Health technology assessment and haemophilia

Area of Science:

• Health Economics
• Rare Disease Management
• Health Technology Assessment

Background:

• Funding for rare diseases like haemophilia faces global economic pressures.
• Health Technology Assessment (HTA) and cost-utility analysis (CUA) are increasingly used for reimbursement decisions.
• Standard CUA methods often deem haemophilia treatments cost-ineffective due to high costs per Quality Adjusted Life Year (QALY).

Purpose of the Study:

• To review the principles of HTA for haemophilia treatments.
• To critique the application of standard CUA in rare disease assessment.
• To propose alternative assessment methodologies for haemophilia therapies.

Main Methods:

• Review of principles used in a Swedish HTA of haemophilia products.
• Analysis of cost-utility analysis (CUA) methodology.
• Comparison of haemophilia treatment assessment with mainstream interventions.

Main Results:

• Standard CUA ranking of haemophilia interventions against general therapeutics is inappropriate.
• Haemophilia treatments, as blood replacement therapies, should be compared to blood transfusions.
• Current methodologies inadequately capture the value of haemophilia therapies.

Conclusions:

• Haemophilia therapies require distinct assessment methodologies.
• Comparisons should be made with similar interventions like blood transfusions.
• Reimbursement policies must acknowledge societal willingness to support rare disease patients.