This article provides insights into the European Medicines Agency’s approach to horizon scanning as a foresight tool to support early identification, analysis and preparedness for emerging trends, technologies and challenges relevant to medicines development and authorization. Emerging topics are identified through a continuous scanning of several sources of information, including key trends identified by leading institutions such as the World Health Organization, the European Commission Joint Research Centre, and the European Innovation Council. This perspective sets out the scope, impact and outputs of horizon scanning within the European medicines regulatory network, which include topic-specific reports, collaborative workshops and webinars, and contributions to regulatory policy, strategic planning, capacity building and research prioritization. It also emphasizes the European medicines regulatory network’s commitment to collaboration and knowledge sharing across European and international organizations. Through these efforts, horizon scanning supports regulatory preparedness and resilience of the European medicines regulatory system.

The full potential of pharmacogenomics to improve drug development and clinical practice has yet to be realized. Here, we present recommendations to address barriers to its implementation. The full potential of pharmacogenomics to improve drug development and clinical practice has yet to be realized. Here, we present recommendations to address barriers to its implementation.

The upcoming revised European Union (EU) general pharmaceutical legislation will incorporate the concept of the platform technology approach. Based on focus group discussions, we provide perspectives on the definition and implementation of the platform technology approach. The definition should enable the use of this approach across the entire medicine lifecycle, and for all types of medicines and disease areas. Regulatory acceptability depends on the availability of robust, platform-specific data, while clinical acceptability requires clear communication to ensure trust. The successful implementation of the platform technology approach further depends on key factors, including stakeholder collaboration to establish a clear and effective EU regulatory framework.

Consistent progress in medicines development has allowed both for de novo treatment options and for the refinement of existing products that improve effectiveness or reduce harm. Nonetheless, unmet medical needs persist, particularly in rare diseases, pediatrics and underserved populations. The many promises of pharmacological, technological, and regulatory innovation spark new hope for patients and doctors searching for treatments, and it is therefore essential that progress made translates into the availability of new and improved medicines. We here present our vision for accelerating the path from innovation to safe and effective medicines. Our vision is driven by the continued and transparent collaboration between regulators, academia, industry, patients, and healthcare professionals. Through concrete examples and strategic initiatives, we illustrate how the EMA works with stakeholders to support continuous evolution of regulatory science, contribute to patient-focused refinements in evidence generation and medicines assessments, and engage in forward-looking initiatives that monitor and examine innovative developments. The ultimate goal is to future-proof the regulatory ecosystem and ensure that scientific progress translates into meaningful health outcomes.

New approach methodologies (NAMs) have the potential to progressively transform medicines development by reducing reliance on animal testing while increasing the relevance of nonclinical data to patients. However, achieving regulatory acceptance of NAMs demands enhanced collaboration, clear guidance and continuous, science-based adaptation of the regulatory environment to accommodate emerging innovation. New approach methodologies (NAMs) have the potential to progressively transform medicines development by reducing reliance on animal testing while increasing the relevance of nonclinical data to patients. However, achieving regulatory acceptance of NAMs demands enhanced collaboration, clear guidance and continuous, science-based adaptation of the regulatory environment to accommodate emerging innovation.

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RNA-based medicines have potential to treat a large variety of diseases, and research in the field is very dynamic. Proactively, The European Medicines Agency (EMA) organized a virtual conference on February 2, 2023 to promote the development of RNA-based medicines. The initiative addresses the goal of the EMA Regulatory Science Strategy to 2025 to "catalyse the integration of science and technology in medicines development." The conference focused on RNA technologies (excluding RNA vaccines) and involved different stakeholders, including representatives from academia, industry, regulatory authorities, and patient organizations. The conference comprised presentations and discussion sessions conducted by panels of subject matter experts. In this meeting report, we summarize the presentations and recap the main themes of the panel discussions.

The EMA Qualification of Novel Methodologies 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. This policy brief analyses QoNM submissions providing policy messages and recommendations to stakeholders on how to better prepare qualification applications in this regard. The recommendations include: 1. Grounding validation strategy using a current standard measure or a distribution technique. 2. Accurately represent pertinent subgroups via accurate inclusion and exclusion criteria. 3. Establish a well-defined and specific CoU with clear descriptions of the use within a development program target population and disease stage. Lastly, it emphasizes role of the QoNM procedure in advancing medicine development methodologies within the EU.

