FDA Direct: Combating Rare Diseases at the FDA

Okay.

We're back for Rare Disease Day
and Rare Disease Week, an exciting time

at the FDA, and tons of folks
who are in this community are going to be

on a virtual town hall that we're
having - a program that's set up by the FDA.

So an exciting time.
And that's why we're here.

Elisabeth and Jim, thanks for joining.

So glad to be here.
Thanks for having us.

Yeah, I was really excited to join.

This is a topic that I'm super passionate
about and I understand, Jim.

You know,
you have struggled with a rare disease.

You've survived a rare disease.

And that's actually kind of
what connected you two to begin with.

So I was really curious if you would share
a little bit, if you’re comfortable,

about your journey.
And can I just jump in?

Jim, we are so lucky to have you
in leadership at the FDA.

And you too, Elisabeth.

So it's just I was really looking forward
to this conversation today.

Thanks.

So thanks for being willing
to be vulnerable and share a little bit.

Yeah. Now, thanks for having us.
Thanks for all you're doing.

Yeah.

We're a rare disease family.

Honestly, for,

years, our family has struggled
with, rare disease.

It affected
not only me, but also our daughter.

The disease is called
primary sclerosing cholangitis.

It's like widening
and narrowing of the bile ducts.

A way to think of this disease is

if you're washing your car
and, you crimp the hose,

it's like that where the bile ducts
narrow in the liver, and then the toxins

that your liver would normally filter
go back onto the liver, cause scarring.

Thus the sclerosing
in the title of the disease.

And you develop cirrhosis.

And then if that's not addressed
or remediated, you get cancer

in the bile ducts.

And it's, it's not a good diagnosis.

Tough disease.  Tough disease.

It's a, it's a very hard disease.

And we didn't know it.

I mean, the journey I think this is
probably true for everybody with rare

is you just know

something's wrong
and you start looking for help.

In my case,

I'm driving home from work
and having to pull off

on the side of the road
because I'm so tired.

And I was telling Gwen, my wife,

you know, while I'm burning the candle at both
ends, going to graduate school, working hard 

Because when you're,
when you're in your s.

In my s.

Yeah, yeah, just out of college.

Played two sports.

So I thought of myself as an athlete,
even though maybe not the best one.

But life was going great.

Everything was great.
Yeah. In fact, we're just married.

New to DC, working in the,
space community at the time.

Just a great job.

You got married in your s?

Yeah, right out of college.
God bless you.  You chose well.

I did, I married up, 
there's no question about that.

Have you met Gwen?
She's the best.

She's. Yeah.
she is something.

Yeah.

In fact, today
she's actually leading a rare

disease advisory committee
for the state of Virginia.

She's the chair of that.

So our story has led to us
getting the chance to serve.

Me at the FDA, 
her with the state of Virginia.

Sometimes, you know, if you

think things are happening to you and
it may turn out they're happening for you.

The harshness of this, 
and I'll just try to give you a summary

was,

the disease progresses in a way
that requires that you get a transplant.

There's no therapy that will cure it.

And, 
and so all of a sudden, you have your,

you're on a timeline
where your disease is progressing

and you know that this is in your future.

You just don't know when

I was on a business trip and my,

doctor from Hopkins called me,
I could still remember it like it was now.

Jim,
we've been treating this for a long time,

but the disease has broken through,
and you're going to need the transplant.

And I remember,

like, not knowing what to do.

I wanted to call Gwen, but I thought,

no, it's
probably better to tell her in person.

So I waited till I flew home and then, 
shared with her what I had learned.

And I think which,
you know, maybe a little embarrassing,

but what I think most patients here
on this journey,

that takes a long time to find out
even what is it you have.

And then when you get to what's required

for you to endure that disease,
it's, it's traumatic.

It's hard on the family.

It's hard on the patient, of course,
but all of the people

that are close to them,
it really takes its toll.

That was true in our family.

So I thanks to the,

you know, my doctors at Hopkins,
I, you know,

I get to a meeting where they said, hey,
you made it onto the transplant list.

