Treatment Comparison: Results from a Systematic Literature Review

by 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

Abstract

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, [...] Read more.

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. Full article

► Show Figures

Figure 1

23 pages, 3449 KiB

Open AccessArticle

Cost-Effectiveness Analysis of Innovative Therapies for Patients with Non-Alcoholic Fatty Liver Disease

by 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

Abstract

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, [...] Read more.

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 stop** its progression. Nonetheless, each of the analyzed therapies could be cost-effective compared with lifestyle intervention at a relatively high price. Full article

► Show Figures

Figure 1

14 pages, 1076 KiB

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

by 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 Ruof Show full author list Hide full author list

J. Mark. Access Health Policy 2024, 12(1), 21-34; https://doi.org/10.3390/jmahp12010004 - 14 Mar 2024

Abstract

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 [...] Read more.

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 sco** 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. Full article

► Show Figures

Figure 1

16 pages, 1182 KiB

Open AccessArticle

Comparative Clinical Effectiveness and Cost-Effectiveness of the Cochlear Osia System and Baha Attract System in Patients with Conductive or Mixed Hearing Loss or Single-Sided Deafness

by Matthias Brunner, Manjula Schou, Robert J. Briggs and Dell Kingsford Smith

J. Mark. Access Health Policy 2024, 12(1), 5-20; https://doi.org/10.3390/jmahp12010003 - 6 Mar 2024

Abstract

The aim of this study was to evaluate the comparative clinical effectiveness and cost-utility of the active transcutaneous Osia^® System versus the passive transcutaneous Baha^® Attract System for patients with conductive or mixed hearing loss or single-sided deafness in an Australian [...] Read more.

The aim of this study was to evaluate the comparative clinical effectiveness and cost-utility of the active transcutaneous Osia^® System versus the passive transcutaneous Baha^® Attract System for patients with conductive or mixed hearing loss or single-sided deafness in an Australian healthcare setting. In the absence of direct comparative evidence, an indirect treatment comparison (ITC) of the clinical effectiveness and utility gains was needed. The ITC was informed by three studies identified through a systematic literature review. A Markov model was developed to evaluate the cost-utility of the Osia System. The literature review identified three studies suitable to inform an ITC: Mylanus et al. 2020 and Briggs et al. 2022 (Osia System) and den Besten et al. 2019 (Baha Attract System). The Osia System was found to be clinically superior to the Baha Attract System, across objective audiological outcomes resulting in a clinically meaningful utility benefit of 0.03 measured by the Health Utility Index with at least equivalent safety. In conclusion, the Osia System is more effective than the Baha Attract System, providing better hearing and health-related quality of life outcomes. In an Australian healthcare setting, the Osia System is cost-effective as demonstrated in a cost-utility analysis versus the Baha Attract System. Full article

► Show Figures

Figure 1

3 pages, 145 KiB

Open AccessEditorial

Welcome to the Journal of Market Access and Health Policy under MDPI Wings

by Mondher Toumi

J. Mark. Access Health Policy 2024, 12(1), 2-4; https://doi.org/10.3390/jmahp12010002 - 1 Jan 2024

Abstract

The Journal of Market Access and Health Policy (JMAHP) is the first peer-reviewed open access journal that focuses on the concept of ‘market access’ from economic, technical, scientific, sociological, anthropological, psychological, and policy perspectives [...] Full article

1 pages, 172 KiB

Open AccessEditorial

Publisher’s Note: A New Chapter for the Journal of Market Access and Health Policy (JMAHP)—Continued Publication by MDPI

by Clàudia Aunós

J. Mark. Access Health Policy 2024, 12(1), 1; https://doi.org/10.3390/jmahp12010001 - 1 Jan 2024

Abstract

The Journal of Market Access and Health Policy (JMAHP) is an open access peer-reviewed publication focused on Market Access and its sub-dimensions [...] Full article

1341 KiB

Open AccessEssay

Discounting Health Gain: A Different View

by Baudouin Standaert and Olivier Ethgen

J. Mark. Access Health Policy 2023, 11(1), 2275350; https://doi.org/10.1080/20016689.2023.2275350 - 2 Nov 2023

Abstract

ABSTRACT At least since the Age of Enlightenment, good health has been a tenet for society. Healthy societies could learn better, work harder, improve their wealth, and live longer. Today societies focus on life expectancy, as we value long and healthy lives. As [...] Read more.

