NCATS Director of Clinical Innovation
Mike's Blog
Orthogonal Perspectives
By: Michael Kurilla, M.D., Ph.D., NCATS Director of the Division of Clinical Innovation
December 29, 2023
Hopefully everyone has sufficiently recharged after the holidays, looking forward to the new year. As Lenin once remarked, “There are decades where nothing happens; and there are weeks where decades happen.” It would appear that 2024 is going to offer us weeks and weeks of decades. NIH is still without a budget, and while our current CR runs through February 2 (although 2/2 being a Friday, one can expect the cliffhanger to drag out over the weekend), there is another CR that runs through 1/19, so we might get an inkling of what we can expect before Groundhog Day. At the same time, there are quite a few other pressing items on Congress’s plate that need dealing with. And let’s not forget the presidential primary season kicks off in January (I dread my television viewing options at the gym in 2024).
The start of a new year should be a time for new thinking and new directions. The CTSAs should be major players for new thinking and new directions for medicine and health care. We’re also nearly a quarter of the way into the current century and so it’s about time to consider how 21st century medicine will be conceptualized, or, closer to home, how this generation of academic medical leaders will be remembered. So far, it looks like we have some work to do. How are we dealing with the worsening antimicrobial resistance issue? By largely attempting to recapitulate science that is nearly 100 years old - discovering another new antibiotic. ‘Let’s replace current drugs losing potency with similar alternatives that are likely to eventually suffer the same fate as our existing limited options.’ What are we doing with AI? Mostly attempting to do everything we do now, but just a little faster, freeing up time for the really important stuff like filing more billing claims. Or for the academic medical researcher, discovering a new treatment for a common disease (with said disease quite possibly due to long term adherence to some other medical treatment) that can make a biotech CEO a billionaire (which is one reason why new antibiotics are so hard to produce since they are generally considered cheap and taken for short periods of time anyway, hence unlikely to be a blockbuster).
It is against this background that I ran across this interesting read, which while undated appears to have been written around 2017 (and all the more intriguing since little has changed). The author describes a concept he calls ‘systematic interface’ where the system is “transparent on the outside, and black boxes on the inside. You don’t have to know anything about how they operate in order to use them” using FedEx and Amazon as examples. Health care systems, whether a hospital facility or an insurance company, are just the opposite. To the outside user they are largely opaque. How much will this procedure cost? ‘We can’t tell you until after it’s over.’ In order to navigate an opaque system, you basically need to know someone who knows someone who knows what to do and that person may still not be the one with any authority to make a decision.
While it’s easy to dismiss these concerns as ‘above my paygrade,’ it’s important to keep these concepts in mind especially for the upcoming LHS Collaborative Workshop in May. In order to realize the potential impact of CTSA activities, methodologies for rapid dissemination and implementation across our diverse and balkanized health care ecosystem are critical to avoid forcing everyone to continually reinvent wheels. At the same time, a medicine cabinet full of therapies that aren’t or can’t be used effectively will have little impact on how people feel, function, survive, and thrive. Any large system is resistant (mostly due to inertia) to change and so it becomes incumbent on us to provide compelling justification across multiple dimensions (patient outcomes, staff workload, economic, etc.) as well as simplified pathways for the transition.
In many instances, investigators are solely focused on individual trees (an individual aspect of a single disease) without ever appreciating the forest (our complex health care ecosystem) around them. All too often, we assume that the methodological reductionist approach which has been so successful for science (as well as science funding), is all that is necessary for translating science into implementable health care solutions. Unfortunately, while this sometimes leads to productive scientific research endeavors (as well as successful grant funding), it never quite seems to lead to cost effective health care solutions. For example, traditional approaches for studying monogenetic diseases direct efforts into understanding the genetics, the biochemistry, the physiology, and the natural history of that disease. Of course, much high-quality science emerges which is valuable as scientific knowledge but that doesn’t always offer direct implementable health care solutions for that condition. On the other hand, a readily implementable gene editing methodology (ideally, in utero), once a specific gene variation has been associated with a specific disease phenotype, would address a monogenetic disease even without full understanding of the underlying gene function or subsequent pathology.
For the CTSAs to truly impact the health care ecosystem, we need to reimagine what health and health care can and should look like in the future as well as recognize and appreciate that the small incremental progress our individual work supports must align and contribute to a larger vision of better health as well as health care for everyone, a vision that the CTSA community understands, accepts, nurtures, and pursues, fearlessly and relentlessly.
Happy New Year!
You are under no obligation to remain the same person you were a year ago,
a month ago, or even a day ago. You are here to create yourself, continuously.
-Richard Feynman, Nobel laureate in physics