Revolutionary sickle cell therapy treatments are available. But at over $1 million, confusion over access persists

NINA MOINI: It's been about two months since the FDA approved two new gene therapies for sickle cell. The treatment could be a cure for more than 1,500 Minnesotans with sickle cell disease and millions of people worldwide. The genetic blood condition causes horrible pain and can lead to deadly health complications, like stroke. People of African descent are more likely to have the gene, and the new treatments could address an urgent health equity problem.

But with a price tag of millions of dollars, it's unclear how or when they'll reach people who need them. So joining me to explain is Rae Blaylark, a president and CEO of the Sickle Cell Foundation of Minnesota, and Dr. Roy Kao, an Assistant Professor of Medicine at the University of Minnesota who specializes in gene therapy for sickle cell. Thank you both for making time for us this afternoon.

RAE BLAYLARK: Thank you for having us.

ROY KAO: Thanks for inviting me.

NINA MOINI: Of course. Rae, I'd like to start with you. I know you have your own family history around this, and you really saw a need and created the Sickle Cell Foundation of Minnesota. What is the reaction that you've been hearing from patients about this treatment?

RAE BLAYLARK: Well, it's a mixed reaction. It's certainly an excitement because this disease was identified more than 100 years ago, and there are very limited treatment options. So something like this is really the idea that we could transform the lives of individuals whose lives have been impacted by this genetic blood disorder.

NINA MOINI: Absolutely. And then on the flip side there, what have you heard from insurance providers?

RAE BLAYLARK: Yeah, the concern that we can build it, but how will they get there? I'm hearing that from all sides. I'm hearing that from both the pharmaceutical industry as well as the community and, in fact, the clinical spaces as well. So communities and individuals in those communities, who are impacted by sickle cell disease, who are interested in seeking out a curative therapy, are very concerned because many understand the challenges that come with insurance and the hoops that you have to jump through.

So the concern is it's here. How do we get to it? How do we ensure that there's affordability, that there's a pathway to making what was once a dream now a reality?

NINA MOINI: And doctor, you're involved in the trials for these treatments. How effective are they? And why are they so expensive?

ROY KAO: Yeah. I think in order to answer that second question, you have to go back and understand how sickle cell disease really-- and just going off of what-- I'm super happy that Rae is here, but going off of what Rae was saying, this is a genetic disease that contributes to a lifetime of suffering for these patients with sickle cell disease.

It's the estimated medical cost for a lifetime for these patients is about $1.6 to $2.0 million.

NINA MOINI: Wow.

ROY KAO: And so if we can if we can improve the lives of these patients, we could potentially not just save that money but allow for these patients to live fuller, and healthier, and more productive lives. So that $1.5 to $2 million, it doesn't include all the time that they're missing from work. It doesn't include lost productivity. It doesn't include lost time with family. It doesn't include lost time feeling like a normal person.

So I think that's the important context to know. What makes this really transformative and potentially curative for people is that, in the clinical trials, of which we were a participating site, it was shown to decrease these episodes, these attacks, vaso-occlusive episodes, in about 95% of patients. That's certainly transformative, certainly so much better than anything else we have right now.

So this is an exciting time for us. But just like Rae is saying, this is tempered by the big question of how we're all going to get this paid for.

NINA MOINI: Yeah. And what does it maybe typically take for the cost of a drug to go down? Or what would it take?

ROY KAO: Oh, that's a great question that I have no--

[LAUGHTER]

no qualifications to answer.

NINA MOINI: What do you think, Rae?

RAE BLAYLARK: From my understanding, bringing a drug to market is a quite expensive process. The research that goes into it requires a lot of dollars on the front end. It requires dollars that are invested into the pathway to get the end result. There's a number of steps.

So we recognize that it takes millions and millions of dollars to bring a drug to market, let alone a cellular therapy that is fundamentally very new from a scientific perspective or newer in the medical space. So the cost is really about making sure that it's done well. It's making sure that it's done in environments that are conducive to the needs of the patient, that have expert physicians and specialists that are able to care for the patient, not just during that process but even after the therapy has been administered, what are the needs that happen both in the hospital as well as outside of the hospital.

Going back and forth to a hospital during a treatment like this can be quite cumbersome. But it also takes up the time, the energy, and the space of institutions as well as families. So when we look at a new therapy that has hit the market and certainly, again, something like cellular therapy, we're talking about a revolutionary medicine. The cost of revolutionary medicine is not cheap.

