Cell Therapy News/Articles, page-8824

Interesting health outcomes research recently published comparing remestemcel and ruxolitinib in steroid refractory aGVHD.

"In the full populations, the STC of risk ratios (RRs) found treatment with remestemcel-L-rknd to be associated with a numerical but not statistically significant improvement in the 28-day ORR versus ruxolitinib. In the grade III-IV aGVHD sub-group, the STC showed significantly improved 28-day ORR for remestemcel-L-rknd versus ruxolitinib (P=0.04). Remestemcel-L-rknd was also associated with improved safety outcomes (P<0.05) in 17 out of 30 AEs, including hematologic events, peripheral edema, muscular weakness, nausea, back pain, and fatigue."

These publications and comparative effectiveness analyses form the evidence packages that are submitted to health technology assessment bodies for single-payer healthcare bodies such as PBAC (for Australia), NICE (UK), CADTH (Cananda) to help inform pricing and reimbursement submissions. For government reimbursed access to remestemcel in Australia, the pathway is as follows: FDA approval -> TGA registration (given the unmet need and rarity - they may be eligble for a priority pathway) -> PBS listing via a pricing submission and negotiations. Mesoblast could also provide remestemcel via a Special Access Scheme (out of their own pocket), during the TGA and PBS registration and listing process.

https://jheor.org/article/19008-comparative-effectiveness-of-remestemcel-l-rknd-versus-ruxolitinib-in-pediatric-patients-with-steroid-refractory-acute-graft-versus-host-disease-using-simulated-treatment-comparisons