Implementing the Generalized Risk-Adjusted Cost-Effectiveness Model for Sickle Cell Disease: A Case Study

Area of Science:

• Health Economics
• Pharmacoeconomics
• Medical Technology Assessment

Background:

• Traditional cost-effectiveness analysis (CEA) may undervalue innovative therapies for severe diseases like sickle cell disease.
• Societal value considers factors beyond direct costs, including quality of life and uncertainty.

Purpose of the Study:

• To quantify the societal value of curing sickle cell disease using the Generalized Risk-Adjusted Cost-Effectiveness (GRACE) method.
• To adjust cost-effectiveness calculations for disease severity, treatment outcome uncertainty, and quality of life-life expectancy trade-offs.

Main Methods:

• Recalibrated a health technology assessment for sickle cell disease gene therapies (lovo-cel, exa-cel) using the GRACE framework.
• Modified CEA by adjusting willingness-to-pay thresholds based on illness severity and treatment uncertainty.

Main Results:

• GRACE implementation reduced incremental cost-effectiveness ratios by 6% for lovo-cel and exa-cel.
• Willingness-to-pay thresholds increased by approximately 50% under GRACE.
• Lovo-cel and exa-cel were found to be cost-effective from both direct payer and societal perspectives.

Conclusions:

• The GRACE method provides a more comprehensive estimation of societal value for innovative therapies.
• GRACE facilitates more efficient and equitable resource allocation in healthcare.
• This study advocates for incorporating GRACE elements into health technology assessments for broader acceptance of life-altering treatments.