Zahara & Kesha Weeks

Clinical and Economic Impact of Sickle Cell Disease

Gene therapies have the potential to revolutionize medicine and improve the lives of individuals living with sickle cell disease (SCD). Additional considerations such as high costs for these therapies and questions regarding insurance coverage remain major challenges to patient accessibility and future development.

The Clinical and Economic Impact Analysis (CEIA) Consortium

The Cure Sickle Cell Initiative (CureSCi) formed the Clinical and Economic Impact Analysis (CEIA) Consortium in 2019 to help identify and evaluate the cost effectiveness of gene therapies for SCD. The Consortium was funded by the National Heart, Lung, and Blood Institute (NHLBI) through awards to the University of Washington (UW) and the Fred Hutchinson Cancer Center (FH). The investigators from both institutions received feedback and guidance from an expert panel consisting of individuals living with sickle cell disease, advocates, clinicians with expertise in pediatric and adult sickle cell disease care and bone marrow transplant, as well as bioethicists, health economists, database analysts, and members of the payer community.

CEIA Mission
The CEIA’s mission was to develop simulation models that can be used to demonstrate the potential national impact of specific curative therapies for SCD, and the distribution of that impact on payers, employers, and families over the lifetime of the patients.

Models for Economic Analysis of SCD
Two independently developed simulation models were designed to assess lifetime outcomes with and without gene therapy for people living with SCD. The FH's Sickle Cell Disease Outcomes Research and Economics Model (FH-HISCORE) and the UW Model for Economic Analysis of Sickle Cell Cure (UW-MEASURE) were published in the Annals of Internal Medicine  in January 2024.

The UW-MEASURE microsimulation model  web portal, which uses the R-Shiny platform, allows users to examine and adjust values that help to estimate healthcare and societal outcomes of SCD gene therapies.

“Both the UW and Fred Hutch models suggest that gene therapies can be life-altering for patients with sickle cell disease. We show prolonged, substantial reductions in pain crises events and reduced morbidity over time, greatly improving patients' prospects for long-term employment, decreasing or possibly eliminating caregiver burden, and substantially improving recipients' life expectancy and quality of life compared to current standards of care. The long-term durability of therapy is an important unknown that will impact cost-effectiveness. Future work comparing the clinical and economic effects of gene therapy versus stem cell transplantation will assist in guiding patients to the most appropriate and cost-effective therapy.”

SCOTT RAMSEY, MD, PHD
Principal Investigator

CEIA Goals

  • Analyze the current SCD economic and standard-of-care landscape to inform the creation of two models that simulate real-world SCD care modalities and SCD disease progression.
  • Use the models to perform clinical and cost-effectiveness analysis for payers/insurers that incorporate societal factors.
  • Share findings through publicly available web-based simulation models, peer-reviewed publications, and conference presentations.

Anirban Basu, PhD

Principal Investigator

Health Economist at the Comparative Health Outcomes,Policy, and Economics (CHOICE) Institute, University of Washington, Seattle

Scott Ramsey, MD, PhD

Principal Investigator

Clinician and Health Economist at the Hutchinson Institute for Cancer Outcomes Research (HICOR), Fred Hutchinson Cancer Research Center

Beth Devine, PhD

Health Services Researcher at the Comparative Health Outcomes, Policy, and Economics (CHOICE) Institute, University of Washington, Seattle

Michael A. Bender, MD

Clinician and Sickle Cell Researcher at the Comparative Health Outcomes, Policy, and Economics (CHOICE) Institute, University of Washington, Seattle

Aaron Winn, PhD

Health Economist at the Hutchinson Institute for Cancer Outcomes Research (HICOR), Fred Hutchinson Cancer Research Center

Kate M. Johnson, PhD

Assistant Professor

The University of British Columbia (Formerly: Post-Doc, The CHOICE Institute, University of Washington, Seattle)

Boshen Jiao, PhD

Assistant Professor

University of Southern California (Formerly: PhD Student, The CHOICE Institute, University of Washington, Seattle)

SELECT ACTIVITIES

Performed a landscape analysis which included review of the following areas: population, interventions, comparators, outcomes, timing, setting (geographic), and study designs (PICOTS).

Conducted a literature search to:

  • Better understand the burden of SCD (comorbidities, treatment complications, costs) and the cost-effectiveness of curative therapies compared to existing types of care.
  • Inform parameters of economic models with most up-to-date values from available literature.
  • Consider costs of curative therapies in other chronic disease areas.

Developed simulation models for SCD disease that:

  • Incorporated epidemiology, biostatistics, decision analysis, and health policy.
  • Used input parameters for costs, outcomes, and probabilities – to study how individuals with SCD progress over their lifetime.
  • Utilized real-world evidence (registries, claims databases, electronic health records), or clinical trials.
  • Validated against observed outcomes for individuals living with SCD. Can be used to project long-term health outcomes and costs.

Conducted an analysis of healthcare claims using Medicare, Medicaid, and Market Scan insurance claims records.

  • Identified SCD patients with public and commercial health plan insurance coverage from 2008-2016.
  • Analyzed data to identify the following by age group: SCD complication rates, healthcare resource use, and direct medical expenditures.
  • Utilized information to help understand the SCD lifetime burden of illness overall and by key sub-groups (e.g. age, disease severity).

Developed a clinical and cost-effectiveness analysis using the University of Washington Model for Economic Analysis of Sickle Cell Cure (UW-MEASURE), and the Fred Hutchinson Institute Sickle Cell Disease Outcomes Research and Economics Model (FH-HISCORE). UW-MEASURE, which is used to assess comparative lifetime outcomes with and without gene therapy can be found here .

Featured Publication

Clinical Trial Listing “Gene Therapy Versus Common Care for Eligible Individuals with Sickle Cell Disease in the United States: A Cost-Effectiveness Analysis,” Annals of Internal Medicine.

Additional Publications

Find other CEIA publications by viewing the Cost Analysis Section of the Publications List found here.