Why rare diseases are on the radar of Nevada officials, plus... -3...2...1 Go Brightline!

-Brightline West breaks ground on a high-speed rail between Las Vegas and Southern California.

That's this week on Nevada Week.

♪♪♪ Support for Nevada Week is provided by Senator William H. Hernstat.

Welcome to Nevada Week.

I'm Amber Renee Dixon.

Ahead, we explore a first-of-its-kind statewide effort to find out how many families in Nevada are impacted by rare diseases.

But we begin with Brightline West.

Construction on the all-electric, high-speed rail system is now underway after a Las Vegas groundbreaking ceremony where U.S. Transportation Secretary Pete Buttigieg said it was President Joe Biden's infrastructure law that made the train possible.

(Pete Buittigieg) To get here, it took a President who showed perhaps the greatest appreciation for railroads of any President since Lincoln.

Two weeks ago a journalist asked me, Why can't we have a high-speed rail?

We want high-speed rail.

I said you sound like my boss.

But my real answer was, We can if we choose to, and now America has decided to.

So on behalf of the Biden administration, it is my great honor to help break ground on what will be the first high-speed rail in American history.

[applause] Officials say Brightline West will take about four years to complete.

The $12 billion rail system is being funded in part by the Bipartisan Infrastructure Bill that President Biden signed into law in 2021.

Where the rest of the money will come from, Brightline West says it's still working on that as well as on how much tickets will cost once the high-speed rail is complete.

-3...2...1 Go Brightline!

-On Earth Day is when federal, state, and local leaders hammered rail spikes, signifying the start of construction on an all-electric, high-speed train connecting Las Vegas to Southern California.

(Jacky Rosen) This high-speed rail project is going to help reduce our carbon emissions by taking approximately 3 million cars off the I-15, 3 million cars off the I-15.

I don't think I have to tell anybody here, during rush hour on those Friday nights and Sundays when people are going back and forth, it's a parking lot.

People spend hours and hours bumper to bumper on traffic heading to or from Las Vegas.

We're going to make a dent in that with this train.

-U.S.

Senator Jacky Rosen, was one of several politicians-- (Dina Titus) It's going to bring more visitors to Nevada.

It's going to make their travel more convenient and expedient.

- --who praised the project-- (Steve Horsford) It's about economic development.

35,000 jobs, good paying union jobs.

- --at a ceremony between Interstate 15 and Las Vegas Boulevard near Warm Springs Road.

This is where Brightline West says its Las Vegas station will be.

That is the I-15 behind me.

That is the Las Vegas Strip.

And it's about a 10-minute drive from here to the Strip.

And then from here to Rancho Cucamonga is where this high-speed rail service will run at speeds up to 200 miles per hour.

Brightline West says it'll be about a two-hour trip, and that Rancho Cucamonga station is going to be next to the Metro link which you can then take into LA.

That's an additional hour, making for a three-hour trip from Vegas to LA.

Brightline West hopes to have this all up and running by the summer of 2028 in time for the Olympics in LA.

Without traffic, the drive from Las Vegas to Los Angeles is about 4 to 4 1/2 hours.

And in a car that gets 25 miles per gallon of gas, a round trip would cost $100 according to Triple A.

A flight from Las Vegas to LA takes a little more than 1 hour and can cost less than $100 round trip according to travel sites like Expedia and Orbitz, which begs the question: Will Brightline West be worth it?

In an interview with the LA Times, Brightline Founder Wes Edens reportedly said he will eventually charge more than $400 for a round trip from Las Vegas to Rancho Cucamonga.

I asked him about that at the groundbreaking ceremony.

(Wes Edens) It's a bit of a misstatement.

If you look at train systems around the world--so London to Paris, Paris to Bayeux, Madrid to Seville, Rome to Milan, so other comparable systems or the Amtrak, the Acela system, from New York to Washington--they tend to all fall in a range on the revenue side between 50 cents and $1 a mile.

That's what I told them.

So that's actually what they do average.

