Among stakeholders and decision-makers in advanced breast cancer, the demand for insights from real-world data (RWD) is increasing. Although RWD can be used to support decisions throughout different ...stages of a breast cancer drug's life cycle, barriers exist to its use and acceptance. We propose a collaborative approach to generating and using RWD that is meaningful to multiple stakeholders, and encourage frameworks toward international guidelines to help standardize RWD methodologies to achieve more efficient use of RWD insights.
The cost-effectiveness of childhood obesity prevention interventions is critical for their sustained implementation. This study evaluated the cost-effectiveness of the Educació en Alimentació (EdAl) ...program, a school-based intervention for reducing obesity.
Total EdAl program implementation costs and per-child costs were estimated. Cost-effectiveness, defined using the incremental cost-effectiveness ratio (ICER), was estimated as the difference between the intervention and control group costs divided by the obesity-related outcome effects for boys (avoided cases of obesity, obesity prevalence, body mass index BMI, and BMI z-score units) for each group. As a significant difference (4.39%) in the reduction of obesity prevalence between the intervention and control groups was observed for boys in the EdAl program, the data were calculated only for boys.
The intervention cost was 24,246.53 € for 1,550 children (15.64 €/child/3 years) or 5.21 €/child/year. The ICERs/boy were 968.66 € to avoid one case of obesity, 3.6 € to reduce the obesity prevalence by 1%, 44.68 € to decrease BMI by one unit, and 65.16 € to reduce the BMI z-score by one unit.
The cost of reducing the obesity prevalence in boys by 4.39% was 5.21 €/child/year, half the cost proposed by the Spanish Health Ministry, indicating that the EdAl program is cost-effective.
This is one of a series of articles that consider the barriers to optimal use of real-world evidence (RWE) in health technology assessment and how to overcome them. The work was performed as part of ...EUreccA 2025, in particular with the RWE workstream embodied within that collaboration. Elsewhere in this issue we described the reasoning and process that led us to develop practical tools to support RWE use, including this taxonomy and explained the methods used to do so. The taxonomy classifies questions that are typically addressed using real-world data in health technology assessment and the data sources typically used to address these questions. In this article, we describe the taxonomy itself. For as many of the pairings as possible, we have provided links to advice and methods on how to address the associated question using those data. We have also provided links to examples of RWE use in practical decision making to answer the questions posed. Our work is not complete, but we believe it is sufficient to demonstrate the value of such a taxonomy and information source if it is completed and curated as a “wiki” by the community that would use it.
In the past decade, there have been increasing calls for greater use of real-world evidence (RWE) and data (RWD), with the explicit goal of enabling faster provision of effective medicines to ...patients in need. The push for decision makers to accept RWE is especially noticeable in the pursuit of regulatory approval, but RWE, particularly when used to estimate the relative effectiveness of interventions, is not always readily accepted by agencies responsible for reimbursement and pricing of new pharmaceuticals and, to a varying degree, is not accepted across jurisdictions. This lack of trust hampers the use of RWE despite a very large and growing literature base on the principles of how RWE should be used. In this article, we suggest an important part of the explanation of why this situation has arisen and make suggestions for its alleviation. Given that problems commonly arise that are particular to the question being asked and the data sources being used, general guidance on the principles of how to use RWD cannot cover all eventualities. Therefore, we are suggesting the creation of an archive, or repository, to record uses of RWD in support of decisions by funding bodies or their advisors. This article introduces a proposed, structured classification of decision types using RWE, around which evidence can be assembled in a curated source (RWD/RWE taxonomy) and thus facilitate judgments on when evidence is “good enough.” This article is part of a series in a special issue of this journal that looks at the barriers to optimal use of RWE in health technology assessment and how to overcome them. We begin significantly to populate our “taxonomy” with examples in an accompanying article. We also propose recommendations for international standards of evaluating the acceptability of RWD governance practices.
This is one of a series of articles that consider the barriers to optimal use of real-world evidence (RWE) in health technology assessment (HTA) as well as ways to overcome them. The work was carried ...out as part of EUreccA 2025 (European Initiative for New Reimbursement and Access Approaches 2025), in particular with the RWE workstream embodied within that collaboration. The starting premises of this workstream were as follows: (1) the acceptance of RWE by HTA agencies and payers in the assessment of drugs is suboptimal and variable between jurisdictions, and (2) if that were not the case, the path of new pharmaceuticals to patients could be quicker and less expensive. Elsewhere in this issue we set out the conclusions we had reached in the EUreccA RWE workstream. In this article, we set out the methodology used to conduct the totality of the EureccA 2025 RWE workstream effort, which led us to those conclusions. The main results, strengths, and limitations of the individual parts are discussed further in separate articles in this supplement. Through scoping work, we generated 4 key topics within which to identify and address the barriers to optimal RWE use in HTA. Through pragmatic literature searches, stakeholder engagement, and case studies, we suggest ways in which the problems identified may be addressed as a contribution to progress in this area.
•Real-world evidence is not always readily accepted by agencies responsible for reimbursement and pricing for new pharmaceuticals.•This article describes how the researchers tackled the vast literature base and used key stakeholder engagement and case studies to produce recommendations.•Using a pragmatic and iterative approach allowed us to develop practical tools in a timely manner, which can support improvements in this area.
Abstract
Introduction
Different remote interventions, such as applications (apps), have been used to continue promoting healthy ageing and preventing disability during the COVID-19 pandemic. The ...growing trend of apps in health is exponential and may facilitate scaling up physical activity prescription. Numerous tools are available, but little is known regarding their appropriateness, validation and recommendation, especially for frail older adults.