ABSTRACT At least since the Age of Enlightenment, good health has been a tenet for society. Healthy societies could learn better, work harder, improve their wealth, and live longer. Today societies focus on life expectancy, as we value long and healthy lives. As illustrated by the provision of COVID-19 vaccines first for the elderly, societies value life-saving actions. Paradoxically, health economic assessments conventionally devalue long-lasting health through the practice of discounting health benefits along with costs. However, health, with its intrinsic and instrumental characteristics, is not synonymous with money cash, a tradeable asset that devalues with time. If improving healthy life expectancy is a societal ambition, it seems counter-intuitive to value future health less as a result of an artificial mathematical construct when evaluating economically new medical interventions. In this paper, we investigate the application of discounting health in healthcare and consider paradoxical findings, especially in relation to disease prevention with vaccination. We argue that there is no economically sustainable argument to discount health gains, except for the benefit of the payer with a goal of spending less on life-saving products. If that is the objective for discounting health, there are other means to achieve the same goal in a more transparent and simpler way. From the long-term perspective of healthcare development, not discounting health gains would encourage research that values long-term effects. This in turn has the potential to benefit the investor, the payer, and the patient/consumer, improving the situation from multiple perspectives. Full article

3082 KiB

Open AccessArticle

Observational Study on the Therapeutic Management and Economic Burden of Adult Patients with Moderate to Severe Plaque Psoriasis in France—The POP Study

by A. P. Villani, N. Quiles Tsimaratos, A. Crochard, A. Gherardi, A. Panes, A. Schmidt, M. Hueber Kollen and I. Borget

J. Mark. Access Health Policy 2023, 11(1), 2270293; https://doi.org/10.1080/20016689.2023.2270293 - 1 Nov 2023

Abstract

ABSTRACT Background: Data on the therapeutic management and healthcare cost of moderate to severe psoriasis in France are scarce. Objective: To assess the therapeutic management and economic burden of patients with moderate to severe psoriasis. Setting: This is a retrospective observational [...] Read more.

ABSTRACT Background: Data on the therapeutic management and healthcare cost of moderate to severe psoriasis in France are scarce. Objective: To assess the therapeutic management and economic burden of patients with moderate to severe psoriasis. Setting: This is a retrospective observational study on the Generalist Beneficiaries Sample of the National Health Data System. Patients and outcome measures: Adults with moderate to severe psoriasis (with a topical vitamin D derivative followed by systemic treatment or hospitalization for psoriasis) were included and followed-up from 1 January 2009 to 31 December 2018. Patients were matched to controls without psoriasis. Patients’ characteristics and healthcare cost from the National Health Insurance’s (NHI) perspective were described. Results: Overall, 1,848 and 5,544 adults were included in the psoriatic and control cohorts, respectively. The most frequent treatments were methotrexate (18.5% to 21.4% of patients by year), phototherapy (29.9% in 2010 down to 6.2% in 2018), and acitretin (25.9% in 2010 down to 8.6% in 2018). Overall, 19% of patients used biotherapies. The mean healthcare costs reimbursed by NHI was €5,365/psoriatic patient (including €2,685 potentially attributable to psoriasis), which was twice as high as in controls. In both cohorts, healthcare costs increased over time. Conclusion: Moderate to severe psoriasis healthcare costs are high. Full article

