Access to Care: Insurance and Reimbursement Support Programs for New PKU Treatments

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Medication for PKU is costly, but there are resources available to help you work with your insurer as well as assistance programs to supplement the cost. In this second program of our “Living with PKU” Special Edition Series, host Andrew Schorr is joined by support experts from two major organizations that assist families touched by PKU in gaining access to care and help with reimbursement and insurance needs. Experts like Maria Hardin, Vice President of Patient Services for the National Organization for Rare Disorders (NORD) and Tony Wicks, a BioMarin Patient and Physician Support program (BPPS) expert, explain their supportive roles and answer listener questions. You also hear from Belkys Prado, a metabolic dietitian discussing her experiences in helping patients with insurance and support programs.

The program begins with AnneMarie DeGeorgio, mother of a child with PKU and President of the Inherited Metabolic Disorders Support Group of Suffolk County. AnneMarie tells her story of being faced with insurance woes and how, with the help of BPPS, her son now gets the treatment needed. Learn how AnneMarie stepped up to advocate for her child and how she’s helping others with PKU in her area.

Tony Wicks and Maria Hardin provide an in-depth look at the role of each of their organizations. The BioMarin Patient and Physician Support program works with providers and patients to make sure you are getting the most out of your insurance coverage. To quote Mr. Wicks “Our job is really to be the single point of contact for that family and to really manage all the different activities that need to happen in order to get them on therapy.” The National Organization for Rare Disorders works closely with BPPS on many cases and helps to arrange for payment of health insurance premiums, deductibles, co-payments to pay for medication, among others, and is funded by the organization through donations from corporate donations, grants from concerned companies, foundation grants, public contributions, and membership dues. If you’re searching for information on how to obtain financial assistance, medical care or simply have questions about what resources are available to PKU patients, this program can provide all the answers you need and, if not, certainly point you in the right direction.

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Andrew Schorr:

Hello and thank you for joining us for the second of our live webcasts on PKU. We had one as many of you know on May 8th, and we discussed the new landscape of treatments. Today we're going to discuss access to care, insurance, and reimbursement support programs for new PKU treatments, and we're going to have an array of guests who will help you understand how you get access to the care you or a loved one need and deserve.

Now remember that on June 19 we'll come back with the third in our series of programs, and that will be living with PKU, patient approaches to the management of PKU. So again then we will discuss more treatment issues, but this is really how do you get the medicine that is right for you.

Now we're in a new era, and as you heard in the last program about PKU, where there is an FDA approved medication Kuvan. Well, what's your insurance these days? Do you have a job? What are the co-pays? What's the coinsurance? What's the cap? All these come into play, and so you're going to hear from people who can give you answers, numbers to call, people to help you on your case, and really make you feel a valued part of the PKU community. Thanks again to the unrestricted grant from BioMarin to help make this series possible. We really appreciate them, and the whole PKU community coming together. I've been learning about it along the way, and I just really feel a tremendous amount of warmth and support as people seek information and try to help each other as we develop newer and better therapies to help people go on with hopefully a long and high quality of life.

First I'd like to introduce you to someone who's lived it, because she has a nine-year-old son, Devon, who has PKU, and that's AnneMarie DiGeorgio. AnneMarie lives out on long island, about 45 miles east of New York City in a place called Center Reach, and then her husband is a New York City policeman and goes into the city, but she's out there with Devon, who is doing really well. But keeping his Phe levels just right has been a challenge, right AnneMarie?


Yes, it's been a challenge.

Andrew Schorr:

So there you are now. You are quite the mom, because not only with your only child, Devon, you're working on all of that, but also you're I know, President of the Inherited Metabolic Disorder Support Group of Suffolk County, New York, and you had a gathering just a week or so ago. Didn't you?


Yes we did at Piccolos Restaurant in Bellmore New York. We had a lot of fun there.

Andrew Schorr:

Yeah, that's great bringing people together. Will we are all together tonight. Tell us a little bit about Devon and about how it was difficult before Kuvan to get those Phe levels right and how that was a problem in his life and yours as well.


Well, doesn't be a variant is in between being a classical and a hyper-Phe, which means he's not as restricted as a classical PKU person would be, but he does need to be on diet and does need formula. So because he has those levels that jump around a lot, there are times where he can eat a lot of Phe, and his level would be fine, then you could go the next week and he'd eat the same amount of Phe, and his levels would jump up. So with the Kuvan, that's balanced out his levels.

Andrew Schorr:

Now, he ended up being in the clinical trial, and so you found out as they were still learning about Kuvan that it was helpful for him.



Andrew Schorr:

And so then the trial ends, and then the drug is approved, and so then obviously there's a cost to the drug. How if you worked that out? We are going to hear all the latest related to the BPPS program in a minute, and we are going to explain what that means, the support program for people who want access to Kuvan and may have insurance issues or if they need some help, but how did that work out for you? How did they help you, because I know that your insurance did have some limitations.


Well with our insurance being self-insured our prescription plan was through the PBA, which is the Police Benevolent Association, and what happened was they were going to cover the drug, but unfortunately we had caps, and the cost of Kuvan exceeded the cap. So the drug would have been out of reach in our case. So what happened was BioMarin stepped in. They put us in the BPPS program, and they've been covering the medicine for Devon so far.

I know a lot of insurance companies are covering it, but some insurance companies have caps where it's really an issue, and it's just unaffordable for the average person.

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