Using a treatment satisfaction measure in an early trial to inform the evaluation of a new treatment for benign prostatic hyperplasia.

OBJECTIVE

This article describes the results of an exploratory analysis of treatment satisfaction data from a phase 2 study to inform the evaluation of a new treatment for benign prostatic hyperplasia (BPH).

METHODS

The study was a randomized, placebo-controlled, double blind trial comparing three doses (low, medium, and high) of a new uro-selective alpha-blocker with an active comparator. A total of 536 men aged 40 years and older with lower urinary tract symptoms (LUTS) resulting from BPH were randomized into the study. Patients' perceptions of satisfaction with efficacy, dosing, and side effects were explored using a validated disease-specific instrument, the Treatment Satisfaction Scale-Benign Prostatic Hyperplasia (TSS-BPH). The TSS-BPH was administered at the end of the study or at withdrawal.

RESULTS

Compared with placebo, the medium and high doses showed the greatest efficacy on the primary endpoint, the International Prostate Symptom Score (IPSS). Patient satisfaction with efficacy at these doses was significantly better than that for placebo. Nevertheless, adverse events were most frequent in the high dose group. Satisfaction with side effects was significantly worse than placebo for this group, and this was reflected in the total scores on the TSS-BPH, with patients being most satisfied overall with the medium dose.

CONCLUSIONS

Exploring patients' treatment satisfaction with medication in early trials using multidimensional measures provides an overall evaluation of efficacy and side effects, and can be used to inform the selection of doses for later trials. This is especially useful for products that require adherence with medication over long periods of time.