The Non-Malignant Channel on VJHemOnc is an independent medical education platform, supported with funding from Agios (Gold). Supporters have no influence on the production of content. The levels of sponsorship listed are reflective of the amount of funding given.
The Sickle Cell Disease Channel on VJHemOnc is an independent medical education platform, supported with funding from Agios (Gold). Supporters have no influence on the production of content. The levels of sponsorship listed are reflective of the amount of funding given.
General Updates | Evaluating barriers to accessing gene therapy for sickle cell disease: insights from a single center
Payal Desai, MD, Levine Cancer Institute & Wake Forest School of Medicine, Charlotte, NC, discusses the findings of an analysis that investigated barriers to accessing gene therapy for patients with sickle cell disease (SCD) in a single institution. Dr Desai highlights that both biological and social barriers exist, including the presence of comorbidities, financial challenges, and concerns around fertility. She believes that many of these challenges could be overcome; however, the community will need to come together to improve the support available for patients. This interview took place virtually.
These works are owned by Magdalen Medical Publishing (MMP) and are protected by copyright laws and treaties around the world. All rights are reserved.
Transcript
We looked at our data for our entire center, which included two peds programs and two adult programs, and we had exactly 999 patients. We needed one more, to make the stats even easier. [laughs]
But we looked at those patients to look at what… we talked to patients the first six months after gene therapy was approved here in the US and said, what’s the uptake? What are people interested in talking about? What are there discussions around? What are their concerns around gene therapy?
And so, what we found were two big groups...
We looked at our data for our entire center, which included two peds programs and two adult programs, and we had exactly 999 patients. We needed one more, to make the stats even easier. [laughs]
But we looked at those patients to look at what… we talked to patients the first six months after gene therapy was approved here in the US and said, what’s the uptake? What are people interested in talking about? What are there discussions around? What are their concerns around gene therapy?
And so, what we found were two big groups. We actually looked at barriers. One was patient-specific biological barriers. So things like the doctor said their kidney disease is too bad or they have way too many red cell antibodies that we could successfully transfuse them. Or they have really bad renal disease, and so we might not be able to get them through chemotherapy. So that was one.
The other group were I think things we don’t always think about in the transplant space, but I think are really important, were kind of more social barriers. Things like we’re worried about this patient’s adherence or the patient noted I have concerns around fertility, I have concerns about who will take care of my children, I have financial concerns. So there was a group of those concerns. So I think it was interesting to see where we were, at least just as a baseline, because the whole point is, if we identify these barriers, maybe we can start addressing them.
And I think one of the interesting things in sickle cell disease, and it’s an observation… it’s an interesting thing in terms of an observation. it’s also very, I think, important in terms of how we can help address it as a larger group, but is that there were things like, who’s going to take care of my children? That, for example, if a patient has other conditions like that, we normally do bone marrow transplant for like malignant conditions, there are resources, there are foundations, there are people that can help financially. Those groups don’t exist currently in the sickle space. And so I think it brought up some interesting things that are barriers for patients, that might be overcomeable, but we need as a community to work on those issues.
And I think the other big thing is fertility. So do they have the coverage for fertility? Do they have insurance to actually cover? Not just the collection, but how do you actually give the treatment back? Because fertility treatments are quite expensive, especially here in the US. And so how do we help people overcome some of those barriers, from both financial support and insurance coverage issues?
This transcript is AI-generated. While we strive for accuracy, please verify this copy with the video.
Read more...
