Report of the European Medicines Agency Conference on RNA-Based Medicines.

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.

Queries Raised During Oncology Business Pipeline Meetings at the European Medicines Agency: A 5-Year Retrospective Analysis.

The European Medicines Agency (EMA) offers guidance and support to pharmaceutical companies through bilateral discussions called business pipeline meetings (BPMs). An analysis of BPMs in oncology over a 5-year period was conducted to identify common topics and recurring queries. The documents of all BPMs available at the EMA regarding the field of oncology from January 1, 2018, to Decemer 31, 2022, were reviewed.

Enabling technologies driving drug research and development.

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.

One Health collaboration with and among EU Agencies - Bridging research and policy.

In the coming decade, Europe will dedicate billions of euros to the necessary research and innovation (R&I) to support a transition to safe and sustainable food systems. EU Agencies, individually and even more so collectively, can make a difference in supporting the European research agenda. EU Agencies are knowledge centres, bringing together know-how to inform policy makers.

Biomarkers in Medicines Development-From Discovery to Regulatory Qualification and Beyond.

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.

Regulatory Science to 2025: An Analysis of Stakeholder Responses to the European Medicines Agency's Strategy.

The pace of innovation is accelerating, and so medicines regulators need to actively innovate regulatory science to protect human and animal health. This requires consideration and consultation across all stakeholder groups. To this end, the European Medicines Agency worked with stakeholders to draft its and launched it for public consultation.

Randomized Controlled Trials Versus Real World Evidence: Neither Magic Nor Myth.

Compared with drugs from the blockbuster era, recently authorized drugs and those expected in the future present a heterogenous mix of chemicals, biologicals, and cell and gene therapies, a sizable fraction being for rare diseases, and even individualized treatments or individualized combinations. The shift in the nature of products entails secular trends for the definitions of "drugs" and "target population" and for clinical use and evidence generation. We discuss that the lessons learned from evidence generation for 20th century medicines may have limited relevance for 21st century medicines.

Are Novel, Nonrandomized Analytic Methods Fit for Decision Making? The Need for Prospective, Controlled, and Transparent Validation.

Real-world data and patient-level data from completed randomized controlled trials are becoming available for secondary analysis on an unprecedented scale. A range of novel methodologies and study designs have been proposed for their analysis or combination. However, to make novel analytical methods acceptable for regulators and other decision makers will require their testing and validation in broadly the same way one would evaluate a new drug: prospectively, well-controlled, and according to a pre-agreed plan.

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.

Gold(I) chloride adducts of 1,3-bis(di-2-pyridylphosphino)propane: synthesis, structural studies and antitumour activity.

The novel water soluble bidentate phosphine ligand 1,3-bis(di-2-pyridylphosphino)propane (d2pypp) has been synthesized by a convenient route involving treatment of 2-pyridyllithium with Cl(2)P(CH(2))(3)PCl(2) and isolation in crystalline form as the hydrochloride salt. The synthesis of the precursor Cl(2)P(CH(2))(3)PCl(2) has been optimized by the use of triphosgene as the chlorinating agent. The 2 : 1 and 1 : 2 AuCl : d2pypp adducts have been synthesized and characterized by NMR spectroscopy and single crystal X-ray studies, and shown to be of the form (AuCl)(2)(mu-d2pypp-P,P') and [Au(d2pypp-P,P')(2)]Cl(.

Long range 1,4 and 1,6-interstrand cross-links formed by a trinuclear platinum complex. Minor groove preassociation affects kinetics and mechanism of cross-link formation as well as adduct structure.

Reported here is a comparison of the kinetics of the stepwise formation of 1,4- and 1,6-GG interstrand cross-links by the trinuclear platinum anticancer compound (15)N-[[trans-PtCl(NH(3))(2)](2)[mu-trans-Pt(NH(3))(2)(H(2)N(CH(2))(6)NH(2))(2)]](4+), (1,0,1/t,t,t (1) or BBR3464). The reactions of (15)N-1 with the self-complementary 12-mer duplexes 5'-[d(ATATGTACATAT)(2)] (I) and 5'-[d(TATGTATACATA)(2)] (II) have been studied at 298 K, pH 5.3 by [(1)H,(15)N] HSQC 2D NMR spectroscopy.