Regulatory Science CBG-MEB

The call for expression of interest for Academia to participate in the new EMA/HMA European Platform for Regulatory Science Research is now open with a deadline of 24 January 2025. There are two ways to join the initiative 1. as nominated member of the steering group 2. as platform participant More information about the EMA/HMA European Platform for Regulatory Science Research including details how to join, can be found below, and on the EMA website: https://lnkd.in/di-Fbx29. The page also includes a video recording of the webinar from 18 November 2024. European Medicines Agency, #regulatoryscience

Drug regulators play a key role in assessing treatment outcomes and uncertainties of new medicines. In a recent study, Anne Taams, Lourens Bloem, Toine Egberts, former MEB colleague Carla Herberts along with EMA colleagues Nikolaos Zafiropoulos and Francesco Pignatti, explored uncertainties about the benefit-risk balance for oncology medicines assessed by the European Medicines Agency between 2011 and 2022.   💡 Key Findings: 🔹 121 oncology medicines, 800 uncertainties with a median of 6 per medicine. 🔹 Top categories of uncertainties: Safety (51%): uncertainties about specific adverse events and safety in subpopulations. Efficacy (40%): uncertainties about effect size and efficacy in subpopulations. 🔹 Most uncertainties observed for: Cell and gene therapies. Conditionally authorised medicines. Medicines authorised between 2019-2022. 🔹 Proposal of an uncertainty classification system Consisting of 5 main categories, 27 subcategories of uncertainties.   📊 Conclusions: Their study provides new insights into the uncertainties of the benefit-risk balance of oncology medicines that remain at the time of initial marketing authorisation. The proposed classification system of uncertainties may help to more consistently describe uncertainties and thereby improve communication among stakeholders, including drug regulatory authorities, pharmaceutical companies, health technology assessment bodies, clinicians, and patients.   Read the full article here: https://lnkd.in/eDkKbnft #Oncology #Uncertainties #RegulatoryScience #EMA #ESMOpen Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht University

The EMA Qualification of Novel Methodologies (QoNM) procedure qualifies methods, technologies and methodologies within a well-defined context of use in a pharma R&D context based on the evaluation of the presented scientific rationale and submitted data. A recent paper from Ana Drmić and co-authors published in Frontiers in Pharmacology analysed QoNM submissions providing policy messages and recommendations to stakeholders on how to better prepare qualification applications. Their recommendations include: ◾ Grounding validation strategy using a current standard measure or a distribution technique. ◾ Accurately represent pertinent subgroups via accurate inclusion and exclusion criteria. ◾ Establish a well-defined and specific Context of Use with clear descriptions of the use within a development program target population and disease stage. Please read the paper here: https://lnkd.in/eGHSy6AQ Riccardo Saccà, Thorsten Vetter, MD, Falk Ehmann #scientificadvice #novelmethodologies, European Medicines Agency

When patients need to take several medications, this can result in interactions that can affect the drug´s efficacy or safety. Therefore, it is important that warnings about interactions are understandable for healthcare professionals so they can act if there is a risk. A team at the Swedish Medical Products Agency assessed the interaction texts in product information and their clinical relevance for drugs approved in Europe. The focus was texts for interactions mediated by the drug transporters OATP1B1, 1B3 and BCRP. The results are now published in the European Journal of Clinical Pharmacology. Numerous interaction texts lacked clinical relevance or were ambiguous, highlighting a potential for improvement. Warnings without clinical relevance could be omitted, and some warnings with clinical relevance could be updated to provide actionable recommendations to the prescribers. The article also presents texts for each interaction scenario, which the authors consider as examples of explicit wordings with actionable recommendations. These are aimed to be a starting point to generate standard texts. This would ensure potential interactions are properly warned for and handled adequately, maintaining efficacy and safety for each patient regardless of concomitant medications. Read the paper here: https://lnkd.in/euNedSUE Valeria A., Erik Bergman, Elin Lindhagen, Sherwood Kim, Gabriel Westman, Fabienne Z. Gaugaz Medical Products Agency Läkemedelsverket

The registration is still open for the Winter Meeting on 13–14 January 2025 of the University Utrecht! This annual event brings together pharmaceutical policy researchers from around the world to discuss their work and insights. On the first day, a plenary session will be held focusing on pharmacovigilance and risk communication, with a highlight being the PhD defense of Per Sindahl. Day two will feature abstract presentations showcasing the latest (ongoing) research. Don’t miss this chance to engage with global colleagues! Register with the following link (deadline 19 December 2024): https://lnkd.in/ecgEuhny. Utrecht Centre for Pharmaceutical Policy and Regulation

🎓 Enrico Costa's PhD defense 🌍 Earlier this year Dr. enrico costa's defended his PhD thesis titled "Globalization of Orphan Drug Development: Integrating Science, Regulation, and Access". Conducted under the Utrecht WHO Collaborating Centre for Pharmaceutical Policy and Regulation, this research bridges the critical areas of rare disease treatment, regulatory frameworks, and global health equity. Enrico's work sheds light on the dynamic landscape of orphan drug development, from regulatory innovations in the Global North to challenges in accessibility in the Global South. His thesis explores: ◾ The role of regulatory ecosystems in accelerating treatment approvals. ◾ The complexities of global therapeutic prioritization. ◾ Strategies to improve access to rare disease treatments in low-resource settings. Enrico’s research not only enhances our understanding of rare disease management but also sets a precedent for inclusive global health strategies. 💡 Learn more about how regulation and science converge to address unmet medical needs worldwide, and read Enrico’s thesis here: https://lnkd.in/ekf3HTNg. Bert Leufkens, Rianne van den Ham, Marieke De Bruin, Toine Egberts, Carla Hollak, Aukje Mantel-Teeuwisse, Fatima Suleman, Utrecht Centre for Pharmaceutical Policy and Regulation, AIFA Agenzia Italiana del farmaco

