The public has been hearing for months about the clinical trials conducted by various institutions and pharmaceutical companies related to the COVID-19 pandemic, but what does it mean to be involved in a clinical trial, and why would the public want to participate? What is the importance of clinical trials, what are the safeguards surrounding any clinical trial, and what do clinical trials offer the public?

Dr. Patrick Flume, is a professor of Medicine in Pediatrics with specialties in pulmonology and critical care medicine at the Medical University. He serves as the Powers- Huggins endowed chair for cystic fibrosis, and is the director of the Adult MUSC Cystic Fibrosis Center. Dr. Flume is heavily involved in MUSC's research enterprise, including the way MUSC manages clinical trial safety and protection of all those engaged in any clinical trial. Dr. Flume, along with many other clinical scientists at MUSC, has been instrumental in several very successful clinical trials, most recently the implementation and success of the COVID-19 trials.

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Hello, everyone, and welcome to a very special

Science Never Sleeps conversation.

I'm your host Loretta Lynch-Reichert

at the Medical University of South Carolina.

Today our guest is Dr. Patrick Flume,

professor of Medicine in Pediatrics

with specialties in pulmonology and critical care medicine

at the Medical University.

He serves as the Powers- Huggins endowed chair

for cystic fibrosis,

and is the director of the Adult MUSC Cystic Fibrosis Center.

Dr. Flume is heavily involved in MUSC's research enterprise,

including the way MUSC manages clinical trial safety

and protection of all those engaged in any clinical trial.

Dr. Flume, along with many other clinical scientists at MUSC,

has been instrumental

in several very successful clinical trials,

most recently the implementation and success

of the COVID-19 trials.

The public has been hearing for months about the clinical trials

conducted by various institutions

and pharmaceutical companies

related to the COVID-19 pandemic,

but what does it mean to be involved in a clinical trial,

and why would the public want to participate?

Today we discuss the importance of clinical trials,

the safeguards surrounding any clinical trial,

and what clinical trials offer to the public.

It is especially important

to understand why we do clinical trials,

and why it is of the utmost urgency

to engage our public.

Beginning this month,

MUSC is changing its approach

to contacting patients about research.

You will soon notice an added emphasis

on educating our patients about research opportunities

and empowering them to participate in studies.

This means that researchers at MUSC

will be taking a more active role in contacting patients

to inform them of study opportunities

that might be of interest to them.

So, Dr. Flume,

MUSC recently rolled out a new plan

for contacting patients about research opportunities.

Can you give us an overview of this plan

and why the institution has decided to go this route?

Thank you, Loretta.

As you said, MUSC is engaged in a lot of research,

and especially clinical research.

These studies, these clinical trials

are geared towards trying to define

novel therapies or devices

that can treat a wide variety of conditions.

There are a lot of reasons why people might

choose to participate in trials,

and there are reasons why they might choose not to,

but one of the largest most frequent reasons

that people choose to not participate

is because they didn't know about it.

They didn't know the opportunity existed.

And so, if we're trying to do a better job

of empowering our patients

to know what's available to them

and to see if they might be interested in participating,

then we've got to find a way to communicate that with them.

What we have done is

changed our ability for investigators

to know who might be eligible

and allow them to reach out directly to those folks.

MUSC research teams

can reach out directly

to those persons that are in our system

who have been identified as being eligible,

and those people can now hear more about the study

and decide whether it's appropriate for them,

or whether they have interest in moving forward.

Actually, a very good thought.

I know several people have come up to me

in the public and said, "Well, I have this issue

or that issue, and I'd really like to know

how I can be part of any clinical trials at MUSC."

We're hoping that this podcast will certainly offer

the information that folks will need

to make those decisions.

I want to also stress one thing,

and it's a particular reason

why we have you on the podcast today.

I would like folks to understand

your expertise and your oversight

of clinical trials at the Medical University

of South Carolina.

