The formula shortage crisis has been devastating for patients with rare metabolic conditions, like PKU, who depend on specialty formulas daily. We hear from patients and families impacted by the shortage, including Lillian Isabella and Sarah Chamberlin, executive director of National PKU News.
But patients have struggled to access and afford these formulas for years, which come with high price tags and aren’t always covered under insurance. A bill in Congress, the Medical Nutrition Equity Act, aims to change that.
And ahead of Memorial Day weekend, Terry and Dr. Bob discuss three health care bills in Congress drafted to improve health care access for veterans.
Dr. Robert Goldberg, “Dr. Bob,” Co-Founder and Vice President of the Center for Medicine in the Public Interest
Kate Pecora, Field Correspondent
Sarah Chamberlin, Executive Director, National PKU News
Lillian Isabella, Patient Advocate
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Sarah Chamberlain (6s):
There are definitely people with rare disorders who are experiencing shortages with formula, and not having access to formula, and it just not being there. It's a crisis for everyone, whether or not you have a metabolic condition, but for people with metabolic conditions, some of these formulas are lifesaving.
Terry Wilcox (24s):
The nations formula crisis is having a devastating impact on some in the chronic disease community. Those stories, and more, are on this week's Patients Rising Podcast. Hello everyone. I'm Terry Wilcox of Patients Rising, an organization that fights for the rights of the one-hundred, thirty-three million Americans living with chronic disease. Every week we break-down the latest policy discussions that impact those with chronic illness. Today, I'm here with my cohost, the guy who is debating whether to accept that starring role in the Jersey Shore reboot, he's Dr. Bob Goldberg, Co-Founder of the Center for Medicine in the Public Interest. I have a confession to make Bob.
Dr. Bob Goldberg (1m 3s):
Terry Wilcox (1m 4s):
I have never ever seen a Jersey Shore, a Housewives, Hollywood, Orange County or Kardashian show. I've never wasted one minute of my life on any of that, but if you were on The Jersey Shore, I would definitely watch it just to see you in the black t-shirt.
Dr. Bob Goldberg (1m 25s):
Thank you. Well, you know, I was working on my spray tan, I got my tank tops tailored, ready to film the first episode, Terry, and read my contract. I needed to get tattoos and piercings, and I had to stop keeping kosher to fit-in, so I said, "That's it!" I drew the line on those three things and I'll let the other guys have the spotlight. Really, the real reason, Terry, is it would take time away from the podcast. I really enjoy meeting you here, virtually, each week to discuss the latest headlines, so that our wonderful community of patients and caregivers know what's going on and know how to advocate for themselves.
Dr. Bob Goldberg (2m 8s):
Of course, like you said, all week until today, now we're talking about monkeypox, it's the baby formula shortage. What isn't getting a lot of press, though, is the impact that the formula crisis has had on those with chronic illness. Much of the baby formula shortage is in those areas and it's a desperate situation.
Terry Wilcox (2m 31s):
I know Bob, at Patients Rising, we've been talking about this with our community, and we've been very worried about the impact. Before we get into our main story of the formula crisis, you know Bob, it is Memorial Day weekend, and our flags are already out to honor those who have fought for, and laid down their lives, for this country.
Dr. Bob Goldberg (2m 53s):
Right, so we thought we'd talk about something that's very near-and-dear to our hearts and that is making sure those who chose to serve, and fight for our country, get the healthcare that they need.
Terry Wilcox (3m 6s):
Yes, this is especially close to our hearts, Bob. Your dad and my dad, are and were, using health services from the Department of Veteran Affairs or the VA. There are several pieces of legislation sitting in Congress that should be swiftly considered and we're going to go through those in honor of Memorial Day weekend. The first is H.R.7500 or Fiscal Year 2022 Veterans Affairs Major Medical Facility Authorization Act. This would authorize major medical facility projects for the Veterans Affairs Department, including 3.4 billion to restore, or build, twelve VA medical facilities.
