Hi Reg,
fantastic that you found Dr Perales et al's original was finally published in the Association's journal in October last. Thanks for posting this, as I was very keen to read it in full.
The TANDEM site (where Dr Kurtzberg gave her own presentations on Reme's 4yr plus survival results etc) had already taken down the earlier posters from the February 2023 meeting for Dr Perales et al: See
https://tandem.confex.com/tandem/2023/meetingapp.cgi/Paper/21357I wonder if Joanne Kurtzberg had time to catch up with Dr Perales at TANDEM, and talk about her solutions to the problem he'd highlighted. They've both worked together on panels previously e.g. on the major report on 'Standardizing Definitions of Hematopoietic Recovery, Graft Rejection, Graft Failure, Poor Graft Function, and Donor Chimerism in Allogeneic Hematopoietic Cell Transplantation: A Report on Behalf of the American Society for Transplantation and Cellular Therapy''. Link
here.
I could only find this other commentary (from Feb. last) in Targeted Oncology:
https://www.targetedonc.com/view/economic-burden-with-allohct-gvhd-leaves-need-for-novel-therapies One of the things I really valued in the original paper was the authors' focus on
overall survival (OS) - apparently they weren't buying the ORR rhetoric that Ruxolitinib's sponsors keep putting forward to justify expanded indications for Ruxolitinib treatment. When one's looking at lifetime costs, a 100-day ORR is a red herring, IMO. It looks like the authors also factored in clinical inputs including relapse-free survival (GRFS) and infections, plus the economic costs of those relapse episodes, infection hospitalizations, maintenance treatments, and end-of-life costs etc. So, it looks to me like a rigorous piece of work from Dr Perales et al.
And it backs up to the hilt what CEO Itescu has been saying to us & patenting for our benefit (as MSB holders) for the past 4 years and more, as
@otherperspective points out. The punch line for me from Dr Perales' TANDEM Conference oral presentation was this:
"
The estimated current per-patient medical cost of allo-HCT was estimated to be $1,247,917 with expected QALYs of 4.7.
Projecting continued diffusion of new cGVHD agents, the future per-patient cost of allo-HCT was estimated to range from $1,370,839 - $1,616,684 with 4.8 expected QALYs.
Aggregating current and future per-patient costs over 10 years, the lifetime burden of 64,461 incident allo-HCTs was estimated to be $93B and $102B - $120B, respectively. The analysis suggests that the small gain in QALYs as a result of new treatment approaches for active cGVHD would cost an additional $9B - $27B."
i.e. if ALL of the new treatments for GVHD as at November 2023 can only produce an expected additional 0.1 QALY per patient during a period of 10 years & at a cost of USD9,000,000 - USD27,000,000, then there's no good reason in the world why the FDA shouldn't give Remestemcel-l a shot at these problems based on the product's proven results now before the FDA.
Feeling like a floodgate is slowly opening here.
Cheers
GLTAH
(20min delay)
