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

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.