4535 KiB

Open AccessArticle

Inappropriate Dosing of Direct Oral Anticoagulants: Findings from a Clinical Vignette Study and Physician Survey

by Ahmet Fuat, Emmanuel Ako, David Hargroves, Douglas Holden, Amrit Caleyachetty, Matthew Carter, James Harris, Carol Roberts, Nnanyelu Nzeakor, Burcu Vardar and Helen Williams

J. Mark. Access Health Policy 2023, 11(1), 2267327; https://doi.org/10.1080/20016689.2023.2267327 - 29 Oct 2023

Abstract

ABSTRACT Objective: Direct oral anticoagulants (DOACs) are first-line therapy for stroke prevention for 1.4 million atrial fibrillation (AF) patients in the UK. However, the rates of DOAC dosing below evidence-based recommendations are estimated between 9% and 22%. This study explores specific patient and [...] Read more.

ABSTRACT Objective: Direct oral anticoagulants (DOACs) are first-line therapy for stroke prevention for 1.4 million atrial fibrillation (AF) patients in the UK. However, the rates of DOAC dosing below evidence-based recommendations are estimated between 9% and 22%. This study explores specific patient and physician factors associated with prescribing inappropriate DOAC underdoses. Methods: DOAC-prescribing physicians within the UK completed both a clinical vignette survey, which contained 12 hypothetical patient profiles designed to replicate DOAC prescribing scenarios, and a physician survey to capture sociodemographic, clinical experience, and prescriber-related beliefs and motivations related to DOAC prescribing. Eight patient factors based on a literature search and an expert consultation process were varied within the vignettes. Associations between the prescribers’ dosing choices and patient factors were explored via multilevel logistic regression. The analysis is focused on the most frequently selected DOACs, apixaban and rivaroxaban, both of which have different dosing guidelines. Results: In all, 336 prescribers (69% male; 233/336) completed the survey, mostly general physicians (GPs) (45%) or cardiology specialists (36%) with a mean of 17.9 years’ experience. Most prescribers (73%; 244/336) inappropriately underdosed at least once; rates between GPs and specialists were nearly identical. Patient factors most strongly associated with apixaban inappropriate underdosing included a history of major bleeding and falls. For rivaroxaban, these were major bleeding and severe frailty. Only 32% (106/335) of prescribers reported DOAC dosing guidelines as the sole influence on their prescribing behaviour. Among prescribers who did not inappropriately underdose, greater prescribing confidence was aligned to increased perception of inappropriate underdose risk. Conclusions: Overall, patient factors such as major bleeding and severe frailty were found to be associated with inappropriate underdosing of apixaban and rivaroxaban. Furthermore, prescribers who were more confident in DOAC prescribing, and were more worried about the risk of stroke, were significantly less likely to inappropriately underdose. These findings suggest that all prescribers, regardless of speciality, may benefit from education and training to raise awareness of the risks associated with inappropriate DOAC underdosing. Full article

1138 KiB

Open AccessArticle

Women and Children’s Real-World Economic and Drug Indicators from 2015 to 2019

by Jun Zou, Che Zhang, Guohua Jia and Wei Lu

J. Mark. Access Health Policy 2023, 11(1), 2270297; https://doi.org/10.1080/20016689.2023.2270297 - 26 Oct 2023

Abstract

ABSTRACT Objectives: To obtain real-world data on rational drug use, pharmacoeconomic regarding women’s and children’s health and the benefits of Hainan free trade port (HFTP) health policies, we retrospectively investigated drug indicators, prescribing trend, and economic data. Method: We retrospectively gathered the data [...] Read more.

