Current through Reg. 50, No. 187; September 24, 2024
As used in this rule, the term "appropriate oncological
specialty" means that where a particular kind of tumor or disease is usually
treated by a subspecialty group within the general discipline of oncology,
those who practice within that subspecialty have had specific input into the
decision making process. Cellular therapies include cellular immunotherapies,
chimeric antigen receptor (CAR) T cells, cancer vaccines, and other types of
both autologous and allogeneic cells for certain therapeutic indications. Human
gene therapy refers to products that introduce genetic material into a person's
DNA to replace faulty or missing genetic material, thus treating a disease or
abnormal medical condition.
(1) Upon
the recommendation of the Bone Marrow Transplant Panel, each of the following
procedures meets a minimum level of evidence based on high quality systematic
reviews of case control or cohort studies, high quality case-control or cohort
studies with a very low risk of confounding bias, or chance, and a high
probability that the relationship is causal, and is considered accepted within
the appropriate oncological specialty and not experimental for the purposes of
Section 627.4236, F.S.
(a) Allogeneic bone marrow transplant for the
following:
1. Acute myelogenous leukemia and
myeloid sarcoma;
2. Acute
lymphoblastic leukemia;
3. Chronic
myelogenous leukemia;
4.
Myelodysplastic syndromes;
5.
Chronic myelomonocytic leukemia;
6.
Myelofibrosis;
7. Non-Hodgkin
lymphoma;
8. Hodgkin lymphoma after
autologous stem cell collection failure or relapsed after autologous transplant
but not progressing on salvage chemotherapy;
9. Chronic lymphocytic leukemia;
10. Severe or very severe aplastic anemia
from HLA compatible siblings and any type of bone marrow transplant for
acquired severe aplastic anemia unresponsive to immunosuppression or bone
marrow failure syndromes;
11.
Severe aplastic anemia and other bone marrow failure syndromes;
12. Thalassemia;
13. Inborn errors of immune system including
severe combined immune deficiencies, primary immune deficiencies, and primary
immune regulatory disorders;
14.
Sickle cell disease.
(b)
Autologous bone marrow transplant for the following:
1. Multiple myeloma (including double bone
marrow transplant), Waldenstrom macroglobulinemia and primary
amyloidosis;
2. Non-Hodgkin
lymphoma;
3. Hodgkin
lymphoma;
4. Acute myelogenous
leukemia (stem cells collected in remission);
5. Neuroblastoma;
6. Germ cell tumor, after failure of first
therapy but not progressing on salvage therapy;
7. Primitive neuroectodermal tumor (PNET),
(including medulloblastoma and pinealoblastoma), chemotherapy sensitive after
first relapse;
8. Medulloblastoma
and other PNET tumors, metastatic, at diagnosis;
9. Ewing sarcoma, chemotherapy sensitive
after first relapse.
(c)
Gene and cellular therapy:
1.
Tisagenlecleucel, a CD19-directed, genetically modified autologous T cell
immunotherapy is medically necessary for the treatment of:
a. Adults with relapsed or refractory large
B-cell lymphoma after two or more lines of systemic therapy including:
b. Diffuse large B-cell lymphoma
not otherwise specified.
c. High
grade B-cell lymphoma and diffuse large B-cell lymphoma arising from follicular
lymphoma.
d. Pediatric patients up
to 25 years old with B-cell precursor acute lymphoblastic leukemia that is
relapsed or refractory.
2. Axicabtagene ciloleucel, a CD19-directed,
genetically modified autologous T cell immunotherapy is medically necessary for
adult patients with:
a. Adult patients with
diffuse large B-cell lymphoma that is refractory to first line
chemoimmunotherapy or that relapses within 12 months of first-line
chemoimmunotherapy.
b. Relapsed or
refractory large B-cell lymphoma after two or more lines of systemic therapy,
including:
(I) Diffuse large B-cell lymphoma
not otherwise specified,
(II)
Primary mediastinal large B-cell lymphoma,
(III) High grade B-cell lymphoma,
and
(IV) Diffuse large B-cell
lymphoma arising from follicular lymphoma.
c. Relapsed or refractory follicular lymphoma
after two or more lines of systemic therapy.
d. Axicabtagene ciloleucel is not indicated
for the treatment of patients with primary central nervous system
lymphoma.
3.
Brexucabtagene autoleucel, a CD19-directed, genetically modified autologous T
cell immunotherapy, is medically necessary for the treatment of adult patients
with:
a. Adult patients with relapsed or
refractory B-cell precursor acute lymphoblastic leukemia.
b. Relapsed or refractory Mantle cell
lymphoma.
4.
Lisocabtagene maraleucel, a CD19-directed, genetically modified autologous T
cell immunotherapy, is medically necessary for the treatment of adult patients
with relapsed or refractory large B-cell lymphoma after two or more lines of
systemic therapy, including:
a. Diffuse large
B-cell lymphoma not otherwise specified;
b. High-grade B-cell lymphoma;
c. Primary mediastinal large B-cell
lymphoma;
d. Diffuse large B-cell
lymphoma arising from indolent lymphoma; and
e. Follicular lymphoma grade
3B.
5. Idecabtagene
vicleucel, a genetically modified autologous T cell immunotherapy directed
against the B-cell maturation antigen called BCMA, is medically necessary for
the treatment of adult patients with relapsed or refractory multiple myeloma
after four or more prior lines of therapy, including an immunomodulatory agent,
a proteasome inhibitor, and an anti-CD38 monoclonal antibody.
6. Ciltacabtagene autoleucel, a genetically
modified autologous T cell immunotherapy directed against the B-cell maturation
antigen BCMA, is medically necessary for adult patients with relapsed or
refractory multiple myeloma after four or more prior lines of therapy,
including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38
monoclonal antibody.
