FDA Direct: FDA Awards Six National Priority Vouchers

All right.

Bigfoot says we're ready.

You like the audio? Okay, good.

Don't cut that out, by the way.
Keep it in.

All right.

They're trying to make us
two polished here.

Sometimes it's like, let's just be raw
and roll and then do it be ourselves.

So good to see you, Mallika.

Nice to see you.

Yeah. So, how's life been?

You know?

Yeah. Busy. 

Yeah. Busy week. 
Exciting times. Yeah. Yeah.

So, we are talking today
about the National Priority

Review product recipients.

We just announced another batch,
and we're going to keep going.

We're going to announce more

as we get the nominations
from the divisions here at the FDA.

When they see
something that meets us, meets

one of our national priorities
that have been outlined in the criteria

making the price of medications
affordable,

equalizing prices
with other developed wealthy nations.

Meeting large unmet

public health need
or an important public health need, or,

domesticating manufacturing,
which is a national security issue.

And we have said very openly
that we like medications

that reduce downstream
health care utilization.

If there's a medication that's going
to reduce the number of dialysis sessions

or the number of injections
you might need or

avert the need for surgery,
which is one of the products today

we're going to talk about,
then that's kind of a game changer.

And that's, something that I think
we don't talk about enough in health care.

So good to see you.

Nice to see you. 
Ready to roll through the list?

Yeah. Yeah.  
So I feel I feel like we're,

the press release beat us to the punch
a little bit.

Yeah, a little bit. Yeah,
but we get to talk about.

It just because this is our playroom here,
this spot, these chairs.

So we like coming here.

And every now and then we'll be like,
hey, let's just go

talk about this on the camera.

But we get so busy
and it gets hard to coordinate.

And it's kind of our treat for,
you know, getting things done.

And so we were able to sneak out here.

We tried to do it yesterday.

We couldn't.  We got we were able
to sneak out here to get to do this today.

So I'm glad we could do it. Yeah. Me too.

Okay. Zongertinib.

This is a,

product that is produced
by Boehringer Ingelheim, and it's for 

HERlung cancer.

Already approved by FDA
as a second line treatment.

And what's exciting here is that we're
going to explore the possibility of it

as a first line treatment.

So I think that's, that's exciting.

There's been some really good
clinical data.

In in the original approval.

There was, by the way, Bigfoot loves
first line treatments.

Don't you? Bigfoot?

Did you hear that first line treatment?

Well,
do you need her to get closer to the mic

For cancer? 
Good.

Yeah. It's good. Okay, good. Yeah.

And, for lung cancer,
some types of lung cancer, HERpositive.

HER

You know, HERlung cancer, HER
positive lung cancer.

I don't know the ideal nomenclature,
but yeah you have the HER

gene that that

mutation that it's,
that subtype of lung cancer.

Yep, yep.

And, the clinical data from, 
so earlier this year found

that about % of patients
with, previously treated.

So this was for the original indication.

Previously previously treated HER
mutant non-small cell lung cancer

with this mutation had a great response,
even sustained at a year. So,

for this to be approved as a first line
therapy would be amazing.

So I'm glad you mentioned that this has
already approved for another indication,

because in this batch,
much more than the last batch of products

that were announced, there are more
that were previously approved,

than in the prior batch. So,

the question might be, well,

it's easier to do a priority
review, get a decision out in weeks.

Well, that is true.

And that is in part why they're chosen,

early in the first two rounds.

Because, we're piloting this program,

and it's really changing the process
and the structure of the review,

retaining the expertise of the division
and the primary review team.

But it's a new workflow.

Yeah. 
So,

there are entirely novel,

medications that are in the program.

But in this batch,
I think it was just or out of the six,

that are entirely novel.

So, so that's a little background
on that question.

That question.

Somebody asked me that question,
so I thought I would.

That's a good one.
It's a good one to answer.

And just to add to that,
I think that it's important for us

to acknowledge that not all kind of major

transformational sort of advances,
they don't need to be hard, right?

It can be a new population or in this case
it's adding something as first line

rather than second line.
So speeding that up. It doesn't.

We don't need to be making
this more difficult than it needs to be.

And we can accelerate things that,
that, that, that are already approved

for other indications
and it can make sense.

Yeah, yeah. There was that study

earlier this year in New England Journal

that found that % of patients
with previously treated HERmutant

non-small cell lung cancer,

with a mutation in the tyrosine kinase
domain treated with

Zongertinib this medication had a confirmed
objective response.

And so that was a bit of the signal.

Right. Really prompted this. Right.

And this was observed
across patient subgroups.

So, they looked at sex, age, previous
treatment, race, mutation types of this.

This was a robust response.

Yeah, yeah.

