Publications List
2026
Background:
Early access programs (EAPs) can provide patients with medical devices before full market access in urgent or high unmet-need situations. Unlike pharmaceuticals, the European Union (EU) has no harmonised early-access framework for medical devices, resulting in fragmented national practices. We use EAP as an umbrella term for two overlapping categories with distinct objectives: (1) early access in the strict sense, authorising exceptional pre-market use under defined conditions; and (2) accelerated access, aiming to shorten time to routine access by streamlining regulatory, reimbursement, or evidence-generation pathways. We map and compare these mechanisms across the 27 EU Member States (MS).
Methods:
We conducted a qualitative document-based analysis of peer-reviewed literature, grey literature (policy papers, regulatory guidance), and legal sources (EU legislation, national laws) published up to June 2025. We searched PubMed and Google Scholar, consulted Eur-Lex, and reviewed national competent authority websites. Search terms (English and local languages, then translated) combined “medical device” with “early access,” “accelerated access,” “compassionate use,” and “derogation,” plus “EU” or MS names. Documents were included if they described mechanisms enabling early use of medical devices. We synthesised and compared key program characteristics.
Results:
We identified early and accelerated access pathways at EU level and within MS. EU law provides derogations from CE-marking requirements, notably Article 59 of the EU Medical Device Regulation (EU MDR), allowing national authorisation of non-CE-marked devices in exceptional circumstances. The EU MDR also enables the use of custom-made devices and certain in-house manufactured devices by health institutions without full CE marking. Nationally, at least half of MS operate compassionate or expanded access schemes for devices, and some implement reimbursement or evidence-development mechanisms (e.g., France’s forfait innovation) to support early use while generating decision-relevant data.
Conclusions:
Early access to medical devices in Europe relies on EU regulatory derogations and heterogeneous national schemes. The landscape remains fragmented and unevenly documented. We recommend: (1) a more structured EU-level route for high-risk and combination products, (2) lifecycle-based national schemes integrating regulatory and payer access, (3) measures to reduce inequities and administrative barriers, particularly in Central and Eastern Europe, and (4) improved transparency and standardised data collection on implemented pathways.
Amid growing calls for sustainability in the healthcare sector, this study examines how and under what conditions environmental, social, and governance (ESG) performance influences research and development (R&D) output. Although existing studies suggest that ESG performance enhances R&D output, the financial mechanisms that enable or constrain this relationship remain underexplored. We address this gap by theorizing and testing the dual role of debt financing as both a mediator and a moderator in the ESG performance and R&D output relationship within the healthcare sector, where innovation is highly capital-intensive and socially consequential. Integrating stakeholder theory and agency theory, we argue that ESG performance promotes R&D output through improved access to reputational and financial resources, whereas high debt levels weaken this effect due to agency conflicts. Using panel data from 2016 to 2022 on healthcare firms in Europe and the United States, we estimate our main models using OLS and applying instrumental variable and system GMM techniques as robustness checks to address endogeneity. Our findings show that debt financing partially mediates the ESG–R&D link and negatively moderates it, revealing its ambivalent role. Compared to existing studies, our findings indicate that the effects of ESG performance on R&D output are conditional and context specific, with stronger impacts observed in Europe than in the United States, reflecting institutional conditions such as stricter European Union sustainability reporting frameworks, notably the Corporate Sustainability Reporting Directive (CSRD), along with robust policy incentives and longer term investment horizons. We also find pronounced effects in the biotechnology and pharmaceutical subsectors. This study contributes to theory by bridging competing views on ESG performance and offering a more nuanced understanding of how debt financing shapes the ESG–R&D output relationship.
Background
Orphan medical devices remain the blind spot of rare disease policy. While the United States has recognized them since 1990 through the Humanitarian Use Device (HUD) and Humanitarian Device Exemption (HDE) framework, the European Union only introduced a non-binding definition in 2024 (MDCG 2024–10), revealing a deeper asymmetry: flexibility is codified in the US but interpreted in the EU.
Methods
We conducted a document-based policy analysis (1990–2025) and examined legislation, regulatory guidance, and academic literature to compare EU and US frameworks across evidentiary standards, access pathways, economic incentives, and reimbursement-post-market alignment. Three illustrative case studies (Berlin Heart EXCOR, Argus II, and the Medtronic Melody valve) were used to assess real-world consequences for access, sustainability, and patient continuity of care.
