Former FDA Deputy weighs in on the push into ‘real-time clinical trials’

Last week the Food and Drug Administration announced the start of two real-time clinical trials. 

The proof of concept trials are a prelude to a pilot program.

To unpack the importance, or lack thereof, of this latest declaration, I talked to Amy Abernethy, former Principal Deputy Commissioner and Acting Chief Information Officer for the FDA, who has pushed for both real-time data in clinical trials and real world evidence. She currently runs Highlander Health, a research and investment platform that is funding innovation in healthcare.   

She said there’s reason to be excited about the news, even if it may seem only incremental. 

The news: The FDA is partnering with VC-backed Paradigm Health, a clinical trial platform, to deliver real-time signals during two separate cancer drug trials. Through this partnership, the FDA believes it will be able to see whether drugs are working, or causing harm, earlier.

The two drug sponsors are Astrazeneca and Amgen. Paradigm is already delivering real-time data about participants in Astrazeneca’s Phase 2 trial of a medication combination for a rare form of blood cancer. Participants are located at two health systems, Texas-based MD Anderson Cancer Center and the University of Pennsylvania Health System. Amgen is conducting a Phase 1b trial of a treatment for limited-stage small cell lung cancer. 

What’s not new: The FDA has been working on speedier clinical trials for some time and has several programs: Real-time Oncology Review, Breakthrough Designation, Accelerated approval, Fast Track, and Priority Review. Last year, the FDA Commissioner also introduced The National Priority Review Voucher pilot. 

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Abernethy tells Second Opinion that she’s optimistic about the FDA’s direction. She says there are technical hurdles the agency needs to overcome to be able to regulate drugs and devices in the future. One of the biggest problems with clinical trials historically is that the core processes remain highly manual, despite all the VC money that has flowed into improving automation.

The conversation below has been edited for clarity. We highlighted in bold the parts that we felt were most crucial from the discussion.

Second Opinion: What was your initial reaction to the clinical trial news?

Amy Abernethy: I think everybody wants me to say it’s incremental, but actually I want to say it’s real momentum. Here's why it's momentum even if it falls on its face or it turns out that there's a zillion problems that get in the way. There is a clear statement, this is the direction we have to move. Like we're not around pussyfooting around, let's start to figure this out. 

There are critical issues or questions, but that doesn't at all diminish in my mind, the high level direction, which is: how are we going to make clinical trials much more real time?

I was at FDA in Trump one, as you know. I was not a political appointee, but I had both the roles Principal Deputy Commissioner and I took on the role as Acting Chief Information Officer for this exact reason— and that was because it was clear to me that in order for FDA to scale in the way that it was going to need to scale, for all the innovative treatments coming down the pipeline and everything else, we're going to have to, as an agency, use data and technology in much more modern ways. So the technology modernization action plan and the data modernization action plans really were the starting points of saying we need to get our house in order.

It certainly didn't go all the way that it needed to go. But [the agency] started to say that it matters to start to build the capabilities. And it's interesting, if I look across what's been happening for this FDA leadership team, you can see alignment with where the space is going now.

Lots of books of work continue across administrations or have been going on for a long time and then get surfaced. But again, if you look at the arc, I think, it was like a year ago that the request for information notices were put out for both the role of JSON for receipt of data, and— that's the mechanisms of the FDA—as well as FHIR standards. And you know, I don't know that we need to get into the technical specifics here, but to me, these are indicators the agency is thinking about, how do we get data inside much more efficiently? How are we going to get ourselves ready to deal with it?

The reason why things like JSON and FHIR are so important is because you can't supercharge through AI unless you have the data transformation or data exchange activities, and frankly, all the pieces are starting to get put in place. So what you've been seeing is stepwise activities, moving in the direction of data now being able to much more easily be transferred to FDA, and FDA starting to think about, ‘can we do this in much more real time ways?’

You really just have to start testing. Like, how are we going to get this done? And so being able to transport, transfer endpoint information, being able to transfer, for example, adverse event information, is key to that proof of concept. It is interesting that they're focusing on early phase trials, which I personally think are very critical for shoring up our national capabilities as we think about reinforcing the U.S.’s place in the world, in the global economy, with respect to the bio economy. It's interesting to see it be focused on early phase clinical trials, but of course, the devil's going to be in details.

Second Opinion: What questions did this announcement raise for you?

Amy Abernethy: What are the core goals to how this work is getting done? Does it line up with what Marty is saying around seamless clinical trials, because this data exchange activity is different than the seamless clinical trial conversation.