Table 4.

Key messages

• The premise underlying this article is that to prevent or delay the onset of T1D we will eventually need to incorporate screening for T1D risk in those with and without family members who have T1D and we will need to have effective therapies with a reasonable safety profile to use in clinical care.

• The rationale for incorporating screening programs in those without family members as part of clinical care at this time is weak. Without therapies, the only clinical benefit that is possible from screening and monitoring is reduction of DKA at time of diagnosis. The low prevalence of T1D in the general population means that even a very highly sensitive screening test will have a low PPV. The costs of screening will be high in aggregate and the benefit to an individual low. The potential “harm” of this approach is both the significant costs of screening and monitoring and the psychological stress on the individual/family, particularly if there is a lack of knowledge among clinicians for how to provide interpretation and support.

• In contrast, reducing DKA through screening and monitoring programs among those with a relative with T1D is a reasonable and achievable goal at this time. Since there are fewer relatives to screen, the aggregate costs to the health care system are less. With a 1 in 20 risk for T1D, the predictive value of a positive test is much higher and thus the potential benefit to the individual is higher. Screening and monitoring of relatives as standard of care would reduce DKA at diagnosis dramatically in relatives at risk for disease.

• At the same time, additional benefits will accrue through this effort to bring screening and monitoring relatives to clinical care. These include creating a competitive marketplace to develop and implement better (easier and cheaper) screening and monitoring approaches and providing “real-world” evidence of the processes, costs, benefits, and harms of screening that will be needed to expand from testing relatives to testing the general population as part of clinical care.

• Critically, screening relatives as part of clinical care will address the concern of industry about the feasibility of developing therapies to prevent or delay T1D. The number of individuals who will be identified through widespread screening of relatives will be sufficient for conducting multiple clinical trials. Positive results in such trials will eventually lead to a clinical indication of such therapies in prevention and thus provide the essential piece to someday justify screening the population without relatives with T1D as part of clinical care.