Methods
In-house, we developed an application that makes both the Apple app Store and the Google Play Store searchable using topic-related keywords and facilitates the extraction of basic app-information of the search results. The study was aimed at apps available to an English-speaking market. The resulting apps were filtered using various inclusion and exclusion criteria. The resultant apps underwent a more in-depth characterisation and searches for scientific publications on each app website and PubMed.
Results
From an initial search result of >2,800 apps, 459 met the initial inclusion criteria. After a more in-depth review of their features, 39 apps remained for possible app in older frail patients. After testing them, 22 apps were excluded. Seventeen apps fit the inclusion and exclusion criteria and were deemed appropriate after peer review. Of these, only one app, Vivifrail, had any type of publication/published evidence.
Conclusion
Apps can be valuable tool in prescribing exercise for frail older adults living in the community. However, few apps seem useful on a large scale, and there is limited evidence to support their effectiveness. It is important to invest in adapting Information and Communication Technologies to this population group.
Real-world data (RWD) and real-world evidence (RWE) can provide extensive information on healthcare for use in health technology assessment and decision making. Nevertheless, there is a lack of ...consensus surrounding the appropriate data governance (DG) practices for RWD/RWE. Data sharing is also a large concern, especially considering evolving data protection regulations. Our objective is to propose recommendations for international standards of evaluating the acceptability of RWD governance practices.
After reviewing the literature, we created a checklist targeting DG practices for RWD/RWE. We then carried out a 3-round Delphi panel, including European policy makers, health technology assessment experts, and hospital managers. The consensus for each statement was measured and the checklist adjusted accordingly.
The literature review identified the main topics regarding RWD/RWE DG practices: data privacy and security, data management and linkage, data access management, and the generation and use of RWE. Members of the Delphi panel (21 experts/25 invited) were presented a total of 24 statements related to each of the topics. Experts demonstrated a progressive level of consensus and importance ratings in all topics and to most statements. We suggest a refined checklist in which the statements rated less important or with less consensus have been removed.
This study suggests how the DG of RWD/RWE could be qualitatively evaluated. We propose checklists that could be used by all RWD/RWE users to help ensure the quality and integrity of RWD/RWE governance and complement data protection law.
The aim of this study was to investigate how innovation is defined with respect to new medicines.
MEDLINE, Embase, and EconLit databases were searched for articles published between January 1, 2010 ...and May 25, 2016 that described a relevant definition of innovation. Identified definitions were analyzed by mapping the concepts described onto a set of ten dimensions of innovation.
In total, thirty-six articles were included, and described a total of twenty-five different definitions of innovation. The most commonly occurring dimension was therapeutic benefit, with novelty and the availability of existing treatments the second and third most common dimensions. Overall, there was little agreement in the published literature on what characteristics of new medicines constitute rewardable innovation.
Alignment across countries and among regulators, health technology assessment bodies and payers would help manufacturers define research policies that can drive innovation, but may be challenging, as judgements about what aspects of innovation should be rewarded vary among stakeholders, and depend on political and societal factors.
The main objective of this work was to explore and characterize the current landscape of mobile applications available to treat mood disorders such as depression, bipolar disorder, and dysthymia.
We ...developed a tool that makes both the Apple App Store and the Google Play Store searchable using keywords and that facilitates the extraction of basic app information of the search results. All app results were filtered using various inclusion and exclusion criteria. We characterized all resultant applications according to their technical details. Furthermore, we searched for scientific publications on each app's website and PubMed, to understand whether any of the apps were supported by any type of scientific evidence on their acceptability, validation, use, effectiveness, etc. Results: Thirty apps were identified that fit the inclusion and exclusion criteria. The literature search yielded 27 publications related to the apps. However, these did not exclusively concern mood disorders. 6 were randomized studies and the rest included a protocol, pilot-, feasibility, case-, or qualitative studies, among others. The majority of studies were conducted on relatively small scales and 9 of the 27 studies did not explicitly study the effects of mobile application use on mental wellbeing.
While there exists a wealth of mobile applications aimed at the treatment of mental health disorders, including mood disorders, this study showed that only a handful of these are backed by robust scientific evidence. This result uncovers a need for further clinically oriented and systematic validation and testing of such apps.
The European Regulation on Health Technology Assessment (EU HTA R), effective since January 2022, aims to harmonize and improve the efficiency of common HTA across Member States (MS), with a phased ...implementation from January 2025. At "midterms" of the preparation phase for the implementation of the Regulation our aim was to identify and prioritize tangible action points to move forward. During the 2023 Spring Convention of the European Access Academy (EAA), participants from different nationalities and stakeholder backgrounds discussed readiness and remaining challenges for the Regulation's implementation and identified and prioritized action points. For this purpose, participants were assigned to four working groups: (i) Health Policy Challenges, (ii) Stakeholder Readiness, (iii) Approach to Uncertainty and (iv) Challenges regarding Methodology. Top four action points for each working group were identified and subsequently ranked by all participants during the final plenary session. Overall "readiness" for the Regulation was perceived as neutral. Prioritized action points included the following: Health Policy, i.e. assess adjustability of MS laws and health policy processes; Stakeholders, i.e. capacity building; Uncertainty, i.e. implement HTA guidelines as living documents; Methodology, i.e. clarify the Population, Intervention, Comparator(s), Outcomes (PICO) identification process. At "midterms" of the preparation phase, the focus for the months to come is on executing the tangible action points identified at EAA's Spring Convention. All action points centre around three overarching themes: harmonization and standardization, capacity building and collaboration, uncertainty management and robust data. These themes will ultimately determine the success of the EU HTA R in the long run.
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