Cold Agglutinin Disease: Treatment Gaps

Payers react to gaps that impact coverage decisions for therapies available off-label for cold agglutinin disease.

Neil Minkoff, MD: Let me use that as a kind of pivot point to say, now I think we’re starting to get a pretty good sense of the spectrum here. We’ve taken the patient from symptom, to the early stage of the journey through differential diagnosis, to diagnosis, to some of the treatment options, behavioral- and molecule-based. What’s missing? What more do you wish you could do for these patients? Where are you seeing the unmet needs in the disease state? And I’m going to start with Dr Stephens on this because I want to get the payer perspective for once first, and then I’ll bring in our clinical colleagues.

Kevin Stephens, Sr., MD, JD: Well, of course the problem we have is the rare presentation, and it’s a fairly rare phenomenal disease. Having a greater number would give you greater metrics and greater optics in terms of what to expect and what to see. What would be very helpful for us is, over time as we collect information, that we could put it all together and make an aggregate data set so that we can see long term what’s the best treatment, what are the best treatment regimens, and those sorts of things. If I had a genie in a bottle, I would fast forward, and then I would reverse and come back to where we are.

Neil Minkoff, MD: Anything to add, Dr Gleeson?

Jeremy Gleeson, MD: I think ultimately we would all love to have comparative data between some of the various treatments that we have, but we’re not likely to get that anytime soon in such a rare disease, where we are going to see a series of 20 to 30 patients, not a series of 3000. Thus, I think we’re going to be challenged for a long time with a lot of data, and we’ll be, as with other rare diseases, having to work with what we’ve got. But that’s what we have to work with. I’m not expecting that to change anytime soon.

Neil Minkoff, MD: Let me push you a little bit, both of you, I’m not trying to put either of you on the spot, but is this a disease state that even hits, as they say, your radar screen when you’re doing your planning, you’re looking at your high-cost disease states, you’re looking at your high-risk diseases or even patients who are flowing into the different disease or care management programs?

Jeremy Gleeson, MD: I think the short answer to that is no, I don’t think it does hit our radar screen, unlike the other CAD, the big one, which of course does hit our radar—coronary artery disease.

Neil Minkoff, MD: There’s another CAD?

Jeremy Gleeson, MD: Yes. I’m afraid this one [cold agglutinin disease] probably is going to be managed, at least for smaller payers, on a very much individual basis. It’s not something that’s probably on our radar screen to a significant extent, not one that we’re probably going to develop a lot of policy around just because of its rarity.

Kevin Stephens, Sr., MD, JD: Well, we have somewhat of a slightly different thing here. It depends on the utilization of the patient, because if the patient is being admitted frequently and having frequent transfusions and more severe disease, then it may show up on our radar. And the second thing is, it’s the cost of care. If the cost of treatment gets to be exorbitant, because in the commercial world, we have fully insured, where the client pays for the medication and the profile. If we have an outlier and someone who’s costing $150,000, or whatever number you want to choose, then that appears on the payer’s spreadsheet and then they will come to us and say, “Why is our premium going up, and what’s happening?” In that sense, it may surface on our radar.

Neil Minkoff, MD: Yes…go ahead, please, Jeremy.

Jeremy Gleeson, MD: Yes, and once again, these patients will come to our attention in much the same way, through if they’re admitted frequently, emergency department visits, we obviously monitor those 2 things closely, and again, case managers who are not going to be disease-specific will become involved. But I think it’s going to be, again, the patients at the severe end of the spectrum who have those events that are going to come to our attention rather than maybe the broader group that does not.

Neil Minkoff, MD: I did want to bring one clarification up just in the way I pushed earlier on the definition of response rate. I assume you want to clarify, Dr Stephens, that when you as a payer say the payers are looking at this, you mean the employers, really.

Kevin Stephens, Sr., MD, JD: That’s correct. Because for the fully insured, the employer pays the premiums, and it’s flexible depending upon our projections of what that cost will be. It’s not a solid number.

Neil Minkoff, MD: I just wanted to make sure it was clear, because there are going to be people, providers especially, listening to this who don’t realize that the payers think somebody else is a payer.

Kevin Stephens, Sr., MD, JD: No, no. We in the commercial world, particularly if you’re fully insured, you have a cost estimate that is calculated based upon your disease burden in your employee base, and that determines your premium. Those are the kind of things the employer will look at us and say, “Why is our premium going up?,” particularly in a case like this, and “What are you doing?,” and those kinds of things. They will want a rational explanation for the pricing of the premiums.

Jeremy Gleeson, MD: The ultimate payer is, after all, the membership rather than even the employer or anybody else. It all comes down to the premium.

Kevin Stephens, Sr., MD, JD: That’s correct.

Transcript edited for clarity.

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