Blindness and Clinical Trials Transcript

Thank you for attending our Blindness and Clinical Trials Webinar on Friday, October 13! Please read on for the full transcript of the webinar.

Welcome to our blindness and clinical trials. My name is Alexandra Luzir. I am the vice president of business development and communications with Accessible Pharmacy Services.

 

And we're so excited to have you all here. And we are super excited to have all of these incredible presenters.

 

Speaking with us today about clinical trials in the blindness and low vision community. I really, really do want to thank everybody for being here with us today.

 

We had close to a thousand people register for this webinar, and we are so grateful for the positive response.

 

For those of you who are joining us for the first time, we are accessible pharmacy services and we are a home delivery pharmacy that specializes in individuals with disabilities.

 

We are the only provider of its kind and we are the largest blind owned healthcare company in the country.

 

We are the pharmacy choice. For individuals across 32 different states and we provide accessible medication management and diabetes management to our patients.

 

We've done a few of these webinars in the past, so if you're joining us again, thank you for coming back.

 

We've done them on a variety of different health care topics from mental health to eye drops to diabetes, glaucoma and breast cancer.

 

And they've all been recorded. They're all available on our website. Accessible Pharmacy.

 

Dot com and they're all available also on our YouTube page by searching accessible pharmacy. Throughout this webinar, Jenny and Holly will be here to supply us with ASL interpretation, so I want to give a huge thank you to them.

 

Yes.

 

And this webinar will also have live closed captioning. After it's over in the next few days, we will be sending out an email that will have links to the webinar.

 

Video and audio recordings as well as the transcript and some resources from our presenters. And also when you get that email, please respond to it with any feedback that you have on the webinar.

 

Which we really, really encourage you to do. So we love hearing from you about feedback from the webinar ideas for future webinars and please, please go ahead and respond to that email.

 

Before we get started, I'm just gonna run through some quick announcements and then we will dive right into the webinar.

 

Okay.

 

To start October is breast cancer awareness month breast cancer is really important to us here at accessible pharmacy. Last year we put on an incredible webinar on breast cancer information and breast cancer health for women who are blind and that was recorded and is available on our website and our YouTube page.

 

We also have a standalone video titled How to Do a Brest Self Exam. That video is narrated by Dr.

 

Kim Kubek. She's an FDA certified radiologist and she serves on the board of breast cancer.

 

Org. That video, the how to do a breast self exam video. It has closed captioning.

 

It has an ASL interpreter on it. It's a wonderful. Resource and it can be found on our website and it can also be found on our YouTube page.

 

I'm also really excited to ask you to register for our second annual blind health expo. The expo is completely virtual.

 

It's a chance for individuals and companies to come together. To talk about different health care information products, services and medication for individuals in the blind and low vision and disability communities.

 

The expo is on Friday, December, the first. It's from 10 a. M. To 4 p.

 

1. It's our second year doing it. We're really really excited about it and we would love for you to join us this year for this year's Expo we are kicking off our first ever Vision Healthcare Awards.

 

We are acknowledging leaders in the blindness and low vision field that just do amazing work in this community.

 

So.

 

And we're going to be announcing those winners next month in early November. And then we're going to be showcasing their achievements in December at the Blind Health Expo.

 

So please join us for the day. It's free to register and you can find that registration link on our website.

 

If you feel like you would like to exhibit, we would absolutely love to have you. If you are far of an organization or a company that's involved in the blindness or low vision space, please consider exhibiting.

 

It's free to exhibit if we would absolutely love to have you, you're able to talk.

 

To attendees throughout the day, host presentations, showcase your products, services, talk to other organizations, other companies.

 

It's really a great fun day to just virtually speak to other people. That are in our community.

 

That exhibitor registration link is also on our website page and we have also have an exhibitor information session coming up.

 

On Wednesday, October, the 20 fifth. So if you're interested in learning more, send us an email at info I NFO at accessible pharmacy.

 

Dot com or if you have any questions about exhibiting or attending, please just send us an email as well.

 

And lastly, if you are an organization that works with patients in the blind and low vision community and would like for us to train your staff about medication and diabetes management.

 

We are scheduling training sessions in the winter in the spring and we would love to train your team. Please just send us an email to start the process.

 

And then also if you would like to become a patient of ours here at Accessible Pharmacy, we would love to have you.

 

As I said earlier, we are the pharmacy choice for patients across 32 different states. So if you would like to get started with us, we and join our community, we would love to have you.

 

It is a really, really simple process and it just starts with a conversation. So just give us a call at (188) 633-7007.

 

Now, I am going to stop talking. Those are all of my announcements and we can finally dive into the webinar.

 

It is my absolute pleasure to introduce our first speaker for today. Ben Shaberman is the vice president of science communications with the foundation fighting blindness.

 

He hosts the I on the cure podcast. He has written 3 books and he has been communicating retinal disease, science and research for the Foundation finding blindness for almost 19 years.

 

He does absolutely amazing work and we are huge fans of the Foundation finding blindness. Personally, one of our co-founders, Dr.

 

Alex Cohen, our chief marketing and accessibility officer is actually the president of the Philadelphia affiliate. So we are personally really grateful for the foundation and we love all the work that they do and we are so thankful that Ben is here today to share about the foundation and their latest news and research.

 

So please Ben, go ahead and take it away.

 

Right. Thank you, Alexandra. We're a big fan of accessible pharmacy as well.

 

So that's mutual and thanks to everyone for taking time out of your data. Learn a little bit about research for retinal degenerative diseases and everything else you're going to learn about from the other speakers.

 

So a little background on the foundation fighting blindness. We are a global leader in driving research for retinal degenerative diseases.

 

Namely, red night is pigmentosa. Usher syndrome, Stargart disease, age related macular degeneration, labor congenital amirosis.

 

The list goes on and on. We've been around for more than 50 years. We've raised nearly 900 million dollars toward our mission of eradicating these diseases.

 

And we're really at an exciting juncture in terms of research advancement. It's really over the past 10 to 15 years.

 

That thanks in part to our funding, support from our donors and commercial interest. We've been able to move much more research into human studies.

 

Into clinical trials. And now we have a about 45 trials underway for emerging therapies for the conditions that I mentioned.

 

And an important role of the foundation is to move research to the point where we can attract outside investment from the commercial sector to move treatments through clinical trials across the finish line.

 

Clinical trials are really expensive. We were to move things into clinical trials and then again attract the outside.

 

Investment and with more than 40 companies invested in our space. We're excited about the progress. There's definitely a lot more work to do.

 

But we have great momentum. So I wanna start off talking about some gene therapies that are in clinical trials.

 

Including a gene therapy that's FDA approved. That's where I'm going to start.

 

But just a quick definition of gene therapy. A lot of the conditions that I mentioned, virtually all of them except AMD, are caused by mutations in a single gene.

 

And once somebody learns, somebody affected by a disease learns what that mutated gene is if there's a gene therapy in development that may apply to them.

 

And excitingly, back in 2,017, the first FDA approved gene therapy. Across the finish line, that's called Luxterna.

 

It's for people with RPE, 65 mutations. And in clinical trials and then after it was approved, it provided.

 

Significant vision improvements to people who had this mutated gene and most of these folks had labor congenital amyrosis, very severe vision laws, some had RP.

 

And thanks to the gene therapy, they were able to put away their white canes, many of them.

 

They could see the faces of loved ones. Some could even see stars in the sky and fireflies.

 

And the company that ultimately, Commercialize this therapy, Spark therapeutics.

 

Was eventually acquired by Roche. And it's really exciting to see the commercial interest now in gene therapy.

 

Well, we're very excited about. What it did for the patients who received the gene therapy, it provided affirmation to the research community that gene therapy was in fact an approach.

 

To treating these conditions a very potentially effective and safe approach. And now we have 3 companies in clinical trials.

 

For gene therapies for X-linked retinitis pigmentosa. Mir GTX with their partner Jansen, Beacon Therapeutics, and 40 molecular therapeutics.

 

The first 2 companies I mentioned I have shown vision benefits with their gene therapies. For XLRP, Jansen is in a phase 3, beacon is in a phase 2.