The European Innovation Network is working to support the European medical innovation ecosystem by facilitating early dialogue between developers of medicines and regulators, as well as providing a platform for regulators to share information, good practices and expertise.

IntroductionThe European Medicines Agency Innovation Task Force (ITF) acts as early point of contact for medicine and technology developers to enable innovation during early drug development stages through ITF briefing meetings.AimTo reflect on the current pace of innovation and to assess the potential of ITF stakeholder interactions, a comprehensive analysis of the ITF briefing meetings held between 2021 and 2022 was conducted with a focus on individual questions raised by the developers and the related feedback provided by the European regulators.MethodsQuestions raised during ITF briefing meetings were extracted and categorised into main and sub-categories, revealing different themes across the whole medicine development process such as manufacturing technologies, pre-clinical developments, and clinically relevant questions.ResultsThere was positive feedback from regulators who gave initial guidance in 85% of the answers, provided concrete examples in 20% of the answers and recommended to continue discussions through additional regulatory procedures in 22% of the answers.ConclusionThis analysis frames the content and the type of topics discussed during ITF briefing meetings. Moreover, it describes the type of regulatory feedback provided to medicine developers and identified potential for improvement of these early interactions. Therefore, this analysis emphasises the role of ITF briefing meetings in fostering innovation in medicine.

One of the strategic goals of the European Medicines Agency (EMA) and the European Medicines Regulatory Network is to support the research and uptake of innovative methods and technologies in the development of medicines. To promote this goal, EMA drew up a list of enabling technologies (ETs), which are novel and fast-growing technologies that have the potential to enable innovation and therefore exert considerable impact on drug development. In this work, enabling technologies identified by the EMA are analysed to measure their impact on drug development by following their journey from publications through early regulatory interactions to clinical trials between 2019 and 2022. This work also reviews the current list of EMA-identified ETs by scrutinising previously unseen innovative technologies identified in EMA submissions data. The analysis shows large variations in the appearance of the various innovative technologies in the different studied data sources, which provided valuable insights into the "Journey of Innovation" that innovative technologies undergo. Several emerging technologies were identified and endorsed for inclusion in the enabling technologies list, whereas some others already on the list were proposed to be excluded due to their low appearance in regulatory interactions as well as clinical trials and publications. Overall, this analysis highlights the relevance and value of continuously scanning and monitoring enabling technologies, supporting Europe's goal to remain a leader in research and development of innovative technologies, methods, and methodologies relevant to drug development.

Biomarkers are important tools in medicines development and clinical practice. Besides their use in clinical trials, such as for enrichment of patients, monitoring safety or response to treatment, biomarkers are a cornerstone of precision medicine. The European Medicines Agency (EMA) emphasised the importance of the discovery, qualification, and use of biomarkers in their Regulatory Science Strategy to 2025, which included the recommendation to enhance early engagement with biomarker developers to facilitate regulatory qualification. This study explores the journey of biomarkers through the EU regulatory system and beyond, based on a review of interactions between developers and the EMA from 2008 to 2020, as well as the use of qualified biomarkers in clinical trials. Of applicants that used early interaction platforms such as the Innovation Task Force, less than half engaged in fee-related follow-up procedures. Results showed that, as compared to companies, consortia were more likely to opt for the Qualification of Novel Methodologies procedure and engage in follow-up procedures. Our results highlight the importance of early engagement with regulators for achieving biomarker qualification, including pre-submission discussions in the context of the qualification procedure. A review of clinical trials showed that all qualified biomarkers are used in practice, although not always according to the endorsed context of use. Overall, this study highlights important aspects of biomarker qualification, including opportunities to improve the seamless support for developers by EMA. The use of qualified biomarkers in clinical trials underlines the importance of regulatory qualification, which will further enable precision medicine for the benefit of patients.

