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Cost Effectiveness and Future of CAR T Therapy

Panelists: Krishna V. Komanduri, MD, Sylvester Comprehensive Cancer Center; David Maloney, MD, PhD, Fred Hutchinson Cancer Research Center; Sattva S. Neelapu, MD, University of Texas MD Anderson Cancer Center; Michael Pulsipher, MD, Children
Published: Wednesday, Mar 14, 2018



Transcript: 

Krishna V. Komanduri, MD: We have, obviously, some very dramatic and important advances, but I can’t let this panel not address the issue of cost-effectiveness because these are dramatic therapies, but with a big price tag. And I have my own thoughts about this, but I want to hear it from others. How do we start to address this issue of cost-effectiveness? Because at the end of the day, we have to have the healthcare system that not only provides advances but is sustainable.

Michael Pulsipher, MD: I think we talked about this before, comparative studies of different agents allowing us to move these earlier into therapy where they won’t get a whole series of very expensive drugs, potential replacement of transplantation. There are a whole series of things that I think can make these cost-effective, but probably the most important thing that is going to make this cost-effective is having a lot of things on the market.

David Maloney, MD, PhD: This is a hot topic, obviously, and none of us are experts in this. But obviously, I think the whole system is broken in how we do this, and it’s very challenging. I think it’s important to remember that the cost of the drug is only one part of it. The supportive care or the actual delivery of the care is probably equally as expensive in many situations. Also, we need to remember it’s not just the cost of the drug, it’s the cost of all the other drugs patients are getting in the course of their disease, and often those are chronic drugs that are on year after year after year for many hundreds of thousands of dollars. And so, all this needs to be taken into context rather than just reacting to cost. Ultimately, it will come down to effectiveness, toxicity, and probably cost. Competition will be a good thing, I think, in that.

Krishna V. Komanduri, MD: I agree. I’m going to throw in some things because I’ve been leading the ASBMT (American Society for Blood and Marrow Transplantation) this year. I’ve been working in a lot of healthcare finance issues, and one of the things I would say first, we have to look at these therapies in lieu of the other therapies that might be used. I think that we have to not only look at the cost-of-healthcare system in terms of the entire range of care cost but also the impact on the patient. I think we can’t just think about that out-of-pocket cost. How this affects the patient’s financial burden is important.

There are measures like quality adjusted life years and the dollars, but I think that we have to be a little bit careful because you can come up with a number that might seem like, OK, this is justifiable. But at the end of the day, it has to be one that public and private payers can absorb and that doesn’t lead to preferential access. I think none of us wants to see great advances and we have only a subset of patients who have the right kind of insurance. It is important for us, even though we can’t answer these questions, that those of us who are experts in this area work with payers, work with the public workers, and work with the manufacturers to make sure that we have models that are sustainable and we have to do this in a fair way.

This has been a great discussion, and I think we covered a really broad range of topics. I know we’re a talkative group, but I want to each give you your opportunities to summarize your thoughts about where we are and where we may be going. We’ll start with David.

David Maloney, MD, PhD: I just think this is an absolutely exciting time for the development of new therapies for cancer. I’ve been involved in antibodies for many, many years in development, and this is really making an antibody really work well. And so, CAR T cells have the most antitumor activity I’ve ever seen in my career, and it’s tremendously gratifying to see going forward. We do need to solve toxicity. We need to figure out how to prevent or head off CRS, and especially we need to work on neurotoxicity. And I think if we can get those things under control, this will be more widely acceptable. Obviously, for this to succeed, the companies that are involved need to be reliable, they need to be able to make a product, they need to make that product 100% of the time, and they need to make it available in a timely fashion, whether that be 2 weeks or 4 weeks, but it needs to be timely. And if we can do this, then we can get these new drugs incorporated into the treatment paradigms or the clinical care pathways and save patients’ lives.

Krishna V. Komanduri, MD: Steve?

Stephen J. Schuster, MD: I generally echo what David said. I remember not so long ago—maybe long but it doesn’t seem long ago to me—that people would argue where T cells could even cure cancer, or treat cancer, or have antitumor efficacy. And the DLI (donor leukocyte infusion) data in CML (chronic myelocytic leukemia) were the first inkling that this whole theory of immune surveillance and antitumor immunity might really be clinically relevant. Well, I can say anybody who has doubt about whether T cells can treat cancer, they need to treat somebody with CAR T cells because T cells can definitely successfully treat cancer in the right situation. It is an exciting time. We have a new approach, a new paradigm. We talked about a couple of targets, and we’ve talked about even using modifications of T cells to make them more universal. There are so many things that are possible now because of the technologies that we have, that I think the future is bright and the times are quite exciting. This is probably the best time in my career.

Krishna V. Komanduri, MD: I want to say first it has been a privilege. You guys have all contributed dramatically to the literature in this area and clinical advances. I’ll just say the anecdote. When I went to UC San Francisco as a Fellow and I wanted to go into a research laboratory, there were actually no cancer immunology laboratories at UCSF, and I ended up doing HIV immunology, which was good for me at that time. But it is great for me, as a hematologist and as a T-cell immunologist, to see the era where, all of a sudden, we’re seeing these dramatic advances in the hands of clinicians and affecting patients. It has been a pleasure so I’m really excited about where we are. Michael, you’ll get the last word.

Michael Pulsipher, MD: I think that I couldn’t agree more with everyone that this is very exciting times where we are doing not baby steps, we are doing giant leaps in being able to take care of the sickest patients. I’m a transplanter—I think most of the people here are transplanters—my goal would be to eliminate transplant, to understand CARs well enough that we know when to turn them on and turn them off and to get toxicity under control.

