Journey into Sickle Cell Disease Advocacy

Lauren: My name is Lauren Sapp, and I’m a Communications Coordinator at NICHQ. Here at NICHQ, our sickle cell disease work spans many years and has been the focal point of several of our projects. In honor of National Sickle Cell Awareness Month, I recently had the pleasure of talking to Dr. Titilope Fasipe, a pediatric hematologist, advocate, and sickle cell warrior. 

Can you tell me a bit about when you started to become interested in sickle cell disease and how it has shaped your professional path? 

Dr. Fasipe: I would say that my experience with sickle cell disease has been for as long as I can remember. I was born with sickle cell disease, and so it has always been a part of me. However, when I first started thinking that I wanted to be a doctor that took care of people with sickle cell disease, it was after I met other people with it. And that first person that I met was my cousin, who was a couple years older than me at the time. She sadly passed away at the age of 17, and I think that was my pivotal moment where I was like, I don’t want this to happen to other people, and how do we prevent this? That was what started my journey into exploring a career path in pediatric hematology, and specifically caring for children with sickle cell disease. 

Lauren: I’m sorry to hear about your loss, but I’m in awe that you were able to take that experience and find what you were drawn to in life with it. That’s a beautiful thing. You have so much experience, and you’re actually a co-director for Texas Children’s Hospital Sickle Cell Program and a team lead on a project that helps provide large-scale sickle cell disease care access in Africa. I was wondering how your work internationally impacts your work in Texas. 

Dr. Fasipe: Thank you for the kind words about my cousin’s passing. I will say that that experience is sadly a very common experience for many people who come from Sub-Saharan Africa. It’s sad to say I know other people that have passed since her with sickle cell disease. This has been a burden that Sub-Saharan Africa has faced for generations, thousands of years. 

When I did become a pediatric hematologist and I was trying to figure out where are the spaces I want to work, I wanted to make sure that the institution that I worked for and got training under had an understanding that sickle cell was more than the rare disease that it appears to be in the United States and that it’s this global burden, something that is quite  

important and challenging, and requires dedicated people, requires resources. So, I would say that when I started working in the pediatric hematology space, I had learned, by that time, several things, and I call this the secondary education about sickle cell disease. Medical school had taught me about the biology, and about why the disease makes people sick, and why it causes complications and problems. But I couldn’t understand the disparity component of it. And I started noticing this first in the United States. I realized that people with sickle cell disease do not have access to quality care. 

It was surprising to me because my lens in Nigeria, where I was born and grew up, I thought it was something about Nigeria being a lower-income country and not having healthcare infrastructure. I thought those were the reasons that contributed to my cousin passing away, and indeed they did. However, I noticed that there were challenges in the United States, too. So that gave me this other education about sickle cell that, in addition to the biology of the disease and the complexities of having the disease, there’s this public health component to it that is based off of society. And whether that society is in the United States or in a country like Nigeria in Sub-Saharan Africa, a person with sickle cell disease, no matter where they live in this world, usually face some kind of disparity that prevents them getting access to good care.  

My early career efforts were focused on understanding the United States’ problem. You mentioned I’m the co-director of the sickle cell program at Texas Children’s Hospital, and that has been a privilege for me to impact care locally in the city of Houston, as well as the state of Texas, even thinking bigger nationally through the country of the United States. I didn’t really think that there was one space that mattered [more than others]. I felt like, somehow, we have to impact all the spaces. But [for] the global health side – it wasn’t quite clear to me where my role was initially. I was quite fixated on Nigeria as a country because that was where I grew up, and I was looking for opportunities to figure out how to do more. I think I saw it as something that would happen later in life, maybe when I was closer to retirement or something, like giving back. 

My journey started during the years of COVID. I started exploring what it looked like to work in the international space in addition to the other work that I had been doing. The advocacy skills and the lessons that I learned on the United States side helped me become an advocate on the international space, too, because the stories are the same. I was able to resonate and share my experiences, and that seemed to be encouraging, and also something that was understood by even the spaces in other countries that are not the United States of America. There’s learning on both sides, but it’s really that story about advocacy. Sickle cell disease, because it’s a public health burden around the world in high- and low-income countries, it requires advocacy, and I’m so happy that I’m able to work in both spaces. 

