Improving the Quality of Life for People with Sickle Cell Disease
For Sickle Cell Awareness Month, Dr. Christine Lee speaks with CAPT David Wong about his ongoing work at HHS to address sickle cell disease.
Health Equity Forum Podcast: Episode 16 - Transcript
Dr. Christine Lee: Hello, and welcome to the Health Equity Forum Podcast, presented by the FDA Office Of Minority Health and Health Equity. I’m your host, Dr. Christine Lee, the Acting Associate Commissioner for Minority Health and Director of the Office of Minority Health and Health Equity at FDA.
As we observe Sickle Cell Awareness Month this September, I am pleased to welcome to the Health Equity Forum, Captain David Wong, who is the Senior Advisor for Sickle Cell Disease in the Office of Policy and Legislation within the Office of the Assistant Secretary for Health, U.S. Department of Health and Human Services. Previously, Captain Wong chaired the inter-agency HHS Sickle Cell Disease Workgroup during 2018-2020, and also led work on community health worker sustainability.
Captain Wong’s past assignments have included the HHS Office of Minority Health, the Centers for Disease Control and Prevention, the National Park Service, and the Indian Health Service. Captain Wong also deployed multiple times during the COVID-19 pandemic.
Captain Wong completed his pediatrics residency at the Children’s Hospital of Philadelphia and started his public health career as a CDC Epidemic Intelligence Service Officer.
Doctor Wong, welcome to the HealthEquity Forum podcast, and thank you so much for joining us today.
CAPT Wong: Thank you so much, it's great to be here.
Dr. Christine Lee: Can you tell our listener how you first became interested in health equity research? What inspired you to dedicate your career to these important issues?
CAPT Wong: Yeah, well, it's interesting because if you really look at my career, I started my career as an infectious disease epidemiologist for the first 16 years. So I did a lot of work coordinating outbreak response, working with CDC, with state health departments, and honestly, it was really satisfying work.
You got to work with a lot of different folks during time of need, got to work with a lot of different special populations. And it really kind of, you know, inspired the adrenaline junkie in me in terms of, you know, really thinking about what you can do on your feet to respond to an outbreak very acutely. I had a change in my career mid-career, and I found a job at the HHS Office of Minority Health in 2016 and that was my first entry into health equity.
And it was a lot of learning for me to be honest at the beginning because it was a totally different approach to public health. There was less this kind of acute, you know, urgency towards outbreak response, but more of these underlying systemic issues that needed to be addressed. And I worked with a lot of new partners like CMS and HRSA.
I had never worked with them before in an outbreak response capacity. And also there was this other element of really engaging with communities, really understanding what their needs were, how we could empower them, how could we address some of these systemic, structural discrimination, racism issues. And it was really energizing for me because I felt like I could really make an impact over these large populations, not just during an outbreak response, but really for their entire lives.
Dr. Christine Lee: For our listeners at home, some of you may know that sickle cell disease is the most commonly inherited blood disorder in the United States. But I'd like you to give us an overview of sickle cell disease, who it affects, and how.
CAPT Wong: Sure. So the underlying issue with sickle cell disease is a genetic mutation that affects the hemoglobin protein which is what carries oxygen and red blood cells. And with this mutation it causes the red blood cells to change shape. So instead of being this disk-shaped spongy form that can easily go in and out of blood vessels, even small blood vessels, the sickle cell blood cell is rigid and shaped very long and elongated like a like a sickle, and that causes many problems.
So first of all, the sickle cell, red blood cell doesn't carry oxygen as well, and that leads to fatigue for many sickle cell warriors, as they like to call themselves. The sickle cell red blood cell also doesn't live as long. So it dies and you end up with anemia, low blood cell counts.
And then probably the biggest problem of all is that these rigid sickle cells end up clogging and blocking blood vessels everywhere in the body. And so the main characteristic of that that you'll hear about are these acute pain crises. Some people call them vaso occlusive episodes or VOEs.
