Lou Philipson, MD: Risk-Benefit of Teplizumab for Type 1 Diabetes

Last week, the US Food and Drug Administration (FDA) voted in support of the benefits-risk profile for teplizumab for delaying the onset of type 1 diabetes (T1D).

The Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) voted 10-7 in favor of teplizumab, noting its major clinical need and benefit to at-risk populations, while noting uncertainties about the safety profile and limited data set.

In an interview with HCPLive, Lou Philipson, MD, Kovler Diabetes Center, University of Chicago, discussed the difficulties of people affected by T1D and ultimately what a preventive drug might do for these patients.

He noted that no agent at this time can delay T1D, which is linked to spectacular costs over lifetime, including effort, decision making, and days lost to care.

“The toll of a chronic illness like this is not obvious to people who are not embedded in it, like those who either have the disease or have a loved one with the disease, or take care of someone with the disease,” Philipson said.

Philipson also spoke about the committee's discussion on risk-benefit of teplizumab. He noted that the understanding of a risk-benefit profile for treatment comes from an understanding of the downsides of dealing with T1D for 10 - 60 years.

“In this case, some people will be sick at the outcome, but so far, I am unaware where someone has died from the initial administration [of teplizumab]," Philipson said.

In particular, he noted a "cognitive dissonance" with risk-benefit of teplizumab when it comes to treating children and overall decision-making for parents.

"The downsides of type 1 diabetes are spectacular and a certain amount of risk that is understood, I think could be tolerated," he said.