Transforming Healthcare: Boosting Real-World Evidence With Digital Health

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Traditionally, data from electronic health records, claims, and billing have been used by researchers in prospective analysis. However, there is a recognition that traditional clinical trials are expensive, lengthy, and do not provide granularity on patient-specific characteristics or treatment preferences. This is where real-world evidence comes in.

At the first plenary session at the ISPOR 2018 conference, May 19-23 in Baltimore, Maryland, panelists updated the audience on how digital progress can influence real-world evidence to allow near real-time data analysis and inclusion of specific patient information, lowering cost and providing higher quality care downstream.

The session was moderated by Rachael L. Fleurence, PhD, executive director, National Evaluation System for Health Technology Coordinating Center, Medical Device Innovation Consortium.

Other panelists included Adrian F. Hernandez, MD, MHS, vice dean, Clinical Research, Duke University School of Medicine; Michelle McMurry-Heath, MD, PhD, worldwide vice president and global head, Regulatory Affairs, Johnson & Johnson Medical Device Companies; Danica Marinac-Dabic, MD, PhD, MMSc, FISPE, director, Division of Epidemiology, Center for Devices and Radiological/OSB, FDA; Sally Okun, RN, MMHS, vice president, Policy & Ethics, PatientsLikeMe.

Fleurence provided several examples of disruptive technology: caring for patients with dementia in real time, an app that feeds into Alexa to help parents decide on children’s doses, and wearable devices and trackers for continued health monitoring. “These technologies provide opportunities for gathering real-world data, during treatment or at home,” without direct clinical supervision, Fleurence said.

But against this backdrop, Fleurence pointed out, are sobering statistics on US healthcare outcomes. “Life expectancy remains low, there are inequalities in health outcomes between states and between counties, among others.” She told the audience that the objective of the plenary session is for panelists to debate if we are at an inflection point: “Can we deliver on the innovations that we have at hand or do barriers remain?” Fleurence asked.

Okun, a trained nurse, provided some background on how her organization, PatientsLikeMe, came to be. “Fourteen years ago, PatientsLikeMe launched a website to provide patients an open social network to share their journey of living with and managing illness,” Okun said, providing them with structured tools for the process. This was unthinkable at that time, she said, given the fact that there was no academic or political regulation, or HIPAA (Health Insurance Portability and Accountability Act of 1996) restraints, and no medical oversight. “This has now become a very helpful tool for patient-generated data and patient insight,” she said. “Our 600,000 members have accelerated and advanced the transformational role of engaged and empowered patients.”

Okun emphasized that the focus of today’s digital advances should be improving patients’ lives. “Several questions remain, however, including, do we need to redefine digital evidence?”

“We have a range of technologies, from orthopedic devices to atrial fibrillation to breast implants,” McMurry-Heath said. “We are trying to find accurate data that provide insight on device performance.” But time is of essence for collecting and acting on these kind of data, she said, especially when the device is out in the real world and also when you are innovating, such as with a new device.

“Part of what we are talking about with real-world evidence is to identify a rapid way to detect the pulse of how the devices are improving patient lives, outcomes, and impacting healthcare overall.” McMurry-Heath said. Pointing out the near-ideal nature of clinical trial design, she said that researchers should “divorce” themselves from the pristine designs of controlled clinical trials. “Our idealistic forms of questioning [with these controlled trials] might not answer some of our questions, or answer them too late.”

From the FDA’s perspective, the regulatory body has not really used observational or surveillance data for regulatory decisions, but this is changing now, according to Marinac-Dabic. “The doors for using real-world evidence have been opened up by the FDA and this is helpful for the ecosystem,” she said. In her opinion, the recognition of premarket and post-market continuum of evidence for a product is a significant and vital change in FDA’s approach to continuous product evaluation.

Marinac-Dabic explained that while traditionally, the FDA has been a regulatory body—holding primary dialogue with the pharmaceutical and device industry—the current approach is to involve multiple stakeholders in the conversation. “We enjoy being research partners with the multiple stakeholders who are a part of the dialogue now,” she said.

Beyond the FDA, there’s opportunity for sister agencies—such as the CDC, CMS, and the Agency for Healthcare Research and Quality—to work much more closely with each other, “and there’s a big push to bring the communities together, especially in the device and drug world,” Marinac-Dabic added.

Hernandez used Duke as an example to provide the perspective of the patient—health system relation. He posed the question: “How do we integrate the data we collect when they are under the health system’s watch to when they are back home and living their lives, but still require surveillance?

A case study followed where he described the case of an 80-year-old patient who suffered multiple fainting spells and consequent visits to the emergency department. Multiple tests failed to provide a firm cause for his episodes. During his second episode, though, the patient sent a note to his care provider through a wearable device that was tracking his health. The patient finally ended up getting a pacemaker after the third episode, after analysis of data monitored by the patient’s wearable device that identified a a low heart rate as the cause of his condition.

When Fleurence asked the panelists to define digital health, Okun said, in her world, it’s information that can quickly answer patients’ questions on how they may respond to a treatment, maybe even without clinical oversight and helps drive them to the right next step that is personalized for them.

What McMurry-Heath’s device company, digital health translates into being able to accumulate cumulative results for analysis and using them for decision-making at the point of care.

Fleurence asked Hernandez to share information on a Duke-Stanford-Google-Verily collaborative: Project Baseline, which is trying to understand small signals that can indicate changes in human health. “Our objective is to understand an individual’s baseline health and follow-up on changes, including genomic information and their microbiome, and how it affects the person’s health trajectory,” Hernandez said. It’s a research platform that continues to live as people have questions to develop proof-of-concept ideas for health.

“Are any of these efforts bright spots that can then be brought to scale?” Fleurence asked.

“It’s been a laborious process so far…we are recognizing that we need to make it easier and we need to turn informed consent to understood consent,” Okun said. “This can help advance research and its efficiencies, and also help ensure patients’ influence on study design and outcomes.”

“We also need to define the value proposition better,” McMurry-Heath said. “We see that we get more data when patients understand and realize how all of the data we gather improves their daily life.”

She added that payers often have a higher bar on data, and with real-world evidence, healthcare is entering into the space where payers have traditionally been. “Having multiple standards or moving hurdles is a barrier…it helps when all stakeholders are evaluating data through the same lens,” she added.

For Hernandez, however, data access seems like a substantial challenge, especially when dealing with novel information. He resounded McMurry-Heath’s sentiments saying that it is important to clearly state the return of investment or value of the health data.

As a parting statement, Fleurence asked the panelists to predict whether the healthcare field will continue to struggle with data 3 years from now.

Emphasizing the need to keep the patient at the center of the conversation, “which will help us be accountable to the patient,” Okun said that she believes that we will be there in 3 years, “but there will be a few slippery stones along the way.”

“The most important point is the internationalization of the information,” said McMurry-Heath. “We have to think of data as being culturally specific…we may be seeing more cross-border pollination of data.”

From the FDA’s perspective, “the future will be in the public—private partnership to continue to work together,” said Marinac-Dabic, who believes that policy changes and alternate paradigms will get us there faster.

“A year ago, I’d have said the answer is easy…now, we have to make sure we balance competing interests,” said Hernandez. While there’s been investment in data accumulation, he pointed to data privacy concerns that have recently been in the spotlight. “If there’s lack of trust, the free-flow of information will be stopped and data silos will be introduced, which will lock up answers. We need to bring that cultural change to be able to see this change in 3 years.”