Good news. You made it on the list.

The bad news is your disease
doesn't score well.  So they do blood work.

And there's an algorithm that dictates
where you are in the transplant list,

and you hope to move up in time
to before your disease overtakes you

and you get like a just in time
transplant.

That's the hope.

And, they said but the problem is,

since your disease doesn't score well,
most likely you're going to,

you won't make it to a transplant.

You're going to have to find
a living donor,

which at the time, I'm like,
what are you talking about?

You only have one liver.

It's not like you go next door
and like you're going to.

It's like, can you imagine
asking for somebody for that?

Like, you know, how's it going?

Can I have half your liver?

A portion of your liver.
Yeah, exactly.

And that's really what happens.
They take, in the surgery,

It's a to hour surgery
for both the donor and the recipient.

They take two thirds of the donor's
liver, transplant it into the recipient.

And you get all of the,
like, the bile ducts,

the arterial flow,
and two thirds of a liver.

And the liver mass regenerates,
which I came to appreciate.

Obviously you knew.  So you end up with
like, a full filter and half the plumbing.

That's a way to think about it.

It's amazing. 
It's amazing that it works.  Yeah.

One of the reasons
why the only food I can’t eat is liver.

Just too close to work.
Me, too.

It's amazing. It's an amazing organ.

Yeah.

You had two liver transplants

and then were the recipient
of a compassionate use authorization.

Yeah.
Maybe the first.

Yeah. 
For OKT?

Yeah, that was one of the drugs.

In the first
transplant, my neighbor saved my life.

We were the fifth to have the surgery
at Hopkins.

First that weren't related.

It's very experimental.

At the time, they told me you'll probably
die, but it's your only chance to live.

I mean, it's like,

And you have a fixed end date.

Like there's a date they pick.

And so when you think about how you live
your life,

also, you have clarity.

I call it,
I don't know, this is terrible, maybe.

But the way I think about
it's like a corridor of death.

You confront your own mortality

and the things that you tend
to spend the most of your time

with, like your job
and your stuff are the first things to go.

You walk a little further
and you say goodbye to your

work, your friends, your extended family.

You go a little further.

You say goodbye to your parents.

In my case, then it's my kids.

Then you say goodbye to Gwen, and
then you're all alone at the edge of life.

It's, incredible place to be.
With a lot of uncertainty.

With a lot of uncertainty.

Yeah,
and you're letting go in the process.

But you have total clarity along the way.

And it is.

Isn't it
amazing how those sort of insights

at that moment in life,

when you're sort of confronting your,
your own mortality, which

none of us are going to live forever.
Right?

It's just a matter of
when we confront that mortality. 

How it instills so much wisdom
about what's important.

Yeah, that's really true.

It's very clarifying.

And the hard part, and I think a lot
of families at this, in rare disease,

you know,
we often think about the patients

trying to accelerate cures
and we forget about the caregivers.

There's like, estimates, million
at least.  

million is the highest number
I've heard.

Of people who were caring for a loved one.

And in the rare disease community, there’s like
million people that have rare disease.

It's not that rare.

Yeah, the conditions are rare,
but the circumstances aren't.

And the effect on the family is dramatic.

Yeah.

And it's enduring.

It's over time. It's not a discrete event.

It's a process that you watch loved
ones endure and you put your life on hold.

This was true of Gwen,
which I wish she was here.

She's the best
part of our story, for sure.

But they put their life on hold to care
for somebody else.

In our case,
Gwen was when I was diagnosed.

And then, as you mentioned,

not not only did I have to,
but our daughter Ashley had to as well.

And our son Jeremy

risked his life to save hers
in the first transplant out of necessity.

Again,
because the disease doesn't score well.

And Gwen was when I was diagnosed.

I can remember, like today.

And Ashley
got her second transplant on her th

birthday,
and Gwen took care of us the whole time.

Just as I was getting well, Ashley
got sick, and Gwen was our caregiver.

And I think that's the journey
of rare disease for a lot of families.

