PMID- 27838913
OWN - NLM
STAT- MEDLINE
DCOM- 20171030
LR  - 20191210
IS  - 1179-2027 (Electronic)
IS  - 1170-7690 (Linking)
VI  - 35
IP  - 3
DP  - 2017 Mar
TI  - Validation of the Economic and Health Outcomes Model of Type 2 Diabetes Mellitus 
      (ECHO-T2DM).
PG  - 375-396
LID - 10.1007/s40273-016-0471-3 [doi]
AB  - BACKGROUND: The Economic and Health Outcomes Model of Type 2 Diabetes Mellitus 
      (ECHO-T2DM) was developed to address study questions pertaining to the 
      cost-effectiveness of treatment alternatives in the care of patients with type 2 
      diabetes mellitus (T2DM). Naturally, the usefulness of a model is determined by 
      the accuracy of its predictions. A previous version of ECHO-T2DM was validated 
      against actual trial outcomes and the model predictions were generally accurate. 
      However, there have been recent upgrades to the model, which modify model 
      predictions and necessitate an update of the validation exercises. OBJECTIVES: 
      The objectives of this study were to extend the methods available for evaluating 
      model validity, to conduct a formal model validation of ECHO-T2DM (version 2.3.0) 
      in accordance with the principles espoused by the International Society for 
      Pharmacoeconomics and Outcomes Research (ISPOR) and the Society for Medical 
      Decision Making (SMDM), and secondarily to evaluate the relative accuracy of four 
      sets of macrovascular risk equations included in ECHO-T2DM. METHODS: We followed 
      the ISPOR/SMDM guidelines on model validation, evaluating face validity, 
      verification, cross-validation, and external validation. Model verification 
      involved 297 'stress tests', in which specific model inputs were modified 
      systematically to ascertain correct model implementation. Cross-validation 
      consisted of a comparison between ECHO-T2DM predictions and those of the seminal 
      National Institutes of Health model. In external validation, study 
      characteristics were entered into ECHO-T2DM to replicate the clinical results of 
      12 studies (including 17 patient populations), and model predictions were 
      compared to observed values using established statistical techniques as well as 
      measures of average prediction error, separately for the four sets of 
      macrovascular risk equations supported in ECHO-T2DM. Sub-group analyses were 
      conducted for dependent vs. independent outcomes and for microvascular vs. 
      macrovascular vs. mortality endpoints. RESULTS: All stress tests were passed. 
      ECHO-T2DM replicated the National Institutes of Health cost-effectiveness 
      application with numerically similar results. In external validation of 
      ECHO-T2DM, model predictions agreed well with observed clinical outcomes. For all 
      sets of macrovascular risk equations, the results were close to the intercept and 
      slope coefficients corresponding to a perfect match, resulting in high R (2) and 
      failure to reject concordance using an F test. The results were similar for 
      sub-groups of dependent and independent validation, with some degree of 
      under-prediction of macrovascular events. CONCLUSION: ECHO-T2DM continues to 
      match health outcomes in clinical trials in T2DM, with prediction accuracy 
      similar to other leading models of T2DM.
FAU - Willis, Michael
AU  - Willis M
AUID- ORCID: 0000-0002-6565-7025
AD  - The Swedish Institute for Health Economics, Box 2127, SE-220 02, Lund, Sweden. 
      michael.willis@ihe.se.
FAU - Johansen, Pierre
AU  - Johansen P
AUID- ORCID: 0000-0002-6287-7508
AD  - The Swedish Institute for Health Economics, Box 2127, SE-220 02, Lund, Sweden.
FAU - Nilsson, Andreas
AU  - Nilsson A
AUID- ORCID: 0000-0002-4604-7766
AD  - The Swedish Institute for Health Economics, Box 2127, SE-220 02, Lund, Sweden.
FAU - Asseburg, Christian
AU  - Asseburg C
AUID- ORCID: 0000-0001-7196-3363
AD  - The Swedish Institute for Health Economics, Box 2127, SE-220 02, Lund, Sweden.
LA  - eng
PT  - Journal Article
PT  - Validation Study
PL  - New Zealand
TA  - Pharmacoeconomics
JT  - PharmacoEconomics
JID - 9212404
MH  - Clinical Trials as Topic
MH  - Cost-Benefit Analysis
MH  - Diabetes Mellitus, Type 2/economics/*therapy
MH  - Guidelines as Topic
MH  - Humans
MH  - *Models, Economic
MH  - Outcome Assessment, Health Care/*methods
MH  - Reproducibility of Results
EDAT- 2016/11/14 06:00
MHDA- 2017/10/31 06:00
CRDT- 2016/11/14 06:00
PHST- 2016/11/14 06:00 [pubmed]
PHST- 2017/10/31 06:00 [medline]
PHST- 2016/11/14 06:00 [entrez]
AID - 10.1007/s40273-016-0471-3 [pii]
AID - 10.1007/s40273-016-0471-3 [doi]
PST - ppublish
SO  - Pharmacoeconomics. 2017 Mar;35(3):375-396. doi: 10.1007/s40273-016-0471-3.