Michael Okun, MD

"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 it’s going to take the whole kitchen table.”

In an effort to raise awareness, Michael Okun, MD, and James Giordano, PhD, recently published an editorial in 

calling for the reformation of the process for deep brain stimulation and neurologic device approval in rare diseases. Patients with rare diseases often do not benefit from the same process that traditional pipeline drugs receive because they represent a small population that is deemed unattractive to invest money into by private companies or industry leaders, they noted.

If these corporations are not incentivized, they will not come to the table even if the device or treatment could satisfy a great unmet need in the disease treatment landscape. 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 works for the Tourette Association of America. He has been at the forefront of trying to raise more discussions about the outdated system to spark a change for the future.

 to discuss how the process for changing the system must begin, which leaders are necessary to the discussion, and why the patient needs to be the center focus when thinking about approving devices.

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The executive director of the Norman Fixel Institute for Neurological Diseases gave his call-to-action on how to change the approval system for neurologic devices for rare diseases.

Michael Okun, MD: Long-Term Plan for Modifying Neurologic Device Approval System

A recently published viewpoint paper by Michael Okun, MD, and James Giordano, PhD, outlined the flaws within the approval process for deep brain stimulation and neurologic device approval in rare diseases. In efforts to balance the system out and allow these devices to have the same benefit that traditional drug development receives, Okun mentioned the increased use of disease-based registries.

He detailed that expanding this process would allow a university and/or foundation to submit a device master file as well as using the data to serve as a coapplicant for full FDA approval. This would ultimately decrease legal risks to industry and provide incentives to manufacturers, who commonly find investing in these devices to be “unattractive,” according to the paper. The hope is that these registries could satisfy the FDA-imposed conditions of approval contingencies reliant on long-term data.

Okun, executive director of the Norman Fixel Institute for Neurological Diseases, claims that if industry is incentivized to run these registries, there would be more cooperation across the board. He sat down with 

 to provide an in-depth explanation on why these registries could be potential game-changers, and what trickle-down effects they could have for clinicians and patients alike.

Okun M, Giordano J. Reforming the process for deep brain stimulation and neurologic device approval in rare diseases. 

. Published online December 9, 2020. doi: 10.1001/jamaneurol.2020.4232

The executive director of the Norman Fixel Institute for Neurological Diseases explained how disease-based registries play a crucial part in changing the approval system for neurologic devices in rare diseases.

Michael Okun, MD: Importance of Disease-Based Registries

"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.”

A recently published viewpoint paper by Michael Okun, MD, and James Giordano, PhD, called for a reformation of the process for deep brain stimulation and neurologic device approval in rare diseases. Within the paper, the 2 authors discuss the use of humanitarian device exemptions (HDEs) as a regulatory pathway for device availability.

Despite the fact that HDEs might represent a solution to this problem, they can be underused because they require an industry partner to submit data accompanying the exemption. Industry partners often shy away from dipping their feet in these waters because they perceive HDEs as “unattractive” due to costs required to develop and maintain the HDE, limitations of profit, tax exclusivity, lack of incentives, and legal liability.

Without incentive, industry partners tend to focus on larger disease populations, leaving those with rare diseases to not reap the same benefit from the approval process. Okun, executive director, Norman Fixel Institute for Neurological Diseases, spoke to 

 about egging on industry leaders to invest in these HDEs, and how readily available funding may incentive those to invest.

The executive director of the Norman Fixel Institute for Neurological Diseases provided his thoughts on changing the perception of humanitarian device exemptions.

Michael Okun, MD: Push for Increased Humanitarian Device Exemptions

"They need to be pushed to say, ‘yes, you do have that burden.’ I would argue this is not necessarily a violation, but a direct conflict to the pre-existing conditions within the Affordable Care Act, because it appears to me that they’re not offering a treatment for a pre-existing condition.”

Insurance companies play a major role in the care process for patients with neurological disorders, mainly because they have the final say on whether to deny or accept coverage for treatments. Neurological therapies have traditionally been developed to appease the “many,” leaving those with rare diseases unable to reap the same benefits from the system. This can be applied to insurance coverage, which has typically favored larger patient populations instead of those with rare diseases, which are characterized by having less than 200,000 people with the same disorder.

The “right-to-try movement,” which essentially allows patients to attempt a new treatment strategy or procedure while having coverage, has been buried within the system. In a recent viewpoint paper, Michael Okun, MD, executive director, Norman Fixel Institute for Neurological Diseases, explains that insurance companies have some fault in the approval system for neurological devices for patients with rare diseases.

If insurance companies are not going to cover the costs associated with these devices, patients will be forced to make the decision to seek other options, and in some cases, there are no other options. Okun’s paper lays out a multitude of issues that lie within the system, including the unwillingness for these carriers to cover payments from humanitarian device exemptions (HDEs), modifications to the Patient Protection and Affordable Care Acts, and overall change of mindset from industry leaders who may be hesitant to invest money towards these devices.

sat down with Okun to get his thoughts on whether insurance companies should take the brunt of the blame, how the system needs to change, and why multi-level cooperation is necessary for this change to come about.

Okun MS, Giordano J. Reforming the process for deep brain stimulation and neurologic device approval in rare diseases. 

. published online November 9, 2020. doi: 10.1001/jamaneurol.2020.4232

The executive director of the Norman Fixel Institute for Neurological Diseases discussed the role insurance companies play within the system for approving neurological devices for rare diseases.

Michael Okun, MD: Need for Expanded Insurance Coverage for Rare Diseases

"On the industry side, people should be given a legal break. Not a pass, but there should be breaks and incentives for them to make these devices available which can end up being life changing innovations for these various disorders. If we don’t do that, we’re going to be in trouble or just sitting on a lot of technology that can’t actually get out to these people with rare diseases.”

Traditionally, a drug in development will have a normal streamlined path, making its way through multiple phases of study before being submitted to the FDA with the financial help of a pharmaceutical company or industry leader. Neurological devices for patients with rare diseases do not benefit from the same process. Despite showing efficacy in clinical trials, industry partners may remain hesitant to invest their money due to the costs required to develop and maintain these devices, limitations of profit, or legal liability.

The outdated system that benefits traditional drugs for the masses and not neurological devices for rare diseases, was highlighted in a recent paper by Michael Okun, MD, and James Giordano, PhD. In the viewpoint, the 2 authors lay out multiple problems that lie within the outdated system, including the issues with insurance companies supporting costs, humanitarian device exemptions slowly being phased out, and changing the perception about investing for the greater good of the patients who need it and not the financial outcomes that result from it.

Okun, the executive direct of the Normal Fixel Institute for Neurological Diseases, and Adelaide Lackner Professor and chair of neurology at the University of Florida, also serves as the medical director of the Parkinson’s Foundation. In an interview with 

, he explains where the disconnect is for devices for rare diseases, and why they do not benefit from the same system as traditional drugs.

The executive director of the Norman Fixel Institute for Neurological Diseases discussed the issues within the current system that prevent approvals for neurologic devices for rare diseases.

Michael Okun, MD

Articles

Michael Okun, MD: Long-Term Plan for Modifying Neurologic Device Approval System

Michael Okun, MD: Importance of Disease-Based Registries

Michael Okun, MD: Push for Increased Humanitarian Device Exemptions

Michael Okun, MD: Need for Expanded Insurance Coverage for Rare Diseases

Michael Okun, MD: Systemic Issues With Approving Neurological Devices for Rare Diseases

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