 

And we're hoping that at least one of these makes it across the finish line. Excellent DRP in the retinitis pigmentosa world is a fairly common form of RP it it's often caused by mutations in the gene RPGR.

 

And, again, we're excited about the progress that's being made there. Another company that we're heavily invested in is called it Cena therapeutics.

 

It was founded by Shannon Boy, a gene therapy pioneer out of the University of Florida.

 

They have a few different gene replacement therapies in in their pipeline. They recently reported results for labor congenital amyrosis one that that clinical trial that disease is caused by mutations in GUC, Y 2D.

 

People getting the highest dose in that trial. Had vision improvements they were better able to navigate a mobility course and they had improvements and retinal sensitivity.

 

So we're very excited. They're talking to the FDA about moving that treatment forward.

 

They also recently launched a clinical trial for people with excellent retinoskees. That's a pretty difficult retinal disease.

 

It causes splitting of the retinal layers. That gene therapy is again just moved into a clinical trial.

 

They're dosing patients. And they're also working on advancing a gene therapy for usher syndrome type one B.

 

And that's a challenging, Jean, the, Miles 7 A, the gene that, that when mutated causes usshorn because it's big and they have to actually deliver it in 2 halves to, get it into the retina.

 

So that is not yet in a clinical trial yet, but they're making good progress there. So everything I talked about is gene replacement.

 

It's replacing the bad gene. With a good gene. Now you can use gene therapy for other purposes and one of those purposes is called neuroprotection and the idea there is that you're just trying to preserve.

 

The retinal cells independent of the mutated gene. And a company that we're also invested in called Sparing Vision.

 

They're a French company has just launched a clinical trial for a gene therapy that's designed to preserve cones.

 

And it could potentially work. It's gene agnostic. It could potentially work for people with retinitis pigmentosa, Usher syndrome and some other conditions regardless of the mutated gene causing the disease.

 

The idea is to preserve cones and cone vision. Cones give you the ability to read and recognize faces and perceive colors and see and lighted conditions.

 

And we're very excited that this is just moved into a clinical trial in Pittsburgh. And in Paris.

 

Now, another gene agnostic gene therapy. Approach that we're very excited about is called optogenetics.

 

Now for people who have lost all of their vision to a retinal condition. Usually they've lost all their rods and combs.

 

Those are the cells that make vision possible. And a lot of therapies aren't going to help those people because there are no rods and cones left to save.

 

Well, researchers observed that there are other cells in the retina that survive after rods and cones are gone and many people.

 

Those are called bipolar cells. And ganglion cells. And what these very.

 

Clever researchers have done is they've designed a gene therapy to make the bipolar cells or the ganglion cells work.

 

Like rods and cones like photoreceptors. It's like using these cells as a backup system.

 

For lost photosceptors. Now bipolar cells and gangling cells normally don't respond to light.

 

But that's what optogenetics does. And there are 3 companies in clinical trials for this approach, gen site, bionic site.

 

And nanoscope all have reported some rudimentary vision improvements for people who are getting optogenetic therapies in their clinical trials.

 

Some can see shapes and movement. They're able to navigate, Let's say a mobility course a little more easily.

 

A fourth company called Kiara. Is doing the same kind of thing but not with the gene therapy with a chemical with a photo switch.

 

And they've reported light perception for their patients as well. It's still early in optogenetic development.

 

There are some more promising approaches that are moving toward clinical trials. And we're just very excited about this space because it provides hope for people with the most advanced vision loss and again it's gene agnostic.

 

And finally, I just wanted to close out talking about cell based therapies. And just to be transparent here, cell based therapies have not moved quite as far along.

 

As gene therapies. But one trial that we're very excited about for people with advanced try AMD, that condition is called geographic atrophy.

 

The trial is taking place at the National Eye Institute. And in people with GA. Again, geographic atrophy.

 

They lose a layer of cells called RPE cells. And these cells are really important for supporting rods and cones.

 

And what they're doing at the National Eye Institute is they're transplanting new RPE cells in for these patients in the retinas of these patients to try to preserve.

 

Those rods and cones. Now what's really cool is the way they're getting these RPE cells.

 

They're taking a small sample of blood from each patient. They're genetically tweaking those cells.

So they revert to a stem cell like state. And then they're coaxing those stem cell like cells.

 

Forward to become RPE cells. So if you didn't quite follow that, the bottom line is they're taking blood and they're making retina out of it.

 

And the real advantage there is that the patients are their own cell donors. So that really limits. The possibility of an immune rejection.

 

They're putting those cells on a scaffold to help their orientation and survival and their transplanting them into the patients.

 

So that's a cell based. Therapy that's in the clinic. It's still at an early stage, but we're very excited about it.

 

There are many, many other trials and and promising research projects under way you can learn about those by going to fighting blindness.

 

Dot org. That's the foundation fighting blindness website. Clinical trials.gov is also an excellent resource for learning about any clinical trial for any condition.

 

I, whatever part of the body. It gives a listing of all trials that are underway in the US and many that are overseas.

 

And with that, I know I've. Good. A lot of information out there. But again, fighting blindness.

 

Dot org to get some more details. And thank you to Accessible Pharmacy and everyone out there for the opportunity to talk about what's going on in retinal degenerative disease research.

 

Awesome. Thank you, Ben. All the links that Ben discussed, fighting blindness.org, clinical trials.

 

Dot gov. Those will all be linked in our email and on our website after the fact.

 

I would like to mention fighting blindness and all the work that you all do across the country is absolutely amazing.

 

Like I said before, we personally love a foundation fighting blindness. One of our co-founders is one of the chapter presidents.

 

We were just at the vision walk in Philadelphia. This past Saturday, it's, it's a wonderful organization and we are personal, huge fans of what you do and what the foundation does.

 

Thank you for sharing all the updates and how the clinical trials process works with the foundation and thank you for being here, Ben.

 

Thank you.

 

Of course. Next, I'd like to introduce Linda Harris. Linda is an amazing member of our community and we actually got the idea for having a clinical trial webinar from her.

 

So she's going to spend a few minutes. Sharing her experience being enrolled in a clinical trial and then she's going to present our next speaker.

 

So please take it away. Linda. I also I'm going to share your photo, Linda.

 

Okay, great. Thanks, Alexa, and thank you and Andy for having this. Webinar, obviously.

 

It's a it's got a wide audience and and really an important important topic for people to understand what clinical trials are about and how they can participate in them.

 

And Ben, I was fascinated by your research. It's very, it does provide a lot of hope for those of us who are at risk of losing our vision.

 

So I want to tell you my, my story. Oh, before first first thing I want to do is to do a shout out for the Expo.

 

I attended accessible pharmacies expo last year. And it was truly amazing. I'm a big supporter of people, people with all kinds of disabilities and One of the things I've learned is that when you design a kind of a universal design for people with disabilities, it helps everybody.

 

And that is definitely the case with the Expo because it's so well organized and you can navigate the various speakers and sponsors and exhibits beautifully so it's a it's a real experience for those people who are who have low vision or blonde or those of us who are who are not blond.

 

I also want to say hello to my good friend Marin who was my colleague at the Department of Health and Human Services.

 

And we did a lot of great work together. And Maggie, who helped me navigate my care at the retina group of Washington.

 

So I was first diagnosed about 5 years ago with macular degeneration and it happened at just a regular eye exam at that the at my optometrist.

 

And he referred me then to a retina specialist. He saw something there that he thought was not right.

 

And so I was able to quickly get into the kind of treatment that I needed. Which was basically.

 

Monthly injections, to stop the spread of, of wet macular degeneration in my, in my right eye.

 

And after a couple of years of my vision holding steady with getting monthly injections in my right eye. The macular degeneration, the web macular degeneration moved to my left eye.

 

And as a result of being qualified for this clinical trial. Was able to begin to get a port. That was implanted in my eye.

 

That. A slow release of the medicine. So Alexandra has put the picture a picture of this.

 

Port on the screen. For those of you who don't see it well, I can I kind of think of it as a miniature space shuttle in my eye.

 

But it's about the size of a grain of sand. I can't feel it. I can't see it.

