“There is so much promise in natural experiments that we could run,” PCORI Executive Director Joe Selby says. “Across the country, there is a lot of variation in how diseases get treated. This is a golden opportunity to study which leads to better outcomes.”
Right now, health-care experts say that doesn’t happen: Most doctor visits don’t do much to inform which treatments are most effective.
“Right now, were spending trillions on health care and 99.9999 percent doesn’t contribute anything to medical knowledge,” says Farzad Mostashari, national coordinator for health information technology at Health and Human Services. “That’s the real vision, that we actually take routine care, the heallth care happening in the real world, and have that contribute to knowledge.”
PCORI will spend as much as $68 million to fund “clinical data research networks,” which will each consist of at least two health networks that cover a minimum of 1 million patients. They will each propose three sets of patients to study: one focused on a common disorder, a third focused on a rare medical challenge and the last looking at patients with obesity or diabetes.
“The two networks might have a different formulary which covers different medications,” Selby says, describing one possible experiment that a network could do. “We could look at where each is typically used, and which one leads to a better outcome.”
The nonprofit will also fund another set of “patient-powered research networks,” smaller patient groups, possibly organized by advocacy organizations, that would cover at least 0.5 percent of a given disease’s population — giving researchers a way to study what works best in treating certain conditions.
While patient advocacy groups and industry officials support PCORI’s mission, they do raise concerns over whether the operation will succeed. Many have questioned whether these new networks will be able to easily share data, given that many electronic health records are not interoperable.
“There are significant technical issues that need to be resolved,” says PhRMA President John Castellani, who spoke at a Tuesday panel on the new PCORI initiative. “This is not a short-term product. I’m concerned that if we oversell the short-term potential, the result will be inaccurate analysis based on short-term data.”
Nancy Davenport-Ennis, founder of the National Patient Advocate Foundation, also raised concerns about whether patients would want to participate in these wide-reaching experiments. She recalled attending a recent meeting in Washington, of about 500 people who work in health care, where they were asked how many would be interested in having their data used for research, provided that it was made anonymous.
Only two attendees raised their hands.
“We need to focus on what the patients want and the information they receive,” she says.
Selby does acknowledge that there are challenges in building a research network like this one — but also great potential to better inform the everyday decisions that doctors make.
“This is a chance to actually begin conducting large scale, comparative effectiveness research,” he says. “It’s kind of amazing how long we’ve been willing to wait for answers on these types of questions. Now we’re going to start getting them.”