The Cost of Gene Therapy Versus Alternatives

Biree Andemariam, MD: Cost and funding is a little different. Regarding costs of combination novel therapies—like Michael outlined, we need studies. We need funded studies to tell us what combination of what and when are ideal for each individual patient. What do you all think about the cost of gene therapy, potentially, compared with the evolving standard of care? Not hydroxyurea alone. We’ve moved way past that. We’ve got 3 other FDA-approved drugs and probably more to come.

Wally Smith, MD: I was looking at this 2009 paper, which is now horribly outdated, that was trying to put brackets around where we stand with gene therapy costs. Their estimate was that if we took 70,000 patients with sickle cell disease, and we were trying to avoid future health services—cost of services—then it would cost about $70 billion of services that we might avoid. If we treated about 3000 patients with curative therapy, they thought that it would cost about $3 billion over 1 to 2 years in the state of Louisiana. Obviously, you can’t afford that.

Having said all that, these are not mass-produced therapies. I don’t think it’s fair to expect that we should talk about global budgets that are going to be broken with a therapy that, as we just discussed, takes a year out of somebody’s life and has to be done in a specialized center.

I now sit on the Cure Sickle Cell Initiative clinical and economic impact analysis panel. We’re taking this up. It is a very difficult modeling process. Dr DeBaun and I go way back. We both have experience with Markov modeling, and this is a Markov modeling process that has so many variables that have not been measured. We’re trying to set up a realistic model to try to ask, “Over the lifetime of a child and over the lifetime of an adult, what are the important questions to ask? Where might the data come to fill in the answers to those questions? If we were to run the numbers and play ‘What if’ now, what answers would we get?”

As you said, evolving therapy changes the base assumption, the health state at which you begin changes if you are on baseline therapy. Let’s just use Dr DeBaun’s hypothesis. The government has already paid for a study to show “3 drugs that everybody should be on,” before you start talking about comparing a pharmacologic therapy with 1 of these so-called curative therapies. It’s very difficult modeling.

Elliott Vichinsky, MD: The financial analysis has always been the thing thrown at sickle cell disease. It’s a problem for me because we don’t have the infrastructure to provide standard of care. That would save a lot of money down the line, but somehow we’re able to go ahead and not give it to them. Everyone has published, including us, that if you had a day unit—an infusion unit for pain—to treat patients and were providing care as an outpatient, you could decrease the hospitalization rate 60% to 80%. But the way fractured care is set up in the United States, there’s an impetus to decrease not necessarily costs but costs to certain cost centers. If we invested in preventive care, that could have a real benefit, but somehow it doesn’t happen that way. If you analyze the costs, you need to look at productivity of an adult being employed and what their contribution is to society vs living with the pain and those other things, like taxes. I was a little disappointed at how the drugs that were coming out, which are expensive and do make money as venture capital, were under attack for sickle cell when we, for the first time, had something to treat them with. It’s a reflection of the health care system. So when you do an analysis of cost, just make sure it’s complete.

Julie Kanter, MD: Right.

Michael DeBaun, MD, MPH: I just want to give a public health picture here: 5% of the children with sickle cell disease live in North America and Europe. The other 95% live in low- or middle-income countries. That’s public health. The second point, which is critical, is that in all this discussion about cure, we’ve never really entertained seriously educating people of color about what their genetic risk is of having a child with sickle cell disease.

For every $1 billion we spend on curing sickle cell disease, I’d like to see $1 billion focus on educating premarital genetic counseling or preconception genetic counseling so those individuals can make informed decisions about having a child with sickle cell disease. This is a strategy where the science has been lacking and leadership in this space has been pushing the agenda for cure, which is primarily driven by science. It’s unfair to talk about cost-benefit analysis for gene therapy, gene editing, and sickle cell disease when the driver is actually state-of-the-art human science. This is state-of-the-art biology that’s being celebrated is not a triumph of how we’re going to overcome this disease in our community. They’re totally separate. We’re confused between focusing on what the state-of-the-art science is and how we are going to address the larger problem of making the lives better for individuals who have this disease or informing individuals about the chances of them having a child with this disease.

Wally Smith, MD: I love Bill Gates—he was in my class as an undergraduate—but he’s misdirected if he thinks that throwing money at the wrong target is going to wipe this disease off the face of the earth. He does have a lot of money to throw around, and we appreciate it. But we need to be careful, as Michael said. If you’re going to take a utilitarian strategy of what’s best for the most people, we should all move to Africa, set up clinics to treat the patients who have disease, and then start educating, building schools, and teaching the children what sickle cell disease is and that you can prevent it.

Elliott Vichinsky, MD: These are not mutually exclusive concepts. We should do both. The problem is we’re not providing the infrastructure for standard care in all those other services. We should push ahead with cures and drug development, but we should also provide families with the education, preventive care, and all the other things. That’s what we need to do together, right?

Biree Andemariam, MD: Without the proper infrastructure and the access to care that’s needed for the masses with sickle cell disease, it’s challenging to do the economic analyses that I had asked you about.

Transcript Edited for Clarity