Pearson, S., Dreitlein, B., Towse, A., Hampson, G. and Henshall, C.
OHE Research Paper 18/02: Real World Evidence (RWE) – This paper provides a framework and specific steps to help both manufacturers and payers meet the challenge of developing observational RWE through a transparent process that can be considered credible by all stakeholders.
It presents a new conceptual framework to address three elements largely missing from these earlier efforts focused on defining “best practices” or “standards” for RWE. These are: 1) how to understand the role that contextual factors play in determining how high the evidentiary standard, or “bar” will be in each situation; 2) how to tailor key process and methodological approaches the height of that evidentiary bar; and 3) how to ensure that broader process principles that support transparency are integrated successfully throughout the course of any RWE initiative.
This paper is a companion piece to a detailed background paper on the use of RWE in coverage decisions developed for the Institute for Clinical and Economic Review (ICER) Policy Summit held in December 2017.
Hampson, G., Towse, A., Dreitlein, B., Henshall, C. and Pearson, S.
OHE Research Paper 18/01: This paper sets out the potential opportunities and important challenges and limitations that must be addressed in considering options for using RWE to inform insurer coverage decisions. The primary purpose of developing the paper was to stimulate discussion at the 2017 ICER Policy Summit meeting. A separate paper is available that summarises the authors reflections and proposed ways forwards based on the discussions that were had at the meeting.
RWE is already utilised for multiple purposes in the US and globally. The focus of the 2017 ICER Policy Summit was on RWE for HTA assessments and payer coverage decisions - both initial decisions and reassessments – and the majority of the paper focuses on this.
Acceptance of an expanded future role for RWE is not universal, particularly if it is seen as reducing the amount of RCT evidence available. Among the challenges associated with RWE explored in this paper are: bias and confounding; incomplete data; data mining; access to data; and the lack of universally accepted methodological standards.
Current uses of RWE can be strengthened by increasing the quality and credibility of RWE (via national registries, data repositories and strict protocols) and establishment of appropriate governance arrangements.
Key opportunities for the future include use of innovative study designs that combine benefits of collecting data from real world settings while incorporating best practice methods (i.e. randomisation methods from traditional RCTs); real time monitoring of patients; and the development of adaptive regulatory pathways linked to coverage with evidence development.
Zamora, B., Parkin, D., Feng, Y., Bateman, A., Herdman, M., and Devlin, N.
This paper reports new methods for analysing the distribution of EQ-5D observations. The Health State Density Index and Health State Density Curve are used to summarise the extent to which people’s self-reported health on the EQ-5D is concentrated on a few health states, or distributed more evenly over a wide range of health states. This information can be useful in understanding patients’ treatment needs, as well as providing a way of comparing the nature of data provided by different patients or collected using different patient-reported outcome measures.
Devlin, N., Shah, K., Mulhern, B., Pantiri, K. and van Hout, B.
Standard methods for eliciting the preference data upon which value sets are based (e.g. time trade-off, standard gamble) generally have in common an aim to ‘uncover’ the preferences of survey respondents by asking them to evaluate a sub-set of health states. The responses are then used to infer their preferences over all possible dimensions and levels. An alternative approach is to ask respondents directly about the relative importance to them of the dimensions, levels and interactions between them
Questions of happiness and well-being have increasingly been drawing the attention of health economists, with the understanding that its measures approximate quality of life, or at any rate is one of its major components. Happiness in surveys is typically reported as a rating scale.
Hampson, G., Lichten, C., Berdud, M., Pollitt, A., Mestre-Ferrandiz, J., Sussex, J., and Towse, A.
The Oxford Biomedical Research Centre (BRC) was established in April 2007. OHE and RAND Europe were commissioned by the Oxford BRC to undertake a programme of top-down evaluations of aspects of the impact of the BRC since its inception.
Cubi-Molla, P., Shah, K.K., Garside, J., Herdman, M. and Devlin, N.
Several studies have found differences in health state values by age. We investigate whether and how age affects respondents’ Time Trade-Off (TTO) and Visual Analogue Scale (VAS) valuations of hypothetical EQ-5D health states using data from the 1993 MVH UK valuation study.
Mulhern, B., Feng, Y., Shah, K., van Hout, B., Janssen, B., Herdman, M. and Devlin, N.
Three EQ-5D value sets (the EQ-5D-3L, crosswalk and EQ-5D-5L) are now available for cost utility analysis in the UK and/or England. The value sets’ characteristics differ, and it is important to systematically assess the implications of these differences for the value generated. The aim of this paper is to compare the characteristics of the three value sets. In this Research Report we analyse and compare the predicted values from each of the three value sets, and also compare EQ-5D-3L and EQ-5D-5L data from patients who completed both measures.
Marsden, G., Towse, A., Pearson, S.D., Dreitlein, B. and Henshall, C.
This report presents an analysis of the significant clinical potential of gene therapy and the unique challenges in developing and evaluating evidence on their effectiveness and value. Special attention is given to pricing and payment mechanisms, including new approaches to payment based on long-term amortization of initial costs.
The aim of this paper is to consider what normative arguments might exist for advocating the use of any given measure of the average in the context of health state values. We begin by providing examples of the importance and implications of the choice of the measure of central tendency in stated preference studies (including both EQ-5D values and corresponding issues in the willingness to pay literature).