Allogeneic Hematopoietic Stem Cell Transplant for Sickle Cell Disease

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 sickle cell disease (SCD) will be covered by Medicare only for beneficiaries with severe, symptomatic sickle cell disease who participate 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, 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 sickle cell disease 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 SCD 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.

Final Decision Memo

Below is a list of Medicare-approved clinical studies we have reviewed and determined meet the requirements for coverage under CED.

Approved Studies

Study Title: Prospective Assessment of Allogeneic Hematopoietic Cell Transplantation in Adolescents and Young Adults with Severe Sickle Cell Disease
Sponsor: Medical College of Wisconsin
Clinicaltrials.gov number: NCT01166009
CMS Approval Date: 10/04/2017

Study Title: Bone Marrow Transplantation vs Standard of Care in Patients With Severe Sickle Cell Disease (BMT CTN 1503) (STRIDE)
Sponsor: Medical College of Wisconsin
ClinicalTrials.gov Number: NCT02766465
CMS Approval Date: 06/14/2016