The Acela service from New York to Washington actually averages about $1.25 a mile.

So those are just good information points about this.

We're 4 years away from actually having a train that's running out here, so we're hardly ready to set prices for it right now.

But I can tell you that those are in dozens of different rail systems around the country.

And obviously-- or around the world.

But they all have to be competitive with flights.

They have to be competitive with driving.

There's factors that go into all that.

But I think, you know, I stand with the truth.

The truth is, 50 cents to $1 is a good-- those are good metrics to kind of look at and figure it out.

But of course as with any transportation system, there'll be very early rides that are cheaper, there's peak rides that are more expensive, or whatnot.

It's a blend of all those things.

-Brightline West itself will cost $12 billion and is backed by 6.5 billion from the Biden administration, 3 billion of which is from the Bipartisan Infrastructure Law.

It's well-deserved federal funding, says U.S. Transportation Secretary Pete Buttigieg, despite Brightline Florida, a high-speed train between Miami and Orlando, struggling to make a profit.

-Brightline Florida, for example, has had two straight years of net losses.

What do you take from that?

-What we've seen in Brightline Florida is they've demonstrated how you can run a train on U.S. soil with a level of service that would pass muster even in places that we envy, like Europe.

This is, of course, a totally different project in many ways.

But you have a team that knows how to build a train, and that's the important part in this.

-And while lawmakers say Brightline West will generate $10 billion in economic impact, Nevada Governor and former Clark County Sheriff Joe Lombardo said the number of lives that will be saved on the roads when people take the train instead of driving is what sold him on supporting it.

(Joe Lombardo) The Secretary and I had a conversation not long ago, right?

And we were discussing the reason why for the train, what it would do for Southern Nevada.

And we talked about tourism, but it wasn't hitting home.

It wasn't hitting home when we talked strictly about tourism.

And then I mentioned the lives that will be saved as a result of the build-out of this train along the I-15 roadway, and it hit home.

It hit home, and he says, That's going to take it over.

That's going to take it over the hurdle.

And today we are here.

-Another safety note: Unlike Brightline Florida, which crosses several streets and sidewalks, much of Brightline West will be built in the median of the I-15, eliminating some of the safety concerns that grade crossings present.

As for the financial solvency of Brightline, it's a topic we discussed further with its CEO, Mike Reininger.

(Mike Beininger) Brightline Florida was really a pioneer.

It was the first time in over a century that a private company introduced a high-speed passenger system in the United States.

Lots to have learned there.

We really had to develop a business model.

We had to learn about our customers.

We had to learn how to develop the service.

I'm very happy to say that in the month of March, we carried over 260,000 people in our Florida operation in just the month of March.

And we became profitable for the first time in the month of March.

So that's the path that we're going to follow here with Brightline West.

This is going to be a very profitable venture.

This is, these are two huge markets we're talking about here between Nevada and Southern California.

50 million people or so a year are already making this trip on an annual basis.

That's a huge marketplace.

-Right now Brightline Florida is in the process of refinancing about $4 billion in debt.

Meanwhile, Brightline's founder told Bloomberg Television he's already envisioning where the next Brightline rail will be, listing Dallas, Houston, and San Antonio as a promising route, as well as Atlanta to Charlotte, Portland to Seattle, and Chicago to St. Louis.

Back here in Nevada, visit a local doctor's office, and you may notice a flyer titled "While You Wait."

It's part of a statewide campaign to find out what impact rare diseases are having on Nevada families.

Here to explain why this information is needed in Nevada is Dr. Joe Lasky, Medical Director of Cure 4 The Kids Foundation; Annette Logan-Parker, Chair of the Nevada Rare Disease Advisory Council; and Tiffany Ruben, a mother of children with rare disease.

Tiffany, I want to start with you.

I said you are the mother of children, plural, with rare disease.

Tell me about your children and the conditions that they are dealing with.

(Tiffany Ruben) Yes, so I have four children, four boys that have the diagnosis of Niemann-Pick type C. It's a genetic disease.

