Need for Multi-Level Cooperation to Change Device Approval System

December 10, 2020
Marco Meglio
Marco Meglio

Marco Meglio, Associate Editor for NeurologyLive, has been with the team since October 2019. Follow him on Twitter @marcomeglio1 or email him at mmeglio@neurologylive.com

Michael Okun, MD, executive director of the Norman Fixel Institute for Neurological Diseases detailed the collaborative effort needed to see sustainable change in the approval system of neurological devices.

This is part 2 of a 2-part interview. To view part 1, click here.

Traditionally, the development of neurological devices has been geared toward larger patient populations, leaving the those with rare diseases subjected to an unfair approval process, thus stalling improvements to clinical care. The issues that lie within the outdated approval system were outlined in a paper published by Michael Okun, MD, and James Giordano, PhD, in JAMA Neurology.

They touch on the use of humanitarian device exemptions (HDEs), which have long been used as a regulatory pathway for device availability, but have been phased out of the regulatory process because industry partners are unwilling to invest their money into something they see as “unattractive.” The reasons for this stance include limitations of profit, lack of incentives, and ongoing upkeep and technical support of these devices.

One of the methods Okun mentions in his paper is an expanded use of registries, which will allow for universities and foundations to submit a device master file and while serving as a co-applicant for full FDA approval. This would decrease legal risks to industry and provide incentives to manufacturers.

Okun, the executive director of the Norman Fixel Institute for Neurological Diseases, and Adelaid Lackner Professor and chair of neurology at the University of Florida, also serves as the medical director of the Parkinson’s Foundation. He feels as though resolution will require full cooperation from the FDA, industry leaders, private investors, clinicians and patient groups to fully understand the issues at hand and how they can be resolved.

In part 2 of this interview, Okun describes using the “kitchen table” method as a way to sit down all parties involved and hash out the issues that remain within the current system. He also mentions how the use of incentives may be key to getting greater cooperation from industry leaders, and why the expansion of data registries is key.

NeurologyLive: How do we begin to change the perception and push for more humanitarian device exemptions (HDEs)? Would you see more push if there was more readily available funding?

Michael Okun, MD: I don’t believe we need to give industry a free pass, but frankly we’ve made it so hard for them to the point where they’ve done a couple of these HDEs and are like ‘I don’t want to do it anymore.’ This is where they’re at. They’re happy for scientists like me to shake it up and ‘say this is wrong.’ This is policy that’s now affecting healthcare. For scientists like us to blow the whistle just shows how bad it gotten. The industry people say ‘fine, we’re out. Let’s focus on our core business.’ It takes the scientists and clinicians to blow the whistle. Insurance companies don’t want to come to the table and talk. They don’t want to talk there.

It’s hard for a patient or clinician to appeal or even pre-authorize for the devices they may need. We pointed out in the piece that there needs to be more enforcement of legislation. Whether that is stating that they have a pre-existing condition and even taking it to a court case. We need to put pressure on them to ensure that they are going to be able to provide because they’re a business too. We’ve got to get the players at the table to align or else we’ll continue to sit on technology that we know can help people but remains unavailable. It’s only going to get worse.

What is the importance of disease-based registries? What trickle-down effect can they have?

We’ve tried this and feel as though it’s pretty successful, especially with the data that’s been publicly available. The question is, how do you aggregate the data that’s necessary to ensure the safety of the consumer while having something ready to give to the regulatory agency. You need to do this in a way so that it’s not just a free-for-all. We haven’t incentivized this, but it can be done in industry. If you incentivize the industry to run these registries, there would be more of a reason for them to do it. Right now, there’s no incentive and industry has backed off. Industry has backed off to supporting most HDE applications because they know if it’s not core business, they’re not going to go there, because of the lack of incentive.

