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Drug repurposing leverages existing medicines to reduce time and costs for exploring new therapeutic indications. However, off-patent medicines do not provide enough private incentives for investment, and prescribing by active ingredient can be a major barrier. Academics and non-profit sponsors also face regulatory barriers. This study combined a structured literature review with a questionnaire completed by 25 drug repurposing experts to identify key challenges and policy solutions across the full lifecycle of off-patent drug repurposing. Public funding is generally available for the early-stage research, where opportunities often arise from off-label uses and real-world evidence. However, limited support for confirmatory clinical trials, combined with the predominance of academics and non-profit sponsors lacking regulatory objectives, reduces the likelihood of successful authorisation to below 30%. Policy solutions include prioritising research into rare diseases and other conditions with unmet health needs, and call for a more prominent role of governments in public investment and in reinforcement of public-private partnerships. Substitution in community pharmacies remains a major challenge, which may be addressed through indication-based differentiation and enhanced dispensing controls. Some innovative public intervention mechanisms, such as Social Impact Bonds and the InterVentional PharmacoEconomics proposal, offer potential, although real-world experience is still limited. Off-patent drug repurposing faces persistent financial and regulatory barriers that require a central role from academics and non-profit sponsors, supported by governmental engagement throughout the lifecycle. Although steps have been taken in recent years, efforts remain insufficient, and structural and political barriers continue to hinder broader implementation.

According to the most traditional economic evaluation manuals, all \"relevant\" costs should be included in the economic analysis, taking into account factors such as the patient population, setting, location, year, perspective and time horizon. However, cost information may be designed for other purposes. Health care organisations may lack sophisticated accounting systems and consequently, health economists may be unfamiliar with cost accounting terminology, which may lead to discrepancy in terms used in the economic evaluation literature and management accountancy. This paper identifies new tendencies in costing methodologies in health care and critically comments on each included article. For better clarification of terminology, a pragmatic glossary of terms is proposed. A scoping review of English and Spanish language literature (2005–2018) was conducted to identify new tendencies in costing methodologies in health care. The databases PubMed, Scopus and EconLit were searched. A total of 21 studies were included yielding 43 costing analysis. The most common analysis was top-down micro-costing (49%), followed by top-down gross-costing (37%) and bottom-up micro-costing (14%). Resource data were collected prospectively in 12 top-down studies (32%). Hospital database was the most common way of collection of resource data (44%) in top-down gross-costing studies. In top-down micro-costing studies, the most resource use data collection was the combination of several methods (38%). In general, substantial inconsistencies in the costing methods were found. The convergence of top-down and bottom-up methods may be an important topic in the next decades.

Background The aims of this research were to provide a better understanding of the specific evidence needs for assessment of clinical and cost-effectiveness of cell and gene therapies, and to explore the extent that the relevant categories of evidence are considered in health technology assessment (HTA) processes. Methods A targeted literature review was conducted to identify the specific categories of evidence relevant to the assessment of these therapies. Forty-six HTA reports for 9 products in 10 cell and gene therapy indications across 8 jurisdictions were analysed to determine the extent to which various items of evidence were considered. Results The items to which the HTA bodies reacted positively were: treatment was for a rare disease or serious condition, lack of alternative therapies, evidence indicating substantial health gains, and when alternative payment models could be agreed. The items to which they reacted negatively were: use of unvalidated surrogate endpoints, single arm trials without an adequately matched alternative therapy, inadequate reporting of adverse consequences and risks, short length of follow-up in clinical trials, extrapolating to long-term outcomes, and uncertainty around the economic estimates. Conclusions The consideration by HTA bodies of evidence relating to the particular features of cell and gene therapies is variable. Several suggestions are made for addressing the assessment challenges posed by these therapies. Jurisdictions conducting HTAs of these therapies can consider whether these suggestions could be incorporated within their existing approach through strengthening deliberative decision-making or performing additional analyses.

Introduction: Advanced Therapy Medicinal Products are a type of therapies that, in some cases, hold great potential for patients without an effective current therapeutic approach but they also present multiple challenges to payers. While there are many theoretical papers on pricing and reimbursement (P&R) options, original empirical research is very scarce. This paper aims to provide a comprehensive international review of regulatory and P&R decisions taken for all ATMPs with centralized European marketing authorization in March 2022. Methods: A survey was distributed in July 2022 to representatives of 46 countries. Results: Responses were received from 20 countries out of 46 (43.5%). 14 countries reimbursed at least one ATMP. Six countries in this survey reimbursed no ATMPs. Conclusion: Access to ATMPs is uneven across the countries included in this study. This arises from regulatory differences, commercial decisions by marketing authorization holders, and the divergent assessment processes and criteria applied by payers. Moving towards greater equality of access will require cooperation between countries and stakeholders, for example, through the WHO Regional Office for Europe’s Access to Novel Medicines Platform.

Background: What constitutes innovation in health technologies can be defined and measured in a number of ways and it has been widely researched and published about. However, while many countries mention it as a criterion for pricing or reimbursement of health technologies, countries differ widely in how they define and operationalise it. Methods: We performed a literature review, using a snowballing search. In this paper, we explore how innovation has been defined in the literature in relation to health technology assessment. We also describe how a selection of countries (England, France, Italy, Spain and Japan) take account of innovation in their health technology assessment frameworks and explore the key methodologies that can capture it as a dimension of value in a new health technology. We propose a way of coming to, and incorporating into health technology assessment systems, a definition of innovation for health technologies that is independent of other dimensions of value that they already account for in their systems, such as clinical benefit. We use Spain as an illustrative example of how innovation might be operationalised as a criterion for decision making in health technology assessment. Results: The countries analysed here can be divided into 2 groups with respect to how they define innovation. France, Japan and Italy use features such as severity, unmet need and therapeutic added value as indicators of the degree of innovation of a health technology, while England, Spain consider the degree of innovation as a separate and additional criterion from others. In the case of Spain, a notion of innovation might be constructed around concepts of \"step-change\", \"convenience\", \"strength of evidence base\" and \"impact on future research & development\". Conclusions: If innovation is to be used as operational criteria for adoption, pricing and reimbursement of health technologies, the concept must be clearly defined, and it ought to be independent from other value dimensions already captured in their health technology assessment systems.

