December 22, 2021

Podcast - Discussing Sickle Cell Research and Care with Dr. James Taylor VI, MD

The Eyes on Washington Podcast Series
Discussing Sickle Cell Research and Care with Dr. James Taylor VI, MD

In honor of National Sickle Cell Awareness Month, Holland & Knight's Public Policy & Regulation Group is proud to partner with the Black Women's Health Imperative (BWHI) for a four-part podcast series on sickle cell disease. Sickle cell disease is a genetic disease that causes red blood cells, which are normally round, to become C-shaped like a sickle. It is a progressive and debilitating disease that can cause pain crises, organ damage and a shortened lifespan, and it disproportionately impacts communities of color, occurring in approximately one in every 365 Black or African American individuals in the United States.

This episode is co-hosted by Holland & Knight Senior Policy Advisor Shawna Watley, Chief Policy Officer & Counsel for BWHI Tammy Boyd and Senior Director for Alliance Development and Government Affairs at bluebird bio Sonya Elling. They are joined by the Director of the Center for Sickle Cell Disease at Howard University, Dr. James Taylor VI, MD who shares insight about the mission and history of the center. He explains what sickle cell centers are, describes why they are so rare in the United States and illustrates how their increased existence could change the course of healthcare for people living with sickle cell. This discussion explores legislation related to sickle cell disease, including the National Sickle Cell Control Act which is celebrating its 50th anniversary next year. Dr. Taylor provides an overview of how funding for sickle cell research and clinical care has historically been distributed, highlighting the disparities in comparison to diseases like cystic fibrosis. During this episode, the group also focuses on discrepancies in care for people with sickle cell when transitioning from pediatric to adult care and how policies can work to better support adults with sickle cell.


Podcast Transcript

Shawna Watley: Hello, I'm Shawna Watley, Senior Policy Advisor with Holland & Knight, and this is our Eyes on Washington Public Policy & Regulatory Group podcast. And we are here and excited to be on our four-part series, this being, the fourth part of our sickle cell awareness episodes. We're very excited to have our co-hosts with us today, Tammy Boyd with the Black Women's Health Imperative, as well as Sonya Elling with bluebird bio. I'm so thankful for our participants on this podcast today, we have Dr. James Taylor, M.D., the Director of  the Center for Sickle Cell Disease at Howard University. And so we'd like to jump right in. Dr. Taylor, can you give us a little bit of background about center and the history of how Howard University got involved with sickle cell?

Background on the Center for Sickle Cell Disease at Howard University

Dr. Taylor: So it's a long history, Ms. Watley, this started actually in 1948 with an amazing man that I have learned more about in the last five years, Roland B. Scott. He was a 1934 M.D. graduate of Howard University. He specialized in pediatrics and had additional training in immunology, returned to Howard and joined the division of Pediatrics. It wasn't a department in 1936 when he joined. And around 1945, he recognized that there was a disproportionate number of children in Washington, D.C. affected by sickle cell anemia that nobody knew very much about. And in 1948, he published his first paper. It was the "Prevalence of Sickling at Freedman's Hospital." That launched his career into becoming an expert in this disease as a non-hematologist. And he is largely credited in the early 1970s with lobbying Congress and was one of the architects with both Congress and the President with Public Law 92-294 that was passed on May 16, 1972. That was the National Sickle Cell Control Act. And amazingly, this is a disease that took an act of Congress to authorize the National Institutes of Health to study it. Around that time, just before that law was passed, the Board of Trustees at Howard University established the Center for Sickle Cell Disease. So 2022 is our 50th anniversary, and the Howard University Center for Sickle Cell Disease and 2022 will be the 50th anniversary of the passage of the National Sickle Cell Control Act.

Amazingly, this is a disease that took an act of Congress to authorize the National Institutes of Health to study it.

Legislation Related to Sickle Cell Research and Treatment

Shawna Watley: Wow, that's pretty impressive. Can you walk us through a little bit about the legislation that was passed? What did it do for sickle cell?

