This white paper discusses the significant burden of diabetes in pediatric populations, including the limited treatment options for pediatric patients with type 2 diabetes mellitus (T2DM). While type 1 diabetes mellitus (T1DM) has historically been the predominant diabetes disease in pediatric populations, T2DM now accounts for more than 10% of the incident cases of diabetes between the ages of 12 and 16 years. Moreover, at 18 years of age the incidence of T1DM and T2DM is nearly identical.
Specific clinical, design, and operational considerations for pediatric clinical trials in this therapeutic area are addressed. These include rational study design and the realization that the protocol for a similar study in adults cannot be adapted merely by changing the age of eligible participants. Designing a protocol for a pediatric clinical trial requires an understanding of developmental physiology, emotional development, and the particular clinical and pathologic manifestations of the disease being studied. Careful attention must be paid to the specifics of the protocol as they pertain to pediatric participants (e.g., age-appropriate clinical endpoints, age-appropriate oral formulations that take into account developmental differences in taste ontogeny). Similarly, Principal Investigator and investigational site selection must consider suitability for and previous experience with pediatric clinical research.
The relevant pediatric regulatory landscapes in the United States and Europe are reviewed. Discussions start with the origins of regulatory discourse focusing on the topic of drug development for pediatric populations, which began in earnest in the late 1990s, and progress to address regulatory considerations specific to diabetes therapies. While there are many
similarities in what is required by EU and U.S. regulations, there are also notable differences that sponsors should be aware of when designing pediatric clinical programs.
Quintiles’ therapeutic expertise is leveraged within its Centers of Excellence (COEs). The specialized diabetes, pediatric, and regulatory expertise utilized during the preparation of this White Paper was drawn from the Diabetes COE and the Pediatric COE.
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