Can you offer folks a little understanding

of what goes on behind the scenes

when an investigator or scientist

is looking to do a clinical trial?

What kind of challenges do they face?

Or, what kind of

protocols, regulations do they need to meet

before they're even allowed to consider a clinical trial?

Ultimately, for a new therapy--

let's say with a new medication

to be approved by the FDA,

to be approved by federal agencies--

you have to demonstrate that that product

is both effective-- meaning it has the benefit

that you thought that it should,

whether it's to improve symptoms

or improve survival--

but also that it's safe.

So there are ways in which you can measure

that a therapy is safe for an individual,

but also the way the study is conducted.

There are a number of guardrails that have been put into place

at the federal level.

The FDA has to grant approvals

for companies or investigators

to study the product

to know that they've proven, to their satisfaction,

that it is safe--

whether it's through pre-clinical studies

in the laboratory,

to early phase studies looking for safety issues.

Essentially, those protocols get approved in a way

to suggest that if they do that study in that way

that would satisfy the regulatory bodies

in terms of their approval.

But then at the local level,

every institution will have an ethics board,

an institutional review board,

whose mission is to protect the safety

of the persons who might be contacted

or participate in the study,

and to monitor those issues.

They look at what the protocol

intends to do,

what the product is supposed to do.

So, when you talk about safety,

it's not merely whether the drug or device

does any harm--

causing liver injury, or other symptoms--

but also does the study itself protect--

is it being done in a way to protect them?

For example, you don't want to draw too much blood

from a person, or subject them to anything

that might put them under undue duress.

At our institution,

our institutional review board

is an independent body

whose task is to review all those materials,

and then if it all seems to be okay,

to grant approval.

But even then,

the investigative teams have to follow those rules

to make sure that they don't

step outside of them.

For example,

if you were being invited to participate,

that we go through the consent forms

so you understand what is going to be done during the study,

what we're looking to learn from this,

and to show how we intend to protect you.

We are also very careful about protecting the information

about those individuals.

It's important to know what the results are,

but it's not terribly important to know who the individual is,

so we try to maintain their confidentiality.

To ensure that, we have a compliance program

that is available to review and assure

that everyone is following the rules

as they're supposed to.

Very comforting.

So, how does a study team determine

if a patient is eligible for a particular trial,

and where are these team members getting

the patient contact information?

For every study,

you have to define

who it is that you intend to invite into the study

and who would not be eligible for the study.

For example,

as you mentioned I run the Cystic Fibrosis Center,

and so I might be doing a study

that is intended to look at a drug

for the purposes of treating complications

related to cystic fibrosis.

So, certainly we want to invite people who have that diagnosis,

but then maybe we're looking particularly for people

whose lung function is not severely impaired,

or they don't have other reasons that would make them

not a good candidate

because they're a little too sick, perhaps.

So you have these, what we call, inclusion

and exclusion criteria.

For some studies,

it's pretty easy to know who you're looking for.

Again, all the cystic fibrosis patients at MUSC

come to our center,

so we know who they are.

But then there is, perhaps,

a study of diabetes or asthma

in which there might be a much larger number

of persons that are seen at MUSC,

but are not confined to a specific clinic.

We have the ability

to look in our electronic medical record

and identify people who might be eligible,

who might fit those criteria.

That then would allow a study team

to look at those individuals

and, if deemed appropriate, to reach out to them

to invite them to learn more about the study

and perhaps choose to participate.

I think it's important to note

that not everyone can look at

clinical records.

I certainly cannot.

You have to be authorized.

It is well-monitored,

and only those people engaged in that research,

if I'm correct,

are given the opportunity

to review those clinical records.

Is that correct?

That is correct.

If I want to get a list of people eligible for a study,

I need to go through our ethics board

so they understand why I might be interested

in getting that information,

and how I intend to use that information.

It's all, again, about protecting the individuals

to make sure that there's nothing malicious being done.

Excellent.

What if someone decides that

they don't want to be contacted about research opportunities?