 

But it holds enough of the medicine. To hold me for about 6 months. And then every 6 months I get a refill of that portal so that I don't need to have monthly injections.

 

The second image you'll see or I can describe to you is an image of an eye, not mine, and you can see that there is a pupil and the implant.

 

Just near the pupil but under the eyelid so you can't see it if you're looking at me.

 

You can only see see the implant. Throw special. Photographs that are taken at the clinic.

 

So with my slow release medicine over time. My clinical trial. Participation is pretty much like standard care. With some exceptions that I think are pretty important.

 

I'm still monitored and I'm only monitored every 2 months because I don't need monthly injections.

 

But the monitoring is really important not only because in my clinical trial, I'm getting evaluated for the port, but I'm also getting evaluated for signs of progress of of the macular degeneration.

 

Not only in my research I, but in my right eye, which is where things started. So, so far my macular generation is on hold thanks to the medication that I'm injections in my right eye.

 

And now the every 6 month. Of filling up of the pork. In my left eye. I wanna just end by saying there are some downsides to the to being in a clinical trial.

 

There's a lot of testing, lots of paperwork for informed consent. But those are also the upsides for me.

 

One, I appreciate that at every step when things changed. As a patient and as a participant in the clinical trial, they have to get my informed consent in order to proceed with me in the trial.

 

I always have the opportunity to opt out of the trial. And I am treated like any other patient. For the most part, except for the extra testing that's required in the clinical trial protocol.

 

I would say the. The best advantage of being in the clinical trial is that monitoring. So I think that without regular monitoring.

 

That came from the clinical trial. The the monitoring of both eyes. Probably has saved. My vision in my ride eye because without symptoms in my right eye, I would probably not have been monitoring it.

 

But because I'm in the clinical trial on my left eye, they've been monitoring both all along the way.

 

So there's nothing better for managing Mac or degeneration than frequent monitoring. And so I'm very grateful forge and intact.

 

For including both eyes in the clinical trial. Just a little word of where things stand with the clinical trial in there and the genentech research.

 

It will be on the market once it's approved by FDA and I imagine that I believe that approval will be happening in the near future.

 

And I would like to just close by giving a couple of bits of advice based on my experience. One.

 

Yeah. And 2, if you have an eye doctor. Ask that doctor. If he or she is involved in clinical research.

 

And if they are, find out if that clinical research protocol is good for you. It works for you.

 

And the last thing I'd say is if you don't yet have a doctor, See if you can find one who is involved in clinical research.

 

Because you can get a step up. In getting innovations in eye care in in I, I, I vision, vision loss prevention by being in, in that research.

 

So I want to thank you all for hearing my story. For letting me tell my story. And I hope that I am encouraging to those of you who have not been involved in clinical trials to take the leap.

 

There's lots of care taken to make sure you're in formed and that your research is safe.

 

I am really happy to be able to be in a position to introduce Maggie. Cashin who is a clinical trial, a clinical research manager with PRISM vision group.

 

With extensive experience in ophthalmology. She oversees the clinical trial operations, manages clinical research coordination, and ensures compliance with good clinical trial.

 

Practices. She is an adept of, technician. And she was.

 

One of the important coordinators of the clinical trial and the research. Who helped me a great deal, navigate my care at the retina group of Washington.

 

So Maggie, it's great to see you and I can't wait to hear what you have what you've been doing lately.

 

Thanks so much, Miss. It's great to see you too. 1 min to just share my slide deck.

 

Give me 1 s here.

 

Okay.

 

Yes. Yes.

 

Yes, good to go.

 

Alex, can you just confirm you see that okay? Okay, Well, thanks so much for the warm introduction.

 

It's just an honor to even have it opportunity to speak at this seminar. Thank you to all of our organizers and thank you to all of our organizers and co presenters and co presenters. I'm really excited to be here.

 

Co-presenters. I'm really excited to be here. So like as Harris said, my name is Maggie.

 

I'll just give a little bit of a background of why I'm excited to be here. So like as Harris said, my name is Maggie.

 

I'll just give a little bit of a background of why I'm super passionate about clinical research, especially in the ophthalmology space.

 

So unfortunately my father, and uncles and a few of my family members have had a history of eye disease, everything from peaks corneal dystrophy to retinal detachment to a traumatic injury of the globe of VI.

 

So, I've had experience with family members going to eye doctors, since a young age and I myself are contact so I'm a frequent flyer at the ophthalmologist and optometrist.

 

I studied biology in college. I was also in clinical research in college and I knew I wanted to be in the health care field not really sure where so I ended up shadowing my dad's surgeon at the retina group of Washington was very fascinated with the sub specialty and retina because patients come in, majority of them have had good division for their whole life and then all of a sudden are diagnosed with something

 

and our providers are really able to help them in real time. You really see kind of that vulnerable situation where a patient comes in, the doctor kind of gives them an overview of their options and really starts them on some exciting treatments.

 

A lot of which we've been involved in clinical trials for. So that means that I, you know, kind of reached my, of the technician, and then I got into the clinical research department of Breton here to Washington worrying that Miss Harris and then also started managing locally for retina group of Washington research.

 

And now I manage for multiple practices within prison vision group. We are a doctor owned and private equity backed administrative organization to provide services on behalf of the medical business practices.

 

So we do a lot of exciting clinical research. I'll just kind of give a high level overview today.

Like Alex mentioned, I will provide my slide deck and resources and contact information, so happy to follow up and have further conversations.

 

Of how we can help support. So that being said, I'll get into my power.

 

Pro presentation describe what's on each slide. So we'll probably begin just kind of a lie of why prison vision group does research, just kind of a lie of why prison vision group does research, why we're passionate about why prison vision group does research, why we're passionate about continuing clinical trials within our practices.

 

And then too, providing care for our patients who are uninsured or underinsured, the pharmaceutical companies that we partner with really design the studies with patients in mind.

 

So they have kind of perks and bells and whistles, things like patient stipend per visit, things like car service transportation, if they make the medication and they usually give that for free to the non study.

 

I would typically only enroll one eye. So first and foremost for our patients to provide excellent care whether that's in the clinic or in a clinical trial.

 

And then secondly for our providers. So for our physicians amongst the practices, our doctors are really excited to be challenged by new medical devices, drugs and discoveries.

 

Many of our providers did not learn and train on some of the devices and drugs that we are using today in our clinical trials.

 

So just kind of fostering that physician morale of continued education. And then also to prestige and publicity for new providers to hopefully join our practices.

 

We do offer fellowship opportunities and are continually acquiescing more practices. So just to foster, new providers, and make that an attractive business opportunity.

 

And then last but not least, we are running a business. So we do partner and negotiate with big pharmaceutical companies who generously compensate our providers for all of their time and effort in the clinical research study.

 

So that does provide additional revenue to our practice. And then again, like I said earlier, also where patients are compensated for their time, and donating their eyes to political trials.

 

Next I have a graphic of how far our geographic footprint spans. So we have multiple research sites in New Jersey.

 

Teaneck, New Jersey, Tom's River, New Jersey, and Edison, New Jersey.

 

We also go down to South Jersey. We have a ophthalmology research practice in South Jersey.

And then we go into the DC in Maryland, Northern Virginia area with threatened group of Washington and retina care center in Baltimore.

 

Are you newest acquisition is, Sign and Breakfast go they are an ophthalmology practice.

 

This summer we actually did our first pediatric clinical trial in prison vision group. It was a peed study and enrolled about 75 pediatric patients.

 

For an anterior segment condition. So very exciting to span not only geriatric adult population, but also, as well.

 

So the main studies that we participate in the retina space are injectables. You heard a little bit about that earlier.

 

It sounds scary. It's actually not as intimidating. The eyes are numbed before any injection procedure or any surgery.

 

So we particularly focus on the diagnosis of age related inocular degeneration. We do both wet trials and also dry clinical trials and then also diabetes is our biggest disease indication.

 

We also have enrolling studies right now for vein inclusion. That's kind of a small stroke in the eyes.

 

So we have kind of a higher dose of what's already at the approved for that disease. And then like I said earlier, we do have ophthalmology studies.