Interesting study.

I do recommend people read it if you're,

a medical journal junkie

hobbyist that likes rummaging through,
the medical journals

on interesting studies that are maybe
not in your field of medicine.

Yeah. Always good to read more broadly.

Yeah. Yeah. Okay, great. Bedaquiline.

This one is Bedaquiline.

Yep. For drug resistant tuberculosis
in younger children.

I'm sorry.
I don't know.

Yeah. You said it right. I don't know.

Probably ask the companies
how they want us to say it.

Yeah, yeah.

And I think what's interesting about this,
it is,

you know, people think about TB as being a

something that that impacts
more other countries than the US.

But I was really interested,
as we were learning about this product,

to learn about, that

rates of TB are actually rising in the US,

even though the overall number of cases
is still fairly low.

In according to the national
TB surveillance system,

the estimated number was around
cases in the US,

but that was a %
increase relative to the previous year.

So, it's it's
something we still need to pay

attention to and have those, treatments
available when needed.

Yeah,
I was a little surprised that the division

that nominated this medication
nominated it.

But look, if it can help some young kids
in the United States

or anywhere in the world,
then they're all God's children.

And I hope it does. Right.

And this is for multidrug resistant TB.

So this is these are patients who have,
you know, are not going to respond

to the usual treatments,
isoniazid and rifampin.

Pretty uncommon in the United States,
but around the world much more common.

Yeah.

And with international travel patients
who interact with,

you know, family members
who might be from other countries.

So it's still relevant.

It's definitely still relevant for our,
for our, you know, for our country.

Edgar Allan Poe die of tuberculosis.

Do I have that right?

Oh. Do you know Ben?

If that's. Ben's on it.  He’s checking.  Now, if

I'm wrong, it's going to be embarrassing,
but we don't edit things out.

So you're going to see.

I was just going to advise that we could.

Yeah. Okay. All right. Okay. So,

All right.

Great. Interesting.

Dostarlimab for rectal cancer.
Fascinating.

And that was the medication I was
referring to that actually eliminated

the need for surgery, chemo and radiation
in some patients with rectal cancer.

I think all the patients in that 
New England Journal study.

Or at least right, patients.

Got it. Well,
why don't I let you explain it?

I don't want to say it was every single
patient averted chemo, radiation, surgery.

But the majority the vast majority did.

I think that's right.

I don't think any anyone did
receive chemo, radiation or surgery.

So this is these are really remarkable
results for this product.

That's, that's amazing.

That's literally having a tumor,
a GI tumor

melt away with a complete response.

Or some people know it as a complete
pathologic

response, also known as a CPR.

Not the kind of.

Yeah.

Pounce on someone's chest, a CPR or a

CR in a patient,
a patient with a GI tumor.

I mean, that is, again,
potentially promising.

I don't want to say this is like,


done deal.

We reserve the right to say, look,
the trial didn't meet the standards

or there was a safety issue.

So this is not, my enthusiasm
around the potential promise.

This medication is not proportional
to the likelihood of approval,

but pretty amazing.

If you can melt a tumor away
without the need for surgery, radiation

or chemotherapy.

Especially in the modern era where we are

struggling with a health care,

system
that's broken and an affordability crisis

whereby if we can reduce downstream
health care utilization, as we are

seeing with this med, then we are taking
an entirely new approach to health care.

Right.

And you think about, you know, not
and you have more experience with this

clinically than, than I would.

But the location of cancer
and how that, you know, the surgery in

for that site
specific tumor can impact the patient.

And being able to avoid that for something
like rectal cancer is, is tremendous.

It can be very debilitating to do surgery

in the rectum, very debilitating
in terms of quality of life.

And and it's pretty amazing
surgical techniques now to reconstruct

the rectum or to, to be able to go
lower down with adequate margins. But

look if a

tumor can
melt away and you can easily surveil it,

which is what they did,
keep keeping an eye on it.

That's that's a pretty amazing thing.
Yeah.

This is this is a an exciting advance.

Yeah.

This is a real I mean, every now and then,
you see, real leap

leaps forward with, in medicine. Yeah.

Now this is a PDblocker I think.

Is that right. Yes. Yeah.

All right.

Great.

Anything else you want to say on Dostarlimab?


No, I think I think,

the main thing is as this is phenomenal
initial results in that study.

So great.

Casgevy. 
I don't know if I'm saying it right.

Casgevy.  I'll go with that one. Okay.

This is for sickle cell disease.

And beta cell, a beta thalassemia.

This produced by Vertex Pharmaceuticals.

This is a cell based gene
therapy using the Crispr technology

that's been, you know, in the news
for a while at this point.

But, but it's exciting to, to see it
make its way

into our therapeutics.