My oldest passed away when he was seven, Jacob.

Then I have my son Mason who's nine and my son Logan who's six and my son Ethan who's four.

-And he passed away as a result of this?

-Yeah.

In 2019.

-What is it like being the mother of children with rare disease?

-So it took us a long time to get the diagnosis.

It took us 3 1/2 years, lots of medical appointments, and testing.

And as a mother, I felt this accomplishment of getting this diagnosis.

And when I saw our local doctor to tell him, like, We finally got this diagnosis, I remember him telling us-- you know, he reminded us of the prognosis of this disease, which is not great.

It's a horrible disease.

And, you know, here I tried to reassure him like, But it's okay because things are happening every day.

We could get a cure.

We could get, you know, things are happening, and we can have hope.

And I remember he stopped me dead in my thoughts, and he said, Why would anybody care about your disease?

It only affects so very few worldwide.

Why would anybody fund or give any money towards your disease?

And I remember feeling the weight of having this rare disease, and I felt very alone.

Right now we are one of two families in the state of Nevada that have this disease.

So there's not a big support system for us.

And I just remember just feeling, just that heaviness of being-- that our army was so small.

-And that has stuck with you?

-Yeah, it resurfaces quite often, actually.

-Annette, as Tiffany mentioned, she is one of two families in Nevada with this particular disease.

But when we're talking about rare diseases, how rare are they really?

(Annette Logan-Parker) Well, rare diseases as a grouping is not that rare.

It's estimated that 1 in 10 Americans have some type of rare disease.

This could be 7,000 to 10,000 different conditions.

So, collectively, rare diseases are not that rare.

-That makes it so difficult, though.

7,000 different types?

I mean, Doctor, do you learn about this enough in medical school?

Is that even a possibility?

(Dr. Joe Lasky) Well, truth be told, even those 7,000 different rare diseases, of course, fall into a whole different number of different subspecialties.

So I specialize in hematology and oncology, but then you have, you know, geneticists and endocrinologists and nephrologists who all of whom are gonna need to be experts in some subset of these diseases.

And so it's very complicated.

-Okay.

So, Annette, this survey, the purpose of it, what you want to determine, tell us about that.

-Sure.

So the Nevada Rare Disease Advisory Council has published what we call the "While You Wait" campaign, and it is designed to collect data statewide.

And we call it While You Wait because we want to have the posters and the flyers in medical facilities, urgent cares, hospitals, doctors offices, so that while people are waiting, they can fill out their needs assessment.

And the needs assessment is really geared towards collecting not only the prevalence of rare diseases, the types of rare diseases, but also the experience of the rare disease so that we can accumulate all of that information and help our lawmakers and our Department of Health and Human Services and the Governor's office understand what can be done to improve the experience, whether that be improved access to lab testing, whether it be access to out-of-state care that we don't currently have here.

It's a challenge to get these families what they need.

And we're hoping that the information that we gather-- it's a three-year campaign.

We're going to be collecting data for three years.

And we'll publish that data annually.

And our goal is to provide high-quality, verifiable information so that people can make good, well-informed decisions.

-You want to find out about the experiences of these families.

We just heard from Tiffany who described her experience with the doctor saying, Why are people going to care?

Why should people in this state care?

-Well, that's a complicated answer.

You know, there's the economic impact of undiagnosed rare diseases and what does that look like, which can be extensive.

Well, other studies have shown that it can be as high as a half a million dollars per patient in unnecessary testing and medical interventions because of a rare disease not being promptly diagnosed.

-Wow!

Tell me more about why people should care.

-Well, and I think that it's the human piece too.

You know, if 1 in 10 Nevadans is suffering and living with and dealing with some type of rare disease, that's a significant part of our population.

And I think that they deserve to have the best access and the best care that we have available.

-Doctor, yes, please go ahead.

-And some of these diseases, rare, genetic, and/or malignant diseases, it's very critical to diagnose them early.

