Health Topics

Listener Questions

Andrew Schorr:

Okay, let's talk about questions. This one came in from Kelly. I think this is going to be for you Tony. 'We are parents of two children with PKU. One is 15. One is 12. They are both on diet and doing very well. But of course they'd love to be able to eat regular foods. My question is about coverage of the drug. We move frequently with my husband's company, and our insurance changes with each move. We are concerned about one company agreeing to the drug, and then if we move the next company turning us down. I realize there's an assistance program to help families when the insurance companies refuse this treatment, but there's a salary cap for this assistance. With two children and no assistance, this treatment is not available to our children and then they'd be devastated not able to receive the drug. What options are available for children with these circumstances?' So again, people move, change jobs, how do you help through that Tony?

Mr. Wicks:

Okay, so there are a number of steps that are going to take place. First of all, it is very important that if you currently are on Kuvan, and you are preparing to move, definitely contact BPPS and let us know because we will assist you through that process of changing insurances. In particular, the thing that we're going to want to do right away is contact your new insurance to figure out what the benefits are going to look like, whether or not they're going to be different from the current coverage, and whether or not it's going to be different from the current coverage that you have. One of the key benefits that you do have as you're transitioning from one insurance to another, if you're already on therapy and you are seeing a clinical response is; the strongest argument that you have for an insurance company covering a drug is the fact that the patient is actually responding to therapy. So it just makes it that much easier sell when we go to an insurance company saying that this patient is actually a good candidate for this therapy, it's working for them, and were able to show an argument of medical necessity.

So, that is the first step that were going to do is we're going to try to figure out what the benefits for that patient will look like on the new insurance, and we're going to make sure we have a very strong clinical argument. If they deny it then what were going to do is we're going to appeal that decision. There may be some involvement necessary from the patient as well as from the physician and dietitian to assist with that, and we will continue to appeal that decision as high as possible. In some cases, you can actually appeal the decisions to outside parties such as state insurance boards or even to employers to try to get coverage.

Typically, in the event that if we run into a situation where we can't get that patient covered, that patient will be rendered uninsured, and at that point they will qualify. If they meet the financial criteria, they will then qualify for free drug via the BioMarin Patient Assistance Program.

Andrew Schorr:

Okay. Here's a question that's for you Maria from Sandra and Melbourne, Florida. She says, 'I've been turned down for Social Security disability and Medicaid. My car is always breaking down, and it's very hard to work without my supplement. What should I do?'

Ms. Hardin:

One thing I will say is don't give up when you are applying for Social Security disability. It's pretty automatic that you're going to get denied the first go round, and we tell people they need to go back and re-appeal, and as they go up the ladder and they re-appeal, the timeframe get shorter, and your chances of getting approved are much better. We deal with this throughout the rare disease community. Most people are chronically ill that have rare diseases, but I certainly would tell her that if she can show that she doesn't qualify for Medicaid or disability that she definitely needs to get in touch with BPPS. We are in constant communication. If they are able to find insurance for her and she cannot afford the premium, she certainly could become a candidate for the NORD program.

Andrew Schorr:

Okay, thank you for that. Now here's another ripple in all this. Jim writes in, and he said, 'How to you deal with self-insured companies for Kuvan coverage, especially with diet and formula being the most prescribed regimen up until this new era of Kuvan. When the insurance administrators see the cost of Kuvan, they will tell the employee that the diet is the best method and will not cover Kuvan.' Tony, you may have encountered that. Help us understand what these self-insured program.

Mr. Wicks:

Well, typically, the self insured fund means that it's actually funded by, it's typically funded by an employer for example, and in those kinds of situations even whether it's a self-insured plan, or even if it's a larger plan, you're going to see these issues play out regardless and especially when it comes to diet and formula versus prescription drug. A really important thing to remember is that those two things are covered under very different benefits, and really it's not up to the insurance company to decide the appropriate treatment regimen. It's really a decision that's made by the provider as well as the patient. So in those situations where an insurance company is saying we are not going to cover it because we think that food and formula is an appropriate regimen for this patient, again, we're going to have to appeal that decision and we're going to probably have to use an argument of why it is medically necessary for this patient be on Kuvan, and that again requires some assistance from your physician as well as dietitian and from you as far as building that argument with the insurance company.