It's a decades long, you give up your life
to and caring for somebody else.

And then to your point,
in my first transplant,

I had multiple episodes of
rejection and one, the FDA

just kind of amazingly, you know,
I didn't know it was an option.

We’re like, we've tried everything else.
We're going to give you a shot.

We don't know if it'll kill you
or kill you.

We'll know in minutes.

And you're like, I'm also a business guy.

And I'm like, what's plan B?

You know, like, there's got to be
something else you got back there.

And, but you take that shot
and it's like rebooting your immune system

and hoping that it'll come back on
and not reject the transplant.

And so to go from
and I don't want to be too dramatic,

but in between the transplant,
I had septic shock.

Where you go right to the brink of death.

I had a hospital acquired infection.

Not Hopkins.

It's okay if it was at Hopkins,
by the way.

No offense taken.

Yeah, but I'm just saying
it's a devastating thing, and it happens.

You know, it's nobody's.

And I was in a wheelchair for a long time.

I didn’t know if I'd ever walk again,
let alone work again, let alone

get to be doing this
today at the FDA, who helped save my life,

you know, some years ago,
not only do I get a second chance at life,

a third chance, and then a chance to serve
here, it's just unbelievable.

I think that's the thing that I hear from
your story is the clarity.

I know you said off the top.

You worked in the space industry,
but you completely transitioned your life

to work in health care, to serve patients.
Yeah. 

Can you talk a little bit
about that part?

Yeah, and this was, when I finally got well,
this is after the second transplant.

I had all these insights from being sick
when we were in,

we would do
health and safety of satellites

like we wrote the software to control
a GPS constellation is a good example.

And you try to do things to optimize
with data to keep them on orbit longer.

So when I was sick,
that's where they're giving me the shot.

I'm like,
I'm going to get smarter about this.

So I start interviewing the residents,
the fellows, the interns.

I become friends
with the attending surgeons

and ask them,
how are you assessing my health?

And every morning they would hand me
the results of my blood work,

and I knew what my medications were.

So looking for some semblance of control,
I start trending my blood work

and medications like we would do in space,

and I come up with an algorithm you could
predict when rejection would occur.

This sounds like you at the FDA.
Making friends, tracking things.

That's kind of important.

You're running out of options.
You want to do something? 

That’s why everyone loves Jim at the FDA.

Yeah.
He's in the workstreams.

Coming up with a few algorithms. 
What has happened, 

so, I developed the symptoms
a third time, I now have.

I know the symptoms are.

So I'm having a mean rejection
episode for the third time,

but I'm looking at my data and
I change my medication based on the data.

Then I call on my doctors at Hopkins,
and it wasn't Dr. Makary.

And they were furious.

But when I showed him the data,
it was obvious that you could treat

to the individual case
and not the therapeutic range of the drug.

And, it saved my life.

And then they were able to, administer

post-transplant care differently
going forward because of the, 

it was their data.

So I had this idea.

Well, I'm going to go create a health
care company to solve this problem.

And I, I was in a large defense firm

and we had like,

$billion a year
to take the state of the art in advance

that are applied in unique ways
for the intelligence community, primarily.

But I could see
how it could be used in medicine.

So I went to the CEO and,

you know,
could I have the chance to do this?

And they said, yes, eventually.

And then I was like, now what do I do?

My first thought was, I'm
going back to Hopkins.

They saved my life.
Maybe they could save my career.

And we did the first ever joint venture.
And that's where I met Marty.

You know, it was at the end of that

when I was up at Hopkins all the time,
and we became friends.

And, you know, you've done
so much for our family ever since as we've

navigated Covid and everything else, so.

Well, Jim, you've had

a very successful business career,
and we're lucky to have you at the FDA.

So I think everybody would say that
in leadership here.

Absolutely.

I think one of the things that I hear
when you're talking about

kind of how you tracked your journey is,
how you must have done that as a parent

for Ashley.

We see that, I am really lucky

I get to help facilitate your meetings,
Dr. Makary.