 

So things like. Interocular lens studies, glaucoma, ocular allergy studies, more of the anterior segment of the eye.

 

I don't man this department alone. I have a great team that supports all of our practices.

 

I partner with my colleagues, Amy and Heather. We do everything from contract and budget negotiation to tracking research revenue.

 

We have kind of a quality assurance data entry check point to make sure we're in regulatory compliance and then we also help our accounting and finance partners make sure our positions are compensated accordingly.

 

To all of our contracts. At each research division we have anywhere between one clinical. Research coordinator up to 6 research coordinators just depending on the patient volume and the amount of studies that we have each division is also manned by a research director.

 

So that's usually a physician who serves as a principal investigator on our clinical trials. And really they just determine if we have the capacity to support a new trial.

 

If you have the patient population, if the contract in budget looks good and patient safety is always first and foremost in mind.

 

So a little bit about the overview of a drug pipeline in the United States. Many of you all might be familiar with the food and drug administration, especially coming off of the COVID vaccine, at the approval.

 

So good a drug administration is responsible for review and approval of all new drugs and devices. It is a very long lengthy process.

 

It takes about 12 to 15 years from an experimental design and ideation to a potential FDA approval, to then be used in the United States by providers and patients.

 

New interventions are not always offered to the public as soon as they're developed. Sometimes there's post marketing safety data analysis that has to occur.

 

Clinical trials ultimately determine whether the drug or device is safe and efficacious. There's typically 5 phases of drug development.

 

So first the pharma company discovers or has a concept for the clinical trial they do pre-clinical research so whenever a patient says oh I don't want to be a guinea pig well the patient is never the guinea pig.

 

We always participate in clinical trials that are never first in human or first in eye. We always want to see very good preclinical results, good pharmacokinetics, good safety data.

 

And good testing on, you know, phase one healthy participants and that also happens after testing on animal models.

 

Then the clinical research happens so that everything from phase one through phase 4 and then FD review and as I said earlier, any post FDA approval safety monitoring and analysis.

 

So kind of going back to what Miss Harris shared, the informed consent process is there per reason.

 

We discuss everything from rights and and standard of care options to risks and potential benefits of the clinical trial. Pre clinical results and also compliance with IRB institutional review board regulations so that group, you know, protects rights and welfare of research participants.

 

So we want to show that we're designing the study and conducting this study with the patient in mind.

 

Unfortunately here in the United States we've kind of had a dark past with clinical trials. So there are international and local guidelines that we abide by.

 

Making sure that the consent is voluntary. We don't just have a referral for a patient and start testing them in the study.

 

We discuss the important concern. Again, the risks benefits. The patient evaluates whether they're a good candidate.

 

We evaluate. Do they qualify with the inclusion, exclusion criteria? So, again, it's a lengthy process, but it's there for a good reason.

 

So that's really the first and foremost part of enrolling in a study is going through that informed consent.

 

So again, why do we do informed consent? Well, it's the law. The qualified team.

 

Member on the research, study department, they, basically give the subject an informed consent discussion. So that's usually the PI, the principal investigator, whoever is responsible for the clinical trial or the delegated study personnel, so the research coordinator.

 

Many elements of the informed consent that we always discuss are what the research is. So why the pharmaceutical company feels it's very efficacious, what the risks are and you know a lot of times we discuss that not all risks are known that's part of the clinical research.

 

Benefits of the treatment. Alternatives to treatment. So what's an option as standard of care? Confidentiality.

 

So we don't share patients names, date of birth, social security number to all the different vendors.

 

We de-identify the patient and kind of code them as a subject number. What to do in the case of research related injury.

 

If the PI or pharmaceutical company discovers that there is an adverse event or a series inverse event related to the study device or drug, the pharmaceutical company covers the cost.

 

And we've never really had instances of that, but it's just important to know that if something goes wrong, pharmaceutical company does pay for that.

 

And then who to contact? We have a 24 h line on that informed consent. So if the subject has any questions or concerns, they can always reach a staff member or an on call doctor.

 

And then again, last but not least, that the clinical trial is voluntary. It's also an ongoing process.

 

So the informed consent discussion doesn't end when the subject signs that informed consent document. It's an ongoing conversation throughout the course of study.

 

Many of our patients are with us 1, 2, 3 or 4 years. With monthly visits or even more frequently.

 

So whenever new information isn't made available, we always share that with the subject. There's an opportunity for reconents during this study if the sponsor has new safety information.

 

And we like to discuss, you know, patient consent before any study procedure. So during these long lengthy visits, for any study procedure.

 

So during these long lengthy visits, you know, patient consent for any study procedure. So during these long lengthy visits, you know, anything from checking vision to doing lab work, vitals, photography, you name it, we talk to the subject about it, make sure that they're volunteering their consent before they participate.

 

So advantages of pharma partnering with our sites within Prison Vision group for clinical trials. We consider ourselves a very premier research organization.

 

We have speed of negotiation, everything from assigning a non-disclosure to starting up a trial.

 

Good regulatory oversight, our contract and budget negotiation. Pretty much everything we do we feel is very streamlined.

 

So when we're approached, you know, one doctor might be approached for a clinical trial. We offer to run the study at multiple research sites, multiple locations.

 

We don't do clinical trials at every single, every single office. So we do like to, every single office.

 

So we do like to, again, kind of put those bells and whistles in our contract and we do like to again kind of put those bells and whistles in our contract and make sure that we can pull from other doctors with nearby nearby sites and so we can kind of span the geography and really make that research, and really make that research, study reach enrollment.

 

So our studies are closely monitored. We have again those regulatory guidelines that we have to meet.

 

Essentially we have auditors that come on site, review our records, make sure the study. Coordinators and the research doctors are abiding by the study protocol.

 

So the monitor comes on site, they come through all of our records with the pretty fine toothed comb, inevitably they find things and we increase our oversight and documentation to provide good clean quality data to the electronic data capture system.

 

We are collecting all of the data and then transcribing it into a system where a third party or delegated statisticians are monitoring the data and making sure patients are responding to the medication.

 

Or the device. And a safe manner.

 

I for some vision group, we also have marketing efforts that we do to publicize our clinical trials. So we do everything from a chart of enrolling studies so all of our physicians can know brief inclusion, exclusion criteria, what diseases were recruiting for.

 

We also send newsletters to our referring doctors. So optometrist and ophthalmologist who may refer to an ophthalmology research site or a research site.

 

Usually they're the ones catching like Miss. Harry said the eye disease for the first time.

 

So not only can they refer to our practices, but they can refer directly to a clinical trial. And then also to our internal providers, we send lots of email blasts and posters and flyers, anything that's approved to just market the study.

 

So just to kind of pause here and encourage you all, how can you do this on a local scale?

 

You know, place contact your provider, your ophthalmologist, optometrist, maybe you have a retina specialist and ask do they know if any clinical research opportunities and then again going back to that clinical trials.

 

Gov website, that's a great resource that list all of the clinical trials going on, not just ophthalmology and retina, here in the US.

 

So you can kind of find out if there is a site that's near you.

 

Okay, so just an overview of what a life cycle of a clinical trial within one of our research divisions looks like.

 

Typically one of our providers is approached directly by the pharmaceutical organization or their regulatory agency that runs the trial, we sign a non-disclosure agreement to start learning about the study protocol and there may be a feasibility process where the pharma company wants to learn more about us and our resources and staffing and we basically give information about everything from our experience to our equipment to our facilities.

 

If selected, there's a study startup process where we provide lots of research. Documents, and we negotiated contract and budget.

 

And then a site initiation that takes place where we have representatives come on site and train our staff. We also make sure that all of our staff are properly certified.

 

So everything that the patient comes in contact with is a certified piece of equipment, a certified technician to check things like vision or photography.

 

So really a very close regulatory oversight to get that study off the ground.

 

A little bit about study visits versus in clinic visits. So I think the biggest difference is quality versus quantity.

 

In the clinic, our providers see multiple patients per day. The study departments kind of max out at maybe 4 patients a day.

 

Our study coordinators really become friends with our patients. They not only know everything about their eyes, but they know about their grandchildren and loved ones and who they prefer to numb their eye, etc.

 

Okay.