Terry Wilcox (3m 47s):
This bill has passed the House. Number two, H.R.5754. This is the Patient Advocate Tracker Act. This would require the VA's Office of Patient Advocacy, to create an IT system, for veterans to file and track complaints related to VA health care services and facilities. This bill has passed the House. Next, we have Senate Bill S.2102, the Dr. Kate Hendricks Thomas SERVICE Act. Now, this would expand eligibility of VA mammogram screenings to veterans who were exposed to burn pits during their service.
Terry Wilcox (4m 26s):
This bill has passed both the House and the Senate. We will link to those bills in the show notes. If you want to write your legislator on any of those bills, to put in your two-cents, we would love for you to do that. These are very important bills for the veterans community. Now, our main story of the week is the formula crisis and it has really put some families, with rare disease, into a desperate scramble. This week we spoke with Sarah Chamberlain, she's the Executive Director of National PKU News, an organization that supports the people, family, and clinicians, who deal with PKU, or phenylketonuria.
Terry Wilcox (5m 9s):
She's also a mom of two girls. One of whom was born in 2013 and diagnosed with PKU. She's well familiar with what the formula shortage means to the PKU community.
Dr. Bob Goldberg (5m 21s):
Terry, we should probably take two minutes to explain what phenylketonuria actually is. It's a condition where your body can't break down an amino acid, which is also kind of difficult to pronounce. Without the treatment, this amino acid builds-up in the blood, causes health problems, behavioral problems, and so on. It's an inherited condition. The reason you need a special formula is, you need a formula that allows you to metabolize the amino acids, which are related to the disease itself.
Dr. Bob Goldberg (6m 1s):
I thought that little science lesson might be of use to people.
Terry Wilcox (6m 6s):
No, I think that's great. So, we asked her to share about a PKU diagnosis and what that means for medical nutritional needs.
Sarah Chamberlain (6m 15s):
What that means is that the core of treatment is a medical nutrition diet, and that is a formula, an amino acid formula usually, then low protein medical foods. My daughter gets about five grams of protein from food per day. That's about two slices of Wonder bread or one slice of American cheese. Not a lot of protein when you look at it and she drinks about 24 ounces of formula per day. That's about 15 cans of your standard-size formula can per month.
Terry Wilcox (6m 51s):
Now, Bob, this is just one disease. What do we know about medical nutritional needs? How many other diseases need medical nutrition?
Dr. Bob Goldberg (6m 59s):
If you go to any number of the websites, that manufactures, of course we'll get to that in a minute, there are several dozen. There are five or six major categories of specialty formula that are customized, and formulated to specific stages of birth, for specific conditions. Premature birth is a specialty formula. Under nutrition failure to thrive is a specialty formula. The inborn errors of metabolism, of which PKU is, is one, it's another thing. Food allergies, and both my grandchildren have that. They were breastfed and there were times when "your's truly"
Dr. Bob Goldberg (7m 42s):
was babysitting and they had to use the special formula. There's protein intolerance and the soy protein intolerance. By the way, the number of formulations customized to optimize the nutrition, to promote healthy development of infants, is growing.
Terry Wilcox (8m 0s):
So, this family can't live without this formula. Right now, we're thinking about, Oh my God, the shelves at Walmart for the general formula, or the shelves at Target, or wherever you shop for formula-Amazon, even. It's all tough. Naturally you know how I'm going to segue this. It's kind of how we think around here. We want to know how much her family is typically spending, not just on formula, but on managing the illness, which is something we don't often talk about or even consider.
Sarah Chamberlain (8m 36s):
My daughter's formula-again, she's eight years old, so she's pretty small, her formula is billed-out to insurance at $1,674.16 per month. On top of that, you have medical foods costs, of the low-protein medical foods. A pound of low-protein pasta is $9. A loaf of low-protein bread can be $11 to $13. All the staples are much more expensive, and if you want to get things like the equivalent of a hot pocket, or a low-protein product, those are going to be about five times the cost of regular store-bought products that are higher in protein.
