Understanding and Addressing Enrollment Challenges in Pediatric T2D Clinical Trials Compared with T1D

Abstract

Background: While behavior and diet modifications are the first line of defense in treating T2D, pediatric patients requiring additional intervention have few options compared with adults with T2D. The disparity in available treatments can be attributed, in part, to enrollment challenges in pediatric T2D clinical trials that delay FDA approval of new therapies. Methods: Established in 2008, the Pediatric Diabetes Consortium has connected 49 diabetes research institutes and Industry partners to address enrollment challenges at a structural level. Yet, we must also address the social, economic and cultural barriers to participation specific to T2D pediatric patients. Barriers: The overall prevalence in 2009 of T1D vs. T2D in youth was 1.93/1,000 compared to 0.24/1,000, with T1D affecting a wider age span. T2D is rarely diagnosed in children <10 years. While there are racial and ethnic disparities in diabetes prevalence overall, they are more pronounced in T2D. Thus, compared to T1D, potential T2D pediatric study participants are fewer, older and more likely to face barriers to participation that include financial concerns, transportation issues, familial disruptions, distrust of research, health illiteracy, language barriers and parents’ inability to miss work. Conclusion: Addressing barriers is critical to ensure clinical trial success and improved quality of life for these patients. Industry sponsors should make translated materials, such as e-diaries available for non-English speakers, particularly for Spanish speakers and consider quality of life assessments as a secondary outcome. Researchers should partner with community leaders in areas with high T2D prevalence, to establish trust between the patient community and research institution. Additionally, incentives that address families’ social and economic needs should be offered, including direct compensation to offset costs associated with trial participation such as travel and lost income. Disclosure A.M. Terry: None. S. Bollepalli: None. L. Smith: None. A.F. Walker: None. R.L. Gal: Research Support; Self; Boehringer Ingelheim Pharmaceuticals, Inc., Takeda Development Center Americas, Inc., Novo Nordisk Inc. L.C. Beaulieu: Research Support; Self; Novo Nordisk Inc., Boehringer Ingelheim Pharmaceuticals, Inc., Takeda Development Center Americas, Inc.. R.C. Edelen: None. J.E. Less: None.