But you know, besides it causing pain from the blockage, it can also cause a blockage of blood flow to every organ in the body. And so you can have problems with your eyes, with your heart, with your lungs, with your spleen, with your kidneys, you know, the list goes on and on. And so you can end up with all kinds of acute complications, chronic complications that builds up overtime. And so people think about sickle cell, maybe the first thing they often think about is pain.
But pain is just one aspect. There are many other issues that people may be dealing with silently with this disease.
Dr. Christine Lee: Can you please tell our listeners about your work on the report by the National Academies of Sciences, Engineering and Medicine and your role on the Sickle Cell Interagency Working Group? Can you also tell us about the working group's report to Congress that came out in December of 2023?
CAPT Wong: Sure. First of all, with the NASEM report that was issued in September 2020, that's a project that I'm very proud of. This was work that was commissioned by the HHS Office of Minority Health and also the Office of the Assistant Secretary for Health and it's the nation's first most comprehensive sickle cell strategic plan and blueprint ever since the disease first received federal funding in 1972.
So we start, we received this report almost 50 years after the first funding. And what I really love about the report and the NASEM process is that it's very objective. Besides a very short paragraph statement of work that we developed together to develop the charge for the committee, all the work was conducted by NASAM on their own. They certainly asked for some input from us along the way. They could take that advice or leave it.
It was really up to them and the body of experts, which was multidisciplinary. It wasn't just sickle cell experts that were on the committee. They brought people from all different disciplines to really contribute to this work. And the result was a 500-page comprehensive plan and blueprint, which includes 25 recommendations across 8 overarching strategies. And I’m very proud to hear that the report, after it was issued in September 2020, has been extremely well received by the community, by providers, by researchers. People are using it truly as a blueprint and we are too as well within the federal government in terms of, you know, fighting areas where we can all rally behind, find synergy across different agencies, including FDA to address some of these priority strategies and recommendations.
And the report to Congress that you referenced in December 2023 was an update, a progress report on one aspect of that larger NASEM report is really focusing on treatment. And so with that process, you know, we, you know, queried the different, different agencies in HHS to provide input. We collated that information into this progress report, and we continue the fight today.
Dr. Christine Lee: Since the report to Congress on HHS’ efforts around sickle cell disease came out last year, what kind of progress have you seen in addressing unmet care needs specifically with the FDA?
CAPT Wong: Well, first of all, we really value the partnership with the FDA. As you know, your office has been a very important contributor over work with sickle cell disease for many years. Besides your office, we also work very closely with CDER, with Doctor Ann Farrell and then also with CBER, with Doctor Peter Marks and his team. And together, you know, you guys contribute in so many different ways. One of them is with the clinical trial endpoints. This is very important. This was work that Doctor Farrell's team led with the American Society of Hematology, I think in 2018 or 2019.
And the issue of clinical trial endpoints and endpoints in general and outcomes is even more important now that the two new cell and gene therapies for sickle cell disease were approved by FDA in December 2023. Because many of the outcome that many of the access models that states and CMS are developing are based on outcomes. And so having a better clarity on exactly what those outcomes are, to then determine payments related to access to these very expensive therapies is an important issue.
And another area I'll just highlight is with clinical trial diversity. I think that's something that your office as well as other parts of FDA and other operating divisions at HHS are working on. But that's so critical because as we continue have more and more therapies for sickle cell disease in the pipeline, we need to have good data for safety and efficacy. And in order to do that, we need to build trust with the community. They need to have awareness about these clinical trial opportunities. They need to understand how they can contribute. They need to understand why their voice is important.
Dr. Christine Lee: Are there any new activities from the Sickle Cell Disease Federal Interagency Working Group that you can share with us?
