Quantifying Patient Preferences for Treatments for Refractory Chronic Spontaneous Urticaria
In recent years, it has become increasingly common to incorporate the patient perspective in healthcare and regulatory decision making. A patient centered benefit risk approach to decision making leads to improved adherence and outcomes, reduced health system costs, and increased satisfaction and quality of life. The United States Food and Drug Administration has particularly requested the use of quantitative patient preference information (evidence) in decision making across the total life cycle of the medical product which includes the development phase, evaluation and post market surveillance. Discrete choice experiment is the most commonly used method to measure/elicit patient preferences and generate quantitative patient preference information. Treatment options for refractory chronic spontaneous urticaria present with varying risks, benefits, mode of administration, and other treatment attributes. To the best of my knowledge there are no studies that quantitatively explores patient preferences (priorities and tradeoffs) for the benefits, risks and other treatment characteristics associated with therapies for refractory chronic spontaneous urticaria. This dissertation aims to use the discrete choice experiment methodology to measure and quantify patients’ preferences for refractory chronic spontaneous urticaria treatments. Results of this study can inform chronic spontaneous urticaria drug development and the evaluation of attributes of future chronic spontaneous urticaria therapies by regulatory authorities. Adult patients in the United States that self-reported with refractory chronic spontaneous urticaria symptoms completed an online discrete choice experiment survey. The survey included 10 choice questions, that asked respondents to choose between 2 hypothetical refractory chronic spontaneous urticaria treatments that were defined by the following attributes: chance of control of symptoms, time to symptom control, return of symptoms (complete remission), allergic reaction, risk of kidney dysfunction, and mode and frequency of administration. Conditional and random parameters logit regression models were used to analyze the preference data. Differences in preferences were explored via subgroups defined by age, gender, and location of hives. The average age of our sample was 51 years, 57% self-identified as female, and the sample largely comprised of White individuals (80%). A majority of the sample reported having a mixture of hives that may itch and swelling (57%), 38% reported having mainly hives, and 6% mainly swelling. Our results show that for the changes in attributes presented in this study, efficacy (symptom control) and mode of administration were the two most important attributes to treatment choice. These were followed by the attributes that characterized treatment safety and time to achieve symptom control. Complete remission of symptoms and risk of allergic reaction were identified as the least important attributes. Findings from our risk tolerance analysis showed that respondents in our sample were willing to accept increased risk of a treatment related side effect (kidney dysfunction) in exchange for improvements in control of symptoms or long-term remission of symptoms. When considering mode of administration (how you take the medicine) attribute, although a topical treatment was the most preferred option, and infusion therapy was the least preferred option, respondents were willing to accept infusion over topical treatment if there were significant increases in treatment efficacy. This study intended to demonstrate a quantitative patient centered benefit risk approach in regulatory decision-making for refractory chronic spontaneous urticaria. The findings from this study provide insights into how respondents with refractory chronic spontaneous urticaria symptoms prioritize the benefits, risks and other attributes, and their willingness to tradeoff between these attributes when considering treatment. The results of this study can inform refractory chronic spontaneous urticaria drug development and the evaluation of future refractory chronic spontaneous urticaria therapies by regulatory authorities.
Economics|Health sciences|Public health|Health care management
Babalola, Olufemi, "Quantifying Patient Preferences for Treatments for Refractory Chronic Spontaneous Urticaria" (2023). ProQuest ETD Collection - Thomas Jefferson University. AAI30638608.