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Table 1 Assumption used to develop the base case analysis

From: Budget impact analysis of the use of extended half-life recombinant factor VIII (efmoroctocog alfa) for the treatment of congenital haemophilia a: the Italian National Health System perspective

Assumption

References

The target population is represented by patients with severe haemophilia A

Product indication

The use of rFVIIIFc is considered only for prophylaxis treatment

Product indication

Product consumption related to the use of drugs for surgery is not included in the analysis because no difference between the two scenario are hypothesized

Assumption based on the opinion of clinical experts

Patients with inhibitors are excluded from the analysis because deserving particular treatment different from those analysed in the present analysis

Product indication

Paediatric patients are exposed only to prophylaxis regimen, no on-demand treatment is considered for these patient according to current clinical practice in Italy

Assumption based on the opinion of clinical experts

According to current clinical practice in Italy, paediatric patients are not exposed to treatment with plasma derived products

Assumption based on the opinion of clinical experts

Age cut-off to differentiate treatment for paediatric and adults patients is considered equal to 12 years old

Assumption based on the opinion of clinical experts, [7]

An equal treatment schedule is considered for both plasma derived and conventional recombinant products

Assumption based on the opinion of clinical experts

The incidence of spontaneous bleeding is equal between patients treated with plasma derived and conventional recombinant products

Assumption based on the opinion of clinical experts

The consumption of products for the management of spontaneous bleeding is equal between patients treated with plasma derived and conventional recombinant products

Assumption based on the opinion of clinical experts

For all products compliance with prophylaxis treatment is considered to be 100%

Assumption based on the opinion of clinical experts

Percentage of patients switching to rFVIIIFc is considered to do not differ among the diverse products

Assumption based on the opinion of clinical experts