The Food and Drug Administration (FDA) has proposed creating a new office for Patient Affairs to serve as a central hub for the agency’s engagement with patient communities. What does that mean? In a nutshell, the agency is trying to make it easier for both sides to communicate with and learn from each other, with the end goal of a regulatory system that works better for patients. Some of the agency’s specific goals include:
- To serve as the official “front door” for patient communities, from official advocacy organizations all the way down to self-organized grassroots efforts;
- To make it easier for patient stakeholders to reach the right people inside the agency by setting up a triage and navigation system for inbound inquiries;
- To make better use of technology to help the agency communicate with patient stakeholders online, as well as to keep track of what they learn from patient communities for future use;
The FDA is looking to understand patient communities better. This means understanding patient communities’ perspectives about specific drugs, medical devices, and increasingly, digital health apps. More broadly, it also means the FDA wants to understand how to get patient groups to weigh in on ongoing regulatory issues, and to understand the regulatory process better.
So, What’s the Fix?
If you’re like me and you don’t know a whole lot about the FDA, all of the above sounds like perfectly reasonable goals for a Federal Agency to have. But if overarching goal is to improve their relationship with patient communities, then a good place to start might be to understand how those communities feel about their relationship with the FDA.
While it’s made strides in recent years to engage with patient stakeholders, the FDA remains difficult for patients to use. The people who work there may have the best of intentions, but as a whole the agency lacks a culture of consumer-friendliness. Patients, parents, caregivers, and other laypeople with important perspectives to share are likely to find the process of submitting feedback or weighing in on proposed regulations confusing and challenging, especially if they have no prior experience in interacting with a federal agency.
Deeper than that, “there has historically been a tendency in the agency to focus on hard data and write off patient experiences as anecdotal,” according to one insider. This attitude mirrors the internal bias of the medical industrial complex more broadly; patients are not considered the experts on their own conditions, their own bodies, or their own health care.
Diabetes offers perhaps the most prominent example of some of the progress – and limitations – of the FDA’s engagement of patient communities to date. Patients with type I diabetes require insulin injections and glucose monitoring throughout the day to stay in optimal health. Unfortunately, not only are the devices used to deliver insulin distinct from the devices used to monitor blood glucose, but both devices tend to come from different manufacturers, with little to no out-of-the-box features to sync or share data. While the FDA recently approved the first fully closed loop system (also known as an “artificial pancreas”) it remains unavailable to purchase, and doesn’t do anything for the millions of individuals who have already purchased glucose monitors and insulin pumps.
And that’s just the way it is, at least for now. Bound by regulations on safety and data access, the FDA wasn’t able or willing to act on the concerns of the patients, parents, and caregivers who wanted tools that made their 24/7 job of managing diabetes easier. When the federal agency responsible for regulating the medical devices that keep you alive tells you to wait, what’s the fix?
Patient-Driven Problem Solving
Over the last four years, patient communities began taking matters into their own hands. Social media hashtags like #wearenotwaiting began popping up as people started hacking and tweaking their own devices to make the data shareable, as well as modify alerts and feedback loops. These do-it-yourself (DIY) home hacks enabled parents to keep tabs on their children’s’ blood sugar levels while the kids are at school. It also helped adults with diabetes tweak their own monitoring tools so they would actually fit into people’s daily (and nightly) lives. An open-source project called Nightscout became an official hub and guide for DIY mobile technology systems; their “CGM In The Cloud” Facebook group has over 23,000 members.
Dana Lewis, one of the featured speakers at the upcoming What’s the Fix conference, is one of the pioneers of the DIY diabetes innovation movement. Diagnosed with type I diabetes at age 14, she grew frustrated with her continuous glucose monitor’s inability to wake her up in the middle of the night when her blood sugar was low. She started tinkering and went on to develop her own “artificial pancreas” by hacking her devices’ data and bridging the communications between her devices. Not only that, but she also helped make code and documentation freely available to help other patients, families, and caregivers who also want to take matters into their own hands, which started the OpenAPS movement.
Dana will share her full story in June in Seattle – but we had a chance to ask her via e-mail about her experiences and insights on the FDA’s efforts to improve community engagement. Her perspective is an optimistic one – the FDA has shown interest in improving patient-friendly regulation, and has been engaged in the right conversations over the last few years; This new proposed office appears to be another step in the right direction. One of her key recommendations is simply for the FDA to approve systems that have been approved by other countries’ regulating agencies. Overall, while the FDA’s progress in improving patient-centric regulations for diabetes has been slow and steady, most patients are still forced to wait for better solutions.
Her remarks, in full, are below – the emphasis added is my own:
“Our first direct experience with the FDA was when we originally presented about DIYPS (my original “louder alarm” and eventual open loop system) at a D-Data Exchange meeting in San Francisco in early 2014. Two attendees from the FDA came over to our booth, and were very interested in what we were doing. They mentioned that the fact that DIYPS was making insulin dosing recommendations would make it a class III medical device, and suggested we might not want to share our code online. Since that time, we have had a number of constructive discussions with them around Nightscout and then later OpenAPS (a DIY hybrid closed loop system, the code and documentation for which we did ultimately decide to make open source), and have been impressed at the degree to which the FDA has made themselves familiar and comfortable with the work of patients advancing medical technology through DIY innovation and a #WeAreNotWaiting spirit.
Despite the FDA and patients’ shared mission and close collaboration, there are a number of areas where the great people at the FDA seem hemmed in by their own regulations and end up slowing down the availability of breakthrough drugs and medical devices that would greatly improve the lives of people living with diabetes. While the FDA has accelerated approval of commercial APS (“hybrid closed loop”) technology, and has not proven to be a blocker there, other devices like the Freestyle Libre and DANA*R insulin pump, and more effective new drugs like Fiasp, are still not FDA-approved for use in the United States. However, they are being actively used by our counterparts with diabetes in other parts of the world.
While it’s difficult to know what the optimal solution would be for reforming a large bureaucracy like the FDA, there is one major change that would make a huge difference: regulatory reciprocity. This is the idea that, if another agency like the European Medicines Agency, Health Canada, Australian TGA, Japanese PMDA, etc. have approved a drug or device, then the FDA should presume it to be safe enough for Americans to use as well. This presumptive approval might be provisional, with a short built-in delay, such that the FDA could place a hold on approval within 30-60 days if they have good reason to suspect that something may have been approved prematurely and may have safety concerns. And the FDA may still wish to go through its full approval process before declaring that a drug or device is considered *effective*, which might be considered by large payers like Medicare and private insurers when decided how such a treatment should be covered. But there is no reason it should be illegal for an American to pay out of pocket for Fiasp insulin, a Libre sensor, and a DANA R insulin pump.
While #WeAreNotWaiting to get access to our diabetes device data and make the best use of the devices available to us, many of use *are* still waiting for the FDA to do its part and make new technologies available to more quickly improve and save as many lives as possible and reduce the burden of Type 1 diabetes. We shouldn’t have to.”
Dana Lewis is a speaker at the inaugural “What’s the Fix” conference, an event designed to highlight stories of patients improving care for themselves or their loved ones. Virtual attendance has been made available for free – more information on how to register is available online.
Share your stories and perspectives with us on social media using #WTFix and #whatthehealthcare.