Editor’s Choice Articles

Editor’s Choice articles are based on recommendations by the scientific editors of MDPI journals from around the world. Editors select a small number of articles recently published in the journal that they believe will be particularly interesting to readers, or important in the respective research area. The aim is to provide a snapshot of some of the most exciting work published in the various research areas of the journal.

Order results
Result details
Results per page
Select all
Export citation of selected articles as:
14 pages, 983 KiB  
Article
Opportunities and Challenges in Cross-Country Collaboration: Insights from the Beneluxa Initiative
by 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
Cited by 4 | Viewed by 1693
Abstract
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 [...] Read more.
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. Full article
Show Figures

Figure 1

5 pages, 422 KiB  
Opinion
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
Cited by 7 | Viewed by 1959
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

23 pages, 1482 KiB  
Systematic 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
Cited by 11 | Viewed by 4236
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

12 pages, 624 KiB  
Article
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
Cited by 5 | Viewed by 1335
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
7 pages, 234 KiB  
Review
Rapid Literature Review: Definition and Methodology
by Beata Smela, Mondher Toumi, Karolina Świerk, Clement Francois, Małgorzata Biernikiewicz, Emilie Clay and Laurent Boyer
J. Mark. Access Health Policy 2023, 11(1), 2241234; https://doi.org/10.1080/20016689.2023.2241234 - 28 Jul 2023
Cited by 39 | Viewed by 5306
Abstract
ABSTRACT Introduction: A rapid literature review (RLR) is an alternative to systematic literature review (SLR) that can speed up the analysis of newly published data. The objective was to identify and summarize available information regarding different approaches to defining RLR and the methodology [...] Read more.
ABSTRACT Introduction: A rapid literature review (RLR) is an alternative to systematic literature review (SLR) that can speed up the analysis of newly published data. The objective was to identify and summarize available information regarding different approaches to defining RLR and the methodology applied to the conduct of such reviews. Methods: The Medline and EMBASE databases, as well as the grey literature, were searched using the set of keywords and their combination related to the targeted and rapid review, as well as design, approach, and methodology. Of the 3,898 records retrieved, 12 articles were included. Results: Specific definition of RLRs has only been developed in 2021. In terms of methodology, the RLR should be completed within shorter timeframes using simplified procedures in comparison to SLRs, while maintaining a similar level of transparency and minimizing bias. Inherent components of the RLR process should be a clear research question, search protocol, simplified process of study selection, data extraction, and quality assurance. Conclusions: There is a lack of consensus on the formal definition of the RLR and the best approaches to perform it. The evidence-based supporting methods are evolving, and more work is needed to define the most robust approaches. Full article
12 pages, 807 KiB  
Article
The Role of Stakeholder Involvement in the Evolving EU HTA Process: Insights Generated through the European Access Academy’s Multi-Stakeholder Pre-Convention Questionnaire
by Lauren Van Haesendonck, Jörg Ruof, Thomas Desmet, Walter Van Dyck, Steven Simoens, Isabelle Huys, Rosa Giuliani, Mondher Toumi, Christian Dierks, Juliana Dierks, Antonella Cardone, Francois Houÿez, Mira Pavlovic, Michael Berntgen, Peter G.M. Mol, Anja Schiel, Wim Goettsch, Fabrizio Gianfrate, Stefano Capri, James Ryan, Pierre Ducournau, Oriol Solà-Morales and Elaine Julianadd Show full author list remove Hide full author list
J. Mark. Access Health Policy 2023, 11(1), 2217543; https://doi.org/10.1080/20016689.2023.2217543 - 4 Jun 2023
Cited by 8 | Viewed by 990
Abstract
ABSTRACT Involvement of all relevant stakeholders will be of utmost importance for the success of the developing EU HTA harmonization process. A multi-step procedure was applied to develop a survey across stakeholders/collaborators within the EU HTA framework to assess their current level of [...] Read more.
