Regulatory and policy issues for T1DM immunotherapy

The development of immunotherapies for T1DM has lagged the development T2DM drugs, but with more clarity around regulatory requirements, large pharmaceutical companies have recently entered the field to support late stage programs. This clarity around regulatory expectations has emerged because of the convergence among regulators and clinical experts in how efficacy of these therapies should be assessed. The key agreement is that the primary efficacy endpoint for treatments directed at the underlying autoimmune cause of T1DM should be endogenous insulin secretion as reflected by standardized C-peptide measurements. Important secondary endpoints include glycemic control, total daily insulin dose and hypoglycemia rates. Most T1DM therapeutic development efforts are directed at new onset disease, which represents a small proportion of the entire T1DM population. A new frontier in T1DM therapeutic development is emerging around combination treatment of established T1DM, a population that far outnumbers those with new onset T1DM. Fully effective therapies of new onset or established T1DM will almost certainly require a combination of two or more therapies. A T1DM prevention vaccine will not be feasible until after extensive experience with the agent as a treatment of new onset and/or established T1DM.