Table 1

Regulatory considerations for advancing precision medicine in T1DM

Stage of DiseaseScientific GapImplications
Primary PreventionIdentifying very high-risk individuals beyond current genetics
Identifying effective preventive interventions with sufficient safety to study broadly
With low disease prevalence, feasible prevention trials would benefit from precision medicine–guided enrichment (and would also inform targeted preventive use clinically)
Broad vaccination programs require interventions that are highly protective and very safe/well tolerated
Secondary PreventionPractical means to identify individuals with subclinical disease
Full understanding of the immune mediators and propagators of β-cell damage
Immune-based interventions carry safety/toxicity risks requiring targeting of use both in clinical trials and subsequent clinical deployment
Better understanding of the inciting and sustaining immune mechanisms would allow for better targeting of intervention vs. broad immunosuppressive approaches
TreatmentNo apparent “precision” considerations in choice of insulin therapy/approachInsulin therapy likely to remain more “personalized” than “precise,” absent new science