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Brainstorm Health Daily: August 2, 2017

“Every revolution in science—from the Copernican heliocentric model to the rise of statistical and quantum mechanics, from Darwin’s theory of evolution and natural selection to the theory of the gene—has been driven by one and only one thing: access to data.”

That was the eye-opening opening of a keynote address given yesterday by the brilliant John Quackenbush, a professor of biostatistics and computational biology at Dana-Farber Cancer Institute who has a dual professorship at the Harvard T.H. Chan School of Public Health and ample other academic credits after his name.

There is also no question that this digital fuel is driving virtually every transformation in healthcare happening today. Speaking at the MedCity Converge conference in Philadelphia, Quackenbush noted that the average hospital is generating roughly 665 terabytes of data annually, with some four-fifths of it in the unstructured forms of images, video, and doctor’s notes.

But the great limiting factor in harnessing all of this information-feedstock is not a “big data problem,” but rather a “messy data problem.”

In sum, in places where there is tons of potentially useful data to examine, we don’t make it accessible in ways that people actually want to use it. Either the data isn’t easy or intuitive to access or it simply isn’t informative. Or it’s in the wrong format. Or it’s incomplete—or created with incompatible “standards” (of which we seem to have an unlimited, irreconcilable supply). Or it captures just one dimension of a multidimensional realm. (“Biological systems are really complex, adaptive systems with many moving parts, that we’ve only begun to scratch the surface of understanding,” he says.)

Or—and this one seems to be a surprisingly common misstep—the data doesn’t really address the question the end user wants to answer. It’s off-purpose, in other words.

Take the case of population-level data, which government and academic institutions routinely collect: “Statistics operate on population data and medical research is driven by population data,” says Quackenbush, “but medical care is driven by individual-level data. So when we’re driving [our data research] to the clinic, we have to think about how we’re going to make that individual-level available in a meaningful format.”

Ultimately, the goal, he says, should be to “create intuitive graphical representations of the underlying data” in ways that allow non-data scientists “to explore it without having to sit at a terminal and type in a bunch of obscure commands.”

“What you want to think about doing when you make data available to people is to create interfaces that allow them to dive in and make sense of that data, using their own intuition,” Quackenbush says.

Without doing that, all of our growing mounds of big data will simply be big blobs on ever-bigger data servers.

What’s to stop that from happening? The incentive for turning all this raw feedstock into a usable fuel “is not going to be enhancing healthcare or making people better,” Quackenbush says flatly. “The driver is really going to be the most important ‘–omics’ science of all: which is economics. We have to show that there’s an advantage to bringing this kind of data and information together if we’re really going to make advances.”

More news below.

Clifton Leaf, Editor in Chief, FORTUNE


23andMe, Lundbeck to collaborate on depression-genetic link study. Drug maker Lundbeck, genetic testing firm 23andMe, and think tank the Milken Institute are joining forces to study the genetic bases and underlying causes of psychiatric disorders like major depression and bipolar disorder. The study will “explore how these disorders and brain functions such as attention and decision-making are related to genetics,” the organizations announced Wednesday. Mental health drugs are a particularly tricky space because patients often react to them in very different ways and can see divergent manifestations of their diseases. The overarching goal here is to facilitate smarter and better-targeted drug development.

UCSF gets $10 million from Zuckerberg, Chan for health data research. Facebook CEO Mark Zuckerberg and wife Dr. Priscilla Chan will donate $10 million to the University of California, San Francisco in an effort to consolidate data silos. To be clear, this isn’t part of the Chan Zuckerberg Initiative—rather, UCSF’s Institute of Computational Health Sciences will receive funding to consolidate health databases among major University of California medical centers. (San Francisco Chronicle)

Gene therapy firm Spark’s shares fly on a strong Q2, hemophilia A data. Spark Therapeutics’ stock soared 20% in Wednesday trading on the heels of its second quarter 2017 earnings report and some (very) early data on the gene therapy-focused firm’s treatment for the blood disorder hemophilia A. While the early-stage company still mounted losses for the quarter, it received more revenue than expected as a result of its collaboration with pharma giant Pfizer. Furthermore, an early-stage study pumping up the dose of its hemophilia A treatment—which aims to be a one-time therapy for the rare blood clotting-related disease—appeared to hit its targets without serious side effects.


Agios lifted by milestone blood cancer drug approval. Agios has pulled off the kind of R&D timeline that bigger biopharma companies envy. The biotech received Food and Drug Administration (FDA) approval for its drug to treat the rare blood cancer acute myeloid lymphoma (AML), which will be marketed as Idhifa, on Tuesday. Strikingly for Agios and partner Celgene, the therapy was in human clinical testing not so long ago (there was just a three year gap between the first human studies and Agios’ regulatory submission to the FDA). Agios shares rose 5.6% in Wednesday trading. (Reuters)

Biotech IPOs: Clementia raises $120 million. The biotech IPO march persists. Clementia, which focuses on super rare bone disorders, raised $120 million in its public offering at $15 per share. The firm has licensed an experimental treatment from Swiss drug giant Roche.


Anthem exits many California exchanges, BCBS of North Carolina reduces its premium hike. The recent legislative drama surrounding Obamacare is a critical political story. But all of the game theory may have obscured the fact that actual firms have to prepare their businesses for this uncertain regulatory landscape. As I’ve been reporting this week, the Trump administration’s threats to cut off insurer payments that help low-income Americans with their deductibles and out-of-pocket medical costs has already had a real-world effect on markets. And a key organization to track is Blue Cross Blue Shield and its federation of health plans (which includes Anthem). Unlike other health insurers who have made high-profile exits from Obamacare marketplaces (but are concentrated in sectors like the large employer market), BCBS plans are essentially a lynchpin of the individual insurance exchanges. And recent activity underscores the complicated Obamacare landscape. In California, Anthem Blue Cross is exiting 16 regions; however, Golden State residents will still have access to at least three Obamacare providers on average. And in North Carolina, BCBS actually cut its average rate hike from 23% to 14%, the firm announced Wednesday. Several prominent insurance companies have cited uncertainty as one rationale for their premium hikes this year. (Axios)

Surgeon General nominee cautious on gun violence/public health question. Trump Surgeon General nominee Dr. Jerome Adams has garnered bipartisan praise for his work on opioid addiction and combating HIV. But Adams carefully balanced his responses on gun violence during a hearing before a U.S. Senate committee. “Guns and gun owners aren’t inherently a public health problem, but the violence that results absolutely is,” he said. Surgeons General dating back to Ronald Reagan appointee C. Everett Koop have consistently cited the medical damage from gun violence as a major public health threat; Adams didn’t reject that logic, but his wording underscores just how politically fraught the issue is. (STAT News)

Biotech stocks on a roll in 2017. As you may have seen, the Dow crossed the 22,000 mark on Wednesday—a record. One sector that’s outpaced the overall market? Biotech. While the Dow, S&P, and NASDAQ are all up anywhere from 11% to 18% year-to-date, major biotech indices are up anywhere from 20% to 30%.


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Produced by Sy Mukherjee

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