Economic return from the Women's Health Initiative estrogen plus progestin clinical trial: a modeling study

Ann Intern Med. 2014 May 6;160(9):594-602. doi: 10.7326/M13-2348.

Abstract

Background: The findings of the Women's Health Initiative (WHI) estrogen plus progestin (E+P) trial led to a substantial reduction in use of combined hormone therapy (cHT) among postmenopausal women in the United States. The economic effect of this shift has not been evaluated relative to the trial's $260 million cost (2012 U.S. dollars).

Objective: To estimate the economic return from the WHI E+P trial.

Design: Decision model to simulate health outcomes for a "WHI scenario" with observed cHT use and a "no-WHI scenario" with cHT use extrapolated from the pretrial period.

Data sources: Primary analyses of WHI outcomes, peer-reviewed literature, and government sources.

Target population: Postmenopausal women in the United States, aged 50 to 79 years, who did not have a hysterectomy.

Time horizon: 2003 to 2012.

Perspective: Payer.

Intervention: Combined hormone therapy.

Outcome measures: Disease incidence, expenditure, quality-adjusted life-years, and net economic return.

Results of base-case analysis: The WHI scenario resulted in 4.3 million fewer cHT users, 126,000 fewer breast cancer cases, 76,000 fewer cardiovascular disease cases, 263,000 more fractures, 145,000 more quality-adjusted life-years, and expenditure savings of $35.2 billion. The corresponding net economic return of the trial was $37.1 billion ($140 per dollar invested in the trial) at a willingness-to-pay level of $100,000 per quality-adjusted life-year.

Results of sensitivity analysis: The 95% CI for the net economic return of the trial was $23.1 to $51.2 billion.

Limitation: No evaluation of indirect costs or outcomes beyond 2012.

Conclusion: The WHI E+P trial made high-value use of public funds with a substantial return on investment. These results can contribute to discussions about the role of public funding for large, prospective trials with high potential for public health effects.

Primary funding source: National Heart, Lung, and Blood Institute.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Aged
  • Breast Neoplasms / epidemiology
  • Cardiovascular Diseases / epidemiology
  • Cardiovascular Diseases / prevention & control*
  • Clinical Trials as Topic / economics*
  • Colorectal Neoplasms / epidemiology
  • Cost Savings
  • Decision Support Techniques
  • Estrogen Replacement Therapy*
  • Female
  • Financing, Government
  • Fractures, Bone / epidemiology
  • Health Expenditures
  • Humans
  • Incidence
  • Markov Chains
  • Middle Aged
  • National Institutes of Health (U.S.)
  • Osteoporosis, Postmenopausal / complications
  • Osteoporosis, Postmenopausal / epidemiology
  • Osteoporosis, Postmenopausal / prevention & control
  • Postmenopause*
  • Quality-Adjusted Life Years
  • United States
  • Women's Health / economics*