The Power of the Patient

Introduction

In this paper, we aim to define the different types of burden of illness studies and highlight the importance of qualitative burden of illness studies as complementary to patient reported outcomes data, and as separate stand-alone research projects that can further support HTA committees in their decision-making.

Health Technology Assessment (HTA) agencies are increasingly accepting of burden of illness (BoI) studies* to support submissions, particularly in rare diseases. However, based on our experience, we find there is some confusion about the various types of BoI studies and when best to use each type to support HTA submissions. In fact, BoI studies are often narrowly thought of as studies generating patient-reported outcomes (PROs) alone. This is confounded by the fact that there are several definitions of BoI studies and multiple different methods for BoI data collection, which can be confusing. For example, in the UK, guidance documents from the National Institute for Health and Care Excellence (NICE), describe BoI in relation to quality-adjusted life year (QALY) weighting and utility scores for PROs1 and potential methods for data collection. This includes ‘ad hoc surveys or primary qualitative research’² or simply ‘qualitative
research’.^{3, 4}