HCP-Patient Conversations on Gene Therapies for rare diseases
Rare Diseases have long been characterized by large unmet needs for patients and healthcare providers alike, often with a lack of resources and a lack of options.
Recent breakthroughs in medical research have paved the way for novel Gene Therapy approaches that offer new avenues of hope for patients.
ZoomRx’s doctor patient dialogue research provides a unique window into these pivotal discussions, gathering audio from real-world clinical appointments as new treatment options bring hope to the discussion.
In this blog, we experience the exciting potential of gene therapy & CAR-T therapy through point-of-care dialogues between doctors and patients with Hemophilia or Diffuse Large B Cell Lymphoma (DLBCL).
Also, read: Lessons from Rare Disease Market Research for Treatments
Introducing Novel Therapies
New treatment approaches offer a beacon of hope for those facing rare blood disorders. From the examples below, we see that both patients and doctors are enthusiastic over these newer treatment options.
Doctors introduce these gene therapy treatments to be potentially curative with improved convenience benefits offering clear benefits.
But as we can see in the third example below, it can be easy for doctors to get excited about how a new treatment works and monologue information which risks less information retention and ultimately patient buy-in.
Patient: No, I haven't. I read about similar stuff in some news articles and stuff, but never about hemophilia.
HCP: Okay, well, we've had our first few patients already receive gene therapy here. It's considered a curative treatment. So, what that would mean is that at least as far as the trials go, with, like, 5-year follow-up, that patients who receive that gene therapy typically don't require any further treatments for at least 5 years. It literally introduces the correct factor gene into your blood cells, so that we don't have to keep giving you the weekly factor. Your body produces it on its own.
Patient: I mean, that sounds pretty good.
HCP: It's quite remarkable. And we're typically starting it with younger folks who don't have a lot of other issues, such as yourself. We're seeing some pretty good results. But once again, this is all very new. With relatively few patients who've been studied. So, it's something if you felt that you are currently feeling kind of like, overwhelmed or frustrated with having to get these infusions weekly, and with the joint pain, it's certainly something we could consider.
Patient: Yeah, it sounds a lot more convenient than what I'm doing now. I mean, I have to drive 3 hours back and forth from the house to the hospital.
HCP: Absolutely. And I think with someone who's working, younger, it definitely makes a lot of sense.
Patient: Okay.
HCP: It used to be patients in your situation would get bone marrow transplants, but this is a lot easier to go through than that.
Patient: Okay. I've been researching that.
HCP: The first thing that we're going to need to do though, is get what we call a manufacturing slot...
Patient: Yeah. Can you tell me more about it, like, how does it work?
HCP: Yeah, CAR-T cell therapy is a very promising new treatment that we have available. And just to tell you, CAR-T basically stands for Chimeric Antigen Receptor T cell therapy. And I don't want you to get too mixed up in kind of the actual what it stands for, but basically, it's a type of cancer immunotherapy that uses your own T cells, you know, your immune cells that we then genetically alter in the lab to allow them and enable them in locating and destroying your cancer cells more effectively. It certainly has been around, although newer, it has been around for a few years now. I have used it in different patients and different leukemias, lymphomas, and multiple myeloma patients. So certainly it has different indications, but I do think that it would potentially be a benefit to you and we could potentially have good results with it and really try to get the lymphoma under control for you.
Patient: So how does it work exactly?
Patient Reactions to Gene Therapies
Patient enthusiasm quickly leads to follow-up questions, especially about the viral delivery and increased risk of cancer. We’ve seen that clinical trial results are heavily relied on by doctors when fielding these questions, balancing their presentation between reassurance of the study rigor and conveying the limitations of current research.
Despite the unfamiliarity with this innovative approach, patients typically trust their doctors and are excited to have a new option, highlighting the transformative impact that gene therapy for rare diseases is shaping their outlook on their treatment journey.
Patient: Yeah, it sounds like it's going to take a while, but it sounds pretty convenient. I'm just worried about if it's a virus, is it going to make me sick in any way, or is it relatively safe? How does that work?
HCP: That's an excellent question. Folks tolerate it very well. There are relatively few side effects. A few people get infusion reactions, temporary fevers, that kind of thing. But so far, in all the trials, people tolerate it very well. And that's why we do these tests ahead of time to make sure that you don't have any immune reaction to the virus.
Patient: Okay. And you said it takes a long, long time. I guess, like, how long does it take from start to finish?
HCP: Yeah, I mean, the reported data from the trials, I think, is like 8 to 10 weeks for manufacturing. In reality, I think it takes a little bit longer. So, realistically, I think if we were, it's January now. If we were to get the ball rolling, this may be something to look at, like the final product around April, March/April. But, I mean, this is a process. We can do the first step, see how it goes.
Patient: Is this like a surgery or something, or what does this entail?
HCP: That's a great question. Thankfully, it's not a surgery. It would entail us harvesting some of your blood cells and sending them off to the lab. That would be a simple procedure, just like a plasmapheresis. And then in the laboratory, they would use a virus to introduce the correct factor VIII gene into your cells, and then we would reinfuse them back. The whole process takes about two, three months, but we would reinfuse them back and your cells would now be able to produce the factor VIII on their own without requiring these weekly transfusions.
Patient: Are there risks for this, like, can playing with my cells give me cancer or something?
HCP: That's an excellent question. I think that's something that a lot of folks have raised concerns about, and that's why this has been studied with some extensive follow up, I think about five year follow up now in the trials. So we can say for certain that, over at least the first five years that people have this, they're not at increased risk of cancer. Now, of course, there's always the consideration that maybe in the long term, this sort of, like, genetic, messing with your genetic material could theoretically increase cancer risk. But the way it's designed is the virus very specifically targets just the factor VIII gene and nothing else.
These examples give us a glimpse into how doctors act as ambassadors of novel treatments for their patients, presenting the information as best as they are able.
By understanding how patients perceive novel therapies at the point of care, pharma brands can help HCPs keep up to speed on recent developments through simple messaging based on clinical trial results to ultimately improve patient outcomes.
ZoomRx’s doctor patient conversation research enables life science companies to hear what’s actually happening at the point of care with their target patient populations.
Click here to access a recent white paper where we look at just how little time some patients get to speak during clinical appointments.
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