Andrew Schorr:

Now PKU is a rare condition, and the first time you go at the insurance company, they may say, whoever you get may say, 'I don't know anything about it,' or maybe they know about the medical food or formula, but as we just heard in the last question. They don't know anything really about the medication or what a difference it can make. So that's when appeals come in. So tell us about how you help navigate the appeals process, and what success you've had with that.

Mr. Wicks:

The way that the appeals process works with Kuvan is actually a little bit unique. The first thing that we actually want to do is make sure that someone is a good candidate for Kuvan. What we do is once we receive at a denial from the insurance that patient will then go on a 30-day trial of Kuvan, and the reason for that is because we want to give the patient and the provider an opportunity to make sure that that person is a good candidate for therapy. So they go on the drug for 30 days. At the end of that 30-day period, they actually measure Phe levels to see if there is a change in Phe levels and really to just gauge whether or not this patient is a good candidate. At that point, they sent that information in to us, and if they want to go forward with the process of appealing, we will then commence that process.

So, what really helps us in that situation is now not only are we drafting an argument about why this patient should be on therapy, but now we've got clinical evidence that this treatment works for this patient. So we then proceed with drafting a letter coming up with our argument for why the patient should be approved for therapy. In some cases we're going to provide some additional educational information about Kuvan and various treatments for PKU just so we can educate the insurance company a little more, and what you'll see is that typically as you go through the appeal process which each level of appeal it's going to be a slightly more clinical more technical person who's going to be reviewing that case, so it can either be a straightforward process, or it can be a pretty long drawn out process, but the key to going through the appeals process is really persistence, staying on top of them, and making sure that you're coming up with a thorough argument, which is what we're able to really do when we engage the physician, as well as the patient in that process.

Andrew Schorr:

AnneMarie, you're a New Yorker. New Yorkers can be pretty feisty, and you run a support group there. What is say to people as they navigate all this? One is I want to get the right treatment for myself or loved one; oh my god, I want to get this financial support of its available, or certainly get what is due from any insurance that I have or my employer does if I have a job. So what advice would you give people?

AnneMarie:

Well, a lot of people with self-insured programs are usually somehow involved with the union, and a big help I know with us was getting in touch with the union rep because they often know somebody at the insurance company that might be higher up that can help you. I'm talking about as far as medical food and formula, they often know somebody that could get you in touch with the right people, because I have to say PKU awareness is a major thing. Nobody knows what it is, like you said. You call, you get some kind of representative from the insurance company, and they have no idea what PKU is. They tell you it's not covered, all kinds of things.

Andrew Schorr:

They give you the quick answer.

AnneMarie:

My advice is to get in touch with somebody from a union or go to a medical management program within the insurance company.

Andrew Schorr:

Right, of course, what's neat is Tony is that you've got people there at BPPS that go to bat for you, and so this can otherwise be very stressful. I think it's a wonderful service. I want to give that number again, 1-877-MYKUVAN, (1-877-695-8226).

Here's a question we got in. It's probably best for you Maria. It's from Laura Duncan who's actually a nutritionist. She has all sorts of letters after her name that she sent in, and she writes in, 'There are some insurance companies that have $1 million cap on reimbursements. We are concerned that some of our patients with PKU may reach this cap when using Kuvan over an extended period of time, especially if they have some other experience which results in unintended medical treatment. What recommendations do you offer?

