Practical Methods to Support Research Prioritisation Decisions: Rapid Assessment of the Need for Evidence

This thesis aims to further the use of value of information (VOI) methods in research prioritisation. It comprises seven chapters and a freely available online tool. The Chapter 1 provides an introduction to VOI and an overview of the methods that have thus far been developed to address the practical barriers to its use. Chapter 2 illustrates the rapid VOI methods which are the focus of this thesis using a case study from one of six retrospective proposals provided by the national institute for health research (NIHR) trials and studies coordinating centre (NETSCC). This provides an estimate of the value of research in terms of research cost required to gain an additional unit of a disease specific binary outcome. Chapter 3 provides an approach to addressing the challenge of prioritising across disease areas by linking disease specific binary outcomes to a generic measure of health outcome: quality adjusted life years (QALYs). The implications of using a generic health outcome in research prioritisation are explored. Chapter 4 extends the methods from Chapters 2 and 3 to allow for analysis of a wider range of outcomes (including continuous and survival outcomes) and develops a method for estimating the VOI provided by a feasibility studies. Chapter 5 introduces the online tool which implements each method described in previous chapters (https://shiny.york.ac.uk/rane/.) Chapter 6 addresses the question of how to make early access decisions without a full economic model. Granting early access means that treatments can be quickly provided without delay. The cost of this is that it can remove the possibility of research. This chapter provides a framework for early access decisions which is built upon the rapid methods described in previous chapters. Chapter 7 concludes by providing a brief overview of the entire thesis and identifies avenues for future research and policy.