FEATURE
CMS Expands Coverage
for Hematopoietic Cell
Transplantation: Proceed
with Cautious Optimism
The recent decision by the Centers for Medicare and Medicaid Services (CMS) to cover
allogeneic hematopoietic cell transplantation
(alloHCT) for eligible patients with sickle cell
disease (SCD), myelofibrosis, and multiple
myeloma (MM) is a boon for hematology.
But, as is often the case with good news, this
National Coverage Determination (NCD)
needs to be taken with quite a few grains of
salt. In this case, the specific criteria needed
to be met that govern exactly how and when
CMS will cover eligible patients are causing many transplanters to slightly curb their
enthusiasm.
“This decision is a very significant milestone that reflects our advances in the art
and science of stem cell transplantation,” said
Krishna Komanduri, MD, director of the
Adult Stem Cell Transplant Program at the
University of Miami Health System’s Sylvester
Comprehensive Cancer Center in Miami,
Florida. “Beyond that, there are some caveats,
including that, within each disease state, there
are some eligibility restrictions for patients
based on their risk level and the severity of
disease.”
The NCD requires the establishment of
clinical trials to assess and validate alloHCT
in Medicare patients with SCD, myelofibrosis,
and MM, and those will take some time to
develop and start enrolling patients, added
Dr. Komanduri, who is also president-elect of
the American Society for Blood and Marrow
Transplantation (ASBMT).
Laura Michaelis, MD, associate professor
of medicine at the Medical College of Wisconsin in Milwaukee, Wisconsin, agreed that this
decision is a clear “win” for clinicians and
patients, but that “there are still aspects [of
the NCD] that need to be finalized.”
“I don’t think these criteria are meant to
be restrictive,” Dr. Michaelis said. “I think
CMS wants to expand coverage, and that’s
great, but they also want to gather information to ensure that this course of action is appropriate. We need to make sure that we work
with CMS to achieve that goal.”
Mary Horowitz, MD, chief of the Division
of Hematology and Oncology at the Medical College of Wisconsin, called the NCD “a
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ASH Clinical News
way forward that removes the
financial barrier to transplant for
a growing population of patients
who can benefit from it.” She also
cautioned that the transplant
community and CMS still have to
agree on what constitutes acceptable clinical
data to support coverage.
Drs. Kumanduri, Michaelis, and Horowitz,
as well as other members of the transplant
community spoke with ASH Clinical News
about the details of this NCD and what it
means for hematologists, transplant specialists,
and patie nts – both now and in the near future.
The Big Picture and the Finer Points
With NCDs such as these, CMS can mandate
or prohibit coverage for specific procedures in
specific indications. Prior to the recent NCD,
CMS was “silent” on the use of alloHCT for
the treatment of SCD and myelofibrosis. That
meant coverage decisions were up to local
Medicare Administrative Contractors, so
patients and transplant centers were put in a
position of potentially taking on the full financial burden of the procedure if coverage was
denied, explained Stephanie Farnia, MPH,
director of payer policy at the National Marrow
Donor Program (NMDP)/Be The Match.
Beyond that, CMS outright prohibited coverage of alloHCT for MM.
Enter the new NCD, with coverage of
alloHCT in these three disease states and the
caveat that reimbursement would be provided
only if the patient is enrolled in a CMSapproved clinical trial designed to evaluate the
benefit of alloHCT in a Medicare population.
Called a Coverage with Evidence Development (CED), this requirement mandates that
eligible beneficiaries meet the following criteria
to receive coverage:
• Symptomatic, stage II or III MM
• Intermediate-2 or High Dynamic
International Prognostic Scoring System
myelofibrosis, plus score in primary or
secondary disease
• Severely symptomatic SCD
Additionally, the CED trial requirements for
alloHCT in these conditions state that patients
must be participating in an approved prospective clinical study that meet the following
criteria:
“There must be appropriate statistical
techniques in the analysis to control for
selection bias and potential confounding by
age, duration of diagnosis, disease classification, International Myeloma Working
Group classification, International Staging System score, Durie Salmon staging,
comorbid conditions, type of preparative/
conditioning regimen, graft-versus-host
disease (GVHD) prophylaxis, donor type,
and cell source.”1
The principal objectives, according to the CED,
are to test whether alloHCT therapy improves
health outcomes, to compare survival outcomes between alloHCT and non-alloHCT
therapies, and to address acute and chronic
GVHD and transplant-related adverse events.
Analysis of quality of life among those receiving alloHCT is an optional component to these
studies, according to CMS.
This isn’t the transplant community’s first
go-round with a CED – in 2010, CMS required
one for coverage of myelodysplastic syndromes
(MDS), although the CED in that setting was
much less demanding, asking that trials:
• Prospectively compare MDS Medicare
beneficiaries who receive alloHCT
therapy and determine if they have
better outcomes than those who do not
get alloHCT treatment.
• Examine how International Prognostic
Scoring System score, age, cytopenias,
and comorbidities predict outcomes
among these patients.
May 2016