Compendia Transparency Document

New Off-Label Uses

Monograph Off-Label Use Proposed Date Participating Panel Member(s) Strength of Recommendation Level of Evidence Off-Label Use Vote Record Panel Member Conflict of Interest Statement
Goserelin Prevention of early menopause during chemotherapy for early stage hormone receptor negative breast cancer 2015.03Diedra Bragalone, PharmD, BCOP, BCPS

Stephanie Minich, PharmD, BCOP

Corey Carter, MD

Omer Koc, MD

Nick Link, PharmD, BCOP
EquivocalBFor the Off-Label Use: 5 of 5 No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

Cisplatin Breast cancer (triple-negative) 2015.04Diedra Bragalone, PharmD, BCOP, BCPS

Stephanie Minich, PharmD, BCOP

Corey Carter, MD

Polly Kintzel, PharmD, BCOP, BCPS

Nick Link, PharmD, BCOP
Equivocal BFor the Off-Label Use: 4 of 5 No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest
Cisplatin Penile cancer 2015.04Diedra Bragalone, PharmD, BCOP, BCPS

Stephanie Minich, PharmD, BCOP

Corey Carter, MD

Nick Link, PharmD, BCOP

Mark Holdsworth, PharmD
EquivocalBFor the Off-Label Use: 5 of 5 No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

Idarubicin AML (Pediatric), relapsed/refractory 2015.04 Stephanie Minich, PharmD, BCOP

Diedra Bragalone, PharmD, BCOP, BCPS

Mark Holdsworth, PharmD

Heidi Trinkman, PharmD

Polly Kintzel, PharmD, BCOP, BCPS

Strong (for off-label use) AFor the Off-Label Use: 5 of 5 No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

Idarubicin AML (Pediatric), newly diagnosed 2015.04Stephanie Minich, PharmD, BCOP

Diedra Bragalone, PharmD, BCOP, BCPS

Mark Holdsworth, PharmD

Heidi Trinkman, PharmD

Polly Kintzel, PharmD, BCOP, BCPS

Equivocal B For the Off-Label Use: 5 of 5 No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

Topotecan Primary CNS lymphoma, relapsed/refractory 2015.06Stephanie Minich, PharmD, BCOP

Diedra Bragalone, PharmD, BCOP, BCPS

Corey Carter, MD

Omer Koc, MD

Nick Link, PharmD, BCOP

Equivocal C For the Off-Label Use: 5 of 5 No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

Nivolumab Melanoma, unresectable or metastatic (without BRAF mutation), first-line, single-agent therapy 2015.07Stephanie Minich, PharmD, BCOP

Diedra Bragalone, PharmD, BCOP, BCPS

Omer Koc, MD

Nick Link, PharmD, BCOP

Polly Kintzel, PharmD, BCOP, BCPS
Strong A For the off-label use: 5 of 5 No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

Palbociclib Breast cancer, advanced (second-line endocrine-based therapy) 2015.10Diedra Bragalone, PharmD, BCOP, BCPS

Stephanie Minich, PharmD, BCOP

Corey Carter, MD

Omer Koc, MD

Nick Link. PharmD, BCOP

Strong A For the Off-Label use: 5 of 5 No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

Fulvestrant Breast cancer, advanced (second-line endocrine-based combination therapy) 2015.10Diedra Bragalone, PharmD, BCOP, BCPS

Stephanie Minich, PharmD, BCOP

Corey Carter, MD

Omer Koc, MD

Nick Link. PharmD, BCOP

Strong A For the Off-Label use: 5 of 5 No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

CabozantinibRenal cell cancer, clear cell (advanced)2015.11Diedra Bragalone, PharmD, BCOP, BCPS

Stephanie Minich, PharmD, BCOP

Corey Carter, MD

Polly Kintzel, PharmD, BCOP, BCPS

Omer Koc, MD

Strong A For the Off-Label use: 5 of 5 No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

Nivolumab Renal cell cancer, clear cell (advanced) 2015.11Diedra Bragalone, PharmD, BCOP, BCPS

Stephanie Minich, PharmD, BCOP

Corey Carter, MD

Polly Kintzel, PharmD, BCOP, BCPS

Omer Koc, MD

Strong A For the off-label use: 4 of 5 No conflict of interest

No conflict of interest

Conflict of interest

No conflict of interest

No conflict of interest

GoserelinBreast cancer, advanced (second-line endocrine-based combination therapy [with palbociclib and fulvestrant] in pre- or peri-menopausal women) 2015.11Diedra Bragalone, PharmD, BCOP, BCPS

Stephanie Minich, PharmD, BCOP

Corey Carter, MD

Polly Kintzel, PharmD, BCOP, BCPS

Nick Link. PharmD, BCOP

Strong A For the off-label use: 5 of 5 No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

Ipilimumab Melanoma, unresectable or metastatic, first-line combination therapy 2015.11Stephanie Minich, PharmD, BCOP

Diedra Bragalone, PharmD, BCOP, BCPS

Polly Kintzel, PharmD, BCOP, BCPS

Omer Koc, MD

Nick Link. PharmD, BCOP

Strong A For the off-label use: 5 of 5 No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

Trabectedin Ovarian cancer, refractory (platinum-sensitive)2016.02Diedra Bragalone, PharmD, BCOP, BCPS

Stephanie Minich, PharmD, BCOP

Mark Holdsworth, PharmD

Omer Koc, MD

Nick Link, PharmD, BCOP

EquivocalBFor the off-label use: 5 of 5 No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

DocetaxelProstate cancer, metastatic, hormone-sensitive2016.03Diedra Bragalone, PharmD, BCOP, BCPS

Stephanie Minich, PharmD, BCOP

Corey Carter, MD

Polly Kintzel, PharmD, BCOP, BCPS

Nick Link, PharmD, BCOP

StrongAFor the off-label use: 5 of 5 No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

DefibrotideHepatic sinusoidal obstruction syndrome, prevention (pediatrics)2016.04Stephanie Minich, PharmD, BCOP

Diedra Bragalone, PharmD, BCOP, BCPS

Polly Kintzel, PharmD, BCOP, BCPS

Heidi Trinkman, PharmD

Mark Holdsworth, PharmD

EquivocalBFor the off-label use: 5 of 5 No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

VemurafenibErdheim-Chester disease, refractory (with BRAF V600E mutation)2016.05Diedra Bragalone, PharmD, BCOP, BCPS

Stephanie Minich, PharmD, BCOP

Corey Carter, MD

Polly Kintzel, PharmD, BCOP, BCPS

Omