Tuesday, June 14, 2016

In Praise Of "Forming Smart Questions": Evidence Based Medicine & The FDA's Role

We have written of this before, repeatedly, but when smart doctors like Harlan Krumholz and Steve Nissen ask uncomfortable questions (even ones to which the answer may turn out to be "no"), then pursue data, and formulate hypotheses from that data -- we all, as consumers of health care, ultimately benefit.

We all benefit, provided that FDA serves its role as well -- and asks for solid evidence as to new drug candidates. The way science more than occasionally works is that we learn new things, on our way to studying other things.

Matt Herper has a nice piece up in Forbes, this morning, describing an ongoing shift in diabetes treatments that was originally animated by doctors asking questions about safety and efficacy, and doing large scale longer term studies. All of which -- even if the questions turned out to be mostly misplaced concerns -- brought Lilly's Jardiance to prominence, in a crowded field of diabetes treatments, a year ago. Here's a bit -- but do go read it all:

. . . .Jardiance, a pill sold by Eli Lilly LLY and Boehringer Ingelheim, presented its positive results just last year. Researchers say that the new results could change the way that doctors treat diabetes, shifting the treatments doctors reach for after metformin, the tried-and-true first-line drug, which is generic.

"There’s a building momentum that maybe we do need to rethink the way diabetes is cared for in America," says John Buse, the University of North Carolina, Chapel Hill researcher who led the study, which was funded by Novo Nordisk.

And doctors and Novo Nordisk itself give credit to the new diabetes data to a surprising source: Tougher regulations for diabetes drugs from the Food and Drug Administration, which many in industry had previously decried, saying it was keeping new drugs from the market and hurting patients.

"I can almost guarantee you that these trials would not have been done if it had not been for the FDA regulations," says Buse, who has been a consultant to many companies for years. "Before the guidance I was constantly pushing on companies to do these trials. . . ."

So it is that often, just being unafraid to ask questions -- smart questions, controversial questions -- ones that no one can answer -- leads to improvements in patients' lives. And we are smiling widely, this fine warmly sunny June morning.

1 comment:

Brian D said...

I appreciate the re-post. I was impressed with design of the study as written in this week's The New England Journal of Medicine. More impressing however, I found that our FDA's Commissioner has had his 'finger on the pulse' of the problem (pun intended)all along. I found a 2014 article, co-authored by the 'very capable' Dr. Robert Califf, very pleasant read and recommend it to anyone interested in the background of these drugs used to treat type-2 diabetes and how their clinical trials assess their potential impact on vascular health (Holman et al., 2014).


Holman RR, Sourij H and Califf RM (2014) Cardiovascular outcome trials of glucose-lowering drugs or strategies in type 2 diabetes. Lancet 383:2008-2017.