Regulatory qualification of biomarkers facilitates their harmonized use across drug developers, enabling more personalized medicine. This study reviews various aspects of the European Medicines Agency’s (EMA’s) biomarker qualification procedure, including frequency and outcome, common challenges, and biomarker characteristics. Our findings provide insights into the EMA’s biomarker qualification process and will thereby support future applications. All biomarker‐related “Qualification of Novel Methodologies for Medicine Development” procedures that started from 2008 to 2020 were included. Procedural data were extracted from relevant documents and analyzed descriptively. In total, 86 biomarker qualification procedures were identified, of which 13 resulted in qualified biomarkers. Whereas initially many biomarker qualification procedures were linked to a single company and specific drug development program, a shift was observed to qualification efforts by consortia. Most biomarkers were proposed (n = 45) and qualified (n = 9) for use in patient selection, stratification, and/or enrichment, followed by efficacy biomarkers (37 proposed, 4 qualified). Overall, many issues were raised during qualification procedures, mostly related to biomarker properties and assay validation (in 79% and 77% of all procedures, respectively). Issues related to the proposed context of use and rationale were least common yet were still raised in 54% of all procedures. While few qualified biomarkers are currently available, procedures focus increasingly on biomarkers for general use instead of those linked to specific drug compounds. The issues raised during qualification procedures illustrate the thorough discussions taking place between applicants and regulators—highlighting aspects that need careful consideration and underlining the importance of an appropriate validation strategy.

Background: An increasing number of medicines authorised in Europe recommend or require biomarker-based patient selection. For some of these the use of a companion diagnostic (CDx), a subset of in vitro diagnostics (IVDs), to identify patient populations eligible for a specific medicinal product may be required. The information and recommendations of use of a medicinal product for which a CDx is required is particularly important to healthcare professionals for correct patient identification. Methods: We reviewed the existing information in SmPCs and European Public Assessment Reports (EPARs) of EU medicinal products approved via the centralised procedure at EMA where reference was made to biomarker testing, including by CDx, for patient selection. Results: The results show that varying levels of detail are provided for the biomarker and the diagnostic test, including variability in where the information was presented. The overall results demonstrate transparent but sometimes heterogeneous reporting of CDx in the SmPC and EPAR. Conclusions: With the introduction of the new Regulation (EU) 2017/746 on in vitro diagnostic medical devices, medicines regulatory authorities' will be required to be consulted during the review of CDx conformity assessment and so, there is opportunity for more consistent and transparent information on CDx to be provided in the SmPC and EPAR.

With rapid advancements in medical science and technology, development of medicines moves ever more into the direction of personalized medicine; often referred to as targeted or precision medicines...

The ability to benefit from knowledge of human genomic data in medicine has been anticipated since the sequencing of the human genome. That promise has experienced some degree of realization, particularly in oncology where biomarker-specific clinical trials and patient treatment specific to the genetics of their tumours now occur. With whole genome sequencing and related technologies becoming more affordable, and the generation and management of vast amounts of data and information, more capable, new opportunities to benefit from these developments lie ahead. Already emerging are many studies describing the association of genomic variation with molecular underpinnings of disease, association with patient response to drugs and informing the nomination of new drug targets. These developments are accompanied by some ethical, legal and regulatory challenges, which we discuss in this article.

Nanomedicines simultaneously present themselves with a wide range of technological, scientific, regulatory, and legal challenges. Each of these challenges requires an early awareness, appropriate scientific and methodological expertise, and consensus on the more suitable and applicable regulatory requirements. By combining mechanical, chemical, metabolic, pharmacological, and immunological properties nanomedicines may provide benefit to patients in new ways not yet explored by classical active substances or traditional delivery systems, while at the same time present significant regulatory challenges concerning the data requirements in support of their quality, safety, and efficacy. This chapter builds on the remaining chapters of this book where the scientific aspects and benefits of the application of nanotechnology in medicinal products are presented in detail. The chapter focuses on the current regulatory framework applicable to nanomedicines. An overview of European Union pharmaceutical legislation will be presented with an emphasis on specific aspects that should be considered by developers of biomedical applications using functionalized nanomaterials. An introductory overview of the current standards of pharmaceutical development and applicable scientific guidance will be provided. And also, how depending on properties of the nanomaterial/nanosystem, different requirements apply, e.g., medicinal product versus medical device, will also be presented. Finally, it will be detailed how the regulatory science is benefitting from the existing collaborative initiatives on nanotechnology.