A METHOD FOR COMPARING DISPARATE OUTCOME MEASURES IN OVERACTIVE BLADDER CLINICAL TRIALS

Author(s)

Casciano R1, Arikian SA1,2, Tarride J-E 2 , Corey R 2,3, Doyle J1, 1The Analytica Group, NY, NY, USA; 2Columbia University, NY, NY, USA; 3University of the Sciences in Philadelphia, Philadelphia, PA, USA

Researchers have reported varying endpoints in assessing treatment options for overactive bladder (OAB). No published trials have compared controlled release once-daily oxybutynin-XL (OXY-XL) and twice daily tolterodine (TOL) using equivalent clinical endpoint for success, making it difficult to compare therapies. OBJECTIVE: Describe approach for comparing disparate OAB treatment outcome measures. METHODS: Clinical trial data supplemented with literature identified randomized controlled and open label safety trials involving OXY-XL or TOL. Patient outcomes were quantified using a correlational matrix to determine relationship between different endpoints. RESULTS: Three OXY-XL randomized clinical trials and one open label safety trial and three TOL trials were identified. All reported rate of reduction of urinary incontinence episodes (R-UI). The OXY-XL trials reported rate of complete continence (CC), defined as percent of patients with no UI episodes for 7 days. Mean CC rates for OXY-XL ranged from 41-50% (mean CC rate = 45.9% + 0.04). OXY-XL mean R-UI ranged from 79.5% to 89.5% (mean R-UI = 84.4% + 0.04). For TOL, R-UI rates ranged from 43.2% to 44.8%, (mean R-UI = 45.7% + 0.02). Computed CC rate for TOL was 24.6%. Correlation across all studies between CC and R-UI was high (r2 = 0.9975, p<0.0001) and consistent; R-UI:CC ratios within trials ranged from 1.8 to 1.9 (mean =1.85 + 0.08). Rate of reduction in UI episodes/week or day is approximately twice that of complete continence. CONCLUSIONS: Rate of complete continence may be derived from a surrogate endpoint by taking approximately one half the rate of reduction in R-UI episodes weekly, allowing direct efficacy comparisons on multiple measures.

Conference/Value in Health Info

1999-05, ISPOR 1999, Arlington, VA, USA

Value in Health, Vol. 2, No. 3 (May/June 1999)

Code

PKU1

Topic

Clinical Outcomes

Topic Subcategory

Comparative Effectiveness or Efficacy

Disease

Urinary/Kidney Disorders

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