ABSTRACT Objectives: To obtain real-world data on rational drug use, pharmacoeconomic regarding women’s and children’s health and the benefits of Hainan free trade port (HFTP) health policies, we retrospectively investigated drug indicators, prescribing trend, and economic data. Method: We retrospectively gathered the data from the database of the hospital information system and the quality indicators of pharmacy; we compared the monthly indicators from 2015 to 2019. Results: In 2017, the HFTP maternal mortality ratio (MMR) was 24.46. In 2019, the HFTP infant mortality rate (‰) was 4.15, and the under-five mortality rate (‰) was 6.19. A total of 1,922,798 prescriptions included in the analysis, the defined daily dose of 2015–2019 ranged from 46.59 to 32.34. In 2019, the proportions of antibiotics prescribed in outpatient, emergency, and inpatient care were 14.19%, 16.68%, and 46.26%, respectively. The injection prescription percentage ranged from 13.08% to 8.08%. The proportion of medicine income to total hospital income of 2015–2019 ranged from 26.66% to 25.31%. Conclusion: According to the analysis of women’s and children’s real-world drug data, economic investment and strict quality control of antimicrobial stewardship programs can lead to the rational use of drugs. Full article

2048 KiB

Open AccessArticle

Ivabradine in Patients with Heart Failure: A Systematic Literature Review

by Zeba M. Khan, Jean Baptiste Briere, Elzbieta Olewinska, Fatma Khrouf and Mateusz Nikodem

J. Mark. Access Health Policy 2023, 11(1), 2262073; https://doi.org/10.1080/20016689.2023.2262073 - 4 Oct 2023

Abstract

ABSTRACT Background: Heart failure is a chronic disease linked with significant morbidity and mortality, and uncontrolled resting heart rate is a risk factor for adverse outcomes. This systematic literature review aimed to assess the efficacy, safety, and patient-reported outcomes (PROs) of ivabradine in [...] Read more.

ABSTRACT Background: Heart failure is a chronic disease linked with significant morbidity and mortality, and uncontrolled resting heart rate is a risk factor for adverse outcomes. This systematic literature review aimed to assess the efficacy, safety, and patient-reported outcomes (PROs) of ivabradine in patients with heart failure (HF) with reduced ejection fraction (HFrEF) in randomized controlled trials (RCTs) and observational studies. Methods: We searched electronic databases from their inception to July 2021 to include studies that reported on efficacy, safety, or PROs of ivabradine in patients with HFrEF. Results: Of 1947 records screened, 51 RCTs and 6 observational studies were identified. Ivabradine on top of background therapy demonstrated a significant reduction in composite outcomes including hospitalization for HF or cardiovascular death. In addition, observational studies suggested that ivabradine was associated with a significant reduction in mortality. Across all studies, ivabradine use on top of background therapy was associated with greater reductions in heart rate, improved EF, and improved health-related quality of life (QoL) and comparable risk of total adverse events compared to those treated with background therapy alone. Conclusions: Ivabradine on top of background therapy is beneficial for heart rate, hospitalization risk for HF, mortality, EF, and patients’ QoL. Moreover, these benefits were achieved with no significant increase in the overall risk of total adverse events. Full article

624 KiB

Open AccessArticle

Patient Perceptions of Copay Card Utilization and Policies

by Dimika Cavalier, Bridget Doherty, Gabrielle Geonnotti, Aarti Patel, Wesley Peters, Steven Zona and Lisa Shea

J. Mark. Access Health Policy 2023, 11(1), 2254586; https://doi.org/10.1080/20016689.2023.2254586 - 7 Sep 2023

Abstract

ABSTRACT Background: Copay cards are intended to mitigate patient out-of-pocket (OOP) expenses. This qualitative, exploratory focus group study aimed to capture patient perceptions of copay cards and copay adjustment programs (CAPs; insurers’ accumulator and maximizer policies), which redirect the copay card utilization benefits [...] Read more.