However, the cost of caring for a condition like sickle cell disease, as you heard from Dr. Kao, is not cheap either. So we have to place the importance and the priority upon human lives and how do we improve their lives, how do we change the trajectory of their quality of life by investing in this medical phenomenon.

NINA MOINI: And I just hear it in your voice, Rae, that you've been such a trailblazer, too, in trying to make sure that people do get the therapies that they need. Can you talk a little bit about, without these therapies, what treatment for sickle cell typically looks like, and if you wanted to share what it was like for you and your son, and what brought you to this point?

RAE BLAYLARK: Yes. I'm glad that you brought that up. I have a 27-year-old son that lives with sickle cell disease, and he started his complications of sickle cell disease at four months old. That was how old he was when he-- when I first watched fentanyl drip into his veins. That was devastating for me. That was the beginning of a new normal that I never expected.

So watching him go in and out of the hospital, watching him miss exorbitant amounts of school and academics, watching him lose jobs as a teenager, and even now as an adult, having to defend what his body is going through and his need for medical attention in order to keep his job. This is quite the change. Prior to options like this, all that you had was a medication that was not created for us, though they found it to be able to be used with us, called hydroxyurea.

It was originally meant for leukemia. It was originally meant for another condition. However, using it in sickle cell disease has-- it's a chemotherapy, so it requires monitoring. It requires going to the clinic and the hospital consistently throughout the entire time that you're on it.

But it's not for everyone. It's not a one-time fix-all. It's not accessible by everyone. It's not tolerated by everyone. So that's just one.

But the other really big issue is that we are in the midst of an opioid overdose crisis. For those who are in the pain community, not just sickle cell disease but definitely those individuals who are suffering from sickle cell disease, being able to get your pain treated has become much, much, much more difficult. And the ability to be treated both respectfully but also believed in the intensity of the pain has made it very difficult and has actually created even more complications for individuals living with this disease. And when there's more complications, now you have increased mortality, and that's what we don't want to see.

NINA MOINI: Absolutely. The American Society of Hematology says sickle cell is the most common genetic blood disorder, and Black people are more likely to carry the gene. And you spoke about it a little bit there, Rae, of how racial bias has affected the treatment. Dr. Kao, is the U doing anything to make sure doctors and doctors in training don't perpetuate this bias?

ROY KAO: Yeah, certainly. I think what we're really doing here at the University of Minnesota with Dr. Boucher, Dr. Ashish Gupta, and many others, especially working with Rae Blaylark, is to help educate the next generation of health care personnel about sickle cell disease and how this is a horrible disease that not just the pathophysiology, for which we have a long track record of research here, but also the clinical care, and also the fundamental impact of race on the patient experience, inside and outside the health care setting.

So these are the things that we want to help new doctors, new nurses, new health care providers understand. And in addition to this, it's really working together with all the blood specialists around the state and in a few extra states as well to really build that capacity and help educate our community.

NINA MOINI: And Rae, I'd like to leave a last word with you here of just anything else that you think is just incredibly needed right now to get sickle cell treatment to where it needs to be, what would your message be?

RAE BLAYLARK: I think that it's interesting that sickle cell disease was the first molecular disease identified in the medical records here in the US. And here we are at a crossroads. We have an opportunity to now sickle cell disease being one of the very first molecular conditions that can be addressed through cellular therapy, like gene therapy.

This is monumental. This is an opportunity to change the trajectory of not just a person's life but a community's life. To have one in 13 individuals identified with sickle cell trait, that means that when you are in your own community, being able to have family planning, being able to determine who you could have children with and build a family with has been challenging. And so we want to change this.

It is the most common genetic blood disorder in the entire world. Here in Minnesota, we have an opportunity to change the trajectory for Minnesotans who are impacted by this disease. So whether you're a patient, whether you're a caregiver, like myself, whether you are a family member, whether you're a neighbor, or whether you are a medical provider, we all play a role in making sure that we have access to medicines that are transformative in nature and [INAUDIBLE].

NINA MOINI: Oh, I think I lost you there, Rae. But thank you both so much. We'll leave it there, and we do appreciate your time. Rae Blaylark, President and CEO of the Sickle Cell Foundation of Minnesota, and Dr. Roy Kao, Assistant Professor of Medicine at the University of Minnesota, thank you both for your time.

ROY KAO: Thanks for having us.

RAE BLAYLARK: Thank you so much.

ROY KAO: Thanks.