2.0 POLICY
2.1 Coverage
for treatment of rare diseases may be considered on a case-by-case
basis. In reviewing the case, the contractor is authorized to approve
coverage when it is determined that the proposed treatment for the
rare disease is medically necessary, including that the treatment
is safe and effective.
2.1.1 In reviewing the case, any
or all of the following sources of clinical literature may be used
to determine if the proposed treatment is considered safe and effective.
2.1.1.1 Trials published in refereed
medical literature.
2.1.1.2 Formal technology assessments.
2.1.1.3 National medical policy organization
positions.
2.1.1.4 National professional associations.
2.1.1.5 National expert opinion organizations.
2.2 If case review
indicates that the proposed benefit for a rare disease is safe and
effective for that disease, benefits may be allowed. If benefits
are denied, an appropriate appealing party may request an appeal.
2.3 TRICARE Encounter Data (TED)
Record Special Processing Code “RD - Rare Diseases”
shall be coded on all TED records where the contractor has approved
treatment for a rare disease. Assignment of Special Processing Code RD will
allow the DHA to identify procedures approved by contractors under
the Rare Diseases policy and will allow bypass of TED edit 2-160-01R
when the procedure code is on the No Government Pay Procedure Code
List.
2.4 The contractor shall provide
a monthly report as described in the Contract Data Requirements List
(CDRL). The report should not include the rare disease treatments
previously approved for coverage and listed herein.
2.5 Off-label use of rituximab
may be considered for cost-sharing for the following treatments:
2.5.1 Effective January 1, 2003,
for recurrent nodular CD20 positive lymphocyte predominant Hodgkin’s
disease.
2.5.2 Effective
March 31, 2005, for Stiff Person Syndrome.
2.5.3 Effective May 1, 2007, for
Immunoglobulin A (IgA) nephropathy (proliferative glomerulonephritis)
to reduce proteinuria.
2.5.4 Effective
March 26, 2010, for neuromyelitis optica.
2.5.5 Effective July 20, 2016, for
N-methyl-D-aspartate (NMDA) receptor encephalitis.
2.5.6 Effective August 22, 2016,
for constitutional (pure) red blood cell aplasia.
2.5.7 Effective September 16, 2016,
for autoimmune sclerosing pancreatitis.
2.5.8 Effective October 6, 2016,
Immunoglobin G4-related disease (IgG4-RD).
2.5.9 Effective October 27, 2016,
for autoimmune hemolytic anemia.
2.5.10 Effective November 1, 2016,
for Graft-Versus-Host-Disease (GVHD).
2.5.11 Effective November 9, 2016,
for bullous pemphigoid.
2.5.12 Effective November 14, 2016,
as a second-line treatment for autoimmune encephalitis.
2.5.13 Effective November 22, 2016,
for cryoglobulinemia.
2.5.14 Effective January 3, 2017,
for Thrombotic Thrombocytopenic Purpura (TTP).
2.5.15 Effective January 19, 2017,
for polymyositis.
2.6 Effective May 13, 2009, Intraperitoneal
Hyperthermic Chemotherapy (IPHC) (Current Procedural Terminology
(CPT) procedure codes 77600, 77605, and 96445) in conjunction with
cytoreductive surgery or peritonectomy for treatment of pseudomyxoma
peritonei resulting from appendiceal carcinoma may be covered on
a case-by-case basis for adult patients when all of the following
criteria are met:
• There is no evidence of distant
metastasis.
• There is evidence of low histological
aggressiveness of the disease.
• The patient has not undergone
preoperative systemic chemotherapy.
• The patient’s condition does
not preclude major surgery.
• The chemotherapeutic agents
used are Mitomycin C, Cisplatin (also known as Cisplatinum), or Fluorouracil.
2.7 Effective January 21, 2009,
External Infusion Pumps (EIPs) for insulin may be considered for cost-sharing
when the diagnosis is Cystic Fibrosis-Related Diabetes (CFRD) with
fasting hyperglycemia. See
Chapter 8, Section 2.3 for policy regarding
EIPs.
2.8 Post-operative
proton beam radiosurgery/radiotherapy (CPT procedure codes 77520,
77522, 77523, and 77525) may be considered for cost-sharing when
the diagnosis is sacral chordoma. See
Chapter 5, Section 3.1 for policy regarding
proton beam radiosurgery/radiotherapy.
2.9 Extracorporeal photopheresis
(CPT procedure code 36522) may be considered for cost-sharing when
the diagnosis is Bronchiolitis Obliterans Syndrome (BOS) that is
refractory to immunosuppressive drug treatment. See
Chapter 4, Section 9.2 for policy regarding
photopheresis.