ABSTRACT Involvement of all relevant stakeholders will be of utmost importance for the success of the developing EU HTA harmonization process. A multi-step procedure was applied to develop a survey across stakeholders/collaborators within the EU HTA framework to assess their current level of involvement, determine their suggested future role, identify challenges to contribution, and highlight efficient ways to fulfilling their role. The ‘key’ stakeholder groups identified and covered by this research included: patients’, clinicians’, regulatory, and Health Technology Developer representatives. The survey was circulated to a wide expert audience including all relevant stakeholder groups in order to determine self-perception by the ‘key’ stakeholders regarding involvement in the HTA process (self-rating), and in a second, slightly modified version of the questionnaire, to determine the perception of ‘key’ stakeholder involvement by HTA bodies, payers, and policymakers (external rating). Predefined analyses were conducted on the submitted responses. Fifty-four responses were received (patients 9; clinicians: 8; regulators: 4; HTDs 14; HTA bodies: 7; Payers: 5; policymakers 3; others 4). The mean self-perceived involvement score was consistently lower for each of the ‘key’ stakeholder groups than the respective external ratings. Based on the qualitative insights generated in the survey, a RACI Chart (Responsible/Accountable/Consulted/Informed) was developed for each of the stakeholder groups to determine their roles and involvement in the current EU HTA process. Our findings suggest extensive effort and a distinct research agenda are required to ensure adequate involvement of the key stakeholder groups in the evolving EU HTA process. Full article
5 pages, 1063 KiB  
Article
Artificial Intelligence and Remote Patient Monitoring in US Healthcare Market: A Literature Review
by Ayushmaan Dubey and Anuj Tiwari
J. Mark. Access Health Policy 2023, 11(1), 2205618; https://doi.org/10.1080/20016689.2023.2205618 - 3 May 2023
Cited by 18 | Viewed by 1461
Abstract
ABSTRACT Background: Artificial intelligence (AI) enables remote patient monitoring (RPM) which reduces costs by triaging patients to optimize hospitalization and avoid complications. The FDA regulates AI in medical devices and aims to ensure patient safety, effectiveness, and transparent AI solutions. Objectives: Identify and [...] Read more.
ABSTRACT Background: Artificial intelligence (AI) enables remote patient monitoring (RPM) which reduces costs by triaging patients to optimize hospitalization and avoid complications. The FDA regulates AI in medical devices and aims to ensure patient safety, effectiveness, and transparent AI solutions. Objectives: Identify and summarize FDA approved RPM devices to provide information for the US medical device industry based on previous approvals and the markets’ needs. Methods: We searched publicly available databases on FDA-approved RPM devices. Selection criteria were established to classify a solution as AI. Technical information was analyzed on pre-identified 16 parameters for the qualified solutions. Results: A total of 47 RPM devices were reviewed, among which 12.8% were classified as a De Novo product and the remaining devices fell under the 510(K) FDA category. The cardiovascular (74%) AI RPM solutions dominated the US market, followed by ECG-based arrhythmia detection algorithms (59.4%), and Hemodynamics and Vital Sign monitoring algorithms (21.9%). The trend observed in the FDA rejected devices was their inability to be classified into clinically relevant categories (Criteria 2 and 3). Conclusion: The market needs more innovative RPM solutions under the De Novo category, as there are very few. The transparency is low on the technical aspect of AI algorithms. The market needs AI algorithms that can effectively classify patients rather than merely improve device functionality. Full article
3 pages, 149 KiB  
Article
Pricing Zolgensma—The World’s Most Expensive Drug
by Mark Nuijten
J. Mark. Access Health Policy 2022, 10(1), 2022353; https://doi.org/10.1080/20016689.2021.2022353 - 29 Dec 2022
Cited by 45 | Viewed by 1099
Abstract
A heated discussion has recently broken out in Europe about the price of Zolgensma, ‘the most expensive drug ever’ [...]
Full article
14 pages, 1564 KiB  
Article
Burden of Illness Associated with Pneumococcal Infections in Japan—A Targeted Literature Review
by Ataru Igarashi, Maki Ueyama, Koki Idehara and Mariko Nomoto
J. Mark. Access Health Policy 2022, 10(1), 2010956; https://doi.org/10.1080/20016689.2021.2010956 (registering DOI) - 27 Dec 2022
