The NCD for Stem Cell Transplantation Multiple Myeloma (MM), Myelofibrosis (MF) and Sickle Cell Disease (SCD), released by CMS on January 27, 2016, concluded that, CMS will cover items and services necessary for research under §1862(a)(1)(E) for a allogeneic hematopoietic stem cell transplant (HSCT) for certain Medicare beneficiaries with myelofibrosis (MF) using the Coverage with Evidence Development (CED) paradigm.
Allogeneic HSCT for myelofibrosis will be covered by Medicare only for beneficiaries with Dynamic International Prognostic Scoring System (DIPSSplus) intermediate-2 or High primary or secondary MF and participating in an approved prospective clinical study. There must be appropriate statistical techniques in the analysis to control for selection bias and potential confounding by age, duration of diagnosis, disease classification, DIPSSplus score, comorbid conditions, type of preparative/conditioning regimen, graft vs. host disease (GVHD) prophylaxis, donor type and cell source.
A prospective clinical study seeking Medicare coverage of allogeneic HSCT for myelofibrosis pursuant to Coverage with Evidence Development (CED) must address the following question:
Prospectively, compared to patients who do not receive allogeneic HSCT, do Medicare beneficiaries with MF who receive allogeneic HSCT have improved outcomes as indicated by:
- Graft vs. host disease (acute and chronic);
- Other transplant-related adverse events;
- Overall survival; and
- Quality of life (optional).
Instructions on how to submit an application for CMS approval of study protocols can be found on Appendix C of the Final Decision Memo.
Below is a list of Medicare-approved clinical studies we have reviewed and determined meet the requirements for coverage under CED.
Study Title: Prospective Assessment of Allogeneic Hematopoietic Cell Transplantation in Patients with Myelofibrosis
Sponsor: Medical College of Wisconsin
ClinicalTrials.gov Number: NCT02934477
CMS Approval Date: 10/17/2016