Ms. Hardin:

Okay, well first of all, in the calls that we have been taking in talking with the patients, there is a misconception out there that the cap on the family plan is based on all of the family. It's each individual. In other words, the child with PKU or the family member with PKU would have to reach that million dollar mark. It wouldn't be an accumulation of other coverage for other members of the family. So we've been explaining that to people because it is something. This million dollar mark, I can remember 15 years ago, if you had a cap of $1 million, you would never reach it in your lifetime, but with all of the new drugs that are coming out now, we are finding that $1 million is nothing. I would really like for that to get out into the PKU community, and I think Tony would agree with me that this is something that is on people's minds, and when they start going on therapy this is one of the big issues that we hear when we're talking with the parents.

Andrew Schorr:

Okay, thank you for that. Here's another question we got in from Mary in Sullivan, Indiana, and she writes in, 'I have a five-year-old son with PKU and I'm wondering about insurance coverage for food. Our insurance currently covers formula that 80%, but we have not explored the option of food coverage yet. Our insurance plan is an ERISA. Are there different steps to follow with these, and what are those steps to finding out if insurance will cover any of the food costs?' Who wants to start with that one?

Ms. Hardin:

I think we need to let our listeners know what ERISA stands for. It's the Employee Retirement Income Security Act, and there's a website that people who are involved in ERISA can go to, and if you'll just wait a minute I'll pull it up here. It's www.DOL.gov/EBSA, (DOL stands for Department of Labor) and when you go on there, there's an article that says "Ten Top Ways to Make Your Health Benefits Work for You" if you are under ERISA, and under item number four. I was really intrigued about this, and they tell you that your plan summary that you get provides a wealth of information that you should read. I know when I get these I never read them, I just tuck them in my file cabinet, and at the end of it, but it's a wealth of information. When you are involved with such a serious situation like this wondering if you're going to have coverage and how much are going to have, and what it's going to cost you a pocket. Tony, I don't know if you want to add any more.

Andrew Schorr:

Tony, anything you want to add?

Mr. Wicks:

I think you pretty much covered it. I mean, I think the other thing that you can definitely do, and I'm sure there probably is a number on that website, but any situation like this, in which you're trying to get an idea of what your coverage looks like for food and formula, the first thing I would do is actually call the number that's normally provided where you can get information about your benefits, simply because they can provide you a little more detail typically regarding what your benefits are going to look like for food and formula on that particular plan.

Andrew Schorr:

Okay, I believe we have a question on the phone. I believe we have Shawna calling in from California. Shawna, are you there?

Caller:

Yes, I am.

Andrew Schorr:

Hi Shawna. So is it you or a loved one who has PKU?

Caller:

I have two boys, 10 and 11, with it.

Andrew Schorr:

Wow, and we just had, you heard there was a question from somebody with two children, 15 and 12, okay, well what's your question?

Caller:

My question is, with the Kuvan if they go on it for a period of time, and then they have to get off of it for some reason, is that going to affect them medically if they happen to be on it for a certain amount of time, and then suddenly having to stop?

Andrew Schorr:

Well, that's a medical question maybe we're not great at answering today, because we had it in our last program, you know all the Ph.D.'s and super MDs, and we're going to do that again, but let's turn that around a little bit if we could. If for any reason from Tony's point of view, because he deals with supporting people, if somebody is on the drug and then for whatever reason between them and their provider they stop, and actually Corinna who we had in the last show, her son did stop for a little while, does not create any problems? You work so hard to get it going and get it paid for Tony, and if for whatever reason there's sort of a hiatus, is it a problem starting again, or how does that work?

Mr. Wicks:

Generally the only risky one is, depending on the amount of time that the patient goes off therapy what could happen is, I don't know, their insurance might change or if they're coming up towards a time where they might be due for a recertification since most of the insurances will cover it for a certain period of time and then set a date at which they reevaluate that coverage. If the patient is off therapy during that period it could just very well be that that patience benefits have changed. Typically, if somebody is going to go off therapy for a while, just notified BPPS, and we'll actually set up a task to follow up at a certain period of time to make sure that none of that information has changed, and if it has then will evaluate what kind of impact it may have.

Andrew Schorr:

Okay Shawna, I hope that helps. We'll get to the clinical issues maybe again in our next program, and hopefully we'll do others, and also listen again to the earlier program we did just earlier in the month with the two medical experts too. All the best to you.