Cited by 8 | Viewed by 756
Abstract
Introduction: Pneumococcal diseases (PDs) are among the leading causes of mortality and morbidity worldwide. However, the evidence on epidemiology, health economic, and patient-reported outcomes has not been systematically reviewed and published in Japan. This study aimed to assess the burden, treatment adherence and [...] Read more.
Introduction: Pneumococcal diseases (PDs) are among the leading causes of mortality and morbidity worldwide. However, the evidence on epidemiology, health economic, and patient-reported outcomes has not been systematically reviewed and published in Japan. This study aimed to assess the burden, treatment adherence and compliance, and serotype distribution associated with PDs in Japan. Methods: One hundred and eight studies were identified between January 2005 and June 2020. The identified studies were mostly regional and with a limited scale, clinical settings, and populations. Results: In 2013–2017, invasive PD incidence rates were 4.98–9.47/100,000 in <4-year-olds, 0.36/100,000 in 5–14-year-olds, 0.46/100,000 in 15–64-year-olds, and 1.50–5.38/100,000 in the elderly. The incidence of invasive PDs in children decreased from 24.6/100,000 in 2008 to 10.7/100,000 in 2013 after the introduction of PCV7 and further declined to 10.3/100,000 in 2014 after PCV13 was introduced. From 2014, the prevalence of PCV13 serotypes decreased across all age groups along with a decrease of PPV23 serotypes, but an increase of PPV23 serotypes not included in PCV13 among adults and the elderly. No study reported health-related quality-of-life data for PDs. In children, direct costs were 340,905–405,978 JPY (3,099–3,691 USD) per pneumococcal bacteraemia, 767,447–848,255 JPY (6,977–7,711 USD) per pneumococcal meningitis, and 79,000 JPY (718 USD) per pneumococcal acute otitis media episodes. In adults and the elderly, the direct cost of pneumococcal pneumonia was 348,280–389,630 JPY (3,166–3,542 USD). The average hospital stay length was 7.2–31.9 days in children, 9.0 days in adults and 9.0–28.7 days in adults and the elderly. Conclusions: The epidemiological burden of PDs remains high in Japan, especially among children and the elderly with invasive PDs accounting for a very small proportion of all PDs. A significant impact of the PCV13 vaccine program was reported, while the PPV23’s impact remains unclear. A substantial decrease in quality-adjusted life years in adults and the elderly and a high economic burden may exist. Full article
13 pages, 923 KiB  
Article
Impact of the COVID-19 Pandemic on the Conduct of Clinical Trials: A Quantitative Analysis
by Wojciech Margas, Piotr Wojciechowski and Mondher Toumi
J. Mark. Access Health Policy 2022, 10(1), 2106627; https://doi.org/10.1080/20016689.2022.2106627 - 9 Aug 2022
Cited by 11 | Viewed by 582
Abstract
Background: Globally, healthcare has shouldered much of the socioeconomic brunt of the COVID-19 pandemic leading to numerous clinical trials suspended or discontinued. Objective: To estimate the COVID-19 impact on the number of clinical trials worldwide. Methods: Data deposited by 219 countries in [...] Read more.
Background: Globally, healthcare has shouldered much of the socioeconomic brunt of the COVID-19 pandemic leading to numerous clinical trials suspended or discontinued. Objective: To estimate the COVID-19 impact on the number of clinical trials worldwide. Methods: Data deposited by 219 countries in the ClinicalTrials.gov database (2007–2020) were interrogated using targeted queries. A time series model was fitted to the data for studies ongoing, initiated, or ended between 2007 Quarter (Q) 1 and 2019 Q4 to predict the expected trials number in 2020 in the COVID-19 absence. The predicted values were compared with the actual 2020 data to quantify the pandemic impact. Results: The ongoing registered trials number grew from 2007 Q1 (33,739) to 2019 Q4 (80,319). By contrast, there were markedly fewer ongoing trials in all four quarters of 2020 compared with forecasted values (1.6%–2.8% decrease). When excluding COVID-19-related studies, this disparity grew further (3.4%–5.8% decrease), to a peak of almost 5000 fewer ongoing trials than estimated for 2020 Q2. The initiated non-COVID-19 trials number was higher than predicted in 2020 Q4 (9.9%). Conclusions: This pandemic has impacted clinical trials. Provided that current trends persist, clinical trial activities may soon recover to at least pre-COVID-19 levels. Full article
Back to TopTop