 

Those subjects are randomly assigned if they need all the eligibility criteria to either receive investigational product or the standard of care.

 

Only one I is chosen as the study eye. The other I is referred to as fellow I or the non study I.

 

And throughout the course of the clinical trial, the biggest difference is safety is of our utmost concern. So patients, are never, you know, sitting around idle.

 

They're really kind of put through all of these protocol assessments to ensure that the drug is doing what it says it's going to do, the patient is responding, how we want them to, and we're keeping patients.

 

Sometimes in clinical trials, we use the term masking. Masking is just a term that kind of reduces bias.

 

So if the patient knew, oh yes, I'm getting the investigational product, the patient and the assessors might have a certain bias.

 

To want the patient to do better. So many of the studies that we participate in, especially in the retina space, are what's called double masks.

 

So I think the end of the study, the patient, comes to know what treatment they received, but during the study they may not know which treatment getting.

 

Again, kind of going back to that difference between clinic visits versus. The patient comes across a lot of different staff members to make this visit happen.

 

Our clinical research coordinators to make this visit happen. Our clinical research coordinators, the boots on the grounds to make this visit happen.

 

A lot of different staff members to make this visit happen. Our clinical research coordinators, the boots on the grounds, they are really kind of a conductor of a big orchestra.

 

They're doing everything from scheduling the patient's ride to making sure they have the right doctors and the right staff at the research location.

 

And typically for optimology studies we have the patient onsite anywhere between 1 2 to 3 h for a retina research patient.

 

We usually see that patient anywhere between 2 to 4 and potentially up to 5 h per visit.

 

So I'll just kind of finish off with benefits of the clinical trial. Many of which we're touched on already, but from a retina research perspective, so patients who have macular generation, diabetes, retinal van, inclusion, we always try to negotiate a car service transportation for our patients.

 

One, patients already have low vision. 2, we dilate them. We dilate their eyes during the research visit.

 

So it, you know, it becomes, a burden to either a caregiver or a friend to come to the research visit.

 

So we always try to negotiate a car service transportation and then again that helps us to pull referrals from nearby offices.

 

Close to our research sites. We also try to negotiate a per visit patient stipend so the patient can buy coffee or lunch or even do what they please with that money.

 

If the sponsor makes the drug, we always try to ask for the drug to be provided not only for the study I but also the non study I, should need it.

 

And then again going back to that very close safety oversight. So we monitor patients very, very closely, almost above and beyond standard of care.

 

Everything from lab work to pictures to vision, vital signs. And many times we discover things that patients may not know about.

 

The patient comes into our clinic, referred by an optometrist for a retinal, we may discover that that patient has high blood pressure or high cholesterol through our labs or through our high cholesterol through our labs or through our final sign checking.

 

So it's really an opportunity to kind of get a holistic picture or high cholesterol through our final sign checking.

 

So it's really an opportunity to kind of get a holistic picture of what's really an opportunity to kind of get a holistic picture of what's going on with the patients to kind of get a holistic picture of what's going on with the patient's health and usually again at free of charge.

 

And then last but not least, I'd love to say that we give the patient a VIP patient experience.

 

So really the research coordinators are that liaison between the patient, the doctor, the pharmaceutical company, the regulatory agency.

 

They wear a lot of different hats, but ultimately they become a confidant and that very vulnerable time where a patient is coming in with an eye problem and all of a sudden they're diagnosed with a very serious condition and then ask.

 

To be in a clinical trial. So pretty much, there's lots of hand holding and safety oversight.

 

And just helping to navigate that patient through this uncharted territory. So, I really enjoyed my time as a research coordinator.

 

I also think that they enjoy what they do. There's a lot of details, a lot of minutia, but it's in all within the patient safety and mind.

 

So lots of good regulatory oversight to keep our patients safe. So with that, thank you so much for this opportunity.

 

Again, I will provide my slide deck, and any resources and contact information after today's presentation.

 

So thank you so much and Alex back to you.

 

Awesome. Thank you, Maggie. Oh my gosh. Everything was so.

 

Fascinating, I think. It was absolutely wonderful. Here, let me make sure that I'm spotted.

 

There we go. It was absolutely wonderful to hear about everything that you all do at Prison Vision Group and, to hear about the process for clinical trials and, just the nuances around it.

 

So thank you for sharing all of that. I know Maggie's team and all of the clinics that they work with, they're all really wonderful.

 

We personally contacted some of them and everybody really is wonderful to work with. And just across all their ophthalmology clinics.

 

So I really do thank you for sharing with us and for being here. And all that information was really, really fascinating.

 

So I will have that slide deck to share on our page afterwards and on our email and I'll have their website where you can find all those clinics as well to share.

 

I also want to thank Linda. Linda and her work with our community is amazing. I want to thank her for sharing her story and her progress and also thank her for the kind words about our expo.

 

We really are so proud of the event. So that was so sweet. So thank you so much for sharing that.

 

And I really do hope that everybody can join us for it. We really are so excited to be able to put it on again for our second year.

 

So thank you, Linda, and thank you, Maggie. Now I am so excited to announce our final speaker of the day, Marin Allen.

 

Marin served as the Deputy Associate Director of Communications and Public liaison and director of public information in the office of the director at the National Institutes of Health.

 

She led the National Institute on Deafness and other communication disorders first, communications, legislation, and policy office programs.

 

She was also previously a tenured full professor at Gallaudet University and she earned 2 Emmy Awards for Deaf Mosaic episodes.

 

She really is a pioneer in her field. She's truly a superstar. I love speaking with her and I'm so, so grateful to have her here today.

 

Just go over clinical trials with her. With us, excuse me. And I really think, everything she, shares with us is really going to round out this webinar beautifully.

 

So thank you for being here. Marin, and please go ahead and take it away. We can go ahead and share your slides again.

 

Okay, great. Thanks so much, Alexandra. And also thanks to Andy and too good to see Linda and the community.

 

Gets wider and wider doesn't it over time.

 

Definitely does.

 

You should be good too. Oh, there you go. Perfect.

 

Good. Good morning to the West Coast. Good afternoon to the East Coast and for the people to explain the next slide.

 

For the people who are in Windy City, it's time for your second cup of coffee.

 

We are going to go through a world mentor of. What's happening in terms of how clinical research works and a lot of different environments and we're going to talk about why it's done and The previous speakers ball had so many nice insights to pieces of this that I will.

 

Not move through them without acknowledging that you've said some things that we have already talked about. So this is going to put it in a context for you.

 

Of the larger subject of clinical research and also just to let you know if there is for the books or watch who are here for the audience who are blind or have low edition.

 

I will describe anything that I don't say. Auditorial, that I can speak, from the slides, cause I'm working from slide debt for everyone.

 

We want to look at why it's done. I think we're gonna do that very quickly.

 

And the clients of clinical research, this we didn't get into some of the things that lead into it, which is the observation.

 

Observational studies and sort of how they've been into the whole picture. I'm recruiting, we've talked a little bit about that.

 

I'll try and give you some, some efforts that are on. For specifically. Out of the.

 

Nih. Paganical trials. Gov and questions you might want to ask about being at a particular study.

 

I the rates and responsibilities for volunteers. The informed consent has been handled very nicely and we'll do a talk about that from a slightly different perspective.

 

I've got one copy out of one warning sort of near the end. And. Also interpreting what you hear about us study.

 

Okay. Sure.

 

Marin, can I interrupt for 1 s? Your mic is a little bit fuzzy. Are you able to adjust it at all?

 

Oh, I'm sorry. Okay.

 

No, that's okay. I didn't know if anybody else was experiencing that. It's just a little bit.

 

Rough.

 

Yeah.

 

I'm sorry?

 

I guess. Am I just fuzzy to name right now also.

 

Oh it is? Do you have an external mic or you just going through your computer?

 

Once you're in my computer. Is that any better?

 

It's not. Let me. Here, try to say something else.

 

Yeah. So. The the definition of clinical research is the study of health and disease in people. I want to emphasize in this moment all people and that's better issue that I think they kind of touched on in her discussion.

 

Mary, let's try. I'm gonna try turning your camera off and seeing if that helps.

