3 FDA topics to watch in 2018

As the year winds down, STAT reporters are taking a look at the stories they’re most eager to track in 2018

It’s been a busy year for the Food and Drug Administration — since Commissioner Scott Gottlieb was confirmed in May, he has led the agency with an ambitious agenda, tackling everything from prescription opioid addiction to tobacco use to dubious stem cell clinics.

And 2018 shows no signs of letting up. Many of the proclamations the FDA has made under Gottlieb will come to fruition, or at least to bud, in the new year.

The agency’s vast mandate means any "top 3" list is bound to reflect only a paltry sampling of what’s on the FDA’s agenda. But here are three areas that STAT is watching:

Tarnishing the gold standard?

As new medicines come out of the lab that target smaller and smaller groups of patients, it is more and more difficult, and sometimes impossible, to uphold the standard of a randomized controlled trial to determine if the drug is safe and effective. That is one of the challenges facing the project of "precision medicine" — if a treatment is specifically designed for a small group of patients, how can you run a huge trial to demonstrate it works?

And yet the phrase that used to describe such a process — the "gold standard" — doesn’t seem to be going anywhere.

Gottlieb signals support for both ‘gold standard’ and expedited review for drug approvals

Gottlieb has said that he’ll uphold the "gold standard" while also pushing the boundaries of what can count as a good clinical trial, making critics nervous.  He gave some examples of this at a congressional hearing at the end of November.

STAT will be watching closely to see how the agency continues to balance the need for evidence with the challenge of acquiring it.

Digital health and medical software

The FDA is puzzling over whether or not to regulate certain medical software — everything from an algorithm that identifies irregularities in medical scans to a computer program that helps a doctor design a treatment plan. Companies don’t want regulation to stifle innovation, but some others are wary of letting products on the market that claim a medical benefit without proper testing.

The agency is trying to address the issues in a number of ways. One fascinating process to watch: The agency trying to determine if it might make sense to issue approvals to companies themselves instead of the products. If a company is certified as being responsible enough, perhaps it could push products onto the market without those products being vetted. Since September, nine companies have been working with the FDA to hash out what this could mean. During the last two days of January, the agency will hold a public meeting to discuss the progress of the program, potentially giving the public an insight into that might make a company good enough to avoid FDA regulation altogether.

Tobacco and electronic cigarettes

In July, Gottlieb announced a plan to reduce the level of nicotine in cigarettes to "non-addictive" levels. But there’s no consensus about exactly what this means, and the agency has yet to actually start the laborious public rule-making process (in mid-December, Gottlieb said that it would begin "soon"). It’s sure to attract attention from tobacco companies, who themselves are moving away from traditional combustible cigarettes toward high-tech nicotine consumption devices — like e-cigarettes and "heat-not-burn" devices, which purport to be safer than regular cigarettes.

That claim will come under scrutiny at a January meeting, when the Tobacco Products Scientific Advisory Committee will discuss an application by Philip Morris International to market a product called the IQOS, which heats tobacco to release nicotine at a lower temperature so that it may not release as many harmful chemicals. Nothing has yet been approved in the U.S. for marketing as a reduced risk product. The January meeting could signal how the agency plans to move forward with alternatives to traditional cigarettes.