How will that be managed?

There are people who

have zero interest in participating in trials,

and that's perfectly okay.

It may be that they get contacted about a study,

and the study just isn't right for them,

but they might still want to know about other studies.

So, we want to make sure when a person says no,

what is it they're saying no to?

If they do not want to be contacted,

if they don't want anybody at MUSC reaching out to them,

we have a number of methods by which that information

can be put into the electronic medical record

so that their name will not show up on any lists.

They can tell the team member,

if one of my staff members reaches out to you

as a potential participant, and you said, "I don't want to,

and I don't want anyone calling me,"

then our study team can enter that information

into the database, and it gets updated immediately

so that no other study teams

would be reaching out to that individual.

Also, a person can go into MyChart

and edit their research contact preferences.

We've tried to set up as many ways as possible,

and as easy as possible,

for people to communicate their preferences.

So, Dr. Flume, with the COVID-19, of course,

the whole issue of bringing in--

being as inclusive as possible in clinical trials

for the different vaccine drugs

has become very apparent.

I know researchers are hoping to see an increase

in patient diversity in all their clinical trials.

Could you talk a little more about that

and why it's important,

and if you have any suggestions

on how to increase that diversity to make sure

every potential person who could benefit from

a clinical trial, therapy, or device

has that opportunity?

When we are developing

a treatment for a condition,

we can think about all the different people

who would benefit from that.

So, again, if I think that

I'm developing a drug to treat cystic fibrosis,

doesn't mean that everyone with CF

is going to benefit from that,

but let's just take, for example,

that that's the target group.

But I can't study everyone who has CF,

so what I'm going to do for my study

is I'm going to try to recruit

a sample of persons with CF

that seem to provide the most information

for the general population.

What we mean is

the people who are in the study

best represent the persons who will likely be treated.

I'll use another example.

If I'm going to do a study

of a treatment of lung cancer,

and I enroll all these people

who are 40 and 50 years old

with lung cancer,

but actually the people who get lung cancer

that would eventually be treated

are more likely to be in their 60s and 70s,

then we might not be as comfortable

that what we learn in those younger subjects

will apply to those who are older.

So, when you think about something like COVID--

so, this virus is affecting everybody.

It's affecting men and women.

It's affecting people of all races and ethnicities.

It's affecting younger people and older people.

In terms of the sequelae,

in terms of the outcomes from that,

it seems to be harder hit in older individuals

and also people from the Black community.

If we want to have a therapy

to treat these people, to protect these people,

like with a vaccine,

then we want to make sure that we enroll

subjects that match those people.

We want to enroll in the vaccine trials

the young and the old,

men and women,

people who are Black, White, Hispanic,

so that when we look at the results,

we have greater confidence

that it will be representative and be effective

in those populations.

How do we do that?

Well, we try to communicate

with the community

that this opportunity exists.

We can do that in a number of ways.

On the one hand, to try to be in the media--

so whether in the newspaper, or the news media--

to try to convey that these studies are ongoing,

to try to reach out to leaders of different communities

to inform them about the positive aspects

of the vaccine

and why people might want to participate.

Then, we also want to try to inform people

about the questions they're likely to have.

There are people who are just nervous about the safety

of something they think is so new,

or that they think has come out too fast.

There are populations that are a little distrustful

about engagement and research.

So, we try to inform them of the safeguards

that we've put in place,

to the best of our ability,

to show that these vaccines are in fact safe--

we still have to measure their safety--

and that, in terms of those that we're inviting to enroll,

that we are looking to protect them.

The more knowledge people have, the better off they are.

Oh, I could not agree more.

In regard to this new process where

patients can opt out

but might be contacted otherwise--

I know that the folks rolling out the process

here at MUSC have sought feedback

from the community leaders and members of the public.

Can you talk more about that,

and also what you are doing to make sure people are aware

of this upcoming change?

We have to appreciate that there can be some sensitivity

about people being contacted

by people they don't know,

and try to understand what they're being asked

to participate in.