Here's a question, we just got in. We just had Shawna, and now we have Shayna, and Shayna writes in, and I don't know AnneMarie whether you can weigh in on this one, but she's talking about her kid in school. Does the school have to pay for special lunches that are offered to students with PKU by the special companies, or is it the responsibility of the parents? She said she had heard that the school should pay since it falls under the Americans with Disabilities Act, but she's also heard that it's the parent's responsibility. Which is true? AnneMarie, you know?

AnneMarie:

I think it varies, but there is some kind of federal law. I never had to deal with it with Devon, but I know other parents that have, and I do believe the school is supposed to provide the lunch. I know Cambrooke's Green Light program, and they have a lunch program also that deals directly with the school, so she might want to call Cambrooke Foods on that, and they might be able to help.

Andrew Schorr:

Maria, do you have any information on that?

Ms. Hardin:

No, but it's a good topic to look into since so many of the patients of school age.

Andrew Schorr:

You know what, we're going to cover that. This is a wonderful opportunity for me because we have another program that's going to come up on June 19th. Take a look on our website in the special edition section of www.patientpower.info, and you'll see June 19th Living with PKU, Patient Approaches to the Management of PKU. So we're going to give family to family information, and some other experts, and so will deal with some of those school issues as well too, so lots there.

Okay, let's continue our discussion. We've got another question for you. This is from Erica in Fall River, Massachusetts. I imagine, probably for you Maria. 'I've been off diet for 32 years, and I started Kuvan about a month ago. Kuvan is expensive for me, and I understand why. My insurance makes you pay a co-pay of $50, which I can't afford. Will Kuvan come down in price?' You may not know that one, 'But in Massachusetts medical food and formula is no charge to you. Insurance covers it all for PKU. Any chance this will be the case for Kuvan? I don't want to have to stop taking Kuvan, because I can't afford it, and what if you change medical insurance companies or lose health coverage or get coverage by the state?'

We'll get to that with Tony in a minute. But what about the question of the co-pay? Let's start with you Maria on that. Help us understand that.

Ms. Hardin:

My first question would be, has she applied to the BPPS program, and she would be referred to NORD, and we would certainly look at her case and possibly relieve her of having to pay that $50 a month, which obviously is hard for her to do. So that would be my first suggestion.

Andrew Schorr:

Now, it's Massachusetts, especially a good place to live related to some of these benefits that the state provides?

Ms. Hardin:

Some people say so. You know, if it's one of those states that covers food and formula at no charge, that's a blessing, because there are, as I say, there are 13 states that don't have any kind of assistance whatsoever.

Andrew Schorr:

Right, it's all over the map. Tony we talked about, where you may be changing insurance companies or you lose your health coverage or you lose your job, or you're in a different state now, but again, it sounds like you are there to help them navigate that.

Mr. Wicks:

That's correct, and also, you know, another question came up about moving over to state programs. We have definitely received approvals from Medicaid in numerous states, and whether it's a state program, versus a commercial plan, we are going to go through the exact same kind of process to try to make sure that a person is able to continue on therapy, and again if we run into a situation where there's no longer coverage, they will be evaluated for patients assistance.

Andrew Schorr:

If someone calls there and then the NORD program comes into play, then you've helped make that connection. On your end, Maria, is there someone like at the BPPS who helps them and sort of takes their case, if you will? How does that work?

Ms. Hardin:

Yes. I believe some of the patients had spoken to Evelyn, and then there is Jane Kane, who is another contact there, and I am certainly available to talk, if they can't reach either of them. As I say, we run 40 programs, and we have a pretty seasoned, well-trained staff. It's just that sometimes you might not get a live voice at that moment, but we do have a policy that we return the call as soon as possible.

Andrew Schorr:

Okay, that's so neat, and of course we mentioned about BPPS as a starting point if Kuvan is part of what therapy is right for you or a loved one, or there is that trial going on as well.

