Yesterday, ICER posted an Evidence Report assessing the comparative clinical effectiveness and value of atidarsagene autotemcel (“arsa-cel”, Orchard Therapeutics) for metachromatic leukodystrophy (MLD). ICER’s Chief Medical Officer David Rind, MD explained:
“MLD is a devastating disease for children and their caregivers. It is extremely rare, and most doctors have never seen a case. Parent testimony shared throughout this report has provided a window into the hope and frustration that families are feeling as new treatments become available. With arsa-cel, we see a gene therapy again filling the promise of possible treatments for conditions that previously were untreatable.” This Evidence Report will the basis for our September 29th virtual public meeting, where an independent appraisal committee will review the evidence, hear further testimony from stakeholders, and deliberate on the treatment’s comparative clinical effectiveness, other potential benefits, and long-term value for money. Register here. |