Table 6 Triangulation with key recommendations on the economic evaluation alongside clinical or pragmatic trials

1

Study design:

Based on well-designed pragmatic/naturalistic trials with fewer strict protocols

2

Selection of subjects and sites:

Seek for proximity to real-world target population and less restrictive patient inclusion criteria

3

Sample size:

Based on important clinical outcome correlated with economic outcome, previous pilot or wider CI for ICER/CEAC

4

Estimates beyond trial:

Appropriate length of follow-up, estimates beyond trial require survival analysis, link to final outcomes or regression

5

Comparator:

Current practice or standard of care should be the comparator, although there may be different standards of care

6

Measures of outcomes:

Direct, single measures are preferred. Utilities collected directly from study subjects at regular intervals

7

Data collection (resource use and costs):

Relevant resource use and cost measures collected with clinical data (case report forms, patient records, patient diaries, interviews, computerized record linkage)

8

Valuation of costs:

May include: microcosting; unit costing; and gross costing

9

Methods for cost and outcome analysis:

Arithmetic mean cost differences, bootstrapping, OLS or GLM for between group comparison; multivariable methods for outcomes; confidence intervals, p values, ICERs on various time horizons; summary measures

10

Reporting:

General description of trial and major findings; economic data collected alongside trial; missing and censored data; methods to construct, compare and project costs and outcomes; statistical methods; results on resource use, costs and outcomes; results within and beyond time horizon of trial.