Putting patients at the center of the pursuit of real world evidence

In the September 2017 issue of Standard Practices (https://www.hpnonline.com/healthcare-standards-make-trip-real-world/), I wrote about how real world evidence (RWE) can help us better understand how medical devices perform in “the wild.” Jeff Shuren, MD, JD, head of the Center for Devices and Radiological Health (CDRH) at the U.S. Food and Drug Administration (FDA) and others have used that term to contrast how medical devices approved for specific uses by the FDA based on clinically controlled trials are used more broadly in routine clinical practice on a wide range of patients and sometimes for uses beyond their originally intended purpose. This is also the only place where we can really find out how those devices perform, i.e., whether they contribute to better quality outcomes and for which patients. But we can only do that if we are collecting data on their performance in a standardized way and from multiple sources, including patients.

I have written for many years about the need to use unique device identifiers (UDIs) to standardize how we identify the products used in patient care, although most of the focus has been on capturing UDIs in electronic health records. We are making progress in this area, with more than 1.7 million UDIs in the Global UDI Database (GUDID) and more providers working to capture the identifiers to meet pending regulatory requirements to share UDIs for implantable devices as part of the Common Clinical Data Set (CCDS).

What I haven’t written about as much (beyond the March 2015 column, “Know Your UDI”)  is the need to gather RWE from patients themselves. That’s changing, as the FDA, manufacturers, hospitals and patient advocacy groups are working to get patients more involved.

It makes perfect sense, given all the talk about the need for patient-centered care.

As a patient representative on the Michael J. Fox Foundation for Parkinson’s Research told those attending the annual public meeting of the Medical Device Innovation Consortium (MDIC) in October, it’s a whole lot easier now with advancements in patient engagement technology. Just a couple of weeks prior to the MDIC meeting, the Duke-Margolis Center for Health Policy released an action plan focused on the development and use of mobile health (mHealth) technology to advance patient-generated RWE.

There are two primary opportunities for patient engagement. One is providing input on how developers can make mHealth technology easier and more acceptable for diverse patients to use. The other is feedback on their experience with the medical devices themselves. Do they meet an unmet medical need? Is it what patients want? Patient advocates say sometimes what manufacturers develop and the FDA approves is not always what patients want or need.

Dr. Shuren says the FDA’s role is evolving from primarily command and control to facilitating collaborative communities for all key stakeholders, including patients. In September, the agency announced formation of its first ever Patient Engagement Advisory Committee.

The medical device manufacturer trade association Advamed has also called upon the Center for Medicare and Medicaid Services to be more patient, and less budget, focused. Advamed president and CEO Scott Whitaker said both the 21st Century Cures Act, passed last year by Congress, and the Medical Device User Fee Amendments, which structure fees paid by manufacturers, include provisions for patient engagement and RWE in the regulatory process. But he says there is not a similar pathway at CMS, which approves if and what it will pay for use of those products. That, he contends, is holding up patient access to innovative new technologies. At the MDIC meeting, he spoke of possible legislation that would allow CMS to give a product a temporary reimbursement code that can be used while more evidence on its performance in clinical practice is accumulated.

Kim McCleary, who leads an effort at FasterCures to increase patient engagement in evaluating the risks and benefits of medical devices, cautioned that individual patients have different opinions on what they personally consider safe and effective and how much risk they are willing to bear. This, she says, requires advancing the science of patient input to take into account these differences.

We need to have confidence in the quality of the data upon which decisions are made, whether by the FDA for market approval, by payors for reimbursement, by physicians and supply chain when sourcing products, and by patients who ultimately bear the risks and benefits as well as an increasing percentage of the costs.

UDIs are fundamental to making sure we are talking about the same product no matter where that data comes from: patient reported data in mHealth technology; hospital reported outcomes data; research conducted on data in registries and claims, and the National Evaluation System for Health Technology (NEST), among other sources. The FDA has tasked the MDIC with building NEST to generate and leverage RWE across the total product lifecycle of medical devices.

Adrian Hernandez, MD, Vice Dean for Clinical Research at the Duke School of Medicine, noted at the MDIC meeting that common (standardized) data systems are essential, but added that not all of the data will be in a single database or system. Wherever that data exists, it needs to be relatable, shareable, and consistent to the extent possible. One of the challenges with the mHealth movement, noted by an angel investor at the MDIC meeting, is that many new start-ups are proliferating data that only exists in their systems, and if that data is not collected in a manner that is standardized, then we have only created more data, not better data. While innovation often takes many roads, the key is a collective appreciation and pursuit of the ultimate goal: better RWE for better value for patients