We, you know, you meet often
with the rare disease community.

It's, it's behind closed doors.

You're not out in the media about it
because we don't want to be exploitative.

Right?

But, we see the parents come in
and they know the disease state better.

They know their child better than anyone.

And we see that advocacy
and it's, it's really, really beautiful.

I'm curious about kind of your advocacy
journey.

Having been a patient yourself,
but also a parent.

Yeah, you've been,

and also you've been very involved
in the rare disease reforms at the agency.

Totally.

And that's kind of it's
kind of a passion project

for you and leadership here
with a whole bunch of other folks

that have been working on this in the Rare
Disease Innovation Hub and others.

And folks in the outside,
you've had great relationships

with, incredible reformers and leaders
out there, John Crawley and others.

Yeah. So it's, it's kind of cool to watch.

I mean, we're cheering on this whole
effort, one of many reform efforts.

But this is this is a big one, because
when you add up the number of Americans

with rare diseases, all of a sudden it's
not rare.  

Totally million people.
Yeah. What's that? % of the population.

And then globally. Right?

If we develop innovations here at the FDA
selling gene therapies or others,

it's going to be used around the world.

So this is really, a hub for innovation
globally.

Absolutely. Yeah.

You think about the impact
not just on the patients,

but I know you both have highlighted
kind of the caregiver, the parents.

It's massive.

Yeah, yeah.

And also,
one of the things that just brings me

great joy is to see some of the reforms
that you're leading,

that enable small populations

to go through the FDA and,

and in search of therapies that end up
helping these, you know, people.

And the beautiful thing about the FDA,
people are so committed.

I mean, that was one of the,

I don't want to say surprises,
but just beautiful things about coming in.

I don't know what I expected,
but the clinicians, the scientists,

the reviewers, everybody that works

here, they're passionate about
making a difference, helping other people.

And I almost think about it
in the, in terms

like you as a surgeon,

you know, you were able to do things

for people who come to you
with a very difficult condition.

And, you know, you've talked about
even in your care for them,

how that's so hard to deal
with even as a surgeon.

But when things go well
and patients recover,

you actually don't get to see them.
You took care of them in a moment in time.

And that's true with the FDA.

They are innovating around,
how do we regulate and

and come up with more, 
like the cell and gene manufacturing

the thing you just announced at JPMorgan.
We got great feedback on that.

Yeah.
But you're right.

There is this natural intrinsic tension
between, oh, the process here is perfect.

It's been tried and true for years.

Don't touch it.

Versus, hey, we walked in getting clocked

by China and Australia
and other places too.

And phase one is now at four times
the rate in China as in the United States.

So we have got to reform.

And what resonates with people,
including the wonderful staff here

that you alluded to.

I see one of them walking in the frigid
cold out there - is common sense, right?

Common sense.

We've got to just use common sense
and not hold on to our processes

for legacy sake.

If they are truly
the best way to do things, then yes,

but we have to be willing to, willing to

challenge some of these assumptions of why
we do things a certain way,

and probably nothing is better,
a better example of that than Baby KJ.

And the new flexibilities
that we announced for selling gene

therapies, specifically 
with the CMC requirements,

you know, getting away from three batch
routine requirements

and allowing Bayesian statistics
that allow you to sort of

identify the optimal dosing better,
especially with pediatric.

Yeah, exactly.

So, so anyway, those are
I mean, I think it's kind

of the ultimate in common
sense is the reforms on rare diseases.

I think that's something
I always really appreciate.

You know, for such a long time.

I think the rare disease population kind
of didn't get the attention it needed.

And then, you know, we saw all this work
with FDA compassionate use, expanded

access, right to try waivers,
accelerated reviews, tax credits,

%, just over % of 
the reviews we do here

now, in the medical product

centers are on drugs
that would treat folks with rare disease.

I think that's super important.
And also.

It's just, it's half or just over
half of the, all the drug approvals

at the FDA in CBER 
here are for rare diseases.

I don't know
about the veterinary medicine.

I think that's different.