And there are some treatments that only work if you start them in the first year of life.

Or if you start them later, they're not as effective, and those children can go on to have bad outcomes.

And so having the knowledge in place for both pediatricians and the public just to know, to be aware that these diseases exist and to have the correct testing and the correct facilities to diagnose these kids is incredibly important.

Again, not just to prevent, you know, healthcare costs, you know, being higher for those patients, but to take the best care of those children and really give them better qualities of life.

-And you are-- yes.

-It took us 3 1/2, 3 years to diagnose my son, and every week-- -Three years.

-Every week we watched digression.

And had we got him something earlier, like it could have turned his trajectory a different way.

There's no way to tell, but we just watched him go a different path.

-And we talked prior to this about every six months, what happens to your family?

-So for the last six years, we've been receiving a treatment at Cure 4 The Kids.

And every six months we get a denial from insurance saying that they can't get their treatment.

So every six months we have to appeal the insurance to get our treatments.

-And that's private insurance?

-We have-- our kids have Medicaid.

-Okay.

And that's common, as you were telling me.

-It's very common.

-Why?

Will you explain that.

-Well, I think that in the electronic system, there's some sort of cue that triggers an automatic denial.

-I was specifically referencing that children with rare disease are Medicaid patients.

Many of them are.

Why is that?

-Oftentimes.

Well, I think-- you know, at Cure 4 The Kids, 54% of our entire patient population is a Medicaid beneficiary.

And then if you pull out and just focus on the rare diseases, that goes as high as 75%.

Who has young children, but young families, right?

And whether they're not employed where they have a robust health care plan available to them or they're working two jobs as they're building their family.

And so young people have children and, oftentimes, they're not adequately insured.

And then when you-- once the rare disease is diagnosed or the symptoms of a rare disease present themselves, so many times one of the parents needs to become a full-time caregiver.

And so the family income decreases significantly, and so they fall into that category.

-I want to go back to the testing.

There is-- okay, diagnosis.

You had talked about genetic testing being crucial to diagnosis, correct?

But what troubles are you facing just in genetic testing?

-So yeah, so there's a number of situations and diseases where there's now some relatively simple PCR-based or genetic testing that could give us the diagnosis very quickly.

And we're putting, you know, dozens of requests in each week to, that has to go through the insurance company to get authorization.

And we're getting denials for those tests very regularly.

And it's resulting in me having to make phone calls and write letters.

And I would say at least 50% of the time, even with all of that effort, we still end up with a denial in the end.

-Is this something you experienced, Tiffany?

-Yeah.

Early on, we actually went down to UCLA.

And when the doctor told us we would need this test, he said, We could go with the insurance route, but it would take us a long time to get approval, or you could just pay $10,000.

-Yeah.

And who just has that kind of money available?

What kind of laws would you like to see enacted, even without this data, prior to having it, which will support your cause once you get that data?

-I really do feel that laws that support access to genetic testing is critical.

I think that, you know, if say, for example, for people who are insuring lives in Nevada, if we could have, through legislation, a statute that says, If you're insuring lives in Nevada, then you need to provide access to these genetic testing.

And that's not one of those where everybody can get a genetic test.

It's when a physician has done the diagnostic process and they feel that this is the best option for a quick diagnosis.

They should be able to do that in an efficient and timely manner.

And that just doesn't happen within our state.

-So the State of Nevada could make a law that said, If you are insuring people in Nevada, you have to approve these genetic tests.

So once you get the diagnosis, then there is the treatment.

Let's talk about the availability of treatment in Nevada.

How available was it for you once you finally got that diagnosis?

-We were the only family in Nevada at the time, so we didn't have a treatment.

So we had to go down to California every two weeks.

We did that for eight months before we could set up a treatment center here in Nevada.

So ever since then, it's been great.

We've been able to get our treatment at Cure 4 The Kids.

-From your experience?

-So the treatment that is given is still investigational.

It's not technically FDA approved.

And so it's given in the form of a clinical trial or an expanded access trial.