 

Okay. Is that better?

 

Bye. It is.

 

Yeah.

 

Hey, great. And I look so much younger. So that we have the different kinds of clinical research is.

 

Not just to find a disease, but also sets of symptoms because symptoms do not been identified. You can see the causation and that situation and so therefore those are helpful.

 

To treat whether or not a treatment will be helped with symptom or condition or also to learn how behavior affects the health of people.

 

It's focused on what we know or don't know about diseases and disorders. Different studies can approach the same problem or same question in different ways than be going on simultaneously, which is one of the reasons understanding about shared data that we'll talk about in a minute is extremely important.

 

Medical research is hard and it's complicated and it's very necessary and it takes a lot of skills of scientists, medical professionals.

 

And most importantly volunteers. I can hear you already thinking, but we asked about clinical trials. So first, I'm going to give you just a couple of minutes on observational studies because The types of observational studies will help you.

 

When you're when you're hearing about research results, understand sort of where in the process the scientists are.

 

Now the case study or case studies, if there are a few patients, are the ones. That are looking at unusual cases.

 

It's the long and complex, terribly careful way of documenting. Something that we haven't seen before or something that hasn't come to the scientific communities view before in order to set up the hypothesis.

 

The idea that can be formally tested. They're looking for causes and risk factors. The ecological studies compares the rate of the disease or condition for a group of people.

 

And it can be people from different towns or places, different incomes. Differing populations. And then moving into cross sectional studies.

 

There are snapshots and one of the things I'm going through these quickly I know because I also know the time is short but I've set it up so that when you go back through the slide deck you can really sort of study each of these elements.

 

So I'm going to try not to add too much conversation to it. Yes, snapshot is how common it condition is and to identify factors associated with it.

 

The case control study is a group of people who have a condition that's compared to a control group of people who don't.

 

And again, risk factors, there's an emerging theme, risk factors. And, classes can, cohort study is a large group of people observed over time.

 

It's like the study that was mentioned earlier today. Some eventually develop the disease or disorder or condition.

 

And again, they're looking for risk factors. I'm going to tell you about what we would call the granddaddy or grandmother of cohorts studies, which is Framingham study.

 

And Frederick Henson earlier cardiovascular. Oh, and there's a reason I use grandmother or grandfather is 3 generations of studies now.

 

And I think that as the scientists emerged, it's fascinating to look at. How's the diseases and disorders were approached and changing populations.

 

So the first generation was in 1,948. Framingham, Massachusetts with 5,209 men and women between the ages of 30 and 62.

 

That was 2 thirds of the population of the town. And the population was given extensive physical exams, lifestyle interviews, and then they come back depending on the protocol every 2 or 6 years for additional lab tests, more physical exams and detailed medical histories.

 

Slip forward generate gen 2 of this is in 197-15-1240f the adult children and their spouses were enrolled.

 

So, by April of 2,002, their grandchildren. World. So the way that this sort of cadre of people were able to give us lots of results over years and some of them that over 3,000 papers.

 

That identified originally identified some of the effect of cigarette smoking on heart disease. Landings and stroke, hypertension, and a more recently sleep apnea.

 

The research you will see has streamed and changed so that genetics, additional populations, new tools like the ones that were dangerous right this morning.

 

By the foundation are you can see the trend of the science and where it's going and who's doing what work.

 

Then here's an important consideration when I said all of the people. Hey, created the Omni Corps cohort in 1994.

 

They enrolled the first omni cohort of 507 women, then of African-american, Hispanic, Asian, Indian, Pacific Islander, and Native American descent.

 

Those were the terms used at the time. In 2,003 a second group of omni participants was enrolled So there was an entire new cadre of people who they were able to track in specific diseases and disorders.

 

One of the big things and this would be helpful, I think, to show to people on the call.

 

Is one does the benefits of the Big Framingham study was from. In the early days of 68.

 

They have been data sharing. And they've been data sharing through an interactive site at the National Park One and Blood Institute at the NIH.

 

To make originally with massive data tables available to outside researchers from the data that had been collected so that that wasn't just going down one narrow path, but it was able to be shared.

 

And NHLPI has continued to make that data available as it's changed. I mean, some of the problems that those early researchers had was, for example, not having good computer support, not enough to be able to do the kind of work that they did.

 

Now it's much more complex. And it's the data sharing is that is a topic that's really important across the field of research because you're not then replicating a duplicating work.

 

It's not for the purpose of replicating. We're duplicating, but simply that people are more informed about what's available, but data sets are available.

 

I've put a link that is a live link in the slide so that if you're interested in looking at the way the data sets are set up, that should be helpful to you.

 

Well, and I have on the screen there is just a One example of a biased specimen repository center coordinating center out of the NHLBI that chose the kinds of information that you can find about the studies and about the resources that are available.

 

Now it's about you. Clinical trials we've defined, you know now, from all the conversation this morning, the kinds of things that include new devices and new ways of dealing with new treatments.

 

Is part of the entire process of why it's done. Sometimes you hear a well designed clinical trial is the gold standard for treatment or a medical approach that works.

 

But clinical trials can't always be used. For example, and this is the ethics part. You can't randomly assign people who live in different places.

 

And you can't. Have people start to, you know, eat an unhealthy diet.

 

I worked on a I headed a research project between the Federal Trade Commission and the Federal Communications Commission.

 

While back with the team and FTC had asked for information about advertising to children. And particularly candies and sugar.

 

Cereals. And they were actually saving money because there was a we did a content analysis of everything in that documentation.

 

It was 10,000 pages. It had everything from folder books full of justifications for the healthy use of Mars candy bars to little postcards the people that sent in in the request for information from the public.

 

Because now we just do it on that computer, but it wasn't at the time. So one of the things you can't do, where the the researchers couldn't do was put a group of 8 to 10 year old kids.

 

On an island for 2 years and feet of nothing but bars bars and cocoa clubs so there's a kind of a dramatic example with the notion of the way that the ethics of this in terms of how people are selected is an extremely part and I will you know, I'll echo Maggie's comment that the safety of the participants is always paramount.

 

The definition of a political trial, I think we've been through that very thoroughly already. This morning and also the description of placebo.

 

But I just wanted to be sure that you had the official definition in your in your depth. Also, I have to do one disclaimer.

 

It was really nice though Alexander to include the NIH on the cover slide but I also wanted to want to say that it is a whole bunch of federal agencies and they are most of them under the Health and Human Services Department of Health and Human Services.

 

And as Maggie pointed out, The FDA's mission is critical to all of the work that's done in clinical trials.

 

So it's thank you for the recognition of the federal work in this area. The FDA, you just did a really nice presentation of the FDA's sort of overall.

 

I'm just going to hit a couple of other points on it. The notion Fracial here is good clinical practice.

 

And that comes right out of all of law and it's something that keeps coming up in conversation, which is, you know, what's the gold standard?

 

So just so that when you hear that, you, you know what it's related to. They do are.

 

They are the regulator authority. You must submit and investigational. Application for an I and D and investigational drug.

 

You have to do it before starting clinical research. But once again, I think as important as FDA offers help at any point along the way.

 

In drug development, it's not just kind of sitting there and saying, okay, give us, give us your application.

 

There is a review team. A very, very structured review team within the FDA that includes all of the chief kinds of scientists and medical officers who are pertinent to that particular.

 

Area or topic. And they Although the entire process of drug development takes long, the actual review team has 30 days to review my and a submission.

 

And that is in order to be sure that the submission is there and and even though the FDA has been tracking along to see what's going on that they have an absolute sort of kind of point.

 

Yeah, I think, and, be able to protect volunteers. Which, there are 2 things the FTA can do.

 

They can approve it right away. Or they can put a clinical hold to delay or stop investigation on it.

 

That's very rare because by the time that the FDA and the and the development folks have worked together.

 

And by the time they're ready to, to submit to the, FDA.

 

Usually things are unfairly good shape. So it's rare and it's important that that's there in case they're still not convinced that there's something that needs to in the quality of the research.

 

The illness is on, the, developer. To let the FDA know if there are any serious side effects in during the trial or after the trial end the research must be submitted in a study to the FDA.