Sarah Chamberlain (9m 20s):
Then we have blood tests. Starting at birth, you have weekly blood tests and that's really to start to ascertain what that enzyme activity is. Talking about the severity of the case, you basically feed protein, and if your blood levels go up, you need to reduce the amount of protein or phenylalanine in your diet. For the first year, or so, those are weekly blood tests. I think I pay about $180 a week, and then I pay about $30 after insurance covers that. Some places, insurance won't cover them.
Dr. Bob Goldberg (9m 54s):
There's a couple of things. First of all, once you have one of these metabolic conditions, they need to be managed throughout your lifespan. The formula is a part, a very critical part of the life cycle, if you will, of nutrition and testing that you need to stay healthy. Also, there are treatment coverage, state-by-state, which may apply to someone with PKU, but not another disorder. On top of that, in some states, they will cover the testing for inborn and metabolisms, but the insurance won't cover the treatment to improve the sustainability of the little baby.
Terry Wilcox (10m 39s):
That's so crazy. You and I think of it as it's always a lot of people, because this is the population that we operate in, and that we're advocating for, right? It's relatively still a small part of the population. You would think that they could figure-out how to, you know...
Dr. Bob Goldberg (10m 60s):
No, it's just the opposite, Terry. They try to make it more difficult, it seems, to get these services and to provide rational, continuity of care, and design a benefit around the treatment. In Japan, from what I understand, the companies that make the formula are also involved in developing the test. The tests are used to identify irregularities or these metabolic disorders. They inform the government and milk producers of what the estimated need will be, much like we do with vaccines. Then, they develop the right amount of formula so that people will have it.
Dr. Bob Goldberg (11m 45s):
I'm just astonished, that apart from the shortage itself, we do not have a more elegant way to help people.
Terry Wilcox (11m 53s):
You and I talked on the last show about how we should have been looking at the crystal ball a little sooner, because we had a warning, you said, in February.
Dr. Bob Goldberg (12m 3s):
We had a warning in February. We knew as far back as September, that there was a problem with this facility. Once the FDA got in, and there was a little pressure put on, they realized that there were many deficiencies. I know the President said, "Well, you can't read minds." Yeah, but you could read the reports from the FDA.
Terry Wilcox (12m 28s):
You can read what's going on at these facilities. Are these facilities making all these different variations, these specialized, so they're not just making the one that goes to Walmart?
Dr. Bob Goldberg (12m 38s):
Right. Exactly. There are a couple of things. Abbott, for example, thirty-percent of the entire infant formula market, but they also have a very large share of the special formula market, which is smaller. One facility goes out, and that's a shortage particularly among people with the conditions that require those kinds of specialized formulations.
Terry Wilcox (13m 6s):
Is Congress doing anything?
Dr. Bob Goldberg (13m 8s):
There's a bill introduced in both Chambers, that would require some nationwide standard of coverage, and providing the FDA with $20 million. Look, we should spend what we need to spend. $20 million to help beef-up inspections, which are now contracted-out to state agricultural agencies, is not ideal. As Joe Gerardi, who was the manager of the Yankees, now the Phillies, would say, "It's not what you want."
Terry Wilcox (13m 35s):
Right. I think Sarah brought this bill up in the conversation and it's called the Medical Nutrition Equity Bill. Here's what she had to say on this, so let's listen to her.
Sarah Chamberlain (13m 45s):
The bill provides a federal floor of coverage, which means that if your state has great coverage already, it's not going to make it any worse. It just says, let's bring everyone up to this floor of coverage, and that is coverage for this medically necessary formula, administered under the care of a physician, and low-protein foods, which are again, defined by the Orphan Drug Act. This isn't something you're getting off the shelf. This is a medical product for inborn errors of metabolism, and specific GI, or food protein allergies. The disorders are actually named in the bill. It's a very specific, narrow bill that says, basically this coverage has been missed and the bill tries to fill that gap.