I'm not, I'm not sure what's going on.

Not sure on that one.  
For humans, the drugs

approved for, in humans,
majority are for rare diseases.

Yeah.

I love though
that you and the Administration

have not been satisfied with that.

We're still on mission, kind of,
you know, driving forward,

pursuing these meaningful cures
and treatments.

I, I'm curious, like, your background
as a as a surgeon, as a doctor.

You know,
I've seen you meet with patients before.

I'm curious

if that's one of the things

that drives you with that sense of urgency
about why we continue

to move forward here.

Well, first of all,
I love your, your whole premise.

And that is we have got to constantly ask,
what else can we do?

Yes, we have all these incentives,
you know, user fee waivers.

And if you get approved
for a rare disease, you get a pot of gold

of $to $million
in the form of a transferable voucher.

So there are a ton of incentives,
but what else can we do?

Or we shouldn't be satisfied
with what we have in place now.

And when you're proximate to the problem,
when you have friends

like Jim, who have been through hell
and his family and met his daughter

and the incredible altruistic donor
Frank, who I had the privilege of meeting,

and you have to break bad news
when you have these experiences,

when you tell somebody that we figured out

what you have, and unfortunately,

there's nothing that we have to offer
for you at this time,

beyond love, support,
walking through this journey with you

and they feel this sense of injustice.

Why me? Why do I have to deal with this?
This is wrong.

They question everything.

They question their faith,
their relationships.

You know, the medical system.

Some people don't accept it
for a while. It.

And it's a normal human reaction. Right?

These are very difficult conditions.

You meet some of the folks
suffering with these conditions,

and there's a spectrum,
but some of the folks who are, 

require a lot of personal care, 
you can't help

but ask yourself,
is there anything humanly possible?

Because that's what they're asking.

So if you have a heart
and you're compassionate,

you're asking the same question
is there anything?

Can we move mountains to get something?

And you meet a lot of the drug developers
in this space,

and that's
what compels them to be in this space.

And so I think it is a question
we have to constantly ask.

And that's why
we are rolling out the plausible mechanism

pathway for the ultra rare
and bespoke conditions.

We want to see more Baby KJ’s 
and we just need to keep listening

to those who are proximate to the issue,
that is,

the patients and the caregivers
and to the drug developers.

And many of them are proximate
to the problem as well.

So that's kind of how I see it.

I, know that
that's the big headline today.

And I think that that's such
an exciting step forward

for the rare disease community
and for the FDA, something that folks

have wanted to do for a really long time
and kind of keeping that promise.

One thing I did want to ask you about is,
compassionate use.

We know that's a hot topic
for a lot of our,

patients and our neighbors
in the rare disease community.

I'm curious, you know, as we continue
to think about ideas for the FDA,

sort of where, where your head is on
compassionate use, expanded access, 

right to try.  
Yeah. I, I believe in all of it.

And so I've signed a % of compassionate
use authorizations

that have come before the FDA.

And it's not enough.

You know, we,
people have a right to be angry

if there's something out there
that could help them.

And there is some bureaucratic, block
that prevents them,

or the company does
not want to make it available

because they are worried
that giving a drug on compassionate use

might mess up an otherwise perfect trial.

They're about to submit to the FDA
because if that person has a reaction,

they feel like, well, that could.
That shouldn't care against them.

That's not right.

But yeah, common sense.  This
is common sense kind of stuff.

So I mean, we have to keep pushing this
like we're doing a ton.

It's the majority of our work in the

in the drug centers
at the FDA is on rare diseases.

There's a ton of incentives.

We're announcing more and regulatory
flexibilities, but we got to keep going.

We got to keep going.
More to come there for sure.

I guess one of the things
I'm curious about, and I've heard you both

talk a little bit about this,
is how being on the inside of the FDA

has changed your perspective on the work
we're doing here.

What would you want
the public to understand about the tension

with speed and safety?

You know, our, our mission here is protect
the public health.

And also because this is urgent, 
innovation and speed is, is vital.

I hear you talk about it in every meeting.

