Rare Diseases, Treatment of

A rare disease is any disease or condition that affects less than 200,000 people in the U.S.

• TRICARE covers medical treatments, drugs, devices, and procedures that are already proven to be safe and effective.
• TRICARE may consider other treatments for rare diseases on a case-by-case basis.

Below are some of the rare diseases and treatments TRICARE may cover based on case reviews. This list isn’t all-inclusive. You can contact your regional contractor to see if you may qualify or for more information.

Rare Disease	Possible Treatment
Acute intermittent porphyria	Orthotopic liver transplantation
Bronchiolitis obliterans syndrome	Extracorporeal photopheresis
Central serous chorioretinopathy	Photodynamic therapy
Crigler-Najjar syndrome, type 1	Orthotopic liver transplantation
Cystic fibrosis	Intrapulmonary percussive ventilation
Cystic fibrosis-related diabetes with fasting hyperglycemia	External infusion pumps for insulin
Hashimoto’s encephalopathy	Off-label use of intravenous immune globulin
Hodgkin’s disease	Off-label use of rituximab
Immunoglobulin A nephropathy (proliferative glomerulonephritis)	Off-label use of rituximab
Laryngeal cleft, type 1	Radiesse® Voice laryngoplasty injections
Liver metastases from gastric cancer	Radiofrequency Ablation
Neuromyelitis optica	Off-label use of rituximab
Primary plasma cell leukemia	Allogeneic hematopoietic cell transplant
Pseudomyxoma peritonei	Hyperthermic intraperitoneal chemotherapy (in conjunction with cytoreductive surgery or peritonectomy)
Pseudotumor cerebri	Intracranial angioplasty with stenting
Recurrent glioblastoma	NovoTTF-100A system
Retinal astrocytic hamartoma in tuberous sclerosis	Photodynamic therapy with Visudyne
Sacral chordoma	Post-operative proton beam radiosurgery/radiotherapy
Unresectable liver metastases from neuroendocrine tumors	Off-label use of selective internal radiation therapy with yttrium-90 microspheres