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Table 7 Incremental costs per year free of end organ damage gained under different scenarios, undiscounted (upper rows) and discounted (lower rows)

From: Cost-effectiveness of enzyme replacement therapy for type 1 Gaucher disease

Scenario

YFEOD

Costs

ICER

 

ERT

no ERT

Δ

ERT

no ERT

Δ

 

Base case

61.70

48.90

12.80

€5,716,473

€171,780

€5,544,693

€434,416

 

37.77

31.97

5.80

1,206,933

50,048

1,156,885

199,559

Production loss included

61.70

48.90

12.80

€5,772,897

€294,226

€5,478,670

€429,243

 

37.77

31.97

5.80

1,216,954

71,956

1,144,998

197,508

25% ERT costs reduction

61.70

48.90

12.80

€4,338,430

€171,780

€4,166,649

€326,449

 

37.77

31.97

5.80

918,801

50,048

868,753

149,857

Historical I*

19.72

17.90

1.82

€5,202,872

€109,342

€5,093,530

€2,803,382

 

14.83

13.70

1.14

2,391,020

54,143

2,336,877

2,057,891

Historical II*

12.10

10.14

1.96

€6,198,258

€112,774

€6,085,484

€3,111,478

 

9.27

8.01

1.26

3,031,062

59,253

2,971,809

2,363,665

  1. YFEOD: years free of end organ damage, ICER: incremental cost-effectiveness ratio. *The historical scenarios were based on the patient distribution in the Netherlands when ERT was introduced in April 1991 and were derived from medical histories of patients referred to the AMC until September 2011. The first historical scenario includes the asymptomatic stage, the second historical scenario excludes the asymptomatic stage. While running the historical scenarios, two model adjustments were made: 1) the probability of death was adjusted for the mean age of 36 (Historical I) or 38 (Historical II) years for patients in the cohort at the time of ERT market introduction in the Netherlands; 2) the time horizon too was adjusted for the mean age of the cohort in 1991 and became 49 (Historical I) or 47 (Historical II) years instead of 85.
  2. €: amount of money in euros.
  3. Δ: difference between between the scenarios with versus without ERT.