So Tony. When can they call, and when do you call them? So how does that work, and how quick, do you get a live person right away? If I were to call now, if someone in my family were diagnosed with PKU, the doctor writes out a prescription and says we want to try Kuvan, then how quickly things move along?

Mr. Wicks:

So you can call us at any time. There are live people on the phones from 6 a.m. to 4 p.m. Pacific Time, and once you call in you will be triaged to your appropriate case manager, who can assist you. As far as initiating the entire process, once the prescription is received, we will actually be reaching out to you. So we will enter all the information into our system, and your case manager will call you and introduce themselves and will give you an overview of what the process entails. For some people, like I said, it can be pretty quick, and in other cases there is a lot of red tape that we may have to go through, but in all those situations there is a case manager that you'll be working with who will guide you through that process.

In regards to Erica's question as far as if, for example, you have a situation where you think you're co-pay is a little bit too high, again, you can reach out to 877-MYKUVAN, and we will refer them over to NORD for assistance.

Andrew Schorr:

Maria, you've been at this a long time. Tony has too, but I know you've been at this many, many years. It sounds like at least in the case of PKU now it's coming together, that there is a really pretty robust support programs that have shaped up for people. How would you characterize it?

Ms. Hardin:

All I definitely would say that it is robust. Again, this is a new kink in the out-of-pocket costs to patients having to pay high co-pays tiered, drugs that are on specialty tiers and the like, it's very disconcerting, so I would say that we are, the wonderful thing is that we are dealing with a company that runs the whole spectrum of the PKU needs, and we're very impressed with that. That's been a wonderful thing to see, and we use this as a model in many of our other programs when companies come to us and they want to establish something for a particular disease state.

Andrew Schorr:

Well, I'm really, on the outside I'm a cancer survivor, but observing this, I think there's tremendous support. Tony Wicks, I want to compliment you and your organization and mention again, you've said the phone number many times, but I think people would be foolish not call. I think it's worth the call. 1-877-MYKUVAN (1-877-695-8826), and Maria Hardin with NORD, thank you for all you do, and then also these programs that you're announcing as well for medical food and formula, that just really rounds everything out, so thank you so much.

Now I want to give the last word to somebody who lives with this every day as she looks at Devon, who's a pretty active kid now, isn't he AnneMarie?

AnneMarie:

Yes, Devon is active.

Andrew Schorr:

Okay, yeah, she said that like an understatement, okay. Now he's going to be a teenager, wait for that, but the blessing is that you are able to keep his Phe levels under control. So I guess nobody should feel alone. You have your support group in Suffolk County, but nationwide, people should feel like we're all in it together and that there is support.

AnneMarie:

There is support out there, and I have to say that BPPS, they've been wonderful with us as far as the Kuvan goes, and they were very helpful, and they kept all their promises. They got Devon covered, and that was the most important thing. Another important thing that I wanted to say was being on the Kuvan, he doesn't need as much formula, which is also a good thing that the insurance can look at too because he doesn't need as much formula is needed before being on the Kuvan.

Andrew Schorr:

Well AnneMarie, you've given me the opening to talk about our next show that is going to be on all these issues related to living with PKU, and there will be a lot of information sharing going on there. I want to wish you all the best with Devon, and may you dance at his wedding and have grandchildren, and he's going to be keeping those Phe levels controlled for a lifetime. All the best to you, and thank you for joining us. Tony Wicks, thank you with BPPS, and Maria Hardin, thank you with NORD.

I want to mention that our next program, the third in our series, is June 19th at 8 p.m. Eastern, 5 p.m. Pacific, Living with PKU, Patient Approaches to the Management of PKU, and the information is all on our website, the special edition section of www.patientpower.info. Thank you so much. Send me an e-mail just at PKU@patientpower.info, let us know how we did, and remember knowledge can be the best medicine of all. Broadcasting live from Seattle, I'm Andrew Schorr. Thanks for joining us.

Please remember the opinions expressed on Patient Power are not necessarily the views of BioMarin, our partners, or Patient Power. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you.