Clinical Trials, Commercial Treatment, and Insurance Coverage
As the ET trial was reaching its final stages, we talked to potential patients about their interest in participating in the trial. Some were ambivalent, in part because they thought it might be preferable to wait until the treatment was approved by the FDA. They assumed that it was a formality that would not take long, and that FDA approval would further validate focused ultrasound as a treatment for ET and provide an additional measure of safety. However, they may not have adequately considered the financial implications of waiting. The time between FDA approval and widespread insurance reimbursement can be lengthy. Although Medicare is now covering focused ultrasound treatment for ET in 16 states, many patients who decided against participating in the trial are still waiting for insurance to cover the procedure in their region.
For most clinical trials, the cost of the care is paid for by the trial sponsor.* After the FDA approved the focused ultrasound procedure for ET, patients had the option to be treated on a “cash pay” basis at an out-of-pocket cost of approximately $40,000.
After FDA approval, it took almost two years for insurance to begin covering treatment costs. During that interim, some patients were able to pay for treatment, but most put their names on waiting lists to be treated after insurance payment was available. One treatment site had more than 100 patients on its waiting list.
Medicare has a two-step approval process – the first step is the “global” Medicare approval and fee determination. The second step requires each Medicare region to also approve the care. An additional challenge that patients encounter is the fact that commercial insurance providers typically cover a procedure only after it has received Medicare approval. The Medicare regions are often subcontracted, and there is no defined time limit for their response. Each region has its own review process that can exceed the requirements of the FDA and global Medicare, if the region so decides.
A Timeline for Essential Tremor
September 2014: Last enrollment for ET treatment in a clinical trial
July 2016: FDA approval
November 2016: Global Medicare approval
April 1, 2018: First Medicare region covering care
July 2, 2018: Additional Medicare regions covering care
Unknown: Complete Medicare regions coverage of care
For potential patients who did not enroll in a clinical trial and were not able to pay out-of-pocket, the only option for care was to be put on a waiting list. Even as some Medicare regions have begun covering focused ultrasound treatment costs, patients living outside an approved region either have to travel for care or remain on the waiting list until their region decides to pay for the care.
Questions to Ask When Considering Participating in a Parkinson’s Trial
The decision to join a clinical trial is complicated and should be made only after a discussion with your physician and loved ones. The questions below are those that I often hear from patients.
Were there any changes in care between the pivotal clinical trial for essential tremor and the commercial treatment that the FDA approved?
No, the commercial care for ET is unchanged from the care provided in the pivotal study.
Will the path from pivotal trial to commercial approval and reimbursement for Parkinson’s follow the same arc as it did for ET? Could the timing be shorter now that focused ultrasound is already approved for ET?
There is no way to know for certain how long it will take for the Parkinson’s treatment to move from the pivotal trial to Medicare reimbursement. Each study follows its own protocol and is evaluated on its own results.
If I am enrolled in the clinical trial, will I definitely receive the procedure?
Yes, although the study does have a blinded sham arm – or group. If you are randomly selected for that arm, you will have a sham procedure (which simulates the real procedure, but no energy is applied). After three months, the investigators will “un-blind” the study and tell you if you received a sham treatment. If you were part of the sham group, you will have the opportunity to have the procedure. For every one patient in the sham group, three patients will be in the treatment group. There is no added cost for sham patients to receive treatment, other than incidental expenses, after they’ve been notified they were part of the sham arm.
Are there any advantages to participating in the clinical trial that may not be present in commercial care?
Clinical trials have numerous follow-up requirements that may not be duplicated in commercial care. The information and attention patients receive in these follow-up visits may be of some value. Another benefit is the personal satisfaction of knowing that your willingness to participate in a clinical trial not only helps you – your participation also helps make this procedure available for other patients in the future.
There are a lot of considerations to weigh prior to joining a clinical trial. Many patients ask their physician, “If you were in my situation, what would you do?” It is good to ask questions and to seek information. The cost for finding out more about the clinical trial is primarily an investment of your time, and it is well worth it.
The better informed you are, the more comfortable you will be with your decision. A great place to start your research is at www.clinicaltrials.gov, specifically here if you are interested in focused ultrasound therapy for Parkinson’s: Clinical Trial #NCT03319485. If you think you meet the requirements, you can call the nearest study site to learn more. There is no cost for inquiring, and there is no pressure to join the trial.
Who knows? You may decide that the potential for finding relief from your Parkinson’s symptoms right away – while minimizing financial costs and the wait time for focused ultrasound treatment to become commercially available – is enough incentive to prompt you to join the trial. The added satisfaction of helping future patients by participating now would feel pretty good as well.
National Clinical Trial #03319485
Focused Ultrasound Foundation Parkinson’s disease pivotal trial landing page
First Patient Treated in Pivotal Trial
Michael J. Fox Foundation Trial Finder
Tim Meakem, MD, is the chief medical officer of the Focused Ultrasound Foundation.
*The Focused Ultrasound Foundation advises patients to confirm potential out-of-pocket expenses with the clinical trial administrator. There may be incidental expenses, such as travel, lodging, etc.