ABSTRACT Background: Copay cards are intended to mitigate patient out-of-pocket (OOP) expenses. This qualitative, exploratory focus group study aimed to capture patient perceptions of copay cards and copay adjustment programs (CAPs; insurers’ accumulator and maximizer policies), which redirect the copay card utilization benefits intended for patients’ OOP expenses. Methods: Patients with chronic conditions were recruited through Janssen’s Patient Engagement Research Council program. They completed a survey and attended a live virtual session to provide feedback on copay cards. Results: Among 33 participants (median age, 49 years [range, 24–78]), the most frequent conditions were cardiovascular-metabolic disease and inflammatory bowel disease. Patients associated copay cards with lessening financial burden, improving general and mental health, and enabling medication adherence. An impact on medication adherence was identified by 10 (63%) White and nine (100%) Black respondents. Some patients were unaware of CAPs despite having encountered them; they recommended greater copay card education and transparency about CAPs. Conclusion: Patients relied on copay cards to help afford their prescribed medication OOP expenses and maintain medication adherence. Use of CAPs may increase patient OOP expenses. Patients would benefit from awareness programs and industry – healthcare provider partnerships that facilitate and ensure access to copay cards. Full article

4190 KiB

Open AccessArticle

Budget and Health Impact of Switching Eligible Patients with Atrial Fibrillation to Lower- Dose Dabigatran

by Tanja Fens, Lisa de Jong, Bregt Kappelhoff, Cornelis Boersma and Maarten J. Postma

J. Mark. Access Health Policy 2023, 11(1), 2247719; https://doi.org/10.1080/20016689.2023.2247719 - 4 Sep 2023

Abstract

ABSTRACT Objectives: To assess the comparative budget and health impact of lower-dose dabigatran versus reduced doses of apixaban and rivaroxaban in atrial fibrillation (AF) patients eligible for a lower-/reduced-dose due to individual patient characteristics in the Netherlands. Methods: A budget impact model was [...] Read more.

ABSTRACT Objectives: To assess the comparative budget and health impact of lower-dose dabigatran versus reduced doses of apixaban and rivaroxaban in atrial fibrillation (AF) patients eligible for a lower-/reduced-dose due to individual patient characteristics in the Netherlands. Methods: A budget impact model was developed in accordance with ISPOR guidelines. A 3-year-time horizon was considered, and analyses were conducted from a Dutch healthcare payer’s perspective. The model applies published data to local AF-epidemiology, allowing calculations to estimate clinical events (strokes and haemorrhages) and costs. The analyses were based on real-world outcomes from patients with AF receiving a first direct oral anticoagulant (DOAC) prescription for low-dose dabigatran (110 mg) and a reduced dose of apixaban (2.5 mg) or rivaroxaban (15 mg). Two situations of switching treatments from one to another DOAC were modelled: switching from apixaban to dabigatran and from rivaroxaban to dabigatran. Base case results were given as savings per 100 patient-year, per total Dutch population, and events avoided. A univariate sensitivity analysis was conducted to explore the uncertainty around epidemiological and event costs input data. Scenario analyses were performed to estimate the effect of different market shares and potential price reductions due to future patent expiry for the total real-world population from the Netherlands. Results: The 3-years outcomes of switching patients eligible for a lower-/reduced-dose due to individual patient characteristics from apixaban or rivaroxaban to dabigatran resulted in cost savings estimated at €157 or €72 thousand per 100 patient-years, respectively, or €146 million per total Dutch population. Looking into the clinical events, dabigatran reflected the lowest number of mortalities, ischemic strokes, major bleeding, non-major bleeding, and haemorrhagic stroke compared to apixaban and rivaroxaban. The sensitivity analysis consistently reflected cost savings, with the ischeamic stroke events having the biggest impact. Accounting for the Dutch situation, both scenarios showed total savings ranging from €45 to €229 million over 3 years. Conclusions: Switching eligible AF-patients from reduced-dose apixaban or rivaroxaban to lower-dose dabigatran has the potential to reduce healthcare payer’s budget expenditures and provide health gains. Cost savings can potentially be further enhanced by market share adjustments and further price reductions. Full article

372 KiB

Open AccessArticle

Systematic Literature Reviews over the Years

by Beata Smela, Mondher Toumi, Karolina Świerk, Konrad Gawlik, Emilie Clay and Laurent Boyer

J. Mark. Access Health Policy 2023, 11(1), 2244305; https://doi.org/10.1080/20016689.2023.2244305 - 21 Aug 2023

Cited by 1

Abstract

ABSTRACT Purpose: Nowadays, systematic literature reviews (SLRs) and meta-analyses are often placed at the top of the study hierarchy of evidence. The main objective of this paper is to evaluate the trends in SLRs of randomized controlled trials (RCTs) throughout the years. Methods: [...] Read more.