Sarah Chamberlain (14m 30s):
What it says is, depending on whatever your plan is, it should cover medical foods the same way. I think, unfortunately, this formula shortage has shown a lot more people how necessary these products are. Our community has been living under this crisis for years. Supply doesn't equal access. As soon as we have the supply, a lot of us will still not have access and that's really what we need people to hear and understand.
Dr. Bob Goldberg (15m 2s):
So, Terry, a little more perspective. One in 10,000 infants every year, are diagnosed with these inborn errors of metabolism. They're inherited. They prevent the body from absorbing nutrients typically provided by breast or powdered milk. This is rare, but not uncommon, and that enzyme deficiency means that you cannot convert the mother's milk, or the existing powdered milk, into energy. That's the fuel. Depending upon the diagnosis, the infants will require a special formula, and not all special formulas are alike, as an alternative to the breast and powdered milk.
Terry Wilcox (15m 44s):
It seems to me if this bill can help that-but you and I both know, the devil is always in the details. We also asked Sarah what her family did when they realized there was a shortage,
Sarah Chamberlain (15m 59s):
For my daughter, we started transitioning her immediately. She gets 154 grams of powder every day, mixed with water. We do it five grams at a time, because these formulas taste so bad and are so distinctive. She drinks it out of a sippy cup, at age eight, because she can't smell it and drink it without retching. She knows it's what keeps her alive. If she's exhausted, or if she can't think straight, or if she's having frustration, she says, "Can I have my milk?", because she knows it is what makes her body and brain work. The idea of her being without it, or not being able to drink another brand, was terrifying.
Sarah Chamberlain (16m 40s):
We immediately started transitioning her, and by about mid-April, we were halfway on an old formula and a new formula, and she did not know. We were like, great, we're going to be out of the woods. We're going to transition to this new formula and then we found out the new formula was on back order, because of the domino-effect of all these patients having to move off the Abbott products. Right now, we've got maybe a month-and-a-half, or two, of product in our pantry. People have sent me product that their kids didn't like a certain flavor and they know my kid is changing to it. There's a lot of mutual support going on, which is lovely and necessary, but right now we can't get anything more.
Sarah Chamberlain (17m 21s):
When I go onto my DME to order, it says, "Feel free to place an order, but it's back-ordered." and we have no date. Right now, we're just in this "waiting game".
Terry Wilcox (17m 36s):
That is terrifying as a parent.
Dr. Bob Goldberg (17m 41s):
Man-oh-man. I was just on a phone call earlier. A friend of mine's granddaughter is five-weeks-old, inborn error of metabolism as well, and is scrounging for shortages. I understand if there's a shortage of X-Box or a Call to Duty, but this? I guess the good news, Terry, is how quickly the community of parents and caregivers have mobilized to get Congress to start doing something. As I alluded to before, this is just the start. We need to combine early screening for this disorder, affecting nutrition with the development of the supply chain, which is something that the FDA has proactively been working on as well.
Dr. Bob Goldberg (18m 26s):
Patients are already way ahead in pushing for this. There are some people, who would say, "Well, parents and patients shouldn't be involved in making these kinds of regulatory decisions." Really? Part of the whole problem here, to my mind, is that you have a culture that says, "Everyone should breastfeed their babies, so who cares that formula is not available!" I mean, that kind of elitist lackadaisical attitude does contribute to the apathy, which is necessary, for these kinds of shortages to increase.
Terry Wilcox (19m 1s):
Right. I think that narrative, you're talking about, is really driven more by the culture and the media-the general media. I don't really feel that's driven as much by the medical community.
Dr. Bob Goldberg (19m 13s):
Oh, not at!
Terry Wilcox (19m 13s):
One thing that's been really awesome to watch, throughout all of this, and even just the standard parents struggling with off-the-shelf formula. Not to mention what's going on over here with those with illnesses, who need specialty formula, and finding it on back order. We say it all the time, and I'm going to say it again today, it's the patient community across the board. Our collective patient voice really does matter. It really matters to policymaking. It matters! It matters! You've seen that really step-up and that's why we want to invite patients to our nation's Capital to tell their stories and advocate for health policies that will give patients more access and affordability.