CAPT Wong: Absolutely. There's several major priorities that we've developed with our leadership. And I specifically want to mention Admiral Rachel Levine, 17th Assistant Secretary for Health, my big boss. She is such a huge champion and supporter of all the work we're doing in sickle cell disease. One of our big priorities this year is to increase meaningful access with the sickle cell community and the warriors, as I said earlier, they like to call themselves, if I look back in the past when I was leading this work in 2018 to 2020, we did some engagement with the sickle cell community, but not nearly enough.
And so what we want to think about with this year is and moving forward is to really bring that sickle cell warrior voice in early. So it's helping to shape projects. We hear the needs, we take that information, and we help it to drive strategy, to drive new programs, new policies versus what, you know, if I'm looking at myself, even back in 2018 to 2020, maybe more of what we did back then was, you know, bring in a patient voice.
But already after a program had been developed and the idea had been formed to just have a warrior there as part of the discussion, but really too late in the game to really make any changes. So we're really trying hard to change that paradigm.
And so one of the ways we're doing that is to have better ways to engage with the community. So last week we launched a new sickle cell resource box, SCD@hhs.gov. And we've never had this before at the Office of Secretary, an e-mail where people could engage with us directly and not depend on a listening session or a more formal aspect to do it. But now they can engage with us ad hoc all the time. So that's one thing.
We're hoping to develop later this year, a sickle cell disease website, a landing page which will help the community to learn about the different HHS agencies, including FDA, what they're doing around sickle cell disease, have all that information in one location.
And then I also want to mention a new series that we are developing called SCD SOAR. And S-O-A-R stands for Services, Opportunities, Activities and Resources. And this is what we're hearing from the community is that besides a lot of the sickle cell programs that they're already aware of, like the Sickle Cell Disease Treatment Demonstration program at HRSA, the Cure Sickle Cell Initiative at NIH, they want to learn about programs that can improve their quality of life.
And right now, their understanding of those agencies and those resources is limited. So we want to change that with this new series. And so we plan this bimonthly virtual series.
The first inaugural session is in August with SAMHSA. They're going to talk about mental health resources, and then our plan is to have HUD talk about affordable housing for the sickle cell community in October.
Every two months, we'll march out to a different federal agency and new resources to improve and address all of the needs of the sickle cell community, not just the needs for their sickle cell disease.
Dr. Christine Lee: Going back to the National Academies report, what sorts of barriers to care were identified and how can people living with sickle cell disease and their caregivers, friends, family members help them to live in an empowered and thriving life?
CAPT Wong: That's a great question. And as I said earlier, there are 25 recommendations across eight strategies in the NASEM report. So there are a lot of different gaps and needs, but let me just focus on a few of them here today.
So one that I would want to mention is with sickle cell data, in order to really respond and develop appropriate programs and interventions and deliver them in the right places, you need to know where the sickle cell community is living. And that's challenging because right now we don't even know for sure how many people living with sickle cell disease are in the United States. And that's because we don't have a comprehensive national surround system for sickle cell disease.
So if I look back at 2018 to 2020, CDC had a program, they still have a program called Sickle Cell Data Collection or SCDC. In 2018, there were only two states participating in that program, California and Georgia. Now with additional funding that CDC has received, they are now expanded that program to 16 states, which includes roughly 50% of the sickle cell population in the United States.
As that program grows, as we get more data, we'll be able to understand what the needs are, where the people with sickle cell disease are living, how close are they to a comprehensive sickle cell center, how easily can they access sickle cell care. And then with that information, we can better target our limited resources. So I think that the data piece really needs to be expanded.
Second, I would say is another area with quality measures. So as we know, having measures that are actionable, that are enforceable help to drive quality of care. While, there are no mandatory or required measures for sickle cell disease for either children or adults, currently. And so I think this is something that the community is continuing to work on to get a buy in on different quality measures and then hopefully that those will be embedded into different programs that then will drive and increase the quality of care over time.
Dr. Christine Lee: As I noted during your introduction, you've been working on sickle cell disease for some time. Can you highlight what you believe are some of the most promising proposed solutions to engage meaningfully with people living with sickle cell disease?