Results
The EU’s Medical Devices Regulation 2017/745 lacks a legal orphan-device designation, dedicated incentives, or a harmonized HTA framework. Access remains fragmented, and recertification under the MDR threatens supply continuity. The US HUD/HDE pathway codifies flexibility through a “probable benefit” standard under IRB supervision and an annual distribution cap, though financial sustainability remains limited by profit restrictions and inconsistent reimbursement. Case comparisons reveal: (i) earlier EU access without post-market safeguards (Argus II); (ii) US capacity to transition from humanitarian to full approval when incentives align (Melody); and (iii) vulnerability of SMEs to compliance costs (EXCOR).
Conclusion
Addressing this policy vacuum requires an inflection point equivalent to the 1983 Orphan Drug Act, one that redefines how innovation for the few is valued. Five policy pillars emerge: a legal designation, adaptive evidentiary pathways, sustainable incentives (e.g., pooled procurement), integrated reimbursement mechanisms, and mandatory continuity-of-access or “exit-insurance” schemes. Without such reform, essential technologies for rare diseases will remain trapped between regulatory rigidity and market abandonment. This policy paper highlights structural regulatory gaps and proposes actionable reforms to ensure equitable access to orphan medical devices.
Antimicrobial resistance (AMR) is one of the greatest threats to public health, and forecasts for 2050 are even worse. In response, the European Commission (EC) is proposing the Transferable Exclusivity Extension Voucher (TEEV), which aims to encourage the development of innovative antimicrobials. It consists of extending data exclusivity for a medicine selected by the beneficiary company or another company to which the voucher can be sold (i.e. not necessarily the antimicrobial). One requirement for obtaining it is to declare any public contributions received for the antimicrobial R&D. Based on a review of the literature and interviews with five AMR experts, a theoretical and real view of how the TEEV works and its estimated costs is presented, in addition to the impact that the requirement for transparency in R&D costs would have. On the one hand, the results show that a (well-designed) TEEV, coordinated by the EC, offers significant public health benefits compared with the cost of inaction and provides predictability for investors, although, by its nature, it delays the entry of generics/biosimilars and does not provide an access scheme to new antimicrobials. Recent assessments estimate the total healthcare cost at €162 million per voucher and the average cost for each Member State at €6 million. On the other hand, transparency of R&D costs could enable policymakers to design policies focused on public health needs and better design the optimal mix of (push and pull) incentives for new antimicrobials, but they run the risk of threatening trade secrets, discouraging private investment and promoting the application of cost-plus pricing regulations. In conclusion, the TEEV could be a great incentive to combat AMR inaction, but it has not yet been implemented and needs to be complemented by other push and pull incentives. Furthermore, more information is needed on the impact of R&D costs transparency in the context of antimicrobials.
Objectives
The integration of environmental impact into health technology assessment (HTA) is increasingly discussed, but its practical implementation remains uncertain. This study explores multi-stakeholder perspectives on the progress, challenges, and equity implications of incorporating environmental considerations into HTA.
Methods
As part of the Horizon Europe project HI-PRIX (ID: 101095593), 13 semi-structured interviews were conducted with experts across academia, HTA agencies, regulatory bodies, and industry in seven European countries. Interview transcripts were analyzed through a thematic analysis conducted manually, using a grounded-theory approach. From the interviews, 101 quotes corresponding to 37 codes were extracted and organized in eight analytical themes.
Results
Stakeholders agreed that environmental impact is relevant but under-addressed in HTA due to fragmented data, methodological uncertainty, and lack of regulatory guidance. Many emphasized that a health system perspective is too narrow, where methods conventionally used to assess health technologies are not necessarily appropriate to also assess environmental impact, calling for a societal approach that reflects lifecycle impacts. Equity consequences linked to the inclusion of environmental impact of health technologies in HTA were specifically recognized as both reasons for its incorporation and potential barriers. At this stage, procurement was identified as a practical approach to account for the environmental dimension.
Conclusions
Integrating environmental impact into HTA will require shifting toward a societal perspective, stronger collaboration across stakeholders, and development of EU-level guidance, expert panels, and standardized methodologies. Methodologies incorporating equity into economic analyses should be explored as a potential direction for future research. Until frameworks mature, environmental impact is likely to remain informative but not yet decisive in HTA.