ABSTRACT Purpose: Nowadays, systematic literature reviews (SLRs) and meta-analyses are often placed at the top of the study hierarchy of evidence. The main objective of this paper is to evaluate the trends in SLRs of randomized controlled trials (RCTs) throughout the years. Methods: Medline database was searched, using a highly focused search strategy. Each paper was coded according to a specific ICD-10 code; the number of RCTs included in each evaluated SLR was also retrieved. All SLRs analyzing RCTs were included. Protocols, commentaries, or errata were excluded. No restrictions were applied. Results: A total of 7,465 titles and abstracts were analyzed, from which 6,892 were included for further analyses. There was a gradual increase in the number of annual published SLRs, with a significant increase in published articles during the last several years. Overall, the most frequently analyzed areas were diseases of the circulatory system (n = 750) and endocrine, nutritional, and metabolic diseases (n = 734). The majority of SLRs included between 11 and 50 RCTs each. Conclusions: The recognition of SLRs’ usefulness is growing at an increasing speed, which is reflected by the growing number of published studies. The most frequently evaluated diseases are in alignment with leading causes of death and disability worldwide. Full article

466 KiB

Open AccessReview

Pharmaceutical Pricing and Reimbursement Policies in Algeria, Morocco, and Tunisia: Comparative Analysis

by Hajer Dahmani, Ines Fradi, Leila Achour, Mondher Toumi and Maghreb Research Group

J. Mark. Access Health Policy 2023, 11(1), 2244304; https://doi.org/10.1080/20016689.2023.2244304 - 20 Aug 2023

Abstract

Objectives: In this paper, we outline and compare pharmaceutical pricing and reimbursement policies for in-patent prescription medicines in three Maghreb countries, Algeria, Morocco, and Tunisia, and explore possible improvements in their pricing and reimbursement systems. Methods: The evidence informing this study comes from [...] Read more.

Objectives: In this paper, we outline and compare pharmaceutical pricing and reimbursement policies for in-patent prescription medicines in three Maghreb countries, Algeria, Morocco, and Tunisia, and explore possible improvements in their pricing and reimbursement systems. Methods: The evidence informing this study comes from both an extensive literature review and a primary data collection from experts in the three studied countries. Key findings: Twenty-six local experts where interviewed Intervieweesincluded ministry officials, representatives of national regulatory authorities, health insurance organizations, pharmaceutical procurement departments and agencies, academics, private pharmaceutical-sector actors, and associations. Results show that External Reference Pricing (ERP) is the dominant pricing method for in-patent medicines in the studied countries. Value-based pricing through Health Technology Assessment (HTA) is a new concept, recently used in Tunisia to help the reimbursement decision of some in-patent medicines but not yet used in the pricing of innovative medicines in the studied countries. Reimbursement decision is mainly based on negotiations set on Internal Reference Pricing (IRP). Conclusion: Whereas each country has its specific regulations, there are many similarities in the pricing and reimbursement policies of in-patent medicines in Algeria, Morocco, and Tunisia. The ERP was found to be the dominant method to inform pricing and reimbursement decisions of in-patent medicines. Countries in the region can focus on the development of explicit value assessment systems and minimize their dependence on ERP over the longer-term. In this context, HTA will rely on local assessment of the evidence. Full article

Journal Description

Journal of Market Access & Health Policy

Latest Articles

Journal Menu

Journal Browser

Highly Accessed Articles

Latest Books

E-Mail Alert

News

Topics

Conferences

Special Issues

Further Information

Guidelines

MDPI Initiatives

Follow MDPI