Clinical and Economic Impact of Molecular Testing for BRAF Fusion in Pediatric Low-Grade Glioma

Clinical and Economic Impact of Molecular Testing for BRAF Fusion in Pediatric Low-Grade Glioma

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Disease Area (Primary)

Pediatric low-grade glioma (PLGG)

Disease Area (Secondary)

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First Developed

06/07/2020

Last Developed

10/27/2021

Software Used

R (e.g., heemod, BCEA, dampack, hesim)

Model Sponsor

Healthcare organization

Intervention

braf_molecular_testing

Model Validation Score

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Results

The life expectancy after diagnosis for individuals who did not receive molecular testing was 39.01 (95% Confidence Intervals (CI): 32.94;44.38) years and 40.08 (95% CI: 33.19;45.76) years for those who received testing. Our findings indicate that patients who received molecular testing at diagnosis experienced a 0.38 (95% CI: 0.08;0.77) gain in QALYs and $1384 (95% CI: $-3486; $1204) reduction in costs over their lifetime. Cost and QALY benefits were driven primarily by the avoidance of long-term adverse events (stroke, secondary neoplasms) associated with unnecessary use of radiation.

Conclusion

What are the key conclusions or current applications of this model?

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Model Review

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Summary
Validation Score

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Internal Comments

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Full review

01 Model Built Reflective yes
02 Model Subgroups yes
03 Model Assesses all comparators yes
04 Model Incorporates costs yes
05 Model assesses all outcomes yes

06 Model structure validated by experts yes
07 Model aligns with or justifies deviation from previous models yes

08 Time in health states yes
09 Consistency with time in states yes
10 Clinical events extractable yes
11 Consistency with number of clinical events yes
12 Impact of adverse events yes
13 Consistency with adverse events yes
14 Life-years reported yes
15 Impact on mortality yes
16a Reasons for mortality differences yes
16b Reasons for mortality differences yes
16c Reasons for mortality differences yes
16d Reasons for mortality differences yes
17 Main driver of incremental life-years yes
18 Consistency with mortality rates yes
19 No technology-specific utilities used yes
20 Main driver of cost-effectiveness yes
21 Extrapolation methods identified yes
22 Adjustable time horizon yes
23 Double counting avoided yes
24 Surrogate vs final outcomes alignment yes
25 Flexibility for treatment effect waning yes

26 Access to deterministic and Monte Carlo results yes
27 Clear trace from inputs to outcomes yes
28 Macros used only for simulation/navigation (Excel) yes

29 QALY equivalence across technologies yes
30 Extreme effectiveness impact on QALY yes
31 Slight effectiveness impact on QALY yes
32 Increased mortality lowers QALYs yes
33 Reduced mortality increases QALYs yes
34 Increased baseline risk lowers QALYs yes
35 Reduced baseline risk increases QALYs yes
36 Zero mortality leads to equal life-years yes
37 Cost change affects only total costs yes
38 Utilities = 1 makes QALYs equal life-years yes
39 No discounting increases QALYs/costs yes
40 Higher discounting decreases QALYs/costs yes
41 Shorter time horizon lowers QALYs/costs yes
42 Inputs switchable across alternatives yes
43 Cost-QALY correlation across simulations yes
44 Strong cost correlation from Monte Carlo yes
45 Strong QALY correlation from Monte Carlo yes
46 Deterministic ≈ Probabilistic results yes

47 Backward trace from results to inputs yes
48 Backward trace from results to inputs yes

49 No use of non-transparent Excel functions yes
50 No hidden sheets, rows, or columns yes
51 No custom formulas inside VBA macros yes
52 Parameters persist after macros yes
53 Transparent input structure in single worksheet yes
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