Dr. Taylor: So what it did in a nutshell, and this is one that I've really thought about intensely for the last five years, we're really trying to delve down into exactly what Dr. Scott did in this process. But it authorized the National Institutes of Health, prior to May 1972 the NIH was spending very little money on sickle cell disease, and most of it was studying how red cells sickle nothing for patients. There were no therapies. It authorized the NIH to establish 10 national centers at universities spread around the country that would be centers of clinical and research excellence. And I believe it was 1974, they appropriated about $40 million for sickle cell disease. So prior to that time, there was less than $250,000 spent a year on sickle cell disease in total. So in a nutshell, that's what it did. But the downside to this, the unintended consequence is the NIH focuses on research. So about 2007 that NIH decided to dismantle the 10 centers, I guess it had grown to 15, but they dismantled the centers because they felt that they were providing too much clinical care and taking money away from research. And to do research, especially patient oriented or translational research, you need both patients and researchers in one place at one time. So this has been, you know, the unintended consequence is, there are inequities in delivering clinical care. Everyone wants to study these patients, but at the same time, it's very hard for them to get clinical care.

Everyone wants to study these patients, but at the same time, it's very hard for them to get clinical care.

What Happens at a Sickle Cell Center and How Prevalent Are They in the U.S.?

Shawna Watley: Wow. So at Howard's Sickle Cell Center are you providing both? Or do you just do the research?

Dr. Taylor: We do both and always have. I guess I'm the one to three four, fifth director of the center, so, every decade there's a new director, and it's been a lot of very prominent, more prominent than myself, excellent clinicians and researchers at the same time. So we provide clinical care to all comers. You know, the downside is we do have to pay attention to insurance. You have to be insured to get clinical care if you're uninsured. We can do some things about it. So we have a social worker on staff to address those types of issues. And at the same time, every day, I guess yesterday I was in clinic. I saw 10, 12 patients and I signed off on five research documents for patients who are doing different research activities in clinic. So in my mind, when I'm doing one I'm doing the other. And it can range from laboratory based studies, genetic studies and drug related trials.

Shawna Watley: And so and how many centers are there like yours in the country?

Dr. Taylor: Very few off the top of my head. And this is not exhaustive, but the ones that stand out in my mind are Oakland Children's Hospital in Oakland, California. They've had a very large sickle cell program for both adults and children at a pediatric hospital. The University of Illinois, Chicago at Cook County Hospital has always had a prominent sickle cell program. The Medical College of Georgia in Augusta, Georgia, has a state sponsored center for sickle cell. At Johns Hopkins University, for decades and decades, maybe not quite as long as Howard University, but has had a very prominent sickle cell program. And I give a shout out to one community based organization that is will this year be celebrating its 50th anniversary, The Martin Center in Indianapolis, Indiana, is a community based organization to advocate for clinical care and research for sickle cell disease. And this is not an exhaustive list. There are other terrific places to go, but they're largely few and far between.

There are other terrific places to go, but they're largely few and far between.

Shawna Watley: Wow. So I'd like to turn it over to my co-host, Sonya Elling. I believe she has a few questions for you as well.

Transitioning From Pediatric to Adult Care While Living with Sickle Cell Disease

Sonya Elling: So I have so many after listening to you Dr. Taylor, and you're very informative and have helped provide more background information for us on a variety of things. One thing we've talked about and heard is that there is a huge gap, if that's the right word, in regards to care and access to care for people with sickle cell when they become adults, when they age out of pediatric care. So I would love to get your thoughts on how we can address that, because that's one thing we've been seeing in the D.C. policy area and all the work that Shawna does and the government affairs policy area, as well as myself and Black Women's Health Imperative, trying to figure out how we can work with policymakers to make some changes. So would love your thoughts on what might be a possibility to try to help really address that huge gap that exists for people with sickle cell as they enter adulthood and leave pediatric care?

