Preclinical research |
• More detailed studies of human T1D pathology are needed |
• Intensive investigation should be carried out into the disease cause/driver (other than autoimmunity) such as aberrant sensitivity of beta cells to metabolic stress, viruses, bacteria, etc. |
• Biomarkers should be developed which could be used to map the natural history of T1D and therefore establish whether the disease follows a relapsing/remitting, primary progressive or secondary progressive time–course (or indeed whether the time course depends on the individual) |
Phase 1 studies |
• Well‐powered studies are required to determine the optimal and rational combinations of therapies for treatment |
• Surrogate markers of beta cell stress/loss need to be developed and tested (e.g. methylated insulin, non‐invasive imaging, immune biomarkers, etc.) |
Phase 2 studies |
• Appropriately‐powered Phase 2 studies are required to account for T1D heterogeneity |
• Biomarkers should be developed to enable the stratification of susceptible patient subsets |
• Children should be enrolled unless there is any compelling argument (safety/mechanism) not to |