Lauren: Absolutely. Touching on your experience as a pediatric clinician a little more, can you speak to the difference in the experience of young people living with sickle cell disease to those transitioning into adulthood, and specifically looking at those who are starting the transition from pediatric to adult care? 

Dr. Fasipe: I first encountered a patient with sickle cell disease in medical school. She was a young woman in her 20s, and that was about my age at the time. I saw that the health care system was quite challenging for a patient like her, and that was when I realized I needed to learn more about sickle cell disease. 

I was quite focused on the pediatric side for two reasons. One reason was I do like children. If I didn’t become a doctor, I would have become a school teacher or working with children in another space. But then I also was overwhelmed and maybe disillusioned, if you will, by seeing how challenging the adult side for sickle cell disease was, and I didn’t even understand it fully. I just knew that if I worked in the pediatric space, we could work on changing the generational view of sickle cell disease so that by the time the children I care for are adults, one, people might think about sickle cell disease differently. But then two, maybe we can build a better adult care system for them. So, I felt my energy would be best on the pediatric side. Now that I know even more about sickle cell disease and the adult side, I’m glad I chose pediatrics. And I have to give a shout-out to all of my colleagues who are adult hematologists that take care of adults, like myself, with sickle cell disease, because it’s quite challenging to do so. 

And so, to your question, pediatrics, we have more of a shared model of how to take care of sickle cell disease. Cancer is much rarer in pediatrics, and so people who train in both cancer and hematology, so pediatric hematology oncology, you’re able to get care for sickle cell disease in any space that has a pediatric hematologist oncologist. But on the adult side, it becomes a little bit more complicated. Cancer is much more common on the adult side, and so you’ll see that there are more oncologists or hematologist oncologists who are more skilled in the cancer side of the world. It’s not as easy to find people who have expertise in caring for adults with sickle cell disease. So that’s one of the challenges that faces individuals when they’re crossing over from the pediatric to the adult side. They realize that there’s less people who understand what sickle cell disease is and can answer their questions and can help them live high-quality lives. That’s one aspect. 

The other aspect is depending on which state you live in, your insurance options become much more stark on the adult side. In pediatrics, most states provide Medicaid or programs to help get children into healthcare, and that seems to be an essential right. Everybody believes children need healthcare. But when you cross over to the adult side, having sickle cell disease is usually not enough to get assistance with your healthcare. If you don’t have a job or you don’t have the privilege of having a family that has private insurance, it becomes quite challenging to access care because you lose the ability to pay for that care. And then the other part of this is children can have complications of sickle cell disease, of course, but those complications might increase, or new complications might occur when they enter adolescence, their teenage years, their young adult years. And so, you’re taking a person who maybe had a decent or good experience with their sickle cell care when they were young, and suddenly they start having bad things happen to them, complications that they don’t understand. And that’s quite scary. Then you’re entering again a health care system that doesn’t seem to understand sickle cell disease. 

It’s quite sad that the period between pediatric and adult care actually is associated with increased mortality. So, more deaths are seen in that transition age period, and it’s earlier than what you would expect for the disease when you think about the biology of the disease. In today’s world, the average lifespan for somebody with sickle cell disease can be in their 50s, yet you see this small bump in individuals that are in that young adult period. So, it’s quite sad, frustrating, and it’s something that inspired me and others to focus on: how do we improve that transition period, the access to care, but also the communication between the pediatric and adult side? How do we ensure that people are plugged into good care and don’t have that gap in their care? 

Lauren: As you were speaking to that, especially the experience of many people not fully understanding sickle cell disease and how access to care becomes more difficult for adults, I was wondering if you have any insight into explaining the importance of including sickle cell disease warriors in care plans and care practices. 

Dr. Fasipe: Yes, thank you for that question. This is an important question. I started fellowship for pediatric hematology oncology in 2013, and then I graduated 2016. During that time, I was trying to understand the advocacy side of things, and I started to learn from various community-based organizations. There’s one organization called Sickle Cell Community Consortium, where I picked up this phrase, ‘nothing for us without us,’ and you may have heard this in other contexts too. I have seen that other rare diseases, such as hemophilia, also have similar phrasings about ‘do everything with the community impacted.’ So, nothing for us without us. That became something that I saw was important, not just in the clinical side, but also the research side. Anything that has to do with improving outcomes for sickle cell disease or for individuals with sickle cell disease must have people with sickle cell disease or their caregivers at the table, helping guide the conversations and sharing from their lived experience. 