Dr. Taylor: Sure. Thank you for the question. Good question. I'll give you my perspective. So I originally trained as a pediatrician and I did a long postdoctoral fellowship in the lab, and I had an amazing opportunity to join an adult sickle cell research and clinical care group at the end of that fellowship. And I jumped at it and I now have more experience. I've never been in a pediatric department and I have more experience in internal medicine now than I do in pediatrics in terms of clinical care. So what is my perspective? Well, one, when you're a child, you go to a children's hospital and most large cities in the country have a children's hospital in Washington, D.C., its Children's National Medical Center. You can think of many different large American cities, and they all have children's hospitals. And what you have in a children's hospital is a concentration of subspecialists in every possible subspecialty of pediatric care. And this means when you have a multi organ disease that affects everything, you have one stop shopping, your parents take you to one place. And when you leave that pediatric care, nobody tells you that perhaps the best sickle cell care in Washington, D.C., is at Johns Hopkins Hospital or at Howard University Hospital, or perhaps Washington Hospital Center, and maybe even the Medical College of Virginia in Richmond. Those are kind of the four places that I would go if I were to send my child if I were a parent of a child with sickle cell. But I think this gets to the larger question on how our health care is organized in the United States, and I have an international perspective.

Since 2018, Howard has had a collaboration with the Republic of Congo. They've started the National Center for Sickle Cell in Brazzaville. And I went there in 2018 and 2019, this whole project's been interrupted by the pandemic, but Congo is a medium income country in Africa. You're either very wealthy or very poor. There's no middle class. Not surprising. And there's no national healthcare system. But sickle cell is such a problem in the country. It affects the gross domestic product, and I heard that straight from the government. So they set up, they found the money to fund a national center for sickle cell. And when you're starting an organization like that to provide care, you have staffing issues, which means you pay salaries and so they are open from nine to five or seven p.m., something like that. They're not open 24 hours and they're not a hospital, but they deliver care like it's a hospital. They have an emergency room there. They have a day hospital. They can treat you. If you're having a pain crisis and you'd be hospitalized in the United States, in Congo, you go home at seven p.m. and you come back the next day. So you're not admitted overnight and therefore they don't have to spend money on 24 hour staffing. So they have significant cost savings. In the United States, it's completely opposite when you have sickle cell disease, let's be blunt, the major manifestation is pain that brings you into contact with the healthcare system. That is 90 percent of hospitalizations. So when you have pain, you go to the emergency room, the emergency room physicians to be very cynical, they think of patients like vectors. They have a direction and a velocity. That velocity is about one hour to make a decision, and the direction is they're going to send you home or they're going admit you to the hospital and they have to make that decision in that period of time. So about a third of the time you're admitted to the hospital and then in sickle cell, you're not cared for by a specialist in sickle cell care. You're cared for by a hospitalist who's a general internal medicine physician who may have expertise in sickle cell but may not have expertise in sickle cell. And when you consider that this is a rare disease, you're not getting the most informed care. So there are national guidelines for sickle cell, and I would submit to you that most hospitalists aren't aware of them or confuse them with other guidelines that apply to general medical patients. So I think that's one of the challenges for young adults in transitioning to adult care is they go from going to one place and when you get in an ambulance as an adult, they could take you to any one of 10 hospitals in the Washington, D.C. area and you have no control over where you go. So you may not necessarily go to a hospital where they know your case.

So about a third of the time you're admitted to the hospital and then in sickle cell, you're not cared for by a specialist in sickle cell care. You're cared for by a hospitalist who's a general internal medicine physician who may have expertise in sickle cell but may not have expertise in sickle cell. And when you consider that this is a rare disease, you're not getting the most informed care.

Addressing the Lack of Sickle Cell Centers and Access to Quality Clinical Care

Sonya Elling: I had no idea about the structure, and what they were doing over Congo. That is fascinating. I'd be curious to also know how you think we could address this. Howard has this great facility. You do the research and you do providing of care. You mentioned how from the 1972 Act, we had these centers of, I'm going to call them Centers of Excellence because that's where my brain is going is, is that there aren't existing centers of excellence for sickle cell. I guess I'd be curious your thoughts on that. To kind of bring back what had originally been established from the 1972 Sickle Cell Act and try to figure out a way to do these centers that might be placed in areas where we know there's a high patient population of people with sickle cell. And whether or not you think that might help begin addressing the problem and some of the things I know, the NASEM report highlighted.

