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.