Krishna V. Komanduri, MD: That’s great. I want to thank you all for your contributions again in this discussion. On behalf of our panel, we thank you for joining us and we hope you found this Peer Exchange® discussion to be both useful and informative. Thank you.

Transcript Edited for Clarity 

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Transcript: 

Krishna V. Komanduri, MD: We have, obviously, some very dramatic and important advances, but I can’t let this panel not address the issue of cost-effectiveness because these are dramatic therapies, but with a big price tag. And I have my own thoughts about this, but I want to hear it from others. How do we start to address this issue of cost-effectiveness? Because at the end of the day, we have to have the healthcare system that not only provides advances but is sustainable.

Michael Pulsipher, MD: I think we talked about this before, comparative studies of different agents allowing us to move these earlier into therapy where they won’t get a whole series of very expensive drugs, potential replacement of transplantation. There are a whole series of things that I think can make these cost-effective, but probably the most important thing that is going to make this cost-effective is having a lot of things on the market.

David Maloney, MD, PhD: This is a hot topic, obviously, and none of us are experts in this. But obviously, I think the whole system is broken in how we do this, and it’s very challenging. I think it’s important to remember that the cost of the drug is only one part of it. The supportive care or the actual delivery of the care is probably equally as expensive in many situations. Also, we need to remember it’s not just the cost of the drug, it’s the cost of all the other drugs patients are getting in the course of their disease, and often those are chronic drugs that are on year after year after year for many hundreds of thousands of dollars. And so, all this needs to be taken into context rather than just reacting to cost. Ultimately, it will come down to effectiveness, toxicity, and probably cost. Competition will be a good thing, I think, in that.

Krishna V. Komanduri, MD: I agree. I’m going to throw in some things because I’ve been leading the ASBMT (American Society for Blood and Marrow Transplantation) this year. I’ve been working in a lot of healthcare finance issues, and one of the things I would say first, we have to look at these therapies in lieu of the other therapies that might be used. I think that we have to not only look at the cost-of-healthcare system in terms of the entire range of care cost but also the impact on the patient. I think we can’t just think about that out-of-pocket cost. How this affects the patient’s financial burden is important.

There are measures like quality adjusted life years and the dollars, but I think that we have to be a little bit careful because you can come up with a number that might seem like, OK, this is justifiable. But at the end of the day, it has to be one that public and private payers can absorb and that doesn’t lead to preferential access. I think none of us wants to see great advances and we have only a subset of patients who have the right kind of insurance. It is important for us, even though we can’t answer these questions, that those of us who are experts in this area work with payers, work with the public workers, and work with the manufacturers to make sure that we have models that are sustainable and we have to do this in a fair way.

This has been a great discussion, and I think we covered a really broad range of topics. I know we’re a talkative group, but I want to each give you your opportunities to summarize your thoughts about where we are and where we may be going. We’ll start with David.

David Maloney, MD, PhD: I just think this is an absolutely exciting time for the development of new therapies for cancer. I’ve been involved in antibodies for many, many years in development, and this is really making an antibody really work well. And so, CAR T cells have the most antitumor activity I’ve ever seen in my career, and it’s tremendously gratifying to see going forward. We do need to solve toxicity. We need to figure out how to prevent or head off CRS, and especially we need to work on neurotoxicity. And I think if we can get those things under control, this will be more widely acceptable. Obviously, for this to succeed, the companies that are involved need to be reliable, they need to be able to make a product, they need to make that product 100% of the time, and they need to make it available in a timely fashion, whether that be 2 weeks or 4 weeks, but it needs to be timely. And if we can do this, then we can get these new drugs incorporated into the treatment paradigms or the clinical care pathways and save patients’ lives.

Krishna V. Komanduri, MD: Steve?

Stephen J. Schuster, MD: I generally echo what David said. I remember not so long ago—maybe long but it doesn’t seem long ago to me—that people would argue where T cells could even cure cancer, or treat cancer, or have antitumor efficacy. And the DLI (donor leukocyte infusion) data in CML (chronic myelocytic leukemia) were the first inkling that this whole theory of immune surveillance and antitumor immunity might really be clinically relevant. Well, I can say anybody who has doubt about whether T cells can treat cancer, they need to treat somebody with CAR T cells because T cells can definitely successfully treat cancer in the right situation. It is an exciting time. We have a new approach, a new paradigm. We talked about a couple of targets, and we’ve talked about even using modifications of T cells to make them more universal. There are so many things that are possible now because of the technologies that we have, that I think the future is bright and the times are quite exciting. This is probably the best time in my career.

Krishna V. Komanduri, MD: I want to say first it has been a privilege. You guys have all contributed dramatically to the literature in this area and clinical advances. I’ll just say the anecdote. When I went to UC San Francisco as a Fellow and I wanted to go into a research laboratory, there were actually no cancer immunology laboratories at UCSF, and I ended up doing HIV immunology, which was good for me at that time. But it is great for me, as a hematologist and as a T-cell immunologist, to see the era where, all of a sudden, we’re seeing these dramatic advances in the hands of clinicians and affecting patients. It has been a pleasure so I’m really excited about where we are. Michael, you’ll get the last word.

Michael Pulsipher, MD: I think that I couldn’t agree more with everyone that this is very exciting times where we are doing not baby steps, we are doing giant leaps in being able to take care of the sickest patients. I’m a transplanter—I think most of the people here are transplanters—my goal would be to eliminate transplant, to understand CARs well enough that we know when to turn them on and turn them off and to get toxicity under control.

Krishna V. Komanduri, MD: That’s great. I want to thank you all for your contributions again in this discussion. On behalf of our panel, we thank you for joining us and we hope you found this Peer Exchange® discussion to be both useful and informative. Thank you.

Transcript Edited for Clarity 
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