Dr. Taylor: So I'd highlight a couple of points. One I've been involved with the National Minority Quality Forum there at the think tank in Washington, D.C., and they've embarked on a really detailed analysis to find out where patients are. And I guess I'm not surprised by the findings, but it's something that hasn't been bluntly stated that most of the patients not exclusively, but most of the patients start in the mid-Atlantic and head into the deep south because that's where the, you know, you find African-Americans living more commonly. And the other thing that struck me is Washington, D.C., on a per capita basis among Medicaid and Medicare recipients or beneficiaries is the number one or two per capita state or jurisdiction in the United States. So it doesn't surprise me. I think that, you know, one of the problems is the delivery of care and essentially what the Republic of Congo has done without realizing it. It's just the way they designed their system, what they have is a glorified day hospital.

Instead of going to the emergency room where you're going to see a new physician every time you come in to a sickle cell center where we know who you are. We know what you're on as far as pain medicine, as an outpatient and we deliver the care.

And what a day hospital is, this is an effort that was pioneered by Memorial Health Care in Fort Lauderdale, Florida, also, Johns Hopkins hospital has had a day hospital for a decade now, and the University of Connecticut has also started a day hospital. And what this is, instead of going to the emergency room where you're going to see a new physician every time you come in to a sickle cell center where we know who you are. We know what you're on as far as pain medicine, as an outpatient and we deliver the care. And from my perspective, if you sit there for eight hours, I'm happy with that. So it's a different decision making process than the emergency room where they want to make a decision in about an hour so that they can turn over a bed. In my mind, my goal is to keep you out of the hospital. So if I let you sit there for eight hours and you can go home. I've accomplished the goal. And so what Johns Hopkins has published on this is that they've lowered their admission rate below 10 percent. And they also incentivize this. So if you're taking care of yourself going to the day hospital is a privilege, you have to come to clinic on a regular basis, you have to be on some type of disease modifying therapy and you have to be a responsible patient to a large extent. We don't get judgy about this. But it incentivizes you taking care of yourself, and it's much more cost effective than admitting you to the hospital. And we probably do far fewer laboratory and imaging studies than most emergency rooms do.

In my mind, my goal is to keep you out of the hospital. So if I let you sit there for eight hours and you can go home. I've accomplished the goal.

Shawna Watley: Well, that's pretty impressive. You're just a wealth of information. I have so many questions. Follow up questions. But I know my other co-host, Tammy Boyd, would like to jump in and ask a few questions as well. Dr. Taylor.

Vision for the Sickle Cell Center at Howard

Tammy Boyd: Yes, Dr. Taylor it's quite impressive, and this is very helpful, extremely helpful information and insightful. A question I would have would be, you know, what is your vision for as you talk about some of the work you've done in Republic of Congo, but what is your vision for the sickle cell center there at Howard?

Dr. Taylor: So our vision for the Center for Sickle Cell Disease, so not to put weight on my shoulders, but I feel an incredible responsibility for the legacy of the center and for the legacy of Howard University and really built on the shoulders of Roland Scott, the guy behind the National Sickle Cell Control Act, and also Charles Drew, who is one of the founders of the field of Transfusion Medicine. He was a faculty member at Howard in 1950, and we use a lot of blood transfusion therapy in sickle cell, so I want to build on their legacy. For 50 years now, The Center for Sickle Cell Disease has largely been funded by federal grants and federal grants are what I call federal, sometimes they're good, sometimes they're bad. Financial downturn in 2008 might be a bad time to apply for a grant where it's more competitive. And I thought about this a lot strategically. We do not have a day hospital at Howard University Hospital. This is going to change very soon. So one thing that we have is we've gotten a center of excellence, a five year grant from the city of Washington, D.C., to provide care for sickle cell patients in Washington, D.C., highest per capita prevalence in the United States. So good thing. Hopefully, we can deliver on that promise. From that will come some of the money that will start a day hospital for us. So that's my first vision.

