A Future European Scientific Dialogue Regulatory Framework: Connecting the Dots.

The regulatory framework of the European Union (EU) offers multiple and valuable options for Scientific Advice (SA). However, at a time of increasing scientific complexity and global competition, navigating the SA landscape may be challenging. Such challenges are related to the technicalities of the framework itself, as well as to fundamental changes in the development of promising therapeutics. This article provides an overview of these challenges and reflects on the ways in which the already available SA options could be consolidated and optimized for building an integrated, easy-to-navigate process. The key elements of the proposal are improved orientation and navigation support, a simplified process of managing parallel interactions with multiple bodies, competitive SA timelines, consistency and harmonization across stakeholders, a strengthened horizon scanning to increase network preparedness, and a mechanism for building an institutional memory. The article builds on ongoing dialogues driven by the European Medicines Agency and the European Medicines Regulatory Network, and contributes the viewpoint of the European Federation of Pharmaceutical Industries on the ways in which the EU SA framework needs to evolve to provide effective Scientific Dialogue throughout the medicine lifecycle. The article is timely because of the current discussion on the future Scientific Dialogue framework and may inform forthcoming legislative changes in the draft General Pharma Legislation revision and how they are practically implemented.

Dynamic Regulatory Assessment: Unpacking the Process Reveals Readiness to Pilot for Europe-An EFPIA View.

The medicines regulatory network of the European Economic Area comprises 30 countries, their National Competent Authorities (NCA), and the European Medicines Agency (EMA). The NCAs and EMA are involved at different stages of the medicine life cycle; not all are engaged in a particular medicine's development discussions. As a result, knowledge management (ie, acquisition and transfer between medicine developer and the NCAs) is fragmented and inefficient. Dynamic regulatory assessment (DRA), a regulatory science concept developed by the European Federation of Pharmaceutical Industries and Associations (EFPIA), could drive increased connectedness supporting more continuous knowledge building. DRA works via iterative release and assessment of discrete data packets (DDPs) at mutually agreed milestones during development, culminating in more efficient development and faster authorization. This commentary seeks to build on an earlier article by unpacking the DRA concept, with a particular focus on DDPs. Its aim is to show how DDPs can support efficient and predictable release of data to encourage development and assessment of promising medicines, and it makes the case for piloting the DRA concept with European regulators now.

Real-World Evidence for Regulatory Decision-Making: Guidance From Around the World.

PURPOSE: Interest in leveraging real-world evidence (RWE) to support regulatory decision making for product effectiveness has been increasing globally as evident by the increasing number of regulatory frameworks and guidance documents. However, acceptance of RWE, especially before marketing for regulatory approval, differs across countries. In addition, guidance on the design and conduct of innovative clinical trials, such as randomized controlled registry studies, pragmatic trials, and other hybrid studies, is lacking. METHODS: We assessed the global regulatory environment with regard to RWE based on regional availability of the following 3 key regulatory elements: (1) RWE regulatory framework, (2) data quality and standards guidance. and (3) study methods guidance. FINDINGS: This article reviews the available frameworks and existing guidance from across the globe and discusses the observed gaps and opportunities for further development and harmonization. IMPLICATIONS: Cross-country collaborations are encouraged to further shape and align RWE policies and help establish frameworks in countries without current policies with the goal of creating efficiencies when considering RWE to support regulatory decision-making globally.

Dynamic Regulatory Assessment: evolving the European Regulatory Framework for the Benefit of Patients and Public Health-an EFPIA View.

The European Union regulatory framework supports development, review, authorization, and maintenance of medicines to benefit public health; however, many elements are 2 decades old and undergoing review. Scrutiny was triggered by the coronavirus disease 2019 pandemic, the need to support future innovative medicines, the digital transformation of data exchange, and the need to address efficiency and capacity limitations. There are also ongoing evolutions in regulatory science for medicines (eg, cell and gene therapies), medical device combinations, and software, as well as the need to fully leverage contemporary information technology (IT). Important initiatives to address these challenges include the European Medicines Agency (EMA) Regulatory Science Strategy,1 the EU Regulatory Network Strategy,2 and the Big Data Steering Group,3 alongside European Commission-led initiatives such as the Pharmaceutical Strategy.4 Dynamic regulatory assessment (DRA) is a concept that seeks to integrate these various elements to re-imagine regulatory review interactions across the product life cycle. DRA calls for iterative regulatory dialogue, data submission, and evidence assessment, enabled by contemporary IT. DRA will facilitate iterative interaction and data assessment as it accumulates over a product's life cycle, bringing significant efficiencies for all product types. The DRA concept primarily evolved through dialogue within working groups of the European Federation of Pharmaceutical Industries and Associations. This article describes the long-term vision of the European Federation of Pharmaceutical Industries and Associations and outlines important strategic elements of progress, including: aligning on a multi-stakeholder vision for DRA in the European Union and across regions; leveraging learnings from ongoing initiatives; and advancing IT, governance, and standards considerations. Ultimately, DRA should consider outcomes that deliver optimal benefits for patients in the European Union and worldwide.