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Table 2 Quality of Health Economic Studies (QHES)

From: A systematic review of the economic evaluations of Enzyme Replacement Therapy in Lysosomal Storage Diseases

 

Castro-Jaramillo (2012)

Kanters et al. (2014)

Kanters et al. (2017)

Rombach S.M. et.al., (2013)

Van Dussen L. et.al., (2014)

NCPE

Was the study objective presented in a clear, specific, and measurable manner?

7

7

7

7

7

7

Were the perspective of the analysis (societal, third-party payer, etc.) and reason for its selection stated

4

4

4

4

4

 

Were variable estimates used in the analysis from the best available source (i.e. Randomized Control Trial –Best, Expert Opinion –Worst)?

8

8

8

8

8

8

If estimates came from a subgroup analysis, were the groups prespecified at the beginning of the study?

1

1

1

1

1

1

5) Was uncertainty handled by: 1) statistical analysis to address random events; 2) sensitivity analysis to cover a range of assumptions?

9

9

9

9

9

9

Was incremental analysis performed between alternatives for resources and costs?

6

6

6

6

6

6

Was the methodology for data abstraction (including the value of health states and other benefits) stated?

5

5

 

5

5

 

Did the analytic horizon allow time for all relevant and important outcomes? Were benefits and cost that went beyond one year discounted and a justification given for the discount rate?

7

7

7

7

7

7

Was the measurement of costs appropriate and the methodology for the estimation of quantities and unit costs clearly described?

8

8

 

8

8

8

Were the primary outcome measure(s) for the economic evaluation clearly stated and were the major short-term, long-term, and negative outcomes included?

6

6

6

6

6

6

Were the health outcomes measures/scales valid and reliable? If previously tested, valid and reliable measures were not available, was justification given for the measures/scale and reliable measures were not available, was justification given for the measures/scale used?

7

7

7

7

7

7

Was the economic model (including structure), study methods and analysis, and the components of the numerator and denominator displayed in a clear transparent manner?

8

8

8

8

8

 

Were the choice of economic model, main assumptions and limitations of the study stated and justified?

7

7

7

7

7

 

Did the author(s) explicitly discuss direction and magnitude of potential biases?

    

6

 

Were the conclusion/ recommendations of the study justified and based on the study results?

8

8

8

8

8

8

Was there a statement disclosing the source of funding for the study?

  

3

3

3

 

Total score

91

91

81

94

100

67