The second is, I see this all the time in terms of curative therapies like transplants. I get letters all the time from other transplant centers asking me to identify people who are eligible, have a match for transplant. They want the patient, they want to transplant them, and then they always say, and we'll send them back when we're done. And so I've talked to some of these centers, and not all of them are like this, but they say, 'Why don't you start your own sickle cell program?' They say, 'Well, we don't do sickle cell here. We just want to do the transplant.' The trouble is that the care for sickle cell is poorly reimbursed, the transplants are well reimbursed and it's a moneymaker. My vision long term is to be able to provide that therapy here at Howard University Hospital to build the infrastructure and even the delivery of transplant and gene therapy and gene editing is going to change over the next five years. I expect more of this is going to be outpatient, which would be perfect for a place like Howard where we may not have all the inpatient specialty care. The next is how to fund this. Yes, we have a five year grant from Washington, D.C.. It is a very generous grant for a center of excellence to build a center of excellence on top of a center. And so we have launched an initiative with development at Howard University to raise an endowment. And I've got a number in mind and I've got an inaugural seven figure donor who's going to give us a lot of money very generously. Something that they approached me without this idea, and I started thinking about what to do with a seven figure donation. We put it in an endowment and so we can live off the income from investments. This is what universities do. And we just do it within our center. And if we reach our goal, I will have enough money to run the center as if we had a large federal grant from the National Institutes of Health. And then on top of that, we'll apply for grants, too. And this will enhance both research and the delivery of clinical care. With an endowment we can, if we have a federal grant for research, we can pay for the research off the grant and use more of the endowment towards clinical care and will give us the option to expand our services.

The trouble is that the care for sickle cell is poorly reimbursed, the transplants are well reimbursed and it's a moneymaker. My vision long term is to be able to provide that therapy here at Howard University Hospital to build the infrastructure and even the delivery of transplant and gene therapy and gene editing is going to change over the next five years.

How Can Community Based Organizations and Advocates Be Helpful?

Tammy Boyd: Wow, that's amazing. An endowment for the sickle cell center. I mean, I would say that would be quite a legacy. That's excellent. It's an awesome vision. So for community based organizations and sickle cell disease warriors and advocates, you know, what can we do to be helpful? I know we're looking at working on some legislation on Capitol Hill. You know, what can we do or what would you like to see the federal government do in terms of sickle cell to sort of advance sickle cell?

Dr. Taylor: Yeah. So I think this is win win no matter what your political leanings are for all sides. Sickle cell care is expensive. I was called down to the Department of Health in Washington, D.C., my first year at Howard, 2017. I had been in the area for a lot longer, but in the district for six months, and they had done an analysis of Medicaid patients about 600 adults in Washington, D.C. I was the number one prescriber of hydroxyurea in the district. It was only 15 percent of that 600 patients, and they wanted to know if that was an accurate number, if that was inaccurate. I said it sounds about right. And then they saw that seventy five percent of their patients were getting chronic opioid therapy, which is treating a symptom and treating the underlying disease. And I said, that sounds about right. And then they told me something really profound. They spent $71 million a year on sickle cell disease. That's one hundred and twenty thousand dollars per patient per year. And I told them that sound about right to me. Now that I've been around it and we're actually collecting data on this. What I see is that 90 percent of the labs ordered on patients, 90 percent of the imaging studies are done in emergency rooms and hospitals. And some of the most severely affected patients are getting blood counts drawn once every two or three days, which is ridiculous. I couldn't justify doing it more than once a week, sometimes three times a day, because they're going to three different emergency rooms. So when you start to see patterns like that, what can you do as a community based organization? Really highlight how expensive and how inefficient and low quality the care is that we're delivering. Nobody would accept this for any other disease and then tell members of Congress and elected leaders about the history of the disease.

What can you do as a community based organization? Really highlight how expensive and how inefficient and low quality the care is that we're delivering.