We want to make sure that we address their needs,

and their worries, their fears.

We've taken a number of steps

to try and make sure that we've asked the right questions

and try to develop strategies around that.

We held focus groups

using members of the public and community leaders.

We asked for feedback on the process itself,

not just the concept of reaching out to people,

but how we're actually managing it,

how best to communicate that.

Through their advice, we've made some changes

to the processes.

We're following their advice on how best to message this--

so you'll see messaging about the policy in local papers,

interviews, commercials,

banners and screens throughout the MUSC campus.

There will be continuous opportunities

for people to give us feedback--

whether it's communicating directly to a research member

who's contacted them,

or coming through our research contact preference coordinator.

We have regular meetings to address these comments.

I want to add that

it's a privilege for us to be able to do

this type of research.

We couldn't do this without engagement

from our patients.

We try to treat our volunteers

with enormous respect and gratitude.

It's our job to make sure that this research participation

is safe, efficient,

and that they have a positive experience,

that a person who finishes a study says,

"You know, I might do another one."

I want to say just a couple of things about

our volunteers.

It's a safe bet to say that every one of us

has taken a medication at some point in our life--

whether it's over-the-counter,

or a prescription medication.

What we should never forget is that

someone volunteered for that.

We have to respect that.

We make sure we say thank you to everyone who participates.

I have a great deal of respect for all of them.

You actually just took the words right out of my mouth.

I was going to suggest that

if it wasn't for clinical trials,

our medical care wouldn't be at

the state-of-the-art that it actually is.

One of the reasons why we do these podcasts,

Science Never Sleeps, is indeed to inform the public

of what a great opportunity and resource

they have at MUSC.

People of your integrity, your competence,

your compassion,

those are the folks that they'll be dealing with.

People who care, who keep asking the questions,

and who want the answers,

and they want to make that difference

in the lives of our citizens,

and hopefully nationally as well.

I think that's really important for folks to know that

you do what you do,

on top of everything else you do,

and so do your colleagues because you care,

and you want to make a diff-- you want to find the answers.

If you just wanted to be a physician,

you could go to any hospital and do that.

But you come to MUSC as an academic health center

to make that difference for all.

That's why these clinical trials are so very relevant to us.

In our final moments, let's pivot a little

and talk about the importance of research participation

when it comes to beating this coronavirus pandemic.

We just recently finished recruiting participants

for one COVID-19 vaccine,

and now we will begin conducting two more vaccine trials.

The question is,

why is MUSC still actively engaged in vaccine research,

even though Moderna and Pfizer

are already approved for emergency use

and starting to be rolled out to the public?

I think, as everyone sees in the news,

that the rollout of these vaccines

is just not where it needs to be,

that although we're vaccinating at a greater rate

as time goes on,

we're woefully short of

the numbers that we need to be vaccinating.

Yes, we have an authorized use for

the Moderna and Pfizer vaccines,

but that's not going to be enough.

Even if they can get to their full commitment of

100 million doses, that's not going to be sufficient

to vaccinate this country.

They cannot increase their production

to meet the needs of this country,

as well as the rest of the world.

So, we need more vaccines.

Yes, we just finished enrolling the AstraZeneca study.

Those subjects are still participating in the trial

because it's a long-term follow-up,

but we have completed the vaccination

in those subjects.

Then, we recently launched two more vaccine studies.

The Janssen study started two weeks ago,

and then this last week, we started the Novavax.

We will be recruiting subjects

for both of those vaccine trials

until we finish that enrollment.

I will add that all of these studies

are enrolling adults

to try and protect them,

and people are calling saying, "When are you going to do

the pediatric population?"

We do have studies that we are looking at

that are intended to start sometime in the near future--

one for women who are pregnant,

and a couple studies for the pediatric population,

including adolescents

and even younger.

It just doesn't move fast enough.

We're working as hard and as fast as we can.

