OBJECTIVES: There is no formal guidance from Health Technology Assessment (HTA) agencies regarding the selection of generic preference-based measures (PBM) for use in clinical studies with pediatric populations. This can lead to challenges in valuing benefits associated with pediatric treatments in economic evaluations. This methodological research aimed to produce a decision-making framework for selection of PBMs for pediatric populations in trials.

METHODS: Available pediatric PBMs were identified from published systematic reviews of pediatric PBMs and catalogued to understand the range of possible measures for which a decision framework was needed. The study team collectively identified the appropriate elements and a process map was created, which documented a decision algorithm for PBM selection.

RESULTS: Fourteen generic pediatric (including adolescent) PBMs were identified. A repository was created, containing a catalog of the PBMs and over two dozen relevant properties (e.g. target age group, concepts covered, country value sets available, etc) to consider during PBM selection. The study team identified choices to be made by the “Users” and “Expert Users” via a “Process Map” of five discrete steps: 1) Understand existing evidence related to PBM for population of interest, 2) Define disease relevant population characteristics (i.e. age, disease concepts, etc.), 3) Select a PBM from the repository based on the defined trial population characteristics, 4) Verify PBM selection considering all relevant properties and “Expert User” consultation, 5) Discussion of pros/cons with the trial team prior to making a final decision.

CONCLUSIONS: Initial assessment of the Process Map suggests that the use of this decision-making framework may enable a systematic and transparent method for optimizing the selection of appropriate existing pediatric PBMs into studies, and an accurate estimation of utilities for pediatric populations. More work is underway to assess the accuracy and usefulness of the tool.