CAPT Wong: So I mentioned earlier some of the activities we've already embarked on to engage the community. I would say, you know, just high level, one of the things that's really important is sincerity. I mean, the community has to honestly believe that you care. You're having these listening sessions, you're creating these resource boxes, you're creating these new series because you want to take that information and turn it into positive change.
And we're in a very unique position here in the federal government. We have a lot of reach in terms of NIH, FDA, CDC, CMS. We all have different missions, but all of it is to really improve lives for American people.
And so I take that, you know, opportunity very seriously. I really think of it as a responsibility to if we're going to have these sessions that we really think strategically and deeply about how we are going to use that information. So hopefully that is coming across with the community, with the different events, the different panels, the different activities that we've done.
I'll mention also we are planning a first ever inaugural HHS Sickle Cell Disease Summit on September 24th in the Great Hall of the HHH or Hubert H Humphrey HHS Headquarters building. This is the first time we've ever had a full day summit on sickle cell, that we know of, and Secretary Becerra will be giving opening remarks.
We plan to have a very robust agenda. We want to have multiple TED talks from sickle cell warriors. We want to have diverse panels with at least one warrior advocate on every panel. And we really want this to be a celebration of the community for the achievements that have been made in sickle cell disease, both in the federal, you know, agencies, but also within the community themselves. Knowing that we've made a lot of progress, but there's still a lot more work to be done.
And the last thing I'll mention about the summit is that we also see this as an opportunity to bring in new voices and new sectors. So there are many other groups that I think once they hear more about sickle cell disease, once they meet, you know, these empowered individuals living with sickle cell disease, it's very easy to root for them. It's very easy to be inspired by the sickle cell community. And so we want to give these sectors a chance to meet them. So sectors like HBCU presidents, Black fraternities and sororities, faith-based leaders, Black and Hispanic media, other kinds of celebrities, influencers that are involved, we want to invite them to this summit on September 24th so that the sickle cell community can expand the groups that they work with and that can help to advocate for them on their behalf.
Dr. Christine Lee: Please tell us about how you and others across HHS are increasing access to care and various therapies for sickle cell disease.
CAPT Wong: Well, I mentioned earlier the CDC programs. I think data is really critical to understanding where the needs are, where, where folks are living, where we need to develop new resources, perhaps new clinical comprehensive sickle cell centers.
Besides that, I really want to give a shout out to HRSA and their sickle cell disease treatment demonstration program and newborn screening follow up program. These are two long standing grant programs that HRSA runs through their Maternal and Child Health Bureau. These are really important, what we call hub and spoke models, where knowing that there's a limit in terms of the number of specialists for sickle cell disease, they're not going to be found in every single city or town in the United States.
So they really work on this hub and spoke where the hub as a comprehensive sickle cell center does additional outreach and education with downstream providers that might be primary care providers. They might be smaller hospitals that don't have their own sickle cell clinical comprehensive center to provide additional expertise, mentorship, direction in terms of making sure that people with sickle cell disease that don't live near some of these comprehensive centers or don't have easy access to those centers, they still can have access to high quality care. So that program continues and hope that it will continue to receive additional funding and resources that it needs.
And then I'll also mention the CMS cell and gene therapy access model, which you know it's in the works with different states and with the manufacturers. The hope is to launch that model in January and that is outcomes-based agreement so that, you know, high level, I don't know the details of it, to really kind of decrease the cost of these expensive cell and gene therapies. And that's a real game changer because this provides access to potentially curative therapies for many individuals with sickle cell disease that qualify for it.
Dr. Christine Lee: Are there any other efforts you would like to highlight on how the department is working to improve quality of life and well-being for people with sickle cell disease?
CAPT Wong: This is so important in terms of what we're hearing from the sickle cell warriors and community themselves is that, you know, the quality of life is something that is so important to them, not just living longer. We're seeing that anecdotally. And I think some recent studies are showing increased life expectancy, but that's really not enough.
