BACKGROUND: Currently, HbA1c is the primary measure of treatment effectiveness for Type 1 Diabetes (T1D). We investigated patient priorities for measures of T1D control in addition to HbA1c.
METHOD: We developed discrete-choice experiment surveys for adult patients and caregivers of children with T1D that presented choices between hypothetical treatments described by 6 attributes with varying levels: HbA1c, time in range, weekly number and severity of hypoglycemia and hyperglycemia events, additional disease management time, and additional treatment cost. The clinical outcomes were defined based on a 2017 consensus report standardizing the definition of outcomes beyond HbA1c. Respondents answered nine treatment choice questions, and the data were analyzed using random-parameters logit.
RESULTS: Recruited through an online market research panel, 300 adult patients and 400 caregivers completed the survey. Patients and caregivers placed the most importance on reducing low and high events. Additional time in range was as important as a reduction of more than ½ point in HbA1c, but both were less important than avoiding weekly lows and highs. Patients who did not use an insulin pump or CGM placed relatively more weight on reducing lows and highs and on improvements in HbA1c and time in range compared to patients who use technology. For patients, avoiding 1 to 5 lows (54-69 mg/dL) per week was 5 times more important than achieving target HbA1c from ½ point above. Avoiding 1 to 5 highs (181-250 mg/dL) per week was 7 times more important than achieving target HbA1c from ½ point above. Caregivers of children < 12 years placed relatively more weight on avoiding lows and highs than those with older children.
CONCLUSION: Overall, patients and caregivers prioritized control of hypoglycemia and hyperglycemia events, including mild to moderate events. The research has implications for outcomes used to measure the benefits of treatment in research, development, and reimbursement of therapies for T1D.