I don't know if you want to you want to
say any more about this, but.

No, I just remember
it seemed like there might also be

a potential indication
for a subtype of beta thalassemia.

Is that correct? Yeah. Yes.

Yeah. That's right.

And sickle cell disease, it's it's very

I wouldn't say it's common,
but it does impact certain subpopulations.

And, you know, it's highly prevalent.

Certain subpopulations of the U.S,

and by making this available to,

a broader age range,

we can we can intervene earlier
in the trajectory of sickle cell disease

and prevent downstream complications
that that happen and emerge at later ages.

So this is really exciting as well.

Cool. Great.

Well, look forward to seeing that process
play out.

Now there are two GLP-drugs
that are on the list.

And the reason they're on the list is that

these companies are radically reducing
their

the price of their medication,
making them more affordable.

You know, in some areas of the country,
obesity is a disease of poverty

in areas where there are food deserts
and so many people who may benefit

the most from the GLP
one medications are the ones

that have the biggest barriers to access.

And so by lowering the price
of these medications from over $

to $$

that is a major advance in the ability
for Americans to get these medications.

And by the way,

they've been priced around that amount in
other wealthy countries around the world.

So this was a, really a monumental step
in terms of the affordability.

We're learning
more about GLP-medications

now for other health
related, obesity related conditions,

health complications that people are
realizing, hey, almost every,

disease
process in the body has some interface

with insulin resistance, glucose
metabolism, and general body inflammation.

All three of which are things that are
believed to be reduced by GLP-

Shouldn't be the first line
should be doing other things.

We should recognize obesity
in some patients as a glycemic addiction.

Yeah, just like other addictions.

And so it's hard to go cold turkey.

It's hard to get people to change
their behavior without support or help.

Some people can do it, others struggle.

But just saying exercise more and,
you know,

eat better is does not work for the vast
majority of people.

So we either need more intensive
behavioral therapy or coaching.

But we also need to be able
to have these options available

because we live in a real world
and people are getting into serious health

complications, because of obesity
in a way that is preventable.

They are avoidable. Right?

So that's one, domestic manufacturing

is another criteria met
by these companies.

They're going to be manufacturing
in the United States.

And it is, in a sense,
addressing a large public health need.

So that reduces downstream health care
complications, reduces downstream,

health care utilization.

So essentially meets almost all criteria
to varying degrees.

And just to add to that, you know,
by making these products more available

and accessible within the U.S, we can
now I think there have been

historically there have been some concerns
about safety for

compounded products that or that may
or may not even be include the ingredient

of, you know, that people are trying
that claiming that they, they are,

you know, making available.

So by making this, you know, product
more available,

through the approved pathways,

we're keeping patients safer as well.

So I think this is, this is this is great.

This.

You know, I firmly believe, you know,
I'm fascinated with certain public health

concepts
like the moral hazard and access to care

being a factor in the efficacy
of, of a health care intervention.

So if you have a medication
that is % effective

but can only reach

% of candidate patients
because of the affordability problem

or access issues,
the real population level efficacy is %.

It's half of the %.

So we need to think about access
to medications

and affordability as a public health
community, not as just an agency,

but as a public health community,
in a new light.

And so I think, that was a bit of
the basis also for what you're describing.

And I just want to also,

briefly touch on it's, it's
neat to see FDA explore

its regulatory authorities to help
with, with, you know, affordability.

This is this is not something I don't

I don't think we've really done it
to this extent before.

I mean we've done it with with generics
and trying to to improve that process.

But this the voucher program is a
is definitely

an interesting path to
to make that happen.

You know, it's
interesting early in the discussions

when we talked about
can we legally make affordability

a criteria
for the national priority reviews.

After all,
it is a national priority in health care.

The initial

thought by some was that, well, it doesn't
really fit the traditional model.

But then people started raising
the public health aspect of affordability,

that it is directly
proportional to access,

and that is directly proportional
to the public health clinical benefit.

Yes. So, I do believe that, the FDA
is not involved in setting drug prices.

We don't have a requirement
for a certain price point,

but, when we see major actions
by a company to increase

the affordability, that is a public health
benefit in our mind.

Yeah, absolutely.

Yeah, yeah.

Do you want to mention the other one,

or did we sort of
cover it by talking about GLP-s?

So I think it's worth a mention.

So this is orforglipron.

Also, you know, for the usual

but we've seen indications obesity
and related health conditions.

Eli Lilly and what's novel about this
is that it's oral.

So people
people hate to give themselves injections.

They hate them. I don't like them.
Yeah. No one, no one likes.

No I'm not I'm not saying I take GLP-s.

And if you do, it's okay.

But I don't like injections.