 

And once the process continues. You have to have adequate data from 2 large control clinical trials before the actual release.

 

And that's something that,

 

Is an additional assurance for the safety of the public.

 

Okay, the phases and every week in the in the arena can to just flip those phrases.

 

The the phrases of phases off as though everybody knew what they were. So just to let you know in the slide deck, you can go back and refer to it.

 

But phase one is the typically if you were than a hundred people. And it's to test a medical approach.

 

And it's to figure out also in phase one appropriate dosages. That's right at the top of the phasing process.

 

Phase 2, the number of people is typically a hundred to 300. So you see the sort of way that this increases in size.

 

Monitor and for side effects. And.

 

When we get to phase 3. As many as needed and that's a calculation based on a lot of categories of concern.

 

How many patients have presented with this disease what they think that the basis of it is what they've learned so far.

 

Do they need it to be because it's a small, rare disease that they, they have a smaller population.

 

So between, the, that and about a thousand people are your phase 3 clinical trials, folks.

 

And then when you get to phase 4, it's thousands. And this is when the the approach is being marketed and you continue to gather information about its effects.

 

And this is of course part of the whole process of making sure before it's released.

 

Now, how do the studies? Sort of compare in terms of cause and effect. The Hello, there's no.

 

Okay. Yes, one slide here.

 

When there's one off.

 

It depends on the size and design of the study. New results may confirm and people say, well, but didn't we already know that?

 

New results may actually confirm. Or. Support or contradict aspects of the scientific understanding. In the end, class and effect is really hard to establish and that's why so much work goes into the process to take it far this far forward.

 

What about you? Okay, where I think all of us trying to protect the patient here and so The message in these lives is to learn what you can expect from the study.

 

You want to know what happens. You want to know your responsibility. You want to know the kinds of test treatment procedures that will happen during the study who will be in charge of your care.

 

Will I be able to see my own doctor? Will I be able to continue with other treatments if I participate?

 

You need to find out that it's the right choice for you. Every study has its own risks and benefits.

 

And when you find out and and you can hear from the tone, at least the ideal tone that we were hearing from, the 2 people who were discussing these trials before was that They're willing and able and anxious to answer questions because they want this to be fair and they want it to be thorough and they want to be sure that they're participants really want to participate.

 

You will not understand. The basis of the study, why they think the treatment might work and why and one question that people I think find helpful is how is this treatment from other treatment?

 

This treatment different from other treatments that are available.

 

Lots of reasons I'm going to do benefits and risks lots of reasons to participate in being in a study and the clear ones are obviously, you know, how mad the treatment helped my condition, how will I benefit from joining the trial.

 

All of the results of the trial benefit other people like me will I be paid for participating?

 

The benefits for society in general are moving knowledge forward and that's actually the key. Element. It's got to be the notion that this is going to be of help and and extending knowledge about a particular disease or disorder.

 

You can benefit individually if you're the first to try and in treatment that successful or You can also benefit several people mentioned genetic disorders in their families in the earlier presentations.

 

But also that information can be not helpful to you, helpful to people who are members of your family. The risks, I think we all sort of the side effects.

 

The long short term. Side effects of treatment, possible. Comparing the possible risk and treatment to their current treatment.

 

And they can't ever promise that everything is okay because we don't know, cause that's why the trials being done.

 

So the doctors nurses and support staff will get your checkups to find any side effects. Make sure you're healthy enough to stay in the trial.

 

And the independent group of experts I refer to on this on the slide is the group from the FDA and they will keep an eye on everything as it goes on in terms of risks and benefits.

 

You've got rights.

 

Again, ask about the trial. Leave the trial at any time. Someone just said that and that's absolutely the case.

 

Your medical information is private and stays anonymous and reports about the trial. Those are things that are right.

 

You have. You also, and I won't repeat this because we've now kind of reinforced it.

 

But any new one sort of important addition is any new information about the treatment that researchers find during the trial or after the trial is over.

 

Volunteers have a right to know what it is. They were in the treatment group or in the control group.

 

People have mentioned cost. And I'm going to use the National Eye Institute, information on this because it's varying.

 

And the important thing is asking the right questions. So for example, people who join the political trials as the one that was mentioned by Ben earlier.

 

At the NIII, iplanic and Bethesda pay nothing for their participation and they now qualify for some travel expenses and other related costs.

 

It's generally true the other federal agencies. A company that makes the treatment or test has its own policy and that's something that you need to know.

 

Up front. Individuals. Also the foundations can support the development of research. So find at universities where usually or often.

 

Funded by a combination of those the federal government or or foundations or individuals. The same thing is true.

 

So the base with organizations. So the basic thing is Before you join in a trial, ask the staff how it will cost for you.

 

Then check with your health insurance company to see if your plan will cover any of the costs that the sponsor doesn't cover.

 

But that's something that you really need to know upfront. And I think that's probably the idea is to be really sure the people when you're discussing trial.

 

People want to know why does it not always cover my age group? The eligibility criteria. It is, it is always significant because It has to do with the design of study, but for example there was a reason for, to leave the drug name out of this.

 

That covered, kids from 5 on up to 18. For an allergy related treatment. And then a different.

 

Don allergy series of problems that included. Skin rashes and diseases was approved for kids who were 12 to 18.

 

So the can who's 10. And clot in between. Is off label for the drug. And it's a special understanding.

 

Yeah, it needs a special accommodation if it's available because that kid isn't approved. For that drug for that purpose even though they're within.

 

The age range of the use of the drug. Now, what happens in that circumstance is essentially it wasn't tested in kids that age yet.

 

And they said, well, what? That doesn't make any sense. Well, it's cause it wasn't, they were tested in that specific corridor.

 

So they can't approve it for that purpose. So the eligibility criteria is very strict for the reasons of controlling the data to be sure that you're absolutely sure that what you're getting is what you're needing.

 

The informant. Yes, please ask everything and it's a document and you sign it and one of the things is kind of interesting that's in the National Library of Medicines database.

 

You can actually read the the examples of informed consent documents. For any study. And that can be informative to you.

 

To see how it compares to a study in another setting. Okay, here was the little morning though.

 

And it's actually from the MEI. And it's a copy and I've got a picture on the screen of a Very nicely colorized human induced pluripotent stem cell collie.

 

And so what we're gonna just mention is Stem cell clinics. And this is a warning from the National Eye Institute.

 

We're learning every day about H stem cells can help. Though I helped. There was some terrific research that just came out of.

 

London this week. But some stem cell clinics are already advertising these treatments as miracle cures.

 

Without the scientific data of knowing whether or not they're safe. They can share and charge thousands of dollars for unproven treatments which aren't covered by insurance.

 

So before you. Respond to anything that sounds like that, I would suggest that you talk to your doctor.

 

He or she can help you understand if the treatment could be right for you and they can find out if it's FDA approved by the end of the presentation, you'll be able to find it out from clinical trials.

 

Dot com. Or if there's a clinical trial you can join. So just be aware that. Some of these things are not being supported by the same kind of structure that the rest of us have been talking about today.

 

Record about implementation science and this was the Sort of an echo of the issue of how long it takes for something to get to the public the seventeen-year issue.

 

Is the idea from the lab to when it gets to your doctor's office and to you. And it's about 17 years in many cases.

 

And implementation science which is being inserted into a lot of the grant requests. Are trying to get that to work faster and figure out why it's not.

 

For example, the example I have on the screen is the colorectal cancer home stool home home stool test.

 

It's convenient. It's fast. It's less. Invasive and difficult than a colonoscopy.

 

Good. An abnormal result. Requires a follow-up within a year. At this point, only half of the people who are doing the testing who get one of these, you need to have a retested in a year are actually doing it.

 

So there's a gap, there's an information gap. And there's also a, how do we get people to do it when they're leaving the physician office to understand that this is.

 

Really important. So it's something that was just published in April. I've given you the citation and it's in the back deck.

 

How do I find out about specific roles? Trials. Dot. This is a need to know about resource.

 

The reason is that it's both a website and a database. And it provides information about studies.

 

It's used by the public. It's used by healthcare providers and it used by researchers.