And then just funding, I think one of the central issues with sickle cell is that more than 75 percent of patients can't work, which means that they're on Medicare and Medicaid. This is the lowest quality insurance, it pays the least. And what hospitals do is they usually want a small percentage of their patient base to have Medicare and Medicaid. Private insurance pays 100 percent of what they bill Medicare, Medicaid about 33 percent. So places like Howard are unusual in that the university has given me the thumbs up on seeing the least insured, the least financial benefit, and told me, have at it see as many patients as you can. That is a rare thing to find. And again, some of the other places that I've mentioned, the University of Connecticut are doing that. It is on us to look at doing this more efficiently, but that's where people like you bothering Congress and saying, 'What are we doing about sickle cell?' In 2021 we have four FDA approved medications for sickle cell. Another comparable disease that is equally terrible is cystic fibrosis. There are eight FDA approved medications, and they were approved a long time ago for all genotypes of cystic fibrosis. We now have two new drugs that are available to all patients with sickle cell. That's been a major improvement. Prior to 2019, 20 percent of patients I could offer them nothing more than symptomatic therapy. Nothing worked. So we need people to be on the front lines and learning how to lobby Congress. I'll tell you one story in 1975, Roland Scott testified in front of Congress. They wanted to expand from 10 to 15 sickle cell centers. And so he brought up this amazing statistic that in 1974, $40 million had been appropriated for sickle cell disease research and clinical care. But the federal government only spent $16 million, and this is what I've learned from other advocates that you got to keep your eye on the money. That money gets spent, it was just spent on something else. So you've got to be looking prospectively about what you want and look at what you're getting and ensuring that that's being spent on the intended recipient of the appropriated funds.

So you've got to be looking prospectively about what you want and look at what you're getting and ensuring that that's being spent on the intended recipient of the appropriated funds.

Disparities in Funding for Sickle Cell Disease Compared to Other Diseases

Shawna Watley: Can you elaborate and provide, you know, your thoughts on the disparity in funding? Like, why do you think that is the case? And just to put it in context, if you know the numbers or data, like  what's being spent on cystic fibrosis, I know there's a lot less cases of cystic fibrosis compared to individuals who have been diagnosed with sickle cell.

Dr. Taylor: Yeah, so there are about 33,000 cystic fibrosis patients in the United States and the NIH spends about $100 million a year on cystic fibrosis research. There are about 100,000 sickle cell disease patients, let that sink in. Cystic fibrosis is a disease primarily, the mutation in the CFTR gene comes from northern Europe. OK, so it's primarily in people of Northern European descent. Sickle cell disease, the sickle cell mutation arose in Africa because it protects from malaria, so you have to be of African descent. It has nothing to do with your skin color. You just have to have some, some segment of your genome on chromosome 11 that is from Africa. And so we see sickle cell disease in Greece, Italy, Sicily, Spain, North Africa and obviously a lot in Africa. And it's all related to how many of your genes come from African ancestors.

Sickle cell disease, the sickle cell mutation arose in Africa because it protects from malaria, so you have to be of African descent. It has nothing to do with your skin color. You just have to have some, some segment of your genome on chromosome 11 that is from Africa.

Why the disparity? I think it's just sickle cell got started late, 1972. And I think that well, another is that a couple of things have happened. Another terrible disease is hemophilia. And the 1970s were an amazing time where a lot of public funding went into these terrible genetic diseases. And in 1971 or 1972, for hemophilia, they established 174 hemophilia treatment centers across the United States. There are about 20,000 hemophilia patients across the U.S., so that translates into about 125 patients per center. And it was passed through the Social Security Administration. So what is happening here is that is entirely funding clinical care, a doctor salary and a nurse's salary for 125 patients. I have a nurse practitioner, a nurse and myself, and we follow at least 255 patients and we right now receive no federal funding. So you can see that's the disparity in care right there. It's an unintended consequence of legislation being passed for another disease that really provides clinical care and that was never done. That was, as I say, the unintended consequence of the National Sickle Cell Control Act. You passed the research portion of it, but the clinical care portion was not done in the same way. And I spend money on hemophilia just the same as any other genetic disease. But it also relies on advocates and people lobbying their members of Congress so we can all think about in hemophilia Ryan White getting HIV in the 1980s, it was a terrible thing. It was a good thing, the legacy is we saw a problem, we fixed a problem with legislation that led to better care for those patients. Hopefully, that will happen for sickle cell.

That was, as I say, the unintended consequence of the National Sickle Cell Control Act. You passed the research portion of it, but the clinical care portion was not done in the same way.

Vision for Sickle Cell Legislation Going Forward

Shawna Watley: So as we look at 2022 being the 50th anniversary for the center as well as the legislation, would you recommend that we really try to bring back some of the funding for the clinicians or the clinical aspect of it as a part of the legislation? And what else would you like to see in the legislation?