7.
Betibeglogene autotemcel, patient's own bone marrow stem cells that are
genetically modified to produce functional beta-globin, is medically necessary
for adult and pediatric patients with ß-thalassemia who require regular
red blood cell transfusions.
8.
Elivaldogene autotemcel, patient's own bone marrow stem cells that are
genetically modified, is medically necessary to slow the progression of
neurologic dysfunction in boys 4-17 years of age with early, active cerebral
adrenoleukodystrophy.
9. Allogeneic
processed thymus tissue-agdc is medically necessary for immune reconstitution
in pediatric patients with congenital athymia.
10. Sipuleucel-T, an autologous T cell
immunotherapy, is medically necessary for patients with asymptomatic or
minimally symptomatic metastatic castrate resistant, hormone refractory,
prostate cancer.
(d)
Cellular therapies that are Food and Drug Administration (FDA)-approved for a
specific indication and are medically necessary, accepted within the
appropriate oncological specialty and not experimental for the purposes of
section 627.4236, F.S. are covered. In
cases where treatment for any of the above conditions includes a clinical trial
that conforms to subsection (6), routine care costs associated with the bone
marrow transplant will be covered.
(2) Each of the following procedures is
considered accepted within the appropriate oncological specialty and not
experimental for the purposes of Section
627.4236, F.S., provided that
the bone marrow transplantation procedure is performed in the context of a
well-designed clinical treatment trial as described in subsection (6).
Routine care costs associated with the bone marrow transplant
will be covered for the following procedures:
(a) Allogeniec bone marrow transplant for
multiple myeloma and other plasma cell dyscrasias; (e.g., Waldenstrom,
amyloid).
(b) Autologous bone
marrow transplant for:
1. Chronic lymphocytic
leukemia;
2. Germ cell tumor, high
risk, at diagnosis;
3. Ewing
sarcoma, after relapse;
4. Wilms
tumor, at relapse;
5. Soft tissue
sarcoma, pediatric, after failure of first therapy;
6. Multiple autologous bone marrow
transplants for pediatric solid tumors;
7. Autoimmune
disorders.
(3)
The following rare diseases, where there are no existing clinical trials
available, are covered for bone marrow transplant when deemed medically
necessary:
(a) Prolymphocytic
leukemia;
(b) Blastic plasmacytoid
dendritic cell neoplasm;
(c)
Systemic mastocytosis
(d) POEMS
syndrome;
(e) Atypical chronic
myeloid leukemia;
(f) Chronic
neutrophilic leukemia;
(g) Juvenile
myelomonocytic leukaemia;
(h)
Paroxysmal nocturnal hemoglobinuria (PNH); and,
(i) Cerebral form of adrenoleukodystoryphy
and metachromatic leukodystrophy;
(j) Mucopolysaccharidoses.
(k) Treatment of other rare conditions for
which there is no definitive data documenting the indication for BMT and cannot
realistically be expected to generate such data, can be covered. There must be
reasonable evidence of benefit from reputable sources and prior practice must
have demonstrated a benefit.
(4) Transplants from living related donors
incompatible for HLA-A, -B, and -DRB1 loci are covered for bone marrow
transplant at BMT CTN core or non-core medical facilities.
(5) Any bone marrow transplant performed
outside of a clinical trial will be covered when all the following criteria are
met:
(a) The plan of care follows a clinical
trial protocol that meets the requirements of subsection (6);
(b) Patient cannot be enrolled in the
proposed clinical trial;
(c) Bone
marrow transplant treatment is medically necessary;
(d) Patient is an appropriate candidate for
bone marrow transplant; and,
(e)
Treatment center is part of the BMT CTN at a core or non-core
center.
(6) A
well-designed and conducted clinical treatment trial is one which includes an
IRB-approved written protocol. At a minimum, such protocol shall have specific
criteria for evaluating the effect of treatment with defined endpoints that are
precise, meaningful, and reliable and which allow valid conclusions to be drawn
about therapeutic efficacy and safety. Protocols should include an adequate
statistical section describing the method of randomization and stratification,
if any, expected outcome parameters relating to response rates, time to
progression, survival times and other relevant information. Such clinical
treatment trials shall be consistent with protocols reviewed and approved by
the National Cancer Institute for scientific merit.
(7) Phases of the BMT Episode:
(a) Evaluation: The evaluation phase includes
services required to assess and evaluate whether a patient and, in the case of
allogeneic BMT, the donor, are suitable for the transplantation procedure. It
may also include evaluations to assess whether a transplantation is an
appropriate treatment option for the patient.
(b) Pretransplantation Care: The
pretransplantation care phase involves care provided from the time a patient is
identified as a candidate for BMT and includes all related care until the
initiation of conditioning regimen.
(c) Transplantation Event: The
transplantation event phase usually starts from the day of starting
conditioning regimen and it can last from 30 to 120 days after transplantation.
This phase covers the hematopoietic stem cell infusion and the transplantation
hospitalization, and it also typically includes graft procurement, stem cell
mobilization, and processing. In some situations, this phase can extend for a
longer period of time (eg, tandem transplantation for germ cell tumors). This
phase also includes any clinic visits associated with providing care to
patients receiving an outpatient transplantation.
(d) Follow-Up Care: The follow-up care phase
of the BMT episode of care starts on completion of the transplantation event
phase that may extend up to one year. However, transplant physicians will
continue caring for patients life long, to deal with late effects of therapy
including chronic GVHD from allogeneic transplant. Therefore, patients are
never truly discharged from transplantation center
follow-up.
Rulemaking Authority 627.4236 FS. Law Implemented 627.4236
FS.
New 11-9-95, Formerly 10D-127.001, Amended 9-26-00,
8-10-05, 7-7-13, 7-12-15, 2-4-19,
9-28-23.