We understand the need, we understand the desire,

and we'll keep plugging along as quickly as we can.

That's great, and I also wanted to highlight one thing,

at least in my understanding,

and you can certainly correct me.

It's important not only to vaccinate,

as you suggested,

the entire population of the United States,

but since we are a global economy

and a travel-hungry world, it's important to make sure

every other nation gets vaccinated,

or that we could have some peril.

Is that true?

That is true.

I encourage people to think that we don't try to vaccinate

the other countries of the world

just because we're being good citizens.

That actually is for our protection as well.

We do want to get back to the global economy,

and desire to travel and visit places,

and they want to come visit us as well.

So, we want to try and do our best

to rein in this pandemic across the globe.

Great point.

Can you touch briefly on some of the benefits and challenges

to MUSC being a site

that's running multiple COVID-19 vaccine trials?

The benefit for MUSC

is really just-- this is what we do.

This is what we're supposed to do.

This is our mission.

If not us, then who?

We need to be able to bring that to everyone--

The benefit for the individuals

is that people have access to these vaccine options,

even before they get authorized

to be available to the general public.

Participating in the study, they are placebo-controlled.

So, yes, there is a chance they will get the placebo.

But as we saw in the AstraZeneca study,

we were able to break the blind and allow

many of the subjects to get

the approved vaccine afterwards.

The challenges, when we do have authorized use,

it does create some havoc

to how we have to communicate with people.

Running these studies,

it has been quite an operation.

I have to give kudos to all the teams

that came together at MUSC to pull this off

because it's not just one small team,

it's multiple teams,

and a lot of people who are trying to

bring subjects in in an efficient manner,

to do it in a way where we don't pack everyone together.

It's like running a busy restaurant.

Everyone has to work well together.

When we're seeing people coming in for their vaccine,

we're also still seeing people who come in

for follow-up visits.

As we continue to enroll people,

there's days later that we have

many, many more people coming back in.

We meet on a regular basis to talk about

how we can keep things moving and efficient.

We are grateful to the hospital

who have been very gracious to help us

with our testing sites

and meeting the needs to try and get folks

into the system.

If there's one pleasure for me,

it's been watching a lot of

smart and talented people work well together

-to get this job done. -Absolutely.

Do you have any final thoughts or messages

about vaccine trial participation?

No, we are grateful for all those who volunteered.

We encourage people to let us know that they're interested,

and we will work our hardest to get you in as soon as we can,

as we do with all of our trials.

Anytime there's new information,

we get it out to those

if it might change their thinking about this.

I want to make sure that folks know

that we have several sites at MUSC

for you to look at

to see what kind of studies

you'd like to participate in.

We have muschealth.org/trials.

We have the scresearch.org for trials,

and we will put all that information out

to our public when we broadcast this podcast.

There are other ways for people to learn about trials

rather than just waiting to see if they might

show up on someone's list and get contacted.

There are a number of websites,

some that are specific to MUSC--

that's the muschealth.org/trials.

To understand about trials that are being run in the state,

that is the scresearch.org site, so they can learn about

other facilities that are also conducting trials

because they might be interested,

and there's a closer facility for them.

The volunteer registry is the researchmatch.org,

where they, if they say,

"We would be interested in being contacted

if there is a study to learn more."

This whole process is about empowering people.

They can choose not to participate, that is fine.

If they want to participate,

we will do our best to try to get them engaged.

It's really just making sure that people understand

what the opportunities are.

We're very grateful.

And again folks,

you can't do this without an excellent

academic health center, which we have

right here in Charleston, South Carolina

that is available to all of our citizens

and anyone else interested in knowing more.

Dr. Flume, thank you again,

as always, for your generosity

of heart and time and helping us out.

Thank you.

Thank you to all of our listeners

for your continued support.

Please join us each month

for the latest MUSC research conversations

on topics that make a difference in your lives

and the healthcare you receive.

In the meantime, stay healthy and safe.

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