So curious
how you two would frame this up.

I mean, it's a balance, right?

It's balance because we
we want to see hope out there.

Yeah.

We don't want to see people harmed

by drugs that have data showing
that they don't work.

That is also,

a bad thing
to be putting in front of somebody.

So there's
I mean, there's never a perfect way

to articulate that balance,
but there is a balance.

Now, we hit a record number of 
cell and gene therapy

approvals, just a month 
and a half ago at CBER.

That was a milestone moment.

And so we want to see more.

We want to see more.

And, you know, if there is not an approval

from the FDA,
if the, if the scientists here

and by the way, I stand by the scientists
here, we've stood behind their decisions.

If they don't

believe that something should be approved,
the public deserves to know exactly

why, which is why we've made our letters
public.

The letters to the companies.
That’s really helpful.  

That should be public.

It creates accountability.

It also.
Creates transparency.

Creates transparency, reduces spin is kind of how
I was going to phrase it. Right?

Because you're, there's
always going to be spin around drugs.

You know, every drug is going to solve

all the world's problems
and has no side effects

until you look at the file
and get deep into that data.

So we want to see more approvals.

We've made, the mission here very clear,
redefining it when I got here.

We want, number
one, more cures and meaningful treatments.

Number two, healthier food for kids.

And so we're going to keep moving on those.
I love that

I think all the time about the links
between chronic disease and rare disease.

And our food obviously we know
plays into the chronic disease piece.

So, those priorities I think are vital.

Do you do you have any thoughts
on, you know, being inside the FDA?

What surprised you,

what you would have folks know
as a member of the of the community?

Yeah, I would say,
it feels like a black box

when you're on the outside
because you don't get to see what happens.

And a lot of it, until you open up
those CR letters

where we could actually communicate out

what how the decisions are being made
was totally opaque.

And now we've got this
transparency in the view.

But for patients, when you're,

when you're wrestling, you're hoping that,

somebody is paying attention to therapies

that could be helpful to your family,
and especially when it's your kids.

You were asking me about my kids.

I did much better when it was me
managing my disease

and how people reacted to me, like I felt
like I could manage the relationships.

I really struggled when it was my kids,
and I,

I have great empathy and,

for parents

advocating on behalf of their children.

You know, I was mentioning Gwen.

She was going to be Ashley's donor,
for example.

And I'll be quick about this, but, and
then got the call that she couldn't be.

It wasn't a structural match.

I've never seen anybody grieve

like at that moment, like that is.

Your wife was going to donate to your daughter?

For Ashley, yeah.

In fact, we were in the kitchen and,
Ashley was looking at her bloodwork.

She goes, I have a plus here. Like,
what’s the?

I got an A-plus. What's?

And Gwen goes, that's great.

That's why I couldn't be my donor.

Because I could be yours.
Because you need the blood type

to match.  Goes through 
all the tests, and then,

we ended up going back to,

my second transplant surgeon

who was at the University of Minnesota
after she was told no.

And, and she tries talking him
into figuring out a way

to make it work for her for fear that

Jeremy would have to be Ashley's donor
or would want to be. 

Jeremy's our son.

And he's two years younger than Ashley.

And he says,

you know, I would do it
if, if we could, if it would work. But

I'll use you to, like
if I won't let Ashley die without trying.

But if there's another option,
we have to look for another option,

because it's not a good scenario
for Ashley.

And, so Gwen left, and, you know, she's.

You know her. You know her.

I hope the folks at FDA
get to know her like we do family day.

Amazing.

Make a point to see Gwen.

I'll make your day, I promise.

But she was so tenacious.

And that's the role of a parent.

You're trying to advocate for your kids.

And then when, Jeremy ended up
being the donor, you know, thinking about

both of our children

going through that surgery
where we both know what they're facing.

And you never in a million years
want them to have to endure that.

You know, you know, so that.

But then coming back to the FDA,

what you're so thankful for is
when you see progress

and you know that this place is hard
at work

on behalf of the American people
and patients globally,

and the things that are coming out of here
are not only offering hope, but help.