I love that the hemophilia community calls their individuals lived experience experts, LEEs, and it’s something that I’ve seen on the sickle cell disease side too, where you have community advisory boards, focus groups, intentional conversations with lived experience experts to help guide the care plans, but also the research expertise. So, when you’re thinking about transition programs or how to prepare people for transition, and even when you’re thinking about what happens on the adult side, it is important to listen to individuals with the disease, ask those that have already had the experience, what were the barriers, what were their challenges, and how they feel things could be optimized and improved. The voice of the person impacted, the voice of the patient, the community voice needs to be front and center, and part of that prioritization includes having them at the table in a meaningful way.  

I think all of us in healthcare feel that we are all about the right thing, right? But we sometimes accidentally neglect important things because we don’t have the experience of having the disease. So, something we think is a great idea, we don’t realize it could be additional burdens on families. And just having them at the table allows you to see the positives and negatives of any idea, any plan, before launching into it and then finding out later that, ‘oh, this actually wasn’t the best idea.’ That’s actually even less important. It’s important for the person to feel valued and that they’re being respected. And I feel like it also provides dignity to impacted individuals, seeing that their health care teams value their voice and are elevating their voice in these ways. 

Lauren: Thank you so much for that answer and for that incredible insight. Wanting to pivot the conversation now to talking more about the emergence of treatment for sickle cell disease, what do you think are the most exciting things about the new treatments that are emerging? And how do you, as a provider, guide patients who are seeking sickle cell disease treatment? 

Dr. Fasipe: So, this is a complicated question. I will say that I have two lenses. In my pediatric hematology lens, every new family that I meet that has a baby with sickle cell disease or young child with sickle cell disease, I am quite excited, and I’m telling them, ‘I’m so happy your child was born in this time period because your child is living in the age of sickle cell innovation.’ Basically, your child will likely have a much more different experience than somebody who was born in the 80s, somebody who was born in the 90s. And so, as a pediatric hematologist, I am quite excited and happy that there are more options coming down the pipeline that will actually be reality for the patients that I take care of now. 

My other hat is me as an adult with sickle cell disease, but also as one who has now seen the history of sickle cell therapeutics. This isn’t a glass-half-empty view. It’s just a realistic view. I have mentioned in other settings that sometimes when I hear about sickle cell treatments, I think of the poem, “The Rime of the Ancient Mariner,” where it says, “water, water everywhere, but not a drop to drink.” I do sometimes feel like the excitement about the therapies and the newspaper reports, media, all the coverage is like, ‘look, lucky you, sickle cell, you have all these treatments, all these wonderful things. You should have a parade, you should have a party.’ But then, when I want to access them, or an individual is trying to get them, there’s barriers. You’re telling me there’s water, and yet I cannot drink it. So, I keep that perspective in mind because it reminds me that we still have a long way to go. I am excited that hydroxyurea will no longer be the only option. Poor hydroxyurea was approved in 1998, and it was by itself for decades. And then when you did have other drugs join it, I will say that there’s been difficulties with those drugs. And so, it keeps on telling us that we have to go further. I’m quite excited about the clinical trials that are still running for sickle cell disease. The more we learn, even the ones that don’t work out the way that are intended, the more we learn about sickle cell disease and the treatments, the better we can serve families. 

