Cost Utilization Analysis in PNH Treatment Pathways

EP: 1.Phase III Apply-PNH Study Overview

EP: 2.Selecting Optimal Treatment Therapies for PNH

EP: 3.Biosimilar Integration into PNH Treatment Landscape

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EP: 4.Cost Utilization Analysis in PNH Treatment Pathways

EP: 5.Unmet Needs Surrounding PNH

Carlos M. De Castro, MD: The cost utilization study looked at health care costs associated with PNH [paroxysmal nocturnal hemoglobinuria]. It was a retrospective. They used a claims database to go and pick patients out with PNH and look at hospitalizations, and the use of blood products. There were other things they looked at in terms of costs, and they broke it down into groups of how many patients got transfused. I think that was 18%, which I found quite high. I think as we’ve added more treatments, I’m finding fewer people needing transfusions.

The biggest costs, of course, are the medications associated with treating the disease because they’re quite expensive. There was a hospitalization rate that was fairly high for newly diagnosed patients, but that usually relates to the initial admission for whatever brings them in with the PNH. Or when the diagnosis is made, some of these patients have infections or sepsis, or other things. They also looked at ED [emergency department] use and other things like that. I think it wasn’t surprising that PNH can be a fairly expensive disease to have. How we bring those costs down is going to be difficult unless we can get better diagnostic tools, which is hard when you have such a rare disease. Doctors don’t think about PNH as the diagnosis that comes to mind when the patients first present, so we have to do a lot more education there.

The data weren’t too surprising. I think the major costs associated with this are the costs of treatment, which hopefully, as we have more drugs, maybe the cost will come down because it’s a competitive market. We’ll have to see. Again, I mentioned transfusions, it surprised me a bit about how many transfusions are being given; 18% seems high to me, but maybe it is that, at least at initial presentation time.

The time cost of transfusion depends on your treatment center and what they’re used to doing. There’s time associated with, A, you have to first get a type of screen, and at some places, they can’t prepare the blood quick enough so you have to come back the next day. There’s a cost inherent to that. And B, the time you have to be in an infusion center. It depends on how many units of blood you get, but it can be anywhere from 1 to 4 hours to get a unit of blood, depending on the procedure of that transfusion unit.

I think iron overload is a rare thing in PNH. In the days before we had effective treatments, patients used to be more iron deficient because they would lose iron through their urine with hemolysis. As we got into the era of complement inhibition, that was no longer the case because they weren’t hemolyzing and losing iron anymore. You had to be careful if they were still getting transfusions, they could build up some iron. Although I think that’s still pretty uncommon because as you’re on these complement inhibitors that are fairly effective, the need for transfusions has gone way down. So I’m hoping not that many patients are going to get iron overload from transfusions with PNH. There are some, however, who are still needing to get blood products. And those are the ones who have to be considered for iron chelation therapy, trying to keep the iron from building up too high because long term that will cause organ dysfunction.

Transcript edited for clarity.