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.

Regulatory Pathways Supporting Expedited Drug Development and Approval in ICH Member Countries.

Regulators and pharmaceutical companies across the world are intensifying efforts to get increasingly complex and innovative drugs to patients with high unmet medical need in the shortest possible time frame. This article reviews pathways to expedite drug development and approval available in member countries of the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use and Australia. It is concluded that the increasing availability of expedited regulatory pathways and associated modernisation of regulatory systems changes the current regulatory paradigm and requires sponsors to rethink drug development and regulatory strategy. A transformation of the current sequence of regulatory submissions, favouring those countries/collaborations that are best regulatory equipped to make innovative medical need drugs available to patients in the shortest time frame is imminent.

Evaluation of patient engagement in medicine development: A multi-stakeholder framework with metrics.

BACKGROUND: Patient engagement is becoming more customary in medicine development. However, embedding it in organizational decision-making remains challenging, partly due to lack of agreement on its value and the means to evaluate it. The objective of this project was to develop a monitoring and evaluation framework, with metrics, to demonstrate impact and enhance learning. METHODS: A consortium of five patient groups, 15 biopharmaceutical companies and two academic groups iteratively created a framework in a multi-phase participatory process, including analysis of its application in 24 cases. RESULTS: The framework includes six components, with 87 metrics and 15 context factors distributed among (sub)components: (a) Input: expectations, preparations, resources, representativeness of stakeholders; (b) Activities/process: structure, management, interactions, satisfaction; (c) Learnings and changes; (d) Impacts: research relevance, study ethics and inclusiveness, study quality and efficiency, quality of evidence and uptake of products, empowerment, reputation and trust, embedding of patient engagement; (e) Context: policy, institutional, community, decision-making contextual factors. Case study findings show a wide variation in use of metrics. There is no 'one size fits all' set of metrics appropriate for every initiative or organization. Presented sample sets of metrics can be tailored to individual situations. CONCLUSION: Introducing change into any process is best done when the value of that change is clear. This framework allows participants to select what metrics they value and assess to what extent patient engagement has contributed. PATIENT CONTRIBUTION: Five patient groups were involved in all phases of the study (design, conduct, interpretation of data) and in writing the manuscript.

Evaluating the "return on patient engagement initiatives" in medicines research and development: A literature review.

BACKGROUND: Showing how engagement adds value for all stakeholders can be an effective motivator for broader implementation of patient engagement. However, it is unclear what methods can best be used to evaluate patient engagement. This paper is focused on ways to evaluate patient engagement at three decision-making points in the medicines research and development process: research priority setting, clinical trial design and early dialogues with regulators and health technology assessment bodies. OBJECTIVE: Our aim was to review the literature on monitoring and evaluation of patient engagement, with a focus on indicators and methods. SEARCH STRATEGY AND INCLUSION CRITERIA: We undertook a scoping literature review using a systematic search, including academic and grey literature with a focus on evaluation approaches or outcomes associated with patient engagement. No date limits were applied other than a cut-off of publications after July 2018. DATA EXTRACTION AND SYNTHESIS: Data were extracted from 91 publications, coded and thematically analysed. MAIN RESULTS: A total of 18 benefits and 5 costs of patient engagement were identified, mapped with 28 possible indicators for their evaluation. Several quantitative and qualitative methods were found for the evaluation of benefits and costs of patient engagement. DISCUSSION AND CONCLUSIONS: Currently available indicators and methods are of some use in measuring impact but are not sufficient to understand the pathway to impact, nor whether interaction between researchers and patients leads to change. We suggest that the impacts of patient engagement can best be determined not by applying single indicators, but a coherent set of measures.