2.10 Effective
May 1, 2008, the off-label use of Selective Internal Radiation Therapy
(SIRT) with yttrium-90 microspheres (resin or glass) may be considered
for cost-sharing for the treatment of unresectable liver metastases
from neuroendocrine tumors. See
Chapter 5, Section 3.2 for policy regarding
brachytherapy/radiation therapy.
2.11 Effective April 15, 2016, Collagen
Cross-linking for the treatment of corneal ectasia due to the rare
disease Keratoconus is safe and effective and may be considered
for cost-sharing.
2.12 Effective
June 1, 2010, Radiofrequency Ablation (RFA), when performed using
an U.S. Food and Drug Administration (FDA) approved electrosurgical
cutting and coagulation device, may be considered for cost-sharing
for the treatment of liver metastases from gastric cancer.
2.13 Effective September 1, 2012,
the NovoTTF-100A system (HCPCS A4555 and E0766) may be cost-shared
for the treatment of adult patients (22 years of age or older) with
recurrent glioblastoma after surgical and radiation options have
been exhausted.
2.14 Effective
February 4, 2011, Radiesse® Voice laryngoplasty injections may be
cost-shared for the treatment of type 1 laryngeal cleft (also described
as supraglottic interarytenoid defects that extend no further than
the true vocal folds).
2.15 Effective November 27, 1995,
Orthotopic Liver Transplantation (OLT) may be cost-shared for the treatment
of Crigler-Najjar Syndrome Type I. OLT may be performed both prior
to the onset of neurological symptoms or after the onset of neurological
symptoms.
2.16 Effective
June 5, 2013, off-label use of intravenous immune globulin for the
treatment of Hashimoto’s Encephalopathy, may be considered in exceptional
circumstances where there is progressive neurologic decline despite
appropriate steroid therapy or where steroid therapy is contraindicated.
2.17 Effective April 30, 2009, Intrapulmonary
Percussive Ventilation (IPV) may be considered for cost-sharing
when the diagnosis is Cystic Fibrosis (CF). See
Chapter 8, Section 16.1 for policy regarding
IPVs.
2.18 Effective
January 4, 2013, allogeneic hematopoietic cell transplant (CPT procedure
code 38240) may be considered for cost-sharing for the treatment
of primary plasma cell leukemia.
2.19 Effective February 1, 2008,
the off-label use of Photodynamic Therapy (CPT procedure code 67221)
with Visudyne (HCPCS J3396) may be considered for cost-sharing for
the treatment of retinal astrocytic hamartoma in Tuberous Sclerosis.
2.20 Effective June 25, 2014, intracranial
angioplasty with stenting (CPT procedure code 61635) of the venous
sinuses may be considered for cost-sharing for the treatment of
pseudotumor cerebri (also known as idiopathic intracranial hypertension
and benign intracranial hypertension).
2.21 Effective February 1, 2012,
OLT (CPT procedure code 47135) may be considered for cost-sharing for
the treatment of Acute Intermittent Porphyria.
2.22 Effective December 1, 2014,
Photodynamic Therapy may be considered for cost-sharing for the treatment
of Central Serous Chorioretinopathy.
2.23 Effective July 22, 2016, chemotherapy
injections (CPT procedure code 96542) may be considered for cost-sharing
for the treatment of Central Giant Cell Granuloma (CGCG) of the
mandible.
2.24 Effective
July 22, 2016, Peg interferon alfa-2A/180 (HCPCS J3490) may be considered
for cost-sharing for the treatment of CGCG of the mandible.
2.25 Effective August 11, 2016,
a Fluorodeoxyglucose (FDG) PET scan (CPT procedure code 78815) may
be considered for cost-sharing for the treatment of Takayasu's Arteritis
(also known as aortic arch syndrome).
2.26 Effective August 22, 2016,
Gammagard liquid injections (HCPCS J1569) may be considered for cost-sharing
for the treatment of branch retinal artery occlusion secondary to
Susac’s Syndrome.
2.27 Effective
October 13, 2016, an autologous bone marrow transplant, the harvest
of autologous stem cells, and the cryopreservation of stem cells
may be considered for cost-sharing for the treatment of recurrent
medulloblastoma.
2.28 Effective
December 15, 2016, a Magnetic Resonance-guided High Intensity Focused
Ultrasound (MRgFUS) may be considered for cost-sharing for the treatment
of Desmoid fibromatosis.
2.29 Effective
January 24, 2017, Stereotactic Body Radiation Therapy (SBRT) (CPT
procedure codes 77435 and 77373) may be considered for cost-sharing
for the treatment of a benign neoplasm of the aortic body and other
paraganglia.
2.30 Effective
February 16, 2016, Proton Beam Therapy (PBT) may be considered for
cost-sharing for the treatment of thymoma.