May patient’s registries, and observational studies underlying, be useful tools for decision making about the cost-effectiveness of new drugs?
A Practical Guide for Using Registry Data to Inform Decisions About the Cost Effectiveness of New Cancer Drugs: Lessons Learned from the PHAROS Registry
Hedwig M. Blommestein, Margreet G. Franken, Carin A. Uyl-de Groot
Decision makers increasingly request evidence on the real-world cost effectiveness of a new treatment. Consideration of costs and cost effectiveness appear to be crucial for decision making on healthcare resources allocation.
In this paper, authors present a practical guide on use of registry data to sustain decisions about the cost effectiveness of new drugs. They discuss the required steps to define a sound economic evaluation, by using the Population-based Hematological Registry for Observational Studies (PHAROS) as an example.
PHAROS is a population-based diseases registry that started in 2010 with three hematologic malignancies (non-Hodgkin lymphoma, multiple myeloma, and chronic lymphatic leukemia) in three regions. Like many other registries, PHAROS was created to serve multiple purposes including measuring and improving the quality of care and evaluating the clinical and cost effectiveness of treatments used in a real-world setting.
Despite randomized clinical trials (RCT) aim to demonstrate the efficacy of interventions and ensure internal validity by randomly assigning which patients receive the new intervention, the cost-effectiveness evaluation based on RCT data, therefore, may not be sufficiently informative for decision makers. In such cases, evidence is needed from other sources, for example patient registries. A patient registry enables the evaluation of specified outcomes for a population affected by a particular disease, condition, or exposure. A patient registry, if it is thoroughly designed and performed, can provide real-world evidence of clinical practice, patient outcomes, safety, and comparative effectiveness.
There are three main issues related to use of 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.
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