I think part of your question might be referring to the gene therapies that were approved in December 2023. This is an exciting time for sickle cell disease. I can’t even believe, you know, we’re living in this time, and yet our innovation has outpaced our ability to care for sickle cell disease. We still need to work on getting comprehensive care for individuals with sickle cell disease, and that will make the access to treatments and the treatments themselves all the more impressive. I am celebrating, but I do so with this pause, I guess, of knowing that there’s work to do to make sure people actually can access these treatments. We talked about the global health side – are these new treatments going to be realistic for people who live in other countries, who don’t have access to the clinical trials and some of the innovation? I want individuals with sickle cell disease and their families to have hope. Things are changing for the better, and we need to continue to advocate so that people realize that you can’t just invest resources in treatments and research. You also have to invest the resources on that public health side. How do we get more people who can take care of individuals with sickle cell disease compassionately and comprehensively, holistically? So yeah, it’s exciting times, and in a decade, I don’t even know what we’re going to be talking about, but I know there’s going to be more for sickle cell disease, and that makes me feel good. That makes me feel continually hopeful that the sickle cell disease I take care of now will hopefully be different from the sickle cell disease that’s being taken care of the future. I already have proof that it works. The children I take care of now are much healthier than the children that folks took care of in the 80s and the 90s, and so, we’ll continue to look forward to increased health and quality of life. 

Lauren: That really put into perspective how far treatment has come in just 30 years, but also how there could be a failure to consider that this could be a double-edged sword. 

Dr. Fasipe: Double-edged sword is definitely the appropriate word. That’s actually how I view it. It’s beautiful, but there is this other side that we have to appreciate. 

Lauren: So, going off of that and continuing the conversation about treatments, the next question I have for you is, do you see any pitfalls or areas of concern as symptom and/or curative therapies develop? 

Dr. Fasipe: My colleague and I actually just wrote a commentary about an article submitted from a group who was trying to figure out: how do we ethically allocate gene therapies? Now that they’re here, how do we actually do it in a way that is ethical, compassionate, and making sure we get it to people who actually need it? We titled this commentary “Reality Bites.” So again, we have these two new gene therapies. There’s some other research trials going on; we might have more soon, and everybody got excited. And then you have the challenges.  

We had already had an inkling to what some of these challenges would be because in 2017 and 2019, we had additional therapies approved for sickle cell that were not gene therapies, and the cost associated with those therapies caused a lot of concern in the community and lots of talks with insurance companies, who gets this covered, who doesn’t, who has access, who doesn’t. Regardless of the price tag, for the family, they may not see $3 million, they may not see $2 million, but it does equate to high co-pays. And there’s also this unhidden cost in addition to the financial burden. There’s also the burden of jobs. The families have to take time off of work, taking care of the other children who don’t have sickle cell disease, you know, there’s so many other things. Coming to the hospital multiple times, living in the hospital, all those things play a role in cost and the financial burden of sickle cell disease, of a person getting gene therapy with sickle cell disease. The other part is these therapies, you’re not able to infuse them in the body without taking your cells out first, and that process also adds another level of burden. The process of stem cell collection, the process of actually doing the gene therapy process steps, all of them have risk for not going the way that you plan. And so, it’s not as straightforward as a donor-based transplant, where you already know where your cells are coming from and there is a process that has been in place for decades. 

So, the barriers include financial cost, not just, again, the cost of the treatment, but just all the costs of life, like all the things that a family has to do. It includes the challenges of the way that innovative therapy is required to be implemented. So, that includes going to a qualified treatment center, that means not everybody will have easy access to that. And then I think it goes back to what I said earlier: if most people, especially adults with sickle cell disease, do not have access to a person with expertise in sickle cell disease, how will they even get access to the gene therapy, you know? If you try to just walk to a gene therapy center, they’re going to ask you who’s your hematologist, and they need to understand certain things about your health before they could even think about doing gene therapy. This goes back to the biggest problem of all. You need comprehensive care for sickle cell disease in order to ethically and thoughtfully provide them innovative treatments. That’s the double-edged sword, if you will, of the gene therapy conversation. It’s exciting, but there’s so much work to do to make it a reality for more people. 

Since the gene therapies were approved in December 2023, it’s not as if there was this huge stampede and multiple people have been getting it. It’s been this steep hill, and center by center will report different challenges, but people are having meaningful conversations about how to address these barriers. You may have heard that CMS, in fact, knowing that most people with sickle cell disease are more likely to have a Medicaid type plan, they’ve engaged a model called the CMMI model where they have crafted a plan with the states and with the providers of gene therapy, the companies, to try to make the gene therapy accessible to individuals with Medicaid, so without commercial-based plans. And so that’s one step in the right direction, but there are so many things that need to be done before we can talk about lots of people getting gene therapy. Right now, we’re maybe talking handfuls of people, and so we need to keep on working towards that future where it’s for more people. Some people feel like the future lies in changing the way gene therapy is given and in something called in vivo gene therapy, and that will probably be wonderful, but I think innovation comes with the challenges of cost, with the challenges of access to the right locations, to the locations that have that expertise. And so, it’s something to keep in mind that, it’s gonna be like the poem again, not everybody will have this immediate access to the novel therapy. It will be challenging for several people. 

