Real-world evidence use in assessments of cancer drugs by NICE.

Ash Bullement,Mark J. Robinson,Anthony J. Hatswell,Eugene Benson,Gemma E. Shields,Tanja Podkonjak,Ross Selby

doi:10.1017/s0266462320000434

Abstract

To establish how real-world evidence (RWE) has been used to inform single technology appraisals (STAs) of cancer drugs conducted by the National Institute for Health and Care Excellence (NICE). STAs published by NICE from April 2011 to October 2018 that evaluated cancer treatments were reviewed. Information regarding the use of RWE to directly inform the company-submitted cost-effectiveness analysis was extracted and categorized by topic. Summary statistics were used to describe emergent themes, and a narrative summary was provided for key case studies. Materials for a total of 113 relevant STAs were identified and analyzed, of which nearly all (96 percent) included some form of RWE within the company-submitted cost-effectiveness analysis. The most common categories of RWE use concerned the health-related quality of life of patients (71 percent), costs (46 percent), and medical resource utilization (40 percent). While sources of RWE were routinely criticized as part of the appraisal process, we identified only two cases where the use of RWE was overtly rejected; hence, in the majority of cases, RWE was accepted in cancer drug submissions to NICE. RWE has been used extensively in cancer submissions to NICE. Key criticisms of RWE in submissions to NICE are seldom regarding the use of RWE in general; instead, these are typically concerned with specific data sources and the applicability of these to the decision problem. Within an appropriate context, RWE constitutes an extremely valuable source of information to inform decision making; yet the development of best practice guidelines may improve current reporting standards.

Highlights

IntroductionA successful reimbursement decision by funding bodies is required for patient access to new treatments
Following regulatory approval, a successful reimbursement decision by funding bodies is required for patient access to new treatments
real-world evidence (RWE) has been used in a multitude of applications to inform healthcare decision making by National Institute for Health and Care Excellence (NICE), in the field of oncology where clinical trials often do not provide all evidence required to inform cost-effectiveness analysis

Summary

Introduction

A successful reimbursement decision by funding bodies is required for patient access to new treatments. Since the inception of NICE in 1999, a total of 544 technology appraisals (TAs, as of January 2019) have been published across a range of different disease areas, of which 240 were of cancer drugs [1]. Additional nontrial data may provide useful information to decision makers, as it is seldom the case that trials are able to provide all evidence necessary to inform decision making. This is prevalent in oncology, where treatments for rare cancers may receive regulatory approval based on data from an uncontrolled (Phase II) trial, which may lack necessary evidence for health technology assessment (HTA)

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