Patient Benefit-Risk Tradeoffs for Radioactive Iodine-Refractory Differentiated Thyroid Cancer Treatments

Background. The aims of this study were to assess patients’ preferences to wait or start systemic treatment and understand how patients would make tradeoffs between certain severe adverse events (AEs) and additional months of progression-free survival (PFS). Materials and Methods. Adults in France, Germany, and Spain with a diagnosis of DTC and who have had at least one RAI treatment completed a direct-elicitation question and a discrete-choice experiment (DCE) online. The direct-elicitation question asked respondents whether they would opt out of treatment when their tumor is RAI-R. In the DCE, respondents chose between 12 pairs of hypothetical RAI-R DTC treatment profiles. Profiles were defined by magnitudes of efficacy (PFS) and safety (severe hand-foot skin reaction [HFSR], severe proteinuria, and severe hypertension). A main-effects random-parameters logit model was estimated. Results. 134 patients completed the survey. Most patients (86.6%) opted for treatment rather than “wait and see” decision. Patients placed a greater weight on the risk of severe hypertension than the risk of proteinuria and HFSR. Conclusions. DTC patients showed preference toward treatment for RAI-R DTC over watchful waiting. Patients’ concerns about the risk of severe hypertension appeared to have had a greater effect on patients’ choice than severe proteinuria or HFSR

Benefit-risk tradeoff preferences for chronic hand eczema treatments

Hand eczema affects approximately 16% of the US population. The long-term prognosis is poor, and 5–7% experience severe chronic hand eczema (sCHE) that interferes with daily activities. Treatments for CHE may be ineffective or associated with adverse events (AEs) that may dissuade patients from pursuing or continuing treatment. For quantification of patient experiences and benefit-risk preferences for outcomes associated with CHE treatments, a web-based discrete choice experiment survey was administered to patients in the United States with a self-reported physician diagnosis of CHE and severe symptoms not resolved with topical agents. Respondents answered a series of treatment choice questions, each requiring evaluation of a pair of hypothetical profiles of medications for sCHE defined by efficacy and risk of several AEs. Improvement in CHE clearing of 25–50% was rated from 1.5 to 3.1 times as important as eliminating a 5% risk of permanent bone problems. The mean maximum acceptable risk of permanent vision problems in exchange for an improvement in CHE clearing of 25–50% ranged from 3.4% to 4.8%. This study demonstrated that patients with CHE rated efficacy improvements associated with treatment of sCHE as more important than eliminating the risks of specific AEs