2025
The development of health technologies involves complex, costly, and risky investments, with significant contributions from both public and private sectors. Recent EU pharmaceutical directives propose transparency on public funding to aid pricing negotiations and affordability. However, questions remain regarding how public investments should be measured and their influence on pricing and reimbursement (P&R) decisions. In this paper, we characterise public sector institutions as “payers,” “R&D investors,” and “regulators”. Through a myriad of agencies and decisions, these institutions directly, indirectly or sometime unexpectedly influence risk and return on private research and development (R&D) through P&R, direct investments, and regulatory policy. P&R decisions by payers for innovative therapies influence risk and expected return of future R&D. Value-based pricing offers a more reliable signal of payers’ priorities than cost-plus or (international) reference pricing. For greatest impact, public R&D investment should be directed to areas where markets are deficient, such as basic science, translational research, real-world studies, and towards emerging fields like AI and gene editing that will play an increasing role in healthcare and drug development. Applied R&D should be conducted on a financially sustainable basis. Licensing arrangements can be used to recover those investments, while promoting spillover benefits to wider society. Market access regulators are aware of the need for scrupulous transparency and neutrality, but other public sector actors (payers and R&D investors) also must recognise their policies affect the level playing field.
Early access to innovative therapies offers a critical lifeline for patients with severe or life-threatening conditions, yet substantial disparities persist across the United States (US), the European Union (EU), China (CH), and Japan (JP). These disparities arise from diverging definitions of unmet medical need (UMN), heterogeneous timelines for regulatory review, and uneven implementation of Early and Expanded Access mechanisms. This policy review provides a comparative analysis of regional frameworks, emphasizing how clinical trial geography, eligibility rules, and access mechanisms interact to shape pre-approval opportunities for patients. While regulatory innovations such as the FDA's Breakthrough Therapy designation, the EMA's PRIME scheme, the PMDA's Sakigake program, and recent reforms in China have accelerated access in selected contexts, cross-border misalignments and fragmented health technology assessment (HTA) processes continue to generate inequities. The EU's new HTA Regulation (2021/2282) represents a step forward by embedding joint clinical assessment and real-world evidence, but persistent structural inconsistencies hinder timely and equitable uptake. This article argues for a paradigm shift from static approval models to adaptive licensing approaches, including live licenses and regulatory sandboxes. It calls for internationally coordinated benefit–risk governance that integrates early access planning into drug development from Phase II onward. By reframing uncertainty as a shared responsibility among regulators, developers, payers, clinicians, and patients, early access governance can be redesigned to promote equity, transparency, and responsiveness particularly for rare and high-burden diseases.
Background
Pricing and reimbursement (P&R) systems do not normally use public investments in research and development (R&D) as criteria when negotiating the prices and reimbursement of health technologies.
Objective
The objective was to find mechanisms that consider public investment in R&D when negotiating P&R or obtaining a fair return on this public investment
Methods
We conducted a scoping review. A total of four databases (PubMed, Embase, Scopus and Web of Science) and a grey literature information source (Google Scholar) were searched. Eligible articles were published before 2024 and described how public sector investment in R&D is considered in price negotiations or how the public sector can obtain a return on R&D investment.
Results
The review found 28 papers referring to mechanisms that take into account public investment in R&D to reduce prices in the P&R negotiation (e.g. delinkage R&D model, advance purchase agreement and government patent use), to obtain a fair return on investment (e.g. royalties and venture philanthropy) or to save costs or share risks (e.g. social impact bonds and prize fund). Examples are provided of health technologies that used these mechanisms.
Conclusions
Policymakers have several resources they can draw from to ensure a fair and efficient use of public R&D funds. However, there is little evidence that these instruments are widely used in practice, and there is no political consensus on what mechanism is the most appropriate and why. In view of the above, it is essential to create a common framework that will ensure a fairer and more affordable system for public health budgets.
