[caption id="attachment_3903" align="alignleft" width="300"]By chance or by design?[/caption]
Interagency coordination at the Department of Health and Human Services is purported to be a routine fact of life nowadays, but there are signs that outside forces are pushing FDA into activity that could have a big effect on another important agency.
This might be strictly a problem of perception rather than reality, but its tough to avoid the conclusion that all the emphasis on speed at FDA might run up against a rather large obstacle elsewhere at HHS.
Breaking through to nowhere
AdvaMed held its annual policy briefing earlier this month, during which the association advocated mandatory Medicare coverage of pivotal clinical trials for any device FDA designates a breakthrough device. This is part of the congressional 21st Century Cures initiative and not necessarily an idea of AdvaMed’s provenance (though it could be), but there is an understandable lack of detail as to how CMS will be involved.
Parallel review is one obvious vector for this kind of collaboration, but one has to wonder whether this is really a game-changer for more than the occasional PMA. If a device is that important to CMS, it’d want to be involved in any event. If device makers have been wary of parallel review up to now because of the additional requirements for such a trial, will they suddenly jump on board now?
They might. Coverage of a pivotal trial might be pretty irresistible (even if the reimbursement proves miserly), but it’s still tough to imagine this kind of thing will suddenly give parallel review a handful of applications each year when it has produced only two applications over the past five years. At any rate, one has to imagine CMS will be very choosy about which applications it will sign on to cover.
The imprecision of precision medicine
The second issue in this FDA/CMS context falls under the President’s Precision Medicine initiative, and deals with those massively parallel genomic sequencing machines. FDA wants to make use of existing and yet-to-surface data repositories to ease its review of next-gen sequencing applications, but this seems to reintroduce us to the analytical-versus-clinical validity quandary.
A massively parallel machine can fly around the genomic space at warp speed all it wants, but development of clinical evidence sounds like more of a subsonic flight proposition to the untutored ear. Not to criticize FDA for trying to amp up its informatics game, but how is CMS not the rate-limiting step (pardon the cliché) here?
Alzheimer’s again out in the cold
I know I’ve ground this axe before, but you just have to love the Precision Medicine Initiative for its same-old/same-old approach. It offers $70 million for cancer and squadoosh for neurodegenerative states, which will cost the American economy twice as much as cancer in the decades to come. How’s that for responsible stewardship of the taxpayer’s dollar?
The more things stay the same, the more they stay the same.
[caption id="attachment_3892" align="alignleft" width="300"] Scientists funded by the NIH BRAIN Initiative will develop tools to simultaneously watch the unique firing patterns of many neurons in hopes of classifying them based on physical characteristics and functional characteristics, such as patterns of electrical activity. Credit: Vincent Pieribone, John B. Pierce Laboratory.[/caption]
It’s truly a futuristic world we live in with technology that not so long ago was only seen in science fiction. Last year, as part of a three-part series Medical Device Daily published, I explored 3-D printing and the potential for that technology, which enables us to make solid objects from a digital file, to change the medical landscape.
Another rapidly emerging area that sci-fi fans should appreciate is neurotechnology. During the annual J.P. Morgan Healthcare Conference in San Francisco last month, Francis Collins, director of the National Institutes of Health, highlighted the Brain Research through Advancing Innovative Neurotechnologies (BRAIN) Initiative. The BRAIN Initiative, announced in April 2013 by President Obama, is a joint effort between four federal agencies – NIH, the National Science Foundation, the FDA, and the Defense Advanced Research Projects Agency. The “dream team” of neuroscientists involved in the effort is led by Cori Bargmann of The Rockefeller University and Bill Newsome of Stanford University.
According to Collins, Bargmann and Newsome spent a solid year trying to map out a series of steps over a 10-year period to advance science to the point of understanding how all brain’s circuits do what they do. “Such things as processing information, such things as laying down a memory and retrieving it. These are fundamentally important functions, but they don’t happen one neuron at a time, they take circuits and it’s the circuits that we want to understand and currently do not,” Collins said.
But the part that really caught my attention was Collins' mention of a workshop that took place to explore the ethics of things like transferring memories from one person to the next, which is, he said, “a thinkable thought.” But, he quickly added, “we better be sure that we know what we’re doing.”