Terry Wilcox (19m 56s):
Next month, we're winding-down our enrollment period. It's called, We the Patients Fly In 2022, and we have so many patients who have already signed-up. It really knocked-our-socks-off this year. This will be happening in a couple of weeks, June 14th and 15th, right here in Washington, DC. We'll be covering what happened at the Fly In, right here on the show, so stay tuned. This episode of the Patients Rising Podcast is brought to you by Patients Rising Concierge, a new service from Patients Rising that helps patients and caregivers find the resources they need to find stability and support throughout their healthcare journey.
Terry Wilcox (20m 38s):
From finding a professional advocate to help with insurance challenges, to legal and tax counsel, to local caregiving resources and so much more. Our team is standing by to help you navigate the healthcare system and connect you to the services you need. To learn more, visit patientsrisingconcierge.org or email us at email@example.com. Up next, Field Correspondent, Kate Pecora, speaks with caregivers and patient advocates across the country.
Terry Wilcox (21m 19s):
This week, we'd like to introduce you to Lillian, who has a diagnosis of PKU and relies on a special formula for her nutrients. Kate has her story.
Kate Pecora (21m 28s):
Today, I'm speaking with Lillian. Lillian is a patient advocate, and she's going to talk with us today about her experience advocating for the Medical Nutrition Equity Act. Great to talk with you today, Lillian.
Lillian Isabella (21m 40s):
Thank you. I'm excited to be on. Thank you for having me.
Kate Pecora (21m 46s):
Lillian, could you start-off by telling me a bit about yourself? What got you into advocacy? What diagnosis you're living with and how it impacts you day-to-day.
Lillian Isabella (21m 55s):
Absolutely. I have phenylketonuria, or PKU, which is a metabolic condition. Basically, I can't break-down the amino acids, phenylalanine, which is found in food that is high in protein. I've never had meat in my life. I can't have cheese, yogurt, eggs, beans, nuts, dairy, any of that stuff. I have a special formula, that I need to have four to five times a day, that gives me everything I'm missing from my regular diet, with the one thing I can't break down, already broken down for me in a lab. I always knew, growing-up, that my mom had some challenges with health insurance and getting the formula, we need, covered by health insurance. It was always a hassle, and a struggle, to get the insurance to do it. That's in New York, a state that requires coverage for it.
Lillian Isabella (22m 35s):
I just found-out about the Medical Nutrition Equity Act, which we've been trying to push, to get passed, for over twelve years at this point. It has been a long time. When I first started to get into advocacy and pushing this Medical Nutrition Equity Act through, maybe four or five years-ago, I heard about it through the National PKU Alliance. They have been putting a lot of effort towards getting it passed for a while, as well as several other really amazing groups.
Kate Pecora (23m 1s):
Talk to me about what it is. Is the bill at a federal level, or a local level, and what is the optimal outcome, if this bill is passed? Where do we go next?
Lillian Isabella (23m 13s):
The Medical Nutrition Equity Act would be at the federal level, and we are trying to push it through the House and the Senate, and get co-sponsors, the main focus that we have. Basically, the Medical Nutrition Equity Act provides coverage for people who would benefit from medical nutrition. I think a lot of people don't know what medical nutrition is. That's something that we kind of come up against when we're talking to representatives and senators. Medical nutrition, basically, is something that is medically necessary and is nutritious. For example, with PKU, I cannot get that formula anywhere else.
Lillian Isabella (23m 53s):
I can't get in a grocery store. It has to be prescribed to me by my doctor and then I get the formula. There's also a lot of other rare diseases that are covered under the Medical Nutrition Equity Act. There's a whole long list of them. If you go to nutritionequity.org, you can see the full list, but there's so many of them. Some, if they don't get this medically necessary nutrition, they'll die. With PKU, the result isn't death, but it is severe mental and cognitive disability. We aren't able to function like normal members of society, thriving members of society. This legislation would allow patients to choose treatment options that are medically appropriate for them based on their specific needs.