 

The the companies like Maggie's who are sponsors of research provide the information about their own about their own studies as to organizations like men's.

 

The scope of the trials database. Is studies through the US and more than 200 nations around the world.

 

So you can also see what's happening in research across across the world. And there's new stuff in that database.

 

Almost every day. So it's something more sort of keeping track of. The national library of medicine.

 

Reported just to give you the scope. 38,837 active trials in the US and we are the largest supporter of clinical research in the world.

 

And they have on in this database also 100,000 205,172 international active trials.

 

So that just sort of gives you the sense of the scope of, and this is from, this is this year's data.

 

Also, the study record includes Not only information about the study, but information about each study is permanently available, so it doesn't disappear on you.

 

The studies over there's there are improving records in terms of the results of the studies. But also you can go back and see any study that's been done.

 

That has been since the the database was set up. In a study record, it has the name of description, person, sponsored it.

 

People are organizations that fund it. It's important to know that disease or health problems studied and it's searchable, you can actually go in and search blindness and little vision and individual diseases.

 

The disease or health problem study start and end dates and where it's located. It'll tell you who can and can't join the study, how many participants are needed, what the intervention is.

 

What they want to learn and, how to contact the study staff so that you can start the, the, willing to start talking about.

 

Participation on the screen right now. Have an example of a. Yeah. Okay.

 

Search card that it describes it when political trial that's that is recruiting. One also tells you whether it's recruiting or finished or interestingly enough about her recruit.

 

In other words, they're in process of clearing the study. And so sometimes it's good to take a look for the about to recruit because you're not looking at maybe being numbered out of it.

 

If it's something that's close to you.

 

Couple of final notes as you're listening to news about breaking research. Listen for one of 2 words.

 

Association or Causation. It's gets very much for people sometimes in a way that the news reports are set up And just because 2 factors occur together doesn't mean that one caused the other.

 

And that's a tough issue for scientists and researchers, but just know when you hear it, whether or not they're talking about an association effect between factors or that they're talking about an association effect between factors or that they have actually developed an association effect between factors or that they have actually developed or identified a cause.

 

Okay, really brief history. What's not science? The, I'm wearing my,ippet and We all learned early on that the concept of taking 10,000 steps a day to maintain health.

 

But it's not rooted in science. It was marketing. And the 1960.

 

Japanese firm. Invented one of the early pedometers. Because the Japanese figure for 10,000 looks like a picture walking.

 

The company called it the 10,000 stepm. Now, I've attached a link to the, Scientific American article about it and, also there's an image of the person walking.

 

It's, you've got to do a little abstraction from that, but people blocked around for a long time.

 

Agencies used the 10,000 step. Because it just sort of floated into the into the universe of things people thought they knew and in fact they have discovered that at different points in age in time.

 

Different step lengths, different numbers of steps are much more effective. I'm gonna just quickly show you what's in the resources section.

 

The FDA and its background if you're not familiar on the special topics. For the NH, I just gave you the homepage with a yellow circle around, put it in the search box because there's a lot of eye research that goes on in institutes other than the NDI, but obviously you want to serve the NAI.

 

You've got here a really helpful book that they did on clinical trials and vision research and on this slide there is a copy of the book and and a yeah Okay, I just lost the work.

 

Friends, skinny.

 

You are cooked.

 

Yeah, QR code.

 

Sure. Okay, okay. And, I put on here there's a really nice piece that just came out in today's and IH record about Rosenberg's work on a half century of cancer.

 

Research and it's sort of gives you a sort of complete feel for political research and how it happens and the discoveries and the bumps along the way in a very human way, but from a really great researcher.

 

And if you just feel like sending that tone, I think it might be helpful to you just take a look at that.

 

Okay, your quick photos. Loneliness and risk of Parkinson disease. This came out last week in JAMA.

 

And the question was, is loneliness associated with an increased risk of developing Parkinson? Now it was, now you know what a big cohort study is.

 

It was a big cohort study with 491,603 people. It was followed up for 15 years.

 

Loneliness.

 

And say, okay. I will tell you the rest of them. Loneliness.

 

Was associated. With Parkinson's after they had a eliminated. Socio economic factors. Social, social isolation.

 

Genetic risk, physical and mental health. And you say, how did they define? Logliness, if it isn't socialized, and if it isn't mental health.

 

So I'm really fascinated to find out more about it. But when you hear it on, when you hear it in the news, or you read it in the news.

 

Just think about how much. You know about now how to parse it. Because you could go, hello, loneliness and Parkinson's I must be.

 

Getting Parkinson's. And that's not what that means. And so it's a matter of being sort of the caveat.

 

All of this is about kind of collecting as much data as you can. Knowing how you feel about it.

 

And knowing that there are protections out there for you. So I just wanna thank you all for listening and I hope that the debt is helpful to you.

 

And the resources because I think it helps you sort of tell the story and I ran her pretty quickly because I knew we were running on time, but I hope that it makes sense for you.

 

And thanks again.

 

Awesome. Thank you, Marin. I knew that everything you were going to share, was going to be Beautiful conclusion to everything that everybody else shared.

 

Awesome. Internet. Okay.

 

So I was so excited to hear from you and I think everything you share was fascinating. I will say that all of the her slides and all of the links and things that were included in her slides.

 

We will be sharing those on our follow up email and then also on our website page. So if you're interested in navigating to any of those links or learning more from them or learning more from her slides.

 

Those will be included on our email and on our website page after the fact. But I do want to say thank you again, Marion, you really are a superstar and we're so grateful that you were able to join us today.

 

Today to discuss clinical trials. We're so grateful for your work overall too. And your commitment to your project and your programs.

 

One again, thank you. And I also just wanna say thank you to all of our presenters and a huge thank you to our ASL interpreters today for just being with us and helping with the accessibility of this webinar.

 

Thank you to Ben. Thank you, Maggie. Thank you, Linda, and thank you, Marin, for sharing all of these amazing resources with us and sharing their experiences and their information from their organizations with us.

 

With that said, that does conclude our webinar. Thank you for joining us. Thank you for tuning in.

 

This entire webinar has been recorded and I will be sending out a follow up email in the next few days.

 

This entire webinar has been recorded and I will be sending out a follow up email in the next few days with all those resources as I said, but it will also have this recording in video audio and transcript format.

 

Thanks.

 

So that will be available in that email on our website accessible pharmacy. Calm and it will also be on our YouTube page.

 

So please go ahead and navigate to that if you want to check back on any of this information or if you want to share it with anybody that wasn't able to join us live today.

 

Of course.

 

And then also when you get that email, please feel free to respond to it, with any feedback that you have about the webinar, any of our presenters about the accessibility of this webinar and how we can continue to make programs like this more accessible in the future, any ideas you have for future webinars.

 

We would love to hear from you. And to be able to continue to provide webinars like this in the future.

 

Please join us also on Friday, December first for our blind health expo. Like I said, it is free to attend for the day.

 

It's also free to exhibit. It's completely virtual. It's from 10 am to 4 p.

 

1. Eastern time. If you have any questions at all about exhibiting or attending, please just send us an email to info.

 

INFO at accessible pharmacy. Calm and lastly I would just like to say before we leave that if you're if you are not a patient of ours here at Accessible Pharmacy, we would love for you to join us.

 

We learn more about how to support our current patients by listening, or excuse me, learn more about how to support more patients in the community by listening to our current ones.

 

So we would love for you to join us so that we can listen to you too and get feedback.

 

From you and learn more about how to be more accessible. And learn more, learn about more services that we should be providing and how to increase.

 

You know, access to different things in the community. If you are a provider and believe that we, if you, that you have patients that would benefit from our services, please don't hesitate to reach out to us.

 

We would love to hear from you. We would love to hear from your patients and discuss more with you.

 

The easiest way to work with us is to simply call us at 18886337007.

 

With that said, that is it. Thank you for attending our blindness and clinical trials webinar. We were so happy to put it together and have you all attend.

 

I'm so, so grateful to all of our presenters. Thank you again and thank you to our ASL interpreters.

 

We will be in touch with our follow up email. Have a great weekend everybody.

 

Thank you, Alexandra.

 

Of course, thanks.