As the year winds down, STAT reporters are taking a look at the stories they’re most eager to track in 2016. We’ll be running these daily through Dec. 31. Look for more New Year’s predictions here.
Big valuations, big investments, and big price tags drove lots of the news in health care in 2015 — and you can bet they’ll do the same in the new year. For signs of where things are headed, keep an eye on these three influencers:
Jeffrey Marrazzo, chief executive of Spark Therapeutics
The chief executive of the up-and-coming Philadelphia startup Spark Therapeutics is at the center of two of the biggest questions in biotech: Can gene therapy work — and can the system afford to pay for it?
The idea that a one-time treatment could replace a broken gene has generated plenty of scientific and commercial interest, but a high-profile patient death in 1999 set the field back considerably. Now, Spark and several other companies are leading a comeback, working on drugs that will likely carry sky-high prices — which some observers fear will stretch the health care system to its limits.
Marrazzo, who has a background in state government, plans to go to regulators in the new year with Spark’s gene therapy for blindness. The FDA may not act until 2017, but in the meantime, watch for Marrazzo to keep testing the waters with talk about potential pricing models, such as “pay-for-performance” plans. On the science side, don’t miss Spark’s expected release of results from a midstage study testing its gene therapy for hemophilia — another possible indicator of the technology’s promise.
Deborah DiSanzo, general manager of Watson Health
The general manager of IBM’s big bet on health care analytics has a busy year ahead. DiSanzo was brought in just four months ago to lead IBM’s newly hatched Watson Health unit, which aims to use high-powered computing to sift through massive amounts of data to find useful and cost-saving nuggets for medical researchers, doctors, and insurers. (You may remember Watson as the supercomputer that made its big debut on Jeopardy! in 2011 and inspired a $1 billion investment by IBM.)
DiSanzo, who spent two years as chief executive of Philips Healthcare before leaving last year as part of a management shakeup, leads a Watson Health team of more than 2,000 people worldwide, including about 700 who will soon move into the unit’s new headquarters in Cambridge, Mass.
DiSanzo will be tasked in the new year with generating results for Watson Health’s list of early customers, which reads as a who’s who of heavy hitters in health care — CVS Health, Johnson & Johnson, and Teva Pharmaceuticals, among others. Watch for whether DiSanzo can make these companies happy and lure new customers, too.
Vivek Ramaswamy, chief executive of Axovant Sciences
The 30-year-old former hedge fund manager became the poster boy of the much-debated biotech bubble back in June when he pulled off the largest-ever biotech initial public offering — on the back of a drug compound for Alzheimer’s disease that GlaxoSmithKline had discarded.
But Ramaswamy, who Forbes says is worth $500 million, has recruited some top Alzheimer’s researchers to join his New York-operated company, Axovant Sciences, where he’s chief executive. And in October they initiated a late-stage clinical trial testing the GSK reject in combination with another Alzheimer’s drug.
Don’t expect that big study — or another test of that same compound in patients with Lewy body dementia — to deliver results in the new year; those aren’t expected until 2017.
But another one of Ramaswamy’s acquisitions — a compound that’s been tested in patients with insomnia — will be put to the test in Lewy body dementia patients with visual hallucinations and is expected to yield results in the second quarter of the year. That could be a first sign of whether Ramaswamy can actually deliver on his drugs and reward investors, who have sent the company’s stock down by about a third since the IPO.