Ensuring equal access to Friedreich's ataxia treatments
Susan Perlman, MD, discusses how practitioners can ensure equitable access to approved treatments for Friedreich’s ataxia and what options are available.
About Susan Perlman, MD
Susan Perlman, MD, is a clinical professor of neurology and the director of the Ataxia Center at the UCLA Medical Center in Los Angeles. Her specialty is ataxias, including Friedreich’s ataxia. She is also the director of clinical trials in UCLA’s Program in Neurogenetics and sits on the Medical and Research Advisory Board at the National Ataxia Foundation.
Transcript
Certainly, the prescribing healthcare provider is going to have to document that the patient has Friedreich’s ataxia. So they’ll have to get a genetic test to confirm that. And they’ll have to make sure that the patient is at least 16 years of age. I’ve had some patients 15 and a half years who have been able to get it. So you want to document this as well. Those are the only two requirements of the FDA on approving this drug.
The company — originally Reata, now Biogen — does have a patient assistance program. So, if for some reason the insurance company is denying coverage or there’s a co-pay that is really untenable, there is patient assistance that is available and can be arranged. There are some income limitations on that. So if you are a wealthy person, you may not qualify for that and the co-pay could be horrendous. It could be $3,000 per month, and there are very few people who could qualify on their own to pay that amount.
So I think part of solving the problem is going to be getting the insurers to be more reasonable and not make up reasons to deny coverage. Some of the reasons they might deny is if somebody’s non-ambulatory. They have pes cavus documented in their medical record.
They have cardiac issues — they may be stable, but they have a history of cardiac issues. And if you go through the list of inclusion exclusion criteria for the MOXIe trial, you get a sense that many of these insurers are going through that list and trying to qualify people to receive a prescription based on entry into a study, which is completely inappropriate.
And, you know, demanding use of the FARs exam scoring, the Friedreich’s ataxia rating scale scoring, which is not done in clinical practice. It’s done for research. It is not typically done in a clinical setting. So, to ask a prescriber who’s unfamiliar with that rating scale to provide evidence that the scale has improved, that the person’s scores are in a certain range, I think is gonna be, again, an unfortunate way to deny coverage.
So I think educating the insurers, potentially if there’s any governmental role that could be gotten in Medicare- and Medicaid-related plans, as well as utilizing patient assistance when it’s available.
David Lynch, MD, PhD, is the director of the Friedreich’s Ataxia Program at Children’s Hospital of Philadelphia.