Philip Hines

Societal progress in everything, from the economy to health care, is largely driven by a combination of scientific advancement and governance. Unfortunately, there are many examples of failures in evidence informing policy, which speak to the need to improve, as do the increasingly complex and globalised challenges facing the EU. This is also the democratic duty of the EU and in the interest of itself its stakeholders and its citizens. Evidence use, scientific advice and the structures and norms that guide them, need reform. This is true across EU institutions and agencies. Even in highly technocratic institutions that are used to using evidence, such as medicines regulation, there is room for improvement but there are also lessons to be learned.

Medicines regulation revolves around the use of evidence in interaction with stakeholders, and as such offers fertile ground for research to improve its own evidence use and to set examples for evidence use by other institutions, in the EU and beyond. Whilst there has been a lot of research conducted into how evidence is used by medicines regulators, there has not been much attention on the translation of these practices into other institutions. This thesis, therefore, looked at evidence use across the life cycle of medicines regulation, how that can be improved, and set out these practices for use by other stakeholders, in other contexts. Whilst this was the bulk of the thesis it also reaches beyond that, stretching from foresight to systematically improving the EU legislative processes.

The first chapter of the thesis looks at horizon scanning as a tool to inform decision making and strategic responses to new trends, challenges and opportunities. Horizon scanning has been proliferating in the bio-medicine sector yet there has been little research on how this should best be conducted. It provides a systematic literature review of the different horizon scanning methodologies and information sources globally. The results of this review were compiled into a map to inform organisations seeking to establish their own horizon scanning. The benefits of horizon scanning were also discussed as a tool to inform policymaking; the lessons learnt in this mapping are applicable across many functions of EU decision-making, particularly those responding to rapidly advancing fields which require a level of anticipation, early evidence and proactivity.

Through applying horizon scanning methods mapped in chapter one, it became clear that there were multiple regulatory knowledge gaps and a need for a regulatory science research agenda driven by the regulators themselves. This chapter sets out the background rationale and proposed mechanism for such a regulatory science research agenda. It details how this could be used to better inform the policymaking of medicines regulators and provides a case for the importance of conducting research to inform policymaking.

Strategic decision-making requires a variety of evidence inputs and methods, the third chapter sets out the methodology used to acquire evidence to inform the EMA’s Regulatory Science Strategy to 2025, and the components of the strategy itself. Part one takes readers through the drafting process of the Strategy, utilising the horizon scanning methods in chapter one. Its final shape after public consultation is then presented in part 2. This is intended to serve as case study in evidence informed strategy development, with the methods applied being replicable in other settings.

The methodology and findings of the public consultation on the draft Regulatory Science Strategy to 2025, as set out in chapter three, part one, were then presented. These social science methods deployed in the consultation, and analysis, are intended to serve as an example of conducting rigorous public consultation. The resulting analysis and underlying consultation data, that fed into chapter three, part two, are then provided as an open science resource for other stakeholders interested in regulatory science. The information provided demonstrates the value of being open about the evidence used to inform decision-making, and about how it was used. This should be valuable for both for stakeholders and decisionmakers themselves.

Transparency in the evidence used in decision-making, and the underlying methodologies, were also explored in the fifth chapter which analysed how the collaboration between regulators and health technology assessment bodies can be improved to facilitate the flow of evidence and decision-making. The chapter lays out a case, with concrete recommendations, of the benefits to improved interface between adjoining decision-makers in terms of optimised evidence generation, evidence use, and evidence transparency. With the streamlined access the medicines benefiting, decision-makers, patients and medicines developers.

The final chapter zooms out from the health sector to EU legislative policymaking in general. The first part outlines the state of play in EU policymaking and critiques the EU’s legislative procedures to highlight their flaws and where there may be room for improvement. The second part of this chapter builds on the findings of the previous chapters to further elaborate on these flaws, and the opportunities for improvement. It then sets out the idea for a European Evidence Ombudsman to correct these flaws and continually improve the use of evidence across European Union institutions, and by extension, the field of evidence informed policymaking.

Each of these chapters have implications for evidence use in EU policy-making. These include those specific to the context of the chapter, and those of general relevance to EIPM in the EU, as set out above. In addition to each chapter’s findings, cumulatively they serve as case studies into how EU institutions currently use evidence. Their recommendations can act as building blocks in advancing the EU's evidence use. And whilst they are by no means comprehensive, they do demonstrate the challenges and opportunities of improved evidence use, and translate lessons for medicines regulation to EIPM more generally; using the lessons learnt from these pieces of research, and building on the growing body of literature on this subject, a deep critique of the EU’s use of evidence is set out.

The findings of the thesis demonstrate a case for continued improvements in EIPM across EU institutions. This would serve the EU’s decision-making efficiency, quality and its legitimacy – something which must be a priority for the enlightened European project.