Lauren: To your point of, you know, some people think this is the way to go, while others may disagree, wanting to turn the conversation back to you and your thoughts and experiences, understanding that you do have a holistic view of this condition. So, given this, what are some of the advocacy efforts you would like to see happen to advance sickle cell disease research and treatment? 

Dr. Fasipe: I started realizing I need to open up my definition of the word research. When I entered medical school, I thought the only way to help sickle cell was curing sickle cell disease. So, I actually wrote in my medical school statement, ‘I wanna enter medical school because I wanna cure sickle cell disease.’ And at the time, I thought you had to be a geneticist to do that, so that’s what I told them I wanted to do. 

Fast forward, when I actually saw the clinical side more, I realized that the research should not just be on, again, a treatment towards impacting the biology of the disease, or what we call disease-modifying therapies. We do need research in those areas, and I’m glad that we have folks working there, but we also need researchers in the area of implementation science. You know the evidence-based guidelines for sickle cell disease – why is there a gap for people getting them? Why is there a gap to access for care? Implementation scientists do research in those areas: how to improve those gaps to get the treatments that already exist, for instance, hydroxyurea, or the important screenings that you need for sickle cell disease. Then there’s also people who work in something called outcomes research. And these are all somewhat in that bucket of public health research. 

I would like to advocate for things that are not just about disease-modifying therapy but are about the gaps of getting therapies to people, to getting good care to people. And then back to the comprehensive care. Comprehensive care isn’t research. It’s just the right thing to do. It’s the ethically right thing to do to say that I believe people with sickle cell disease should have access to good care. [This is the] realm of quality improvement, you know, at these highest levels. Actually, a lot of work that I do is in the area of quality improvement, and it overlaps in that outcomes and implementation science space as well. I feel like that’s what we need to advocate for, because for every new drug or exciting idea that comes, it’s that bite. How will people get this? How will people get access? We need to expect more for the disease. For years, I think we just accepted poor quality care for sickle cell, like that’s just the reality. And I think if we change our viewpoint and say, ‘no, this should be our [new] standard,’ it allows us to work towards approaching that goal and not just settling for a status quo that is, again, unethical. I’m so excited that people are putting money into the gene therapies and to other exciting therapies, and I want to continue to see that, but I also want to see research in areas that help people get that care. 

Lauren: I wanna give you any room to say anything that I might not have asked about, or any other important thoughts you have to close us out. 

Dr. Fasipe: I am grateful to share this time in Sickle Cell Awareness Month September with you guys. I’m quite humbled and I have a lot of gratitude for your team for elevating sickle cell disease. I do feel that it’s kind of frustrating or hurtful to seem like you accidentally landed on the good side of a coin, and that your ability to live a quality life with sickle cell disease involves chance, involves luck. I don’t like that. And I want people to recognize more and more that there are things we can do to help people having good outcomes, not just be by chance, but to be intentional and to be more far-reaching for everyone. I’m grateful on the pediatric side, I’m able to see that. I am desperate, along with my fellow warriors who are adults with sickle cell disease, to also have more of that. And I already mentioned great gratitude to those that work in that space. There’s so many heroes and champions in that space that I’m happy about. 

And for those that are still learning about sickle cell disease or want to learn more, I hope you’ll continue to expand on your knowledge and looking for where in your own space, how can you help sickle cell disease, and that does not have to be necessarily in the clinical space. People who are blood donors, you’re helping sickle cell disease. People who are in the marrow registry, y’all are helping sickle cell disease. If you look intentionally for community-based organizations in your area or your local hospitals that work with sickle cell disease, those are all ways to support the efforts of sickle cell disease. So yeah, thank you for this time, and happy sickle cell disease awareness month. 

Lauren: Thank you so much for your time and for joining us this month. It was great to have you.