Artikel 57 der vorgeschlagenen Arzneimittelverordnung (RL) der Europäischen Union (EU) verpflichtet Antragsteller auf Marktzulassung, jegliche direkte finanzielle Unterstützung für Forschung und Entwicklung (F&E) durch öffentliche Institutionen offenzulegen. Unsere Forschung zu öffentlichen Beiträgen zielt darauf ab, Kategorien zu identifizieren, die zur Erfassung direkter und indirekter öffentlicher Beiträge zu F&E erforderlich sind. Damit soll eine Systematik für die standardisierte Berichterstattung über öffentliche Beiträge geschaffen und Unklarheiten bei der Interpretation von „direkten“ und „indirekten“ öffentlichen Beiträgen reduziert werden. Letztendlich wollen wir die Operationalisierung von Artikel 57 unterstützen. Acht Kategorien öffentlicher Beiträge zur Arzneimittelentwicklung können entlang der Wertschöpfungskette (von der Grundlagenforschung bis zur Marktüberwachung) identifiziert werden. Die Informationen sind oft nicht umfassend dokumentiert, wenngleich einige Quellen wertvolle Hinweise und Fakten bieten. Die nächsten Schritte sind EU-weit abgestimmte Richtlinien für eine transparente und standardisierte Berichterstattung in ausreichender Detailliertheit über öffentliche F&E‐Investitionen und Konditionen bei der Veräußerung akademischen Wissens, resp. beim Einkauf, um diese Informationen in Preisverhandlungen auch nutzbar zu machen.
Background
Within the context of increasing transparency around public contributions, a framework for reporting and analysing public contributions to research and development (R&D) was previously developed and is piloted here using the example of antibiotics. The aim of this work is to check whether the category system is feasible, to revise and adjust the granularity of the category system where necessary, and to expand the range of sources for detailed analyses.
Methods
All antimicrobial medicinal products in development, discontinued and approved in the last 10 years were identified in the literature. Thereafter clinical trials and company information was searched generating a list of 56 compounds where primarily small to medium-sized enterprises (SMEs) were involved in antibiotics development. Information on clinical trials, university spinouts and public funding for SMEs was then gathered from various sources. The framework for classifying public contributions was then applied.
Results
We found that around one-third of antibiotics are developed by SMEs. We identified numerous public funding sources for SMEs that develop antibiotics. At both early-stage and late-stage development, public research funding is the most common public funding reported by SMEs, ahead of other public sources like public equity funds, private-public partnerships and philanthropic sources. A deep-dive into one antibiotic drug, Venatorx, revealed public funds investment of approximately $655 million, dwarfing private investment funds. We found the classification framework generally practicable and we suggest recommendations to improve its granularity and applicability.
Conclusion
In this paper we piloted and revised a framework that has been developed to classify types of public contributions to pharmaceutical products at different stages of development. The framework, together with work we have done on identifying sources for funding, can be applied to support pharmaceutical price negotiations that reflect the level of public contribution to product development.
Multi-indication drugs pose pricing challenges, affecting therapeutic benefits and patient access. We model how a manufacturer strategically chooses clinically eligible patient populations for each indication, recognizing that broader populations increase demand but reduce expected therapeutic benefits and thus allowable prices. We identify mechanisms that induce the manufacturer to maximize total benefit while ensuring profits cannot exceed benefit generated: indication-specific prices equal to expected benefits, population-weighted uniform prices, and two-part tariffs. This holds when indications are introduced simultaneously or sequentially, provided prices fully adjust as indications are added. Price caps distort patient selection, explaining empirically observed strategies.
Background and Objective
Article 57 of the proposed European Union (EU) Pharmaceutical Legislation (PL, Directive) will require market authorization applicants to publicly declare any direct financial support for R&D received from public authorities. Our research aims to identify the categories needed to capture direct or indirect public contributions to R&D, provide a framework for standardized reporting of public contributions, and reduce ambiguity in the interpretation of “direct” and “indirect” public contributions.
Methods
An iterative mixed-methods approach is applied: a targeted literature review was conducted, complemented by interviews with representatives of different stakeholder groups to identify categories of public contributions to R&D, followed by searches for relevant data sources.
Results
26 publications on primary data relevant to analyses of public contributions were identified, finding that between half of all drugs approved and >90 % of drug targets are associated with public sector institutions and/ or their spin-outs. Eight categories of public contributions to medical innovations were identified along the value chain (from basic research to post-market surveillance).
Discussion and conclusion
The framework offers a structured and systematic approach for identifying data on public and philanthropic contributions to developing medical products (medicines and devices). This information is often not comprehensively documented. Therefore, aligned public policies enforcing transparent and standardized reporting in sufficient granularity on R&D investments and conditions are key.