It's so empowering to hear warriors, you know, ask for, in fact, demand a higher quality of life. They deserve that. And so, as I said earlier, we have this SCD source series as one way to help connect folks to different federal agencies and the resources that can help support warriors, social needs, you know, other aspects of their life. That's one big effort that we're leading.
But I think in general, it's really just, you know, having other kinds of awareness building around quality of life. So we've had several panels. One that we had in July was on mental health and sickle cell disease. I would say that's the number one quality of life issue, if I had to pick one for the sickle cell community, is supporting their mental health.
You know, it's a very challenging for anyone with any chronic disease to kind of keep their strength, their resiliency. That's true really across the board with chronic disease. But then with sickle cell disease, you also have, you know, additional traumas with receiving discriminatory care, biased care.
Often in the emergency department is the anecdotes that we hear where they may be accused of being drug seekers. They don't receive the pain medication that they need for their crises. People doubt and, and don't believe them, and don't respect them, you know, in terms of the story and the request that they're making of the health care community, it's very sad to hear these stories and anecdotes over and over again.
But that kind of trauma that you experience builds up overtime. There's a there's an ongoing toll and burden of that that really needs to be addressed. And so that's something that came up very loud and clear in the mental health panel that we held in July. So I think, again, this is going to take time.
We want to continue to support the community. We want to hear what it is. Quality of life is different for any given person. What matters to them? We're going to have a session on quality of life at the Sickle Cell Disease Summit in September, on September 24th, and we'll continue to explore with the community what it is, more, that we can do with the federal government to support this very important area.
Dr. Christine Lee: As you consider the results of your work thus far, how do you see this work supporting your efforts to continue to advance health equity?
CAPT Wong: Absolutely. So, everything that we do with sickle cell disease, in my opinion, is something that can be done broadly for health equity. And so, for example, the SOAR series I talked about earlier, it's really targeted for the sickle cell community. We're going to work with federal agencies to really curate the best programs, the best resources that align with what we're hearing are the needs from the sickle cell community.
But why couldn't a similar series like that be done for other conditions? I think that people like to hear about information in a very tailored way. It really speaks to them. They can take that information and then in turn advocate with others, use that to have conversations with their providers, for example. And it's very difficult, in my opinion, for people to find that information on their own. Just Googling, going through a massive bureaucratic website like we have at many of our agencies, it's very hard to drill down to the specific information that you need, particularly if you're not working in that agency.
And I can tell you, even though I'm working in Office of the Assistant Secretary for Health or Office of the Secretary, I don't have visibility on all the programs just within that tiny slice of government. So how can we expect the community to find these resources on their own? It's not easy.
So I think we need to continue to challenge ourselves to build those educational tools as one tool to reduce disparities. But then I think there's so much work that we need to do in partnership with the community itself to address some of the implicit biases, the structural issues that are, you know, you know, impeding progress for sickle cell community.
I'll just mention one other, which is really a shortage of hematologists who specialize in sickle cell disease. This is a chronic problem, and there are a lot of financial disincentives for hematologists to choose careers in sickle cell disease versus more lucrative options that are in front of them. And we need to think about how to reduce those disincentives or think about other ways to incentivize hematologists and other specialists who want to work with sickle cell disease. Those are recommendations in the NASEM report as well.
A little harder for us to operationalize, to be honest with you, but I think those are very critical underlying issues that, again, will help address equity.
Dr. Christine Lee: That's great. Thank you so much, Doctor Wong, for being with us today, and of course thank you for your leadership at HHS. We truly appreciate the opportunity to collaborate with you on this important work.
I also want to let our listeners know, if they would like more information about the Health Equity Forum podcast series, visit our website at www.fda.gov/HealthEquity. While you're there, check out our library of resources and sign up for our newsletter. Also, don't forget to follow us on Twitter, @FDAHealthEquity.
Remember, during this Sickle Cell Awareness Month, working together, we can create a world where Health Equity is a reality for all.