C Pelletier

Treatment patterns, health state, and health care resource utilization of patients with radioactive iodine refractory differentiated thyroid cancer.

Background Patients with differentiated thyroid cancer (DTC) often respond well to treatment but some become refractory to radioactive iodine (RAI) treatment, and treatment options are limited. Despite the humanistic and economic burden RAI refractory disease imposes on patients, published research concerning treatment patterns and health care resource utilization is sparse. Methods Data were collected from an online retrospective chart review study in the US and five European Union (EU) countries (France, Germany, Italy, Spain, and UK) with physicians recruited from an online panel. Physicians (N=211) provided demographics, disease history, treatment information, and health care resource utilization for one to four of their patients with radioactive iodine refractory differentiated thyroid cancer (RR-DTC). Results The majority of the patients with RR-DTC (N=623) were female (56%), and their mean age was 58.2 years. In this sample, 63.2% had papillary thyroid cancer and 57.0% were in Stage IV when deemed RAI refractory. Patients with RR-DTC experienced regional recurrence in the thyroid bed/central neck area (25.3%) and had distant metastatic disease (53.6%). At the time data were collected, 50.7% were receiving systemic treatment. Of those, 78.5% were on first-line treatment and 62.7% were receiving multikinase inhibitors. Regional differences for prescribed treatments were observed; the US was more likely to have patients receiving multikinase inhibitors (79.2%) compared with UK (41.2%) and Italy (17.1%). Additional details regarding treatment patterns and resource utilization are discussed. Conclusion The current study aimed to obtain a greater understanding of RR-DTC treatment globally. These results can assist in the development and implementation of treatment guidelines and ultimately enhance the care of patients with RR-DTC.

Health state utility valuation in radioactive iodine-refractory differentiated thyroid cancer.

Purpose The aim of this study was to elicit utilities for radioactive iodine-refractory differentiated thyroid cancer (RR-DTC) and evaluate the impact of treatment response and toxicities on quality of life. Patients and methods RR-DTC health states were developed based on data from a previous qualitative study and iterative review by clinical experts. Following piloting, health states underwent valuation by 100 members of the UK public during time trade-off interviews. Mean utilities and descriptive distribution statistics were calculated, and a logistic regression analysis was conducted. Results The demographic characteristics of the study sample were generally reflective of the UK population. Clear differentiation in valuation between health states was observed. No response/stable disease had an adjusted utility value of 0.87, with a corresponding gain of +0.04 following a treatment response and a decline of −0.35 for disease progression. Adverse events were associated with utility decrements between −0.47 (grade III diarrhea) and −0.05 (grade I/II alopecia). Conclusion The trade-off interviews derived utility weights show clear differentiation between RR-DTC health states in response to treatment. The values reported in this study are suitable for cost-effectiveness evaluations for new treatments in RR-DTC.

Incremental Quality Adjusted Life Years (Qaly) Analysis in Absence of Head to Head and Health Related Quality of Life (Hrqol) Data: a Case Study in Thyroid Cancer

• Head-to-head clinical trials are a powerful way to test medicines against each other. But designing these types of trials presents unique challenges. • Head-to-head trials often not required and not conducted prior to market authorization. • Along with traditional measures of efficacy and safety (e.g., survival, tumor response), HRQoL is another important factor to capture patient perspective and understand the value associated with a drug. • Over the years, the healthcare industry has witnessed regulatory approvals of innovative technologies in oncology without head-to-head clinical trial evidence or Health Related Quality-of-Life (HRQoL) data. • This yields a challenge to decision-makers who require a cost-effectiveness analysis (CEA) to conduct Health Technology Assessments. • We present a case study using recently approved drugs lenvatinib and sorafenib for management of radioiodine-refractory differentiated thyroid cancer (RRDTC) in order to demonstrate methodological approaches and potential data sources to address these gaps. RESULTS