Would it be easier if it was incentivized for industry to run their own registries? Absolutely. That would be another solution to the problem. They would have the device and more ability to do what they want with it, but there’s no incentive. The burden is then put back on us. Let’s take the Tourette example. Why have we been fighting for a decade to claw out of the underbrush if industry has no reason to back us and provide help for us. We’ve gotten our own grant and own partners and tried to move it through that way. We suggest that’s a path for progress, but a more robust and sustainable long-term plan would be better on outcomes. This includes a partnership, handled by the industry, who’s good at regulating this stuff as well as collecting device-related side effects.

We feel like we’re at the table right now, but only the patients and doctors are left at the table. We’re struggling at this table trying to move something for the patients, even with the data and registries we do have. We’re struggling trying to get other partners. We’ve spoke to industry and they can be a willing partner if they’re incentivized to do so. They will be distracted by this for the greater good of everyone else involved. As for insurance companies, it’s all just “No, no, no” right now without any real comment They’re going to have to be forced to the table by the government, regulatory agencies, and law makers to come out and say you have a role to play in this process. When Obamacare came out, there was no omnibus. It sat there without any tune-ups to it.

Hypothetically, there could be that 1 amendment that says, “Rare Disease Device Amendment,” but there just hasn’t been anything like of sorts that I’m aware of. I’m unaware of any omnibus that has been added to legislation, and in most cases, we should be refining the law to make it better. We’ve been at this for a decade and we’re trying to create the advocacy. We’re trying to create a voice that is practical. When people read the piece, I don’t think they would say, ‘oh, my mom would disagree with that.’ Most people go, ‘no, that makes perfect sense.’ It’s not always the logic that wins the day, it needs to have a better ground game. I didn’t imagine myself being in a position to run the ground game on reforming the device game for rare diseases. But if I don’t run the ground game with neuroethicists like James Giordano, then what’s going to happen? Somebody has to step up and start to push for this. We’re trying to work with the FDA, and they have been good partners, but that’s only 1 partner to try to tip the system a bit. We need reforms to help. I don’t think the FDA has a lack of empathy, but we need some people a few pay grades higher to start to pay attention.

What is your call to action that hopefully changes the system? How do we begin to get the wheels turning on reforming this outdated process?

The call to action is that we need the lawmakers to help us create a better system that works for everyone, including those with rare diseases. We need to make sure that we make devices available for people with rare diseases, people with common diseases, and rare symptoms of common diseases. We have the technology to modulate circuits, we just need to wake up and realize that this is exciting and can save the healthcare system in the long run. We need to put a system into place and its going to take the whole kitchen table. Right now, that just has the healthcare team and the individual with a rare disease, but we need legislators, insurance companies, industry, industry’s regulatory agencies, the FDA, and some funding agencies to come to the table. These funding agencies need to provide funding for registries to further rare diseases or at least provide incentives for industry leaders.

It’s forward thinking by the Tourette Association of American and their CEO to have supported me for 5+ years to do this. It could end up a big failure on their books, but they’ve taken the risk to try to move this forward. They’re willing to come to the table and fill the gap that needs to be filled. We need a sustainable solution. We need to hold insurance companies accountable so that they don’t have so many barriers that can be avoided, and actually be forced to be a part of the conversation. My impression is that industry wants to come to the table every time we want to talk to them, and they seem eager to solve this problem. It’s frustrating for them to develop these devices, but not have them being used. You don’t have to force industry to the table, you just have to understand that they are still a business and their time needs to be worthwhile.

Insurance companies don’t want to be at the table, so they need to be forced there. The regulatory agencies empathize with us and would like to see lawmakers help create the solution. If we see the picture in the way as a kitchen table, with the discussion centered around the person with a rare disease, we can get it done. Our philosophy at the university I work at is that the person is the sun, and we should revolve around them. If we put that person in the center, we need to get the right team around them to lift them up. People don’t understand the simple fact that we’re in a new era. It’s not just disease treatment approval, we can now modulate circuits in the brain. This is totally different mind-blowing stuff. If you accept that, then you have to accept that we need a new process.

Transcript edited for clarity.