Yeah.

Like to your point.

Yeah.
That's what's beautiful about this place.

I mean, the people here want to see.

Yeah.
People do better.

They want to see an end to suffering.

That's why they're here. 
It's amazing.

They're amazing folks.  Amazing. 
It’s precious.

And you can't possibly meet all,

you know, plus employees here.

But I've.

I've focused a little bit
on the scientific community

involved in drug approvals.

And I'm just blown away, like,
as if you can't meet more phenomenal

altruistic people making a lot 
of personal sacrifices to work here.

These are salt of the earth people.

Yeah, the best of the best. 
Yeah.

These are not angry librarians.

They want to see,

they want to see the drugs
come across the finish line.

Yeah, they're working hard.

And if they don't come
across the finish line

and because of some issue.
They protect the American people.

Yeah.

Or there's

a safety or manufacturing issue
or there's some violation or something.

Then you see how they try to create a path
to just do this and come back.

Yeah. So that's kind of impressive too.

They could just say sorry. You know. 
Yeah.

They don't.
They care too much.

I think that's the greatest privilege
of getting to work

here is getting to partner with our
our fed colleagues and hear from them.

I any conversation I have,
I always feel like I learned something,

but also seeing that connection to the
the mission and the values that I know we

all share, it's,
it's like the most meaningful

part of getting to come here every day.

Yeah. It's cool.
I love it.

Well, we're thankful to have both of you,
here at the FDA.

I hear so many compliments
from the staff about both of you.

So it's kind of cool.
We can take this time.

I know that,
we thought this would be a 

minute conversation, but
it was so compelling what you shared, Jim.

So, I'm blown away by it,  Elisabeth.

I don't know about you but it’s pretty amazing.
Thank you.

I have one more thought for the folks
at the FDA, if I could. Yeah.

And it's how,
when we finish the transplant

with the kids,
they're rebranding the hospital,

and they had the surgeon
and our family were sitting on the couch

and were being interviewed by an Academy
nominated director.

And the guy says to Jeremy,

to hours in a surgery, 
you have to go

hours before your sister.
Didn't even know if you could do it.

You had to be afraid.

What was your greatest fear?

And he said, without hesitating,

that I would wake up
and Ashley wouldn't.

And then they said to Ashley,
you've been sick for so long,

you probably can't remember
what it's like to be well.  What's next?

And she said, I could dream again.

And that is the

spectrum of the rare disease

community, from fear to hope.

And the people of the FDA,

because of the commitment,
because of the leadership and your vision,

the transparency, the let's do it
faster, let's help more.

Your empathy as a clinician.

I mean, and I could say this
because I've known you for so long.

I knew you were a great doctor.

I didn't know if you were a great leader

until I came to work for you.
Oh, we're trying our best.

Yeah, I know you are.
And I really appreciate.

I love serving alongside of you and,

and FDA people, but that's what we're
delivering here is help

and hope, for American people
and patients everywhere.

Thanks, Jim.

Incredible story.

Thank you for sharing and being vulnerable
and doing it here with the camera on.

And I know it's not easy.

I think Elisabeth and I were talking about
how much we were looking forward

to hearing from you.

So it's, it's great. So.

Next time, let's have Gwen.
I like this.

This is a good plan.

I was going to say
the Secretary often says,

a healthy kid has a thousand dreams.

A sick kid has one. 
Yeah.

And it speaks to the sort of yearning
that people have to get better,

and they're willing to do everything,
including advocate and support.

And so that's what you're seeing this week
with Rare Disease Week.

You are seeing that groundswell of people,
you know, on the front lines,

people who are caring for folks
who are suffering with a rare disease,

who are thinking about it,
who are just, believers in this cause,

who are proximate enough to say that
we think this is important.

So thank you for your voices.

Thanks for all of the ideas that we get

from the drug development community
and from the patient community.

And, we're just going to keep going.

So thank you, guys.
Thank you so much.

Good to see you.
Thanks.