P306: Perceptions of the Benefits and Risks of Novel Therapies for Type 1 Diabetes: A Qualitative Study
Poster Presenter
Stephen Karpen
Senior Director, Regulatory Affairs
Breakthrough T1D United States
Objectives
This study sought to understand perceptions of the benefits and risks of novel therapies like disease-modifying (DMTs) and cell replacement therapies (CRTs), which may improve therapeutic outcomes in people with type 1 diabetes (T1D), among people with T1D and caregivers.
Method
Semistructured interviews were conducted and analyzed using a qualitative content analysis approach. A description of the benefits and risks of DMTs and CRTs, developed with a steering committee of patients and clinicians, was presented to facilitate discussion among people with T1D and caregivers.
Results
We interviewed 26 individuals: 12 adults and 4 adolescents living with T1D, and 10 caregivers of children living with T1D (aged 3-13 years). Interviewed participants’ mean age was 35.6 years (range, 15-71). Approximately half of the adolescent participants (n=2; 50.0%) and two-thirds of adult participants (n=8; 66.7%) were diagnosed = 5 years ago. All caregivers’ children with T1D were diagnosed = 2 years ago.
Most participants (n=22; 84.6%) reported they would have been willing (for themselves or their child) to try DMTs at diagnosis. The main perceived benefit of DMTs, cited by 18 participants (69.2%), was reduced reliance on exogenous insulin for the following reasons: (1) minimizing daily burden of insulin management; (2) mitigating uncertainty and adverse effects associated with exogenous insulin; and (3) lowering out-of-pocket costs. Nearly half (n=11; 42.3%) reported that an extended honeymoon period, the time after diagnosis when the body still has insulin-producing cells and is often associated with reduced burden of care, would have better prepared them for life with T1D. Regarding the potential risks, participants reported concerns about an increased risk of cancer (n=13; 50.0%), cytokine release syndrome (n=6; 23.1%), mild side effects (e.g., rash, headache) (n=3; 11.5%), and lower white blood cell counts (n=1; 3.8%). For all but 4 participants, the stated benefits of DMTs outweighed the potential risks.
All participants expressed willingness to consider CRTs, citing insulin independence as the primary benefit. Perceptions of this benefit aligned with 2 key themes: freedom from the constant demands of T1D and standard therapeutic approaches, and an enhanced sense of normality in daily life. Regarding CRT risks, participants reported concerns with the use of immunosuppressants (n=11; 42.3%), the possibility of cell rejection by the body (n=10; 38.5%), and life-threatening complications related to bleeding (n=9; 34.6%).
Conclusion
This study found that a reduction or elimination of daily exogenous insulin was perceived to be a key benefit of novel therapies. For DMTs, reduced insulin dependence was valued, as it would reduce the time spent managing daily insulin administration and offset the negative effects of insulin therapy and uncertainty around exogenous insulin’s impact on glucose levels. Insulin independence in the context of CRTs was highly valued, as it was perceived to offer freedom from the constant demands of T1D, including the need for frequent decision-making. These findings suggest that addressing insulin requirements is of utmost importance to those most affected by the development of novel therapies for T1D, thus supporting the inclusion of reducing or eliminating insulin dependence as a key outcome for clinical research assessing novel therapies.
This research also elicited preliminary trade-off information, which has implications for drug development and regulatory decision-making. Despite concerns with the risks and drawbacks of novel therapies, most participants reported they would have considered trying DMTs, and all reported that they would consider trying CRTs. These data indicate that, as presented to participants, the benefits of novel therapies could potentially outweigh the perceived risks.
There is a strong desire for novel therapies beyond insulin, despite the potential risks of side effects. When considering and developing new therapies, it is essential to consult people living with T1D or caring for someone with T1D. Understanding their perspectives on potential new treatments, particularly their perceptions of treatment benefits and tolerance for risks, is of utmost importance to inform medical product development and regulatory decision-making. To further understand risk thresholds, an upcoming quantitative stated-preference survey informed by these qualitative findings will quantify the risk-benefit trade-offs.
