Hilde Stevens

Rebalancing innovation, affordability, and access for orphan drugs in the European Union

Hilde Stevens, Georges Siotis, Micael Castanheira, Mathias Dewatripont et autres

Orphan drug policy in the European Union faces a double price-and-innovation gap: a small fraction of rare diseases receive important resources while the overwhelming majority are under- or un-researched, leaving most rare disease patients facing high unmet medical needs. The European Commission's …

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OP10 Global Access Licensing: An Expert Interview Study with Scientists-Inventors and Directors of Technology Transfer Offices Involved in the Development and Commercialization of COVID-19 Vaccines

Shiri Mermelstein, Hilde Stevens

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Early access disparities in innovative therapies across the US, EU, China, and Japan

Sanae Akodad, Hilde Stevens

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 …

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Societal participation in the development of orphan drugs: a systematic review

Sanae Akodad, Delphine De Smedt, Hilde Stevens

The development of orphan drugs (ODs) remains constrained by structural barriers, including limited scientific knowledge, high clinical uncertainty, and fragile commercial incentives. In parallel, an alternative ecosystem has emerged in which non-commercial societal actors (patients, advocacy groups, and philanthropic entities) contribute to …

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Cognition and communication in patients with spinal muscular atrophy: A systematic review

Sanae Akodad, Delphine De Smedt, Simon Baijot, Hilde Stevens et autres

•Synthesizes evidence from 12 studies on cognitive and communicative impacts in SMA, focusing on nuanced functional outcomes.•Highlights cognitive variability in SMA1, revealing subtle challenges in SMA2 and 3, and stresses tailored assessment methodologies.•Identifies communication barriers in SMA, emphasizing the urgency of investigating …

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A novel approach to boost drug development in paediatric oncology

Sam Daems, Hilde Stevens, Mathias Dewatripont, Hans‐Georg Eichler et autres

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What matters most to patients with multiple myeloma? A Pan-European patient preference study

Rosanne Janssens, Tamika Lang, Ana Vallejo, Jayne Galinsky et autres

Introduction Given the rapid increase in novel treatments for patients with multiple myeloma (MM), this patient preference study aimed to establish which treatment attributes matter most to MM patients and evaluate discrete choice experiment (DCE) and swing weighting (SW) as two elicitation …

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TRIPS to Where? A Narrative Review of the Empirical Literature on Intellectual Property Licensing Models to Promote Global Diffusion of Essential Medicines

Shiri Mermelstein, Hilde Stevens

Governed through the World Trade Organization Agreement on Trade-Related Aspects of Intellectual Property Rights (TRIPS) since 1995, the current medical R&D system requires significant trade-offs between innovation and high monopoly prices for patented drugs that restrict patient access to medicines. Since its …

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Patient Preferences for Multiple Myeloma Treatments: A Multinational Qualitative Study

Rosanne Janssens, Tamika Lang, Ana Vallejo, Jayne Galinsky et autres

Background: Investigational and marketed drugs for the treatment of multiple myeloma (MM) are associated with a range of characteristics and uncertainties regarding long term side-effects and efficacy. This raises questions about what matters most to patients living with this disease. This study …

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Human Papillomavirus Is Dangerous—But a Vaccine Can Save You!

Marie Neunez, Pierre Robert Smeesters, Hilde Stevens

Human papilloma virus (HPV) is a virus that can cause disease in the skin and mucus membranes of both women and men. There are more than 100 types of HPV. While most of them cause harmless infections, some types of HPV are …

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Market access of gene therapies across Europe, USA, and Canada: challenges, trends, and solutions

Eline van Overbeeke, Sissel Michelsen, Mondher Toumi, Hilde Stevens et autres

• Challenges blocking market access of GTMPs are highly interrelated. • Developers should seek support and early joint interactions with regulators and payers. • Conditional marketing authorization and reimbursement mechanisms should be explored. • RWE infrastructure and requirements should be developed on …

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Editorial: Medicine and Society

Pietro Ghezzi, Arianne Shahvisi, Hilde Stevens

SCOPUS: ed.j

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