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Related Experiment Video

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

Marlon Graf1, Joris Kleintjens2, Md Tahsin Hasan1

  • 1Precision AQ, Bethesda, MD, USA.

Value in Health : the Journal of the International Society for Pharmacoeconomics and Outcomes Research
|May 25, 2025
PubMed
Summary

The Generalized Risk-Adjusted Cost-Effectiveness (GRACE) method enhances cost-effectiveness analysis for sickle cell disease gene therapies. This approach reveals these treatments are cost-effective, improving societal value and resource allocation.

Keywords:
cost-effectiveness analysisgeneralized risk-adjusted cost-effectiveness (GRACE) methodhealth technology assessmentsickle cell diseasevalue flower

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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.