Quantifying patient preferences for treatments for refractory chronic spontaneous urticaria

Abstract

Background

In recent years, it has become increasingly common to incorporate the patient perspective into drug development and regulatory decision making.

Objective

This study aimed to measure and quantify patient preferences (priorities and trade-offs) for attributes that characterize current and emerging refractory chronic spontaneous urticaria (rCSU) treatments.

Methods

Adult patients with self-reported rCSU symptoms completed an online discrete choice experiment survey. The survey included 10 questions that asked respondents to choose between 2 hypothetical rCSU treatment profiles having similar attributes with varying levels. The attributes included the following: chance of control of symptoms, time to symptom control, return of symptoms after discontinuation of therapy (complete remission), allergic reaction, risk of kidney dysfunction (usually reversible), and mode and frequency of administration. Relative attribute importance and maximum acceptable risks were calculated.

Results

A total of 213 subjects with a mean age of 51 years completed the survey. Efficacy (symptom control) and mode of administration were the 2 most important attributes to treatment choice, followed by risk of kidney dysfunction and time to achieve symptom control. Complete remission of symptoms and risk of allergic reaction were identified as least important. With regard to mode of administration, topical treatment was the most preferred option and infusion therapy was least preferred. Respondents who were presented with a scenario of refractory and severe chronic spontaneous urticaria were willing to accept increased risk of reversible kidney dysfunction in exchange for improvement in symptom control or complete remission. Respondents were willing to accept infusion over topical treatment if there was significant increase in treatment efficacy.

Conclusion

These study results can be used to inform development and evaluation of future rCSU therapies by product developers and regulatory authorities, respectively.