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.