The European Medicines Agency’s goals for regulatory science to 2025

It is increasingly important that regulatory agencies catalyse the translation of scientific and technological innovation into improved patient-centred health care. Here, we highlight the strategic goals of the European Medicines Agency to advance regulatory science and optimize the opportunities arising in areas such as cell and gene therapies, big data and artificial intelligence.</p

Regulatory Science and Innovation Programme for Europe (ReScIPE):A proposed model

Regulatory science underpins the objective evaluation of medicinal products. It is therefore imperative that regulatory science and expertise remain at the cutting edge so that innovations of ever-increasing complexity are translated safely and swiftly into effective, high-quality therapies. We undertook a comprehensive examination of the evolution of science and technology impacting on medicinal product evaluation over the next 5–10 years and this horizon-scanning activity was complemented by extensive stakeholder interviews, resulting in a number of significant recommendations. Highlighted in particular was the need for expertise and regulatory science research to fill knowledge gaps in both more fundamental, longer-term research, with respect to technological and product-specific challenges. A model is proposed to realise these objectives in Europe, comprising a synergistic relationship between the European Medicines Agency, the European Medicines Regulatory Network and academic research centres to establish a novel regulatory science and innovation platform.</p

EMEA and Gene Therapy Medicinal Products Development in the European Union

The evaluation of quality, safety, and efficacy of medicinal products by the European Medicines Evaluation Agency (EMEA) via the centralized procedure is the only available regulatory procedure for obtaining marketing authorization for gene therapy (GT) medicinal products in the European Union. The responsibility for the authorization of clinical trials remains with the national competent authorities (NCA) acting in a harmonized framework from the scientific viewpoint. With the entry into force of a new directive on good clinical practice implementation in clinical trials as of 1 May 2004, procedural aspects will also be harmonized at EU level. Scientifically sound development of medicinal products is the key for the successful registration of dossiers and for contributing to the promotion and protection of public health. The objective of this paper is to introduce the EMEA regulatory processes and scientific activities relevant to GT medicinal products

Regulatory Science and Innovation Programme for Europe (ReScIPE):A proposed model

Regulatory science underpins the objective evaluation of medicinal products. It is therefore imperative that regulatory science and expertise remain at the cutting edge so that innovations of ever-increasing complexity are translated safely and swiftly into effective, high-quality therapies. We undertook a comprehensive examination of the evolution of science and technology impacting on medicinal product evaluation over the next 5-10 years and this horizon-scanning activity was complemented by extensive stakeholder interviews, resulting in a number of significant recommendations. Highlighted in particular was the need for expertise and regulatory science research to fill knowledge gaps in both more fundamental, longer-term research, with respect to technological and product-specific challenges. A model is proposed to realise these objectives in Europe, comprising a synergistic relationship between the European Medicines Agency, the European Medicines Regulatory Network and academic research centres to establish a novel regulatory science and innovation platform.</p

Genetic tests and genomic biomarkers: regulation, qualification and validation

The identification of new risk factors for specific diseases is an enduring theme in medical research. Advances in molecular biology, genetics, and computational biology are accelerating the pace of this work. The research seeks to increase our understanding of the causes of diseases, but there is also hope that the recognition of new risk factors will lead to improved methods for identifying persons who are in the early stages of, or at high risk for, the diseases of concern. Research has shown, however, that a genomic biomarker must have a much stronger association with the disease outcome than we ordinarily see in etiologic research if it is to provide a basis for early diagnosis or prediction in individual patients. However, even if the literature contains ~150,000 reports of disease-associated molecular markers, there are still very few validated biomarkers of proven and robust clinical utility. At present there is no established, standardized means for validating the association between a marker (or set of markers) and clinical outcomes. The Regulatory Authorities have undertaken a number of initiatives in order to enhance the use of biomarkers in drug development, to promote a more informed drug development and maximise the benefit of innovative medicines to the patients

Scanning the horizon:a systematic literature review of methodologies

Society is confronted with the rapid emergence of innovation in science and technology. To manage this, horizon scanning is being adopted globally to identify, assess and prioritise innovations and trends at an early stage of their development. This enables decision-makers to be better informed and to prepare for change. The aim of this paper is to systematically identify and evaluate horizon scanning methodologies employed in the healthcare and biomedical fields.Methods A systematic literature review was performed using PubMed and Embase and was supplemented with grey literature searches (2008-2018). The principal methodologies used in horizon scanning were extracted.Results Approximately 100 articles were summarised in a literature map. The search revealed many examples of horizon scanning across disciplines. Challenges, such as the need to refine prioritisation criteria, manage uncertainty inherent in the findings and improve the dissemination of identified issues, have been highlighted.Conclusion Horizon scanning, when performed appropriately, is a flexible and potentially reliable tool, with a wide variety of methods. Horizon scanning can inform and influence decision-making, through identifying opportunities and challenges, from an organisational to an international level. Further research to identify the most effective methodologies available would add depth to this landscape and enable the evolution of best practice to most efficiently anticipate novel developments and innovations