In the weeks that have passed since hearing Collins speak about the BRAIN Initiative during J.P. Morgan, I haven’t been able to get that thought out of my head. Transferring memories from one person to another? And the country’s top neuroscientists believe that is actually a “thinkable thought.”
I’ve tried to imagine how such a thing would even be possible. Would it be like loading memories onto a flash drive and then sharing them with another person’s brain, similar to the way data files can be shared via a thumb drive and USB port? Or would we simply, by that point in time, be capable of accessing parts of our brain that we’ve never been able to access before, enabling some sort of telepathic transfer of memories? Or maybe it would be similar to a bio-feedback game I once played with my son at the WonderWorks amusement park in Panama City Beach, Florida called Mind Ball.
Mind Ball is designed to test mind capacities and is based on EEG technology. Each player wears a headband with electrodes that records and evaluates brain activity and the objective is to use calmness and relaxation to mentally gain control over a small ball. Perhaps in the future we’ll be able to put one of these EEG headbands on and allow it to record and transfer our memories to the person wearing the other headband?
However the capability may unfold, at this point in time the idea seems almost too sci-fi for sci-fi. But then again, not that long ago I would have said the same thing about 3-D printed body parts.
[caption id="attachment_3878" align="alignleft" width="320"]Ever heard a newspaper publisher say 'there's no news today'?[/caption]
It’s fun trying to report the news, especially when there’s so much non-news masquerading as news. But I’m sure you’re tempted to say to yourself, “this old-news/new-news/non-news dilemma is news?”
Point taken. Anyway, let’s take a look at a couple of recent stories of note, newsy or not.
Old news; the combo products GMP guidance
FDA has issued a combination products draft guidance for good manufacturing practices compliance, but this is not the first such attempt and surely will not be the last.
FDA has taken several cracks at this topic, including a draft guidance in 2004, which came a scant two years after the agency formed the Office of Combination products. Following the 2004 effort, FDA announced in the Sept. 23, 2009, Federal Register that the GMP situation called for rulemaking when addressing single-entity and co-packaged combination products.
Barely four more years would pass when FDA announced yet again in the Jan. 22, 2013, Federal Register that it had finalized the rule developed in 2009, and the latest guidance points to the 2013 rule, noting that a set of GMP requirements had already been in place for human cells and other tissue-based therapeutic products.
This will prove to be one of the more iterative FDA guidances as the 21st Century unwinds if only because materials science is enabling a lot of advances that were previously just pipe dreams. There are a lot of advances in basic research of interest, including a story I wrote for the Dec. 17, 2014, issue of Medical Device Daily on an effort to create synthetic neural circuits based on mesenchymal stem cells (MSCs). Would the use of nanomaterials in addition to the MSCs make this a device or a biologic?
FDA will have to answer that and a whole lot of other questions as science pushes the envelope completely out of shape in the years ahead.
New news; Scientists aren’t always right
To be perfectly frank, this is hardly new news, but what’s a blog for if not the exercise of artistic license? There’s an article at the online portal for Perspectives on Psychological Science questioning whether science is as reliable as is often claimed.
Author John Ioannidis of the Stanford University Prevention Research Center takes up the question in the context of psychology, but anyone who attends FDA advisory meetings on a regular basis might see some of this in other branches of medical science, too. Among Ioannidis’ observations is that history “suggests that major catastrophes in scientific credibility are unfortunately possible,” and he stated further, “the argument that ‘it is obvious that progress is made’ is weak.”
I bring this up only because it is remarkable at times how routinely policymakers and the general public are inclined to accept the idea that a question is beyond dispute just because a scientist say "it’s this and not that."
Three cheers for civil religions, eh?
Non-news; guidance for trials for nail fungus
“Surely you jest, Mark,” you might say upon reading this. Nope, surely I don’t jest. FDA actually has a draft guidance out for clinical trials for treatments of fungal nail infections, said to be distinct from trials for products that clear nails that have been clouded by whatever, fungal nail infections included.
In addition to being stunningly small in scope, this draft guidance has an ick factor that is just off the charts. Oh, marone!