Treatment of Children with Severe Haemophilia A and Inhibitors: a Health Economic Evaluation for Germany

Abstract

Background:

Decision makers request increasingly for high levels of evidence when allocating resources in medical care. This is hardly feasible for rare diseases. The objective was to analyze clinical and economic aspects of different immune tolerance induction (ITI) strategies for children with severe haemophilia A and inhibitors.

Methods:

A decision model, time frame 18 years (base case: 2 year old boy), was constructed from a German statutory health insurance (SHI) perspective. Compared were high-dose (HD) ITI, low-dose (LD) ITI, ‘ITI with risk assessment’, on-demand (OD) treatment with bypassing agents. Clinical data were derived from structured literature research and expert opinion. Sensitivity analyses were conducted for parameters with wide statistical ranges.

Results:

Base case analysis: total costs for HD ITI amounted to €3.4 million with 40.9% ITI costs, 51 joint bleeds, 36 hospital days; LD ITI, €2.4 million with 21.4% ITI costs, 74 joint bleeds, 52 hospital days; ‘ITI with risk assessment’, €2.7 million with 27.6% ITI costs, 53 joint bleeds, 37 hospital days; OD treatment, €1.7 million, 146 joint bleeds, 104 hospital days. Incremental costs per bleed avoided with HD ITI decreased from €1 million to €0.15 million with increase of joint bleeds from 3 to 20 per year, when compared to ‘ITI with risk assessment’ in sensitivity analysis.

Conclusion:

‘ITI with risk assessment’ is cost-saving with comparable outcomes to HD ITI. However, patient-related factors like bleeding frequency have to determine treatment decisions in individual patients. More clinical data is needed to increase the significance of model ­calculations.

Zusammenfassung

Hintergrund:

Entscheidungsträger fordern zunehmend hohe Evidenzgrade bei der Allokation von Ressourcen im Gesundheitswesen. Für seltene Erkrankungen ist dies kaum machbar. Ziel war es, klinische und ökonomische Aspekte verschiedener Immuntoleranzinduktions-Strategien (ITI) bei Kindern mit schwerer Hämophilie A und Inhibitoren zu analysieren.

Methoden:

Die Analysen basieren auf einem Entscheidungsmodell (Zeithorizont 18 Jahre, Basisfall: 2 Jahre alter Junge) aus der Perspektive der gesetzlichen Krankenkasse (GKV). Verglichen wurden Hochdosis (HD) ITI, Niedrigdosis (LD) ITI, ‘ITI mit Risiko-Assessment’, On-demand (OD) Behandlung mit Bypass-Agentien. Klinische Daten basieren auf strukturierter Literaturrecherche und Expertenmeinungen. Sensitivitäts­analysen wurden für Parameter mit großer statistischer Breite durchgeführt.

Ergebnisse:

Basisfallanalyse: Gesamtkosten für HD ITI betrugen 3,4 Mio. € mit 40,9% ITI Kosten, 51 Gelenkblutungen, 36 Krankenhaustagen; LD ITI, 2,4 Mio. € mit 21,4% ITI Kosten, 74 Gelenk­blutungen, 52 Krankenhaustagen; ‘ITI mit Risiko-Assessment’, 2,7 Mio. € mit 27,6% ITI Kosten, 53 Gelenkblutungen, 37 Krankenhaustagen; OD Behandlung, 1,7 Mio. €, 146 Gelenkblutungen, 104 Krankenhaustagen. Die inkrementellen Kosten pro vermiedener Blutung mit HD ITI sanken von 1 auf 0,15 Mio. € bei einer Zunahme der Gelenkblutungen von 3 auf 20 pro Jahr verglichen mit ‘ITI mit Risiko-Assessment’ in Sensiti­vitätsanalysen.

Zusammenfassung:

‘ITI mit Risiko-Assessment’ ist kostensparend mit vergleichbaren ­Outcomes zur HD ITI. Allerdings müssen patientenbezogene Faktoren wie Blutungsfrequenz die Behandlungsentscheidungen bei individuellen Patienten bestimmen. Mehr klinische Daten werden gebraucht, um die Signifikanz von Modelberechnungen zu erhöhen.

Key words

costs - haemophilila A - inhibitors - immune tolerance induction - ITI - outcome

Schlüsselwörter

Kosten - Haemophilie A - Inhibitoren - Immuntoleranzinduktion - ITI - Outcome

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