And so we had to put through, you know, IRB materials and regulatory stuff and a lot of hoops to be approved as a site to even administer that, this medication.

-It took months, months for it.

-As you're watching.

-No.

So we were going down to California.

-Okay.

-Traveling every two weeks.

-Every two weeks.

-Which has risks.

-Yeah.

With three little kids, back and forth.

-The Nevada Rare Disease Advisory Council, this was established back in 2019 during the legislative session.

And at that session, there was a mother who testified she was the mother of a six-year-old son who had passed away, he had leukemia, and she said, quote, Our family was shocked that our highly trained, capable, and trusted pediatrician was not able to identify and diagnose any symptoms of the childhood disease.

We were even more shocked to discover that our pediatrician was himself a childhood leukemia survivor.

Without an established Rare Disease Advisory Council within Nevada relating to the prevalence and treatment of rare childhood diseases, our pediatrician was not able to pick up the symptoms that could have been addressed and potentially saved Carson's life.

Doctor, is this a Nevada issue solely?

-I would say it's certainly not a Nevada issue solely, but I think the answer, you know, again, has lots of layers and is more complicated than something easy.

Yeah.

And so one important thing to understand is that leukemia, just like a lot of other genetic diseases, is a rare disease.

And if a child presents with fever or a headache, the chances that they have leukemia is still really, really low.

And if you're a pediatrician who's seeing 50 patients a day, many of whom have fevers and headaches, it can be difficult to separate out a significant leukemia from just a cold or the flu.

And, you know, I think one of the things that could improve upon that in this state is just getting more pediatricians here.

We rank at the bottom of the list when it comes to per capita pediatric care, as well as for pediatric subspecialist care.

And so something that could help that is by getting more medical schools here in the state, getting more academics here in the state, and then recruiting more pediatricians and pediatric subspecialists to really help out to care for this rapidly growing population we have.

-We know that if there are residencies available, there's a likelihood that someone who's doing a residency in a certain location will end up staying and actually practicing there.

What is the state of that in Nevada as far as pediatricians?

-So at this point, we have one small pediatric residency program just in Las Vegas, and that's really for the entire state.

And so that's obviously not enough.

-What would you add to that, Annette, as far as the amount of support here for physicians involved in pediatrics?

-Well, I think that a big part of a root cause analysis would show that the reimbursements, particularly for Medicaid, is not favorable to attract physicians to an incumbent practice here.

So you know, that's an unfortunate part because they're coming here to an environment that doesn't have good infrastructure and good support for their practice.

And then they're also paid at a much lower rate than some of their colleagues across the country would be paid to provide the same service.

And so that doesn't create an incentive for people to want to come here.

-And so then, the data from this survey, could that change that?

-Well, I think that it could change it because we'll have a clearer picture of what is happening here.

And like Dr. Lasky had mentioned, so many of these rare diseases require pediatric subspecialists.

And in order to attract pediatric subspecialists, we need a solid infrastructure for them so that they can practice their skill, you know, safely.

So there's things that need to happen in order to improve things for pediatric healthcare in Nevada.

I think that the needs assessment will create the foundation that will establish some important conversations with our lawmakers and our Department of Health and Human Services.

And I think that all progress starts with a conversation.

-Okay.

And I have one last question that I can fit in.

Tiffany, tell me how your children are doing and how this disease impacts them on a day-to-day basis.

-So like he said, it's, our treatment is just a treatment.

It's not a cure.

So we do see disease progression.

In our disease, they lose their life function.

So my sons are losing their ability to walk.

They're losing their ability to talk.

So we do see disease progression.

So we just take life day by day and live with hope that, you know, things can happen and with things that are happening, you know, we might have more people that are coming and different things like a cure that could happen and you never know what will happen.

-Thank you so much for sharing your story with our viewers, and thank you to our panel.

Thank you for watching.

For any of the resources discussed, go to our website, vegaspbs.org/nevadaweek, and I'll see you next week on Nevada Week.

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