FDA must make smarter use of big data
A bipartisan think tank is calling on Congress to enable the Food and Drug Administration to use hospital electronic health records and crowd-sourced patient experience data to transform the drug and medical device approval process.
[See also: FDA calls for medical device oversight]
On Monday, the Bipartisan Policy Center kicked off its five-point "FDA: Advancing Medical Innovation" initiative, focusing on the agency's role in biomedical research and how big data – currently soloed in healthcare IT systems and patient social networks – can improve personalized medicine, genomics, molecular pathways and other cutting-edge treatments.
"Our inefficient, less-than-modern, drug discovery and device approval process drives up cost and delays treatment," said the initiative's co-chair, former Senator Bill Frist. "We must accelerate the process of getting safe and effective drug and medical devices to patients."
[See also: Big data doesn't have to be 'Star Wars']
In particular, Frist noted that the FDA's current review processes fail to reflect advances in the aggregation and analysis of real-time patient data at hospitals and healthcare, as well as from patient-focused social networks.
"The FDA saw its last major update (in its authorizing statute) back in 1997, when I was serving in the Senate," he said. "That was 18 years ago, and what has happened since in our own lives – the technological advances have been really mind-boggling. They really don't fit, at least in a good way, into a 20th-century regulatory framework."
As a result, more drug discovery and device development is taking place overseas, Frist said, and private U.S. venture capital investment in medical innovation has declined by 42 percent since 2007. Just as importantly, he said, American patients may be waiting for treatments that other countries have already vetted and approved.
How big data can improve the FDA
At the kick-off event in Washington, D.C., panel members Marc Boutin, CEO of the National Health Council, and Mark McClellan, former administrator for CMS and commissioner of the FDA, and now a senior fellow at the Brookings Institute, agreed that the FDA's primary reliance on pre-market clinical trials to review the risks and benefits of medical innovations needed to catch up with innovations in healthcare data and systems.
But they noted that the FDA is constrained by the current rules in place and by the resources it has been given.
Boutin, whose organization advocates for stronger patient involvement in healthcare delivery, urged Congress to create legal "guard rails" so that medical researchers working for drug and device makers could tap into crowdsourced patient views – aggregated in patient social networks – without fear of being fined for marketing unapproved products.
"They should be able to engage in sophisticated methods (or data collection and analysis) to understand patient preferences," Boutin said, noting that many other fields – from consumer marketing to electoral politics – had adopted such techniques to great effect. "But we're not taking these well-established methods and plugging them into drug and device development."
While Boutin focused on pre-market big data opportunities, McClellan emphasized post-market surveillance opportunities.
"There's been a sea change in both science and the data systems in our country and around the world," he said, pointing to the FDA's own Sentinel Initiative that draws on existing automated healthcare data from multiple sources (including 150 million Americans) to actively monitor the safety of medical products continuously and in real-time.
"But these methods aren't being used for device safety," he added. "And they're not looking at the social networks that patients are using now to discuss effective treatments."
McClellan said collaborating with health plans and health delivery systems by "collecting longitudinal data from individual patients" could enable the FDA to make better decisions about the safety and effectiveness of devices and treatment with the limited resources it has.
Janet Marchibroda, BPC's health innovation director, said the medical innovations initiative will result in concrete policy recommendations to Congress.
"We're moving pretty fast down the road here and will be delivering our first recommendations in late spring," she said.
The recommendations will focus on five key areas:
- Improving the time and cost associated with the discovery, development, and delivery of safe and effective drugs and devices for patients;
- Reviewing the scope of activities within the FDA;
- Advancing a more efficient and effective regulatory framework for medical products;
- Strengthening FDA's ability to carry out its mission; and
- Maintaining U.S. global leadership in medical innovation.
The initiative is being released at a time when the industry is becoming increasingly focused on regulatory issues in Washington D.C. A recent survey from Peak 10, an IT infrastructure and cloud services provider, found that 95 percent of health IT professionals say complying with regulations influences is the chief driver of their decision-making. And three in five say too many rules are having a negative effect on healthcare IT.
In addition to the concerns over content of the FDA's guidance, there are also several bills in Congress that would pre-empt the agency's ability to regulate the industry at all. Those bills include the SOFTWARE Act – part of the the House Energy and Commerce Committee's 21st Century Cures draft legislation – sponsored by Rep. Marsha Blackburn (R-Tenn.). A Senate version of the bill is sponsored by Orrin Hatch (R-Utah) and Sen. Michael Benet (D-Colo.).
Initiative co-chair Bart Gordon, a former member of the U.S. House of Representatives, said he was convinced the BPC's work would be reflected in final legislation likely to pass in the next 12 months.
"With these bipartisan leaders, It's just like slopping the trough – when you bring them good ideas, that's what they want."