Background: The objective of this case study is to pilot a framework of public contributions examining the origins of products from two major European pharmaceutical companies, Novartis and Novo Nordisk, that received approval from the European Medicines Agency (EMA) between January 2014 and May 2024. Our primary focus was to investigate the extent of public contributions, including government grants, public-private partnerships, and other forms of public funding, that supported the development of these products. Additionally, we explored whether these companies primarily relied on in-house research and development (R&D) capabilities or acquired these products at various stages of their development.
Methods: We conducted a thorough analysis of the products approved during the specified period, identifying the origins of each product. The analysis included detailed examination of public databases, financial disclosures, and scientific publications to trace the flow of public funding. We built on a list of sources from our previous studies to increase the level of detail.
Results: Novartis demonstrated a tendency to acquire promising products and technologies from smaller biotech firms and other pharmaceutical companies, particularly in therapeutic areas where it sought to strengthen its market position like oncology (16 out of 25 products acquired, licensed or co-developed). Conversely, Novo Nordisk predominantly advanced its products through internal R&D efforts, although it also engaged in selective acquisitions to complement its core capabilities (two out of six products acquired, licensed or co-developed). For Novartis eleven products received public support, for Novo Nordisk one product did.
Conclusion: Our findings reveal that both Novartis and Novo Nordisk use strategic acquisitions with Novartis relying more heavily on it than Novo Nordisk. Our framework for analyzing public contributions was sufficient for the product portfolios of the firms analyzed and helped us identifying public contributions.
2024
Background and Objective
In a context of growing clinical and financial uncertainty, pricing and payment schemes can act as possible solutions to the problems of affordability and access to health technologies. However, a comprehensive categorization of the available schemes to help decision makers tackle these challenges is lacking. This work aims at mapping existing types of pricing and payment schemes, and proposes a new approach for their classification, in order to help decision makers and other stakeholders select the best type of scheme to meet their needs.
Methods
A Preferred Reporting Items for Systematic Reviews and Meta-Analyses extension for Scoping Reviews (PRISMA-ScR)-compliant scoping literature review was performed between 2010 and 2023 in three databases (PubMed, Web of Science, Scopus). The search strategy was developed around two groups of keywords, “pricing/payment schemes” and “scheme innovativeness”. Eligible studies were those illustrating the unique design and features of each scheme type, which were extracted by two independent reviewers, and synthesized using a narrative format, including a detailed tabular description of each type of scheme.
Results
A total of 70 unique types of pricing and payment schemes were identified. Around one third (33%) was only specified in principle, while two thirds (67%) had been implemented in practice. About half of the scheme types were proposed for drugs (34/70, 49%), and the vast majority were not designed for a specific therapeutic area (55/70, 79%). Each scheme type was categorized based on distinctive characteristics: the objectives, the outcome component, the timing/modalities of payments, and the evidence collection requirements.
Conclusions
Instead of trying to fit the retrieved schemes into a rigid taxonomy, we propose a new approach that suggests a flexible need-driven use of the available scheme types, driven primarily by the specific objective that one might have, and allows leveraging of the other key characteristics of each type of scheme.
Introduction
Innovative pricing and payment/reimbursement schemes have been proposed as one part of the solution to the problem of patient access to new health technologies or to the uncertainty about their long-term effectiveness. As part of a Horizon Europe research project on health innovation next generation pricing and payment models (HI-PRIX), this protocol illustrates the conceptual and methodological steps related to a scoping review aiming at investigating nature and scope of pricing and payment/reimbursement schemes applied to, or proposed for, existing or new health technologies.
Methods
A scoping review of literature will be performed according to the PRISMA guidelines for scoping reviews (PRISMA-ScR) guidelines. The search will be conducted in three scientific databases (i.e., PubMed, Web of Science, and Scopus), over a 2010–2023 timeframe. The search strategy is structured around two blocks of keywords, namely “pricing and payment/reimbursement schemes,” and “innovativeness” (of the scheme type or scheme use). A simplified search will be replicated in the gray literature. Studies illustrating pricing and payment/reimbursement schemes with a sufficient level of details to explain their characteristics and functioning will be deemed eligible to be considered for data synthesis. Pricing and payment/reimbursement schemes will be classified according to several criteria, such as their purpose, nature, governance, data collection needs, and foreseen distribution of risk. The results will populate a publicly available online tool, the Pay-for-Innovation Observatory.
Discussion
The findings of this review have the potential to offer a comprehensive toolkit with a variety of pricing and payment schemes to policymakers and manufacturers facing reimbursement and access decisions.