A Practical Guide for Using Registry Data to Inform Decisions About the Cost Effectiveness of New Cancer Drugs: Lessons Learned from the PHAROS Registry

Abstract

Decision makers increasingly request evidence on the real-world cost effectiveness of a new treatment. There is, however, a lack of practical guidance on how to conduct an economic evaluation based on registry data and how this evidence can be used in actual decision making. This paper explains the required steps on how to perform a sound economic evaluation using examples from an economic evaluation conducted with real-world data from the Dutch Population based HAematological Registry for Observational Studies. There are three main issues related to using registry data: confounding by indication, missing data, and insufficient numbers of (comparable) patients. If encountered, it is crucial to accurately deal with these issues to maximize the internal validity and generalizability of the outcomes and their value to decision makers. Multivariate regression modeling, propensity score matching, and data synthesis are well-established methods to deal with confounding. Multiple imputation methods should be used in cases where data are missing at random. Furthermore, it is important to base the incremental cost-effectiveness ratio of a new treatment compared with its alternative on comparable groups of (matched) patients, even if matching results in a small analytical population. Unmatched real-world data provide insights into the costs and effects of a treatment in a real-world setting. Decision makers should realize that real-world evidence provides extremely valuable and relevant policy information, but needs to be assessed differently compared with evidence derived from a randomized clinical trial.

Real-world cost-effectiveness of rivaroxaban compared with vitamin K antagonists in the context of stroke prevention in atrial fibrillation in France

“…Many uncertainties remain regarding the relevance of the results of RCTs in a real-world setting. Real-world evidence (RWE) may provide additional information to decision-makers [8]. Indeed, RWE sample size is not limited as it is the case of RCTs.…”

“…It is expected that between 2015 and 2035, there will be a 34% increase in total number of stroke events in the European Union [7].Oral anticoagulants including vitamin K antagonists (VKAs) or non-VKA oral anticoagulants (NOACs) such as rivaroxaban, dabigatran, apixaban, and edoxaban, have been established as a cornerstone of management in patients with AF and to reduce stroke incidence and mortality [2] in randomized clinical trials (RCTs) [2].Many uncertainties remain regarding the relevance of the results of RCTs in a real-world setting. Real-world evidence (RWE) may provide additional information to decision-makers [8]. Indeed, RWE sample size is not limited as it is the case of RCTs.…”

Levels of research evidence in health policy assessment in Malawi

“…Reliance on registry and internal data as opposed to reliable health research evidence has received condemnation especially on where to place studies derived from registries within the hierarchies of health research evidence (Roovers, 2007). Debate centres on the assessment of registry and internal data regarding methods of grading health research evidence, underlying assumptions, shortcomings in assessing types of evidence and consistency in evaluating the evidence itself (Blommestein, Franken & Uyl-de Groot, 2015).…”

Nursing : 7 key cancer trends

“…Economic assessments can compare the costeffectiveness of alternative therapies and are therefore particularly important for making decisions about the cost of expensive new drugs [17].…”