Dr. Taylor: I think the research funding $100 million per year, well on a per capita basis, it's underfunded. And if you compare to other diseases like cancer, you know again the 1970s, Richard Nixon declared war on cancer and created the National Cancer Institute that is the largest institute at the National Institutes of Health has the most funding. That's why cancer care is in private practice, so lucrative, because there are so many drugs and so many clinical trials and so many companies focusing on it. You have a whole institute dedicated to a terrible and unfortunately very common disease breast cancer. I am always amazed at how common it is. It's not to minimize that, but in sickle cell we have the clinical care portion of it, and that makes Ms. Elling's endeavors at bluebird bio that much more difficult because they want to run clinical trials and find out if their therapy is effective so they can apply the FDA and actually sell it to people. Well, to run a clinical trial, you have to have participants and you have to have them organized networks of centers. The National Cancer Institute has comprehensive cancer centers designated all across the country, so when they do a clinical trial, they may have 200 centers participating in a trial. Even pediatric cancer, amazingly rare disease 16,000 children across the country. But there's a network of phase one centers for new drugs to be used in children with cancer. We need some type of network like that. And that's a clinical care network. So I'd say the National Sickle Cell Control Act, as it stands, is great for research and that will lead to innovation. However, if you look at clinical care, that's where the real problem is. So we need another act, and I'd say it should be separate, focused entirely on delivering clinical care and more efficient. And what I'd argue is that we could probably save federal dollars, federal and state dollars in a big way by delivering more efficient care. Just ask yourself, what if we got 90 percent fewer laboratory studies? I don't know what a complete blood count cost. I don't know maybe $150, maybe more dollars, every time we do it, we're doing it every two, three days on a patient. Think about the cost savings there. So some type of clinical care network that could also be integrated with research would be outstanding. It would make Ms. Elling's efforts at bluebird bio and every other company that is developing therapies for sickle cell become much easier because then they know exactly where to go, and these would be integrated centers. I think it would benefit patients because like the Children's Hospital model, you have this big building and everyone knows if my kid is sick, I go there. If you have these centers, at least in sickle cell, you know general public wouldn't know this, but they'd know I'm going to go to the comprehensive sickle cell center and this would also put other doctors on notice. You know, if you don't have the expertise in treating this disease, maybe you should refer them to one of these centers. We see patients who are very, very sick and we don't want to see them when they're very sick. We want to make interventions before they get sick. So it's a different perspective. I think that would be the best advice I could give for the future.

We need some type of network like that. And that's a clinical care network. So I'd say the National Sickle Cell Control Act, as it stands, is great for research and that will lead to innovation. However, if you look at clinical care, that's where the real problem is. So we need another act, and I'd say it should be separate, focused entirely on delivering clinical care and more efficient.

Shawna Watley: Well, that's very, very helpful. I'm just thrilled that you are willing to talk to us today. I know Sonya and Tammy and I are going to come together to figure out how we can incorporate your excellent recommendations into policy recommendations as we talk to members on the Hill.

Sonya Elling: I just want to say thank you for being such an amazing and articulate advocate for the sickle cell community, and I'm sure Dr. Roland Scott would be incredibly impressed with all the work that you're carrying on in his footsteps. And I look forward to seeing you up testifying before Congress because you are an amazing advocate. So thank you for all that you do.

Tammy Boyd: Yeah, I think you all have said it and really articulated it well. And again, I really commend you on your vision for the Howard Sickle Cell Center there and the endowment, those are very, very amazing, you know, goals and accomplishments. So thank you so much for taking your time to talk to us today.

Dr. Taylor: Well, thank you all and thank you for all you do in pharma and in community based organizations. We depend on all of you and and we get donations from all types of organizations, individuals, CBOs and pharma. And it's all very helpful in putting forth our mission and caring for our patients.

Shawna Watley: And so once again, thank you for joining us today to discuss sickle cell disease. We hope that this discussion shed light on the challenges of living with sickle cell disease and the barriers that must be addressed in order to ensure equitable access with the sickle cell disease community.

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