Lillian Isabella (24m 39s):
The financial strain on families, to afford this medical nutrition, has only worsened during the pandemic. These families and their children can't afford to wait. Something else we say when we're talking to senators and representatives is, children get this disease through no-fault of their own. Their parents are on-the-hook to foot the bill for the medical foods, which sometimes costs five times as much as what a regular grocery store trip would cost, for example.
Kate Pecora (25m 8s):
There is a pretty critical supply shortage, within the US, as it relates to infant formula. I know infant formula and medical formula are slightly different. There are some conditions, especially within the medical community, that not only rely on medical nutrition, but they also rely on infant formula to supplement their nutrition. Could you talk to me about whether you have seen any changes in the situation as it relates to supply shortages around medical nutrition or formula?
Lillian Isabella (25m 37s):
There's the recent baby formula shortage that has been covered everywhere in the news. CNN, New York Times, The Cut, Politico, it's all over the place. Abbott is one of the facilities that had to shut-down due to FDA regulations and they needed to stop sending their formula out. There is at least one formula, coming out of Abbott, that is related to phenylketonuria and treating phenylketonuria. There are definitely people with rare disorders who are experiencing shortages with formula, not having access to formula,, and it just not being there. It's a crisis for everyone, whether or not you have a metabolic condition, but for people with metabolic conditions, some of these formulas are life-saving.
Lillian Isabella (26m 17s):
To not have access to them is tragic and very scary for parents, because they don't know how they're going to literally keep their kids alive. It's not just for babies. It's not just for kids. People like myself, we need formula every single day in order to thrive. Once the formula shortage crisis ends, our metabolic community is still going to be in crisis, because a lot of us cannot access those life-saving formulas due to health insurance restrictions or denial of coverage. Right now, coverage for these medical nutrition formula, low-protein foods is determined on a state-by-state basis and there are some states that just completely deny coverage for phenylketonuria.
Lillian Isabella (27m 2s):
There are some states that say, we'll only cover it for you until you're 13, or you're 18, or this very arbitrary kind-of-thing. It's not the reality of the medical situation. The medical situation is that you need it for the rest of your life. It doesn't make sense to give insurance companies the ability to deny something that is necessary for your life, and currently, they are able to. The reason that it's so important it's covered by insurance is, because the individual cost is so extreme. For example, the formula I need to have every month, if I didn't have insurance, would cost me a thousand dollars a month.
Lillian Isabella (27m 43s):
That is not in the budget. It's just not in the budget, but I need to have it monthly. Right now, you could be paying so much money for a monthly health insurance plan. You have a child, your child has PKU, and that insurance, you're paying so much money towards, will refuse to cover it. You're kind of stuck. What do you do? Do you get a new job? Do you move to a different state? All of a sudden, your life is totally up-ended. It's extremely important that this gets passed so there's adequate access to care. Right now, if you're not able to get it through insurance, and you're well-off, no problem, right? You can still buy the formula you need to take care of your kid, but the people this disproportionately affects, are the people who are middle-class, the people who are poor and the people who cannot outright buy it, which is the majority of the United States of America.
Dr. Bob Goldberg (28m 46s):
Thank you so much for sharing your story with us. Now, if other people out there want to share their story, and become our next patient correspondent, send an email to Terry and me at firstname.lastname@example.org.
Terry Wilcox (28m 55s):
We are so happy you tuned into today's episode. If you like what you hear, we'd love if you would share this show with a friend, coworker, patient advocate or anyone in your community. Spreading the word of our show helps us reach more of our chronic and rare disease community.
Dr. Bob Goldberg (29m 13s):
Don't forget to click the "follow" button, that way you'll be notified as soon as the latest episode is released.
Terry Wilcox (29m 18s):
That will be next week. Meet us right back here again next Friday for another fresh new episode on health policy. Until then, for Dr. Bob and everyone at Patients Rising, I'm Terry Wilcox stay healthy!