When “Self-Reported” Research Is Reported By Itself

There are some of us, to be sure, who are inveterate chroniclers—natural-born Homers (the Greek, not the Simpson) who can relate from memory how many stairs were climbed or paces were paced in any given hour or day. But most of us, well, aren’t. We’re imperfect scribes with imperfect memories.

Which is why this report from JAMA Cardiology last week was so significant—more significant, I would say, than the amount of attention it received. In the study, Stanford researcher Euan Ashley and colleagues set out to determine if they could automatically measure “objective, real-world physical activity patterns, fitness, sleep, and cardiovascular health” from consenting volunteers who downloaded a Stanford Medicine-developed iPhone app called MyHeart Counts (which is based on Apple’s open-source framework, ResearchKit).

Lots of people downloaded the free app after it was made available in March 2015. No shocker there. But what was surprising, at least to me, was how many of those app users consented to participate in the study: 48,968 of them—which included individuals in all 50 states and the District of Columbia. And more surprising still is that nearly 82% of those folks, or 40,017 volunteers in all, actually uploaded data to the research team.

Though only 9% or so of consenters (4,552 people) uploaded a full week of reports on their physical activity, that’s still a big sample size for an objectively measured, data-driven research study. The “always-on, low-power motion chips” in the smartphones captured what no human memory ever could.

While one aspect of Stanford study involved self-reporting (some participating volunteers filled out a questionnaire on their perceptions of their own heart health), the most significant finding of the study didn’t come from that.

Rather, in the words of the researchers, it’s that both “mobile consent” and “real-time gathering of sensor and survey data from a large ambulatory population” are eminently feasible. And in a smartphone-driven world, that has changed public health research overnight.

More news below.

DIGITAL HEALTH

athenahealth says it wants to bag a return on electronic records. The HITECH Act helped bring about the era of electronic health records back in 2009. Since then, providers have been grappling with how to turn a critical advancement in digital health into a functioning system that delivers value, not just to patients, but their own bottom lines. And at least one firm, athenahealth, says that's the goal for 2017. "Our big moonshot work here in 2017 is getting back to a true ROI," athenahealth's chief medical officer Dr. Todd Rothenhaus told Healthcare IT News in an interview. "We’re working to get past the idea that an EHR is a necessary expense. Instead it’s a tool for automation." Part of the strategy? The firm is aiming to help its clients fully implement the new ICD-10 medical code platform and not charge for the service unless it's successful. (Healthcare IT News)

Hobbled gene therapy pioneer uniQure gets (another) new CEO. uniQure, famous for becoming the first company in the world to bring a gene therapy to market, has decided to name interim chief Matt Kapusta as its official next CEO. "Matt has provided exceptional leadership over the past two years, most notably in his capacity as interim CEO where he led a comprehensive strategic planning process that has focused our product pipeline and streamlined our operations," said uniQure board chair Philip Astley-Sparke in a statement. uniQure's milestone European approval for Glybera, a gene therapy to treat the extremely rare disorder lipoprotein lipase deficiency, has faced massive hurdles thanks to its $1.2 million cost. Sales have been, literally, almost non-existent, with insurers across Europe balking at the price tag. Kapusta will be the company's third CEO in a year.

The world's first stem cell therapy trial for Parkinson's is back on track. California's  International Stem Cell Corporation (ISCO) has treated a second patient in its pioneering clinical trial for Parkinson's disease. The biotech's procedure involves transplanting millions of stem cells in order to make up for the dopamine-making cells that are destroyed by the disease. The early-stage trial involves a dozen patients, and was temporarily put on hold because of a supply chain problem, according to the company. ISCO hopes to have preliminary efficacy data available by early 2017. (FierceBiotech)

INDICATIONS

AstraZeneca oncology head jumps ship to lead small, French biotech. French biotech Innate Pharma has poached Mondher Mahjoubi, the head of AstraZeneca's oncology therapeutics space, as its next CEO. The biotech is one of dozens that's working on experimental cancer therapies; but Mahjoubi's exit from AstraZeneca comes at a particularly important time for the pharma giant, which is hustling to try and get its next-gen "checkpoint inhibitor" cancer drug into the market after rivals Merck, Bristol-Myers Squibb, and Roche beat it to the punch. As we've previously noted, big pharma companies in particular have been facing road bumps in their development programs, and new drug approvals in the U.S. fell to a decade-long low in 2016.

Could Botox treat... Depression? Allergan's flagship cosmetic treatment, Botox, has been on the scene for years. But its star power and ability to draw sales won't be dying down any time soon as the pharma giant continues rack up new approved indications for the treatment—and now, Allergan is hopeful that Botox could become a viable option for treating depression. A mid-stage trial in Germany found that the injectable could be just as effective as antidepressants in patients (many antidepressants aren't effective, which is why patients often have to try out a number of different medications to find one that works). Botox is already commonly used for a number of non-cosmetic purposes, including chronic migraines. (FiercePharma)

THE BIG PICTURE

Obamacare had its busiest sign up day ever. President Obama announced during his final end-of-year news conference that Healthcare.gov had its busiest day of all time last Thursday, with 670,000 people signing up for coverage in a single day. The enrollment rush may have something to do with the prospect of the incoming administration, which has promised to make repealing the health law a top priority, as Americans scramble to make sure they have insurance for at least the next year. The spike also encouraged the Obama administration to extend the signup deadline to this evening in order to accommodate the approximately one million people who are still in the midst of the process. (The Hill)

Hospitals are getting aggressive on controlling drug spending. The Wall Street Journal reports that hospitals are getting more aggressive in their perpetual tug-of-war with pharmaceutical companies over drug spending. Providers are now employing methods such as voluntarily reducing their medication inventories and opting for cheaper generic therapies. One potentially problematic method? A more judicious use of when treatments are administered. Hospitals say the steps are necessary as national spending on drugs continues to rise. (Wall Street Journal)

REQUIRED READING

Why Drug Costs Will Keep Rising in 2017, by Bob Kocher and Bryan Roberts

The Creepy Virtual Assistant That Embodies Japan's Biggest Problems, by David Z. Morris

Here's How the Galactic Empire Fails at Cybersecurity In 'Star Wars', by Robert Hackett

SoftBank Invests $1 Billion in Satellite Startup, by Reuters