Earlier this year the Regulatory Science Network Netherlands (RSNN) organised an expert meeting with the title ‘Companion Diagnostics (CDx) – Challenges and Opportunities’. The goal of this RSNN Expert Meeting was to retrieve information from the field in terms of developments, missed topics or technologies, challenges and opportunities related to CDx. The invited experts shared their knowledge, experiences, and personal views in accordance with the Chatham House Rule. The group comprised 18 experts from various backgrounds including academia, industry, governmental bodies and health care professionals (HCPs). The pre-defined questions were: 1. What sort of issues do you foresee to play an important role from a regulatory perspective with respect to CDx in the coming decade? 2. Which technological developments do you expect to pose a challenge or opportunity in the coming decade? a. With respect to emerging IVD technological developments? b. With respect to developments in personalized medicine? 3. CDx play an important role in oncology. Are there other therapeutic fields where CDx may play an important role in the coming decade? 4. What regulatory challenges and opportunities do you foresee in the context of co-development of CDx and the corresponding medicinal product? You can read the meeting report here: https://lnkd.in/dp9TCn8N #IVDR #CDx #companion #diagnostics

The environmental risk assessment (ERA) evaluates a medicine’s environmental impact, and it is mandatory in the EU regulation for medicines for human use. However, its submission is difficult to enforce by regulators, because the EMA guideline on the ERA states that ‘Under the current requirements, the outcome of the ERA should not constitute a criterion for refusal of a marketing authorisation.’   Jim Zinken, a former MEB-intern, wrote his thesis on stakeholder perspectives on the current position, conduct and assessment of the ERA, as well as future perspectives. The thesis was further developed and expanded since with his co-authors and resulted in a publication in Drug Discovery Today.   Ten stakeholders were interviewed and a dataset with 478 responses to the public consultation regarding the ongoing revision of the EU pharmaceutical legislation was analysed. The findings show that stakeholders largely agreed that the currently limited role of the ERA in EU medicines regulation is problematic. To increase the role of the ERA, stakeholders provided multiple recommendations, including giving the ERA a more prominent role in medicines evaluation, enhancing possibilities for data-sharing, extending the scope of the guideline, and providing more guidance for risk mitigation measures.   Read the paper here: https://lnkd.in/eeNKTmgk Lourens Bloem, Jarno Hoekman, Marjon Pasmooij, Twan Ederveen, Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht University, Utrecht Centre for Pharmaceutical Policy and Regulation #ERA #environmental #risk #assessment

Can monitoring the X/Twitter social network serve as an early warning system for medicine shortages? Early identification is critical for mitigating the impact of medicine shortages on patients. Doerine Postma and colleagues have explored whether a routine analysis of data from the Twitter social network can detect signals of a medicine shortage and serve as an early warning system. The researchers collected Twitter posts about medicine shortages reported between January and December 2019. The first posts per shortage were analyzed for their timing versus the national catalog Royal Dutch Pharmacists Association (KNMP) Farmanco. Of the 341 medicine shortages included in the research, 102 (29.9%) were mentioned on Twitter. However, only 4 (1.2%) of these were mentioned on Twitter more than 14 days before publication by KNMP Farmanco. On average, posts were published with a median delay of 37 days to publication by KNMP Farmanco. The researchers therefore concluded that the Twitter social network is not a suitable early warning system for medicine shortages. Twitter primarily echoes already-known information rather than spreads new information. However, Twitter or potentially any other social media platform provides the opportunity for future qualitative research in the increasingly important field of medicine shortages that investigates how a larger population of patients is affected by shortages. Read the full paper: https://lnkd.in/eBTmNY7n Doerine Postma, Magali Heijkoop, Peter De Smet, Kim Notenboom🇨🇭, Bert Leufkens, Aukje Mantel-Teeuwisse #medicineshortage #shortage #farmanco #Twitter #socialmedia

An article that was recently published in Molecular Therapy has examined the regulatory decision-making process for orphan designations by the Committee for Orphan Medicinal Products (COMP), part of the European Medicines Agency. The retrospective analysis focused on gene therapies for rare non-oncological conditions, based on viral vectors. The authors found that a large portion of submissions for orphan designation relied on non-clinical in vivo data. Mouse models were used in the majority of submissions and played a critical role in generating proof-of-concept data. The majority of submissions relied on non-clinical data only, with just 13% including some preliminary clinical data mainly from a few patients or early stage clinical trials. Significant benefit was applicable in 54% of the submissions. This indicates that about half of the sponsors are focusing gene therapy development in areas of unmet medical need, particularly where there are no authorized medicines available. Former COMP chair and MEB colleague Violeta Stoyanova-Beninska contributed to this analysis by Gloria Palomo Carrasco. https://lnkd.in/eXyfndBZ Kristina Larsson, Frauke Naumann-Winter, enrico costa, Dinah Duarte, Maria Elisabeth Kalland, Eva Malikova, Darius Matusevicius, Dinko Vitezic, Armando Magrelli, Segundo Mariz