Purpose: Preference studies ascertain what is important to individuals when weighing benefits and risks of interventions. The purpose of this study was to investigate parents’ preferences for treatments to delay the onset of insulin dependence in children who will develop Stage III T1D.
Methods: A web-based discrete-choice experiment (DCE) survey measured the preferences of U.S. parents of children with T1D and without T1D. In a hypothetical scenario where their child (<18 years) would become insulin dependent in 6 months or 2 years, respondents were offered a series of choices between hypothetical treatments that would delay insulin dependence or no treatment (monitoring only). Treatments were defined by 6 attributes with varying levels (years until insulin dependence; reductions in the risks of long-term complications, diabetic ketoacidosis (DKA), and serious infection; injection site reaction; and nausea). Random parameters logit analysis evaluated the relative importance of benefits and risks. Maximum acceptable risk (MAR) was calculated.
Results: Parents of children with T1D (n=600) and parents with children without T1D (n=901) from an online consumer panel completed the survey instrument. Most parents chose an active treatment (2% always chose monitoring only for all 8 choices). For parents without a T1D child, delaying insulin dependence and reducing the risk of long-term complications and serious infection were the most important treatment attributes, while parents with a T1D child valued these 3 attributes and reducing the risk of DKA as most important. MAR of serious infection ranged from 1%-7% for an additional 2 to 4-year delay in insulin dependence.
Conclusions: When told to assume their child would develop Stage III T1D, most parents considered hypothetical treatments to delay progression. The preferences expressed over the benefits and harms in this survey provide guidance on acceptable tradeoffs for future treatments to delay insulin dependence in T1D.
J. Hedrick: Employee; Self; Johnson & Johnson. Employee; Spouse/Partner; PTC Therapeutics. J. Sutphin: Other Relationship; Self; Multiple Companies. R.L. DiSantostefano: Employee; Self; Janssen Research & Development. K. Gallaher: Other Relationship; Self; Multiple companies. C. Mansfield: Other Relationship; Self; Multiple companies.
Janssen Research & Development, LLC