BackgroundEconomic evaluation of health technologies requires healthcare resources, procedures and services to be valued at their opportunity cost. In practice, many economic evaluation studies use official databases of hospital Diagnosis-Related Groups (DRGs) as inputs where unit costs are required. This study describes the available data on costs of hospital DRG from official, publicly available sources in nine European countries (England, France, Germany, Italy, Poland, Portugal, Slovenia, Spain and Sweden), critically examines and compares the methodologies used to construct these databases and comments on the appropriateness of such unit cost data for economic evaluation.MethodsA standardized semi-structured questionnaire was developed in order to obtain both official publicly available sources of inpatient DRG databases and documents explaining the costing methodology used in calculation of unit costs available in those databases.ResultsEngland stands out as a benchmark in terms of good practice. Other countries face more challenges in one or more items, whether in documenting and auditing processes, guaranteeing methodological rigour, including all relevant economic items such as depreciation of buildings and equipment and capital costs, conducting the process annually and completely, publishing the costs as well as tariffs and recognising sampling uncertainty or variation.ConclusionAnalysts should evaluate carefully whether DRG costs or tariffs published in each country are appropriate for use in economic evaluation.

This article presents part of the work within Work Package 3 (WP3) of Impact HTA (Improved methods and actionable tools for enhancing HTA), a H2020 EU-funded research project, intended to enhance and promote collaboration in HTA across EU MS. Amongst other objectives, and in close collaboration with WP4, WP3 addressed setting up a multi-country unit-cost database: the European health care and social costs database (EU HCSCD). The purpose of the database is to facilitate the transference of healthcare economic evaluation analyses across countries, jurisdictions and settings. WP3 concentrates on healthcare costs; WP4 on social costs. This paper discusses the state of the art on this topic, building an appropriate conceptual and theoretical framework for Database development. We conducted a broad, but not systematic, literature and gray-literature review (LR), identifying existing practices and problems, and their implications, described in the Results section. We discuss practical implications and draw important conclusions behind the construction, and future evolution, of this database.

From both the methodological point of view and standardization of methodology, little attention has been paid to the estimation of direct costs in evaluation of healthcare technologies. The objective is to revise the recommendations on direct costs provided in European economic evaluation guidelines and to identify the commonalities and divergences among them. To achieve this, a comprehensive search through several online databases was performed resulting in 41 documents from 26 European countries, be they economic evaluation guidelines or costing guidelines. The results show a large disparity in methodologies used in estimation of direct costs to be included in economic evaluations of health technologies recommended by European countries. A lack of standardization of cost estimation methodologies influences arbitrariness in selecting costs of resources included in economic evaluations of medicinal products or any other technologies and, therefore, in decision making process necessary to introduce new technology. In addition, this heterogeneity poses a major challenge for identifying factors that could affect the variability of unit costs across countries.

Background: External reference pricing (ERP) is used to set pharmaceutical prices to improve affordability, but its application may have negative consequences on patient access—thus, equity—across countries and on global innovation. With the United States contemplating ERP, negative effects could be magnified. Our aim: identify and quantify some major consequences of ERP. Research design, methods : Besides relying on databases and ERP modelling, we developed a heart failure case study. 4-step approach: 1) review ERP policies; 2) establish worldwide “price corridor”; 3) quantify patient access and health outcomes impact by ERP; 4) estimate ERP impact on innovation. Results: Our ERP referencing analysis highlights its perverse effects especially in lower-income countries. As counterstrategies to protect their revenues, manufacturers often implement tight list price corridors or launch avoidance/delays. Consequences include suboptimal patient access—hence, worse outcomes—illustrated by our case study: 500,000 + QALYs health loss. Additionally, the ensuing revenue reduction would likely cause innovation loss by one additional medicine that would have benefitted future patients. Conclusion: This research provides key insights on potential unintentional consequences of medicine price setting by ERP worldwide and under a new proposal for the United States. Our results can inform stakeholder discussions to improve patient access to innovative medicines globally.

Introduction Costs are one of the critical factors for the transferability of the results in health technology assessment and economic evaluation. The objective is to develop a cost database at the European level to facilitate cross-border cost comparisons in different settings and explains the factors that lead to differences in healthcare costs in different countries, taking into account the differences between health systems and other factors. Methodology The core of the database is compounded of three main categories (primary resources, composite goods and services, and complex processes and interventions) organized into 13 subcategories. A number of elements providing as detailed information of unit cost as possible were identified in order to mitigate the problem of comparability. Consortium partners validated both the database structure and selected costing items. Results Twenty-seven costing items included in the EU HCSCD resulted in 1450 unit costs when taking into account all item subtypes and countries. Cross-country differences in costs are driven by the type of resources included in the costing items (e.g., overhead costs in case of complex processes and interventions) or by the variety of existing brands and/or models and the type of unit value in most of the primary resources. Conclusion The EU HCSCD is the only public unit healthcare and social cost database at European level that gather data on unit costs and explains differences in costs across countries. Its maintenance and regular data updating will enable establishing specific systems for generating and recording information that will meet many of its current limitations.