I don't like needles personally.

And not liking something translates
to adherence.

Right.

So you're actually you're potentially
improving adherence

and health outcomes
as a result of changing the formulation.

So this is a big this is a big advance
even though it's the same ingredient

or similar.

Yeah.

You could use the same principle
if you have a medication that is

% efficacious. But

half the

patients who are candidates
don't feel comfortable using a needle.

The medication on a population level
of candidate

patients is really only % efficacious.

So it's the same principle.

Yeah.

And you also think about you know
injection site reactions.

And in this population patients
who have comorbid diabetes.

And that can really you know
injections can translate to infection.

So this is there's, there are a lot
of potential benefits with something as

what might seem as simple
as just a dose formulation change.

It's it's more than that.

Yeah. Again,
not a first line treatment GLP-s.

We want to emphasize
that we're pushing a very aggressive

healthy food agenda
and make America healthy again agenda.

So that's important.

Anything else
you want to say on this stuff?

No, I think, it's an exciting
second batch.

Yeah, it's a great second batch. Good job.

Great job with this program.

And to all the scientists here
and managers and people handling

the workflow that are willing to try
something new in this pilot program.

It's it really warms
my heart to see them excited about it.

And them say, hey,
we've got this medication in the pipeline

that we think
might be a good candidate for this.

So it's just it's fun to see that, chatter
in at the agency here.

So, Ben was I right about Edgar Allen Poe?

Mother died of tuberculosis.

I don't know if he died of it.

Interesting.

Yeah. Okay.

Probably, though maybe. Maybe.

Yeah, it's a good guess.

Yeah.

I'm curious if,
maybe it was around the same time,

but I think, you know,
he was in Baltimore.

He had a home near Johns Hopkins.

So, there's a little interest in Edgar
Allen Poe

at, at, in Baltimore
because he's a he's a Baltimore guy.

He was not a Ravens fan because the Ravens
were not there at the time.

Although there was a Raven,
I think, in one of his stories.

Wow. Is that. Right?
That's right. Yeah. Yeah.

Interesting. Yeah.

That's my
that's my common knowledge for the day.

For people or people who.

Thank you.

You've had many contributions
to this conversation.

But that's that was a good one.

So, the Ravens,

name is interesting because
I don't want people out there to think

that Baltimore just has a flock of ravens
constantly circulating around it.

It's like the Buffalo Bills.

Like, I don't think that Buffalo's.

Maybe one at one time.

There was probably.

Yeah, yeah, but not now. Broncos.

Maybe that maybe there's
there are Broncos.

That area.

Although Broncos I believe
are not native to the United States.

Oh.
Yeah.

I'm not going to make you fact check
that one Ben. 

I might be very wrong on that.

And Bigfoot's laughing, which is good.

We're going to get Bigfoot on set.

People say we don't know
if we believe in Bigfoot.

Yeah. And I say, I get it.

You don't see his face on the on the set.

But at some point
we're going to invite Bigfoot to come on

camera and show his birth certificate.

You can see it probably says Bigfoot

on the birth certificate,
it is the name that it goes by.

So are you pleased? Bigfoot? Yeah.

I have one question.
GLP-’s not first line.

I know you like first line medications.
Second. Yeah.

Second or third. Yeah.

All right. I have one question.
Behavioral interventions.

Before we end this
to get you back for the last podcast.

Okay.

Are you more afraid?

Oh, boy. 
Of spiders or snakes?

Snakes. Okay. Talk about that.  For sure.

Well,

sometimes when my golf

ball goes far into the rough and,

of course, in the Midwest,
United States, I'm looking for my ball,

which has a market value of about $.

And I keep hunting for it, hunting for it.

And I can hear some snakes sometimes,

and I perceive
that I should still look for my ball.

And then I do a Pascal's wager of the risk
benefit

ratio and recognize
that what I'm doing is insane.

I'm trying to find my ball.

In order and taking serious risks. So.

So it snakes, hands down. Snakes for sure.
Yeah.

Okay. Yeah. I'm not afraid.

Not afraid of spiders at all. Wow.

Not even tarantulas.

Not even tarantulas.

I've never seen one.

Okay.

Outside of a zoo or something like that.

Yeah, okay.

We're even now. Okay. Yeah.

Good. We're even. Keep coming.

All right. Good to see you.

Yeah. Mallika.  Thanks for doing this.

I hopefully this was.

You enjoyed this as much as we.

I wanted to say
hopefully you learned something,

but some some people may decrease
in knowledge from hearing our banter.

Oh, that's okay, that's okay.
We're just having fun.

This is therapeutic for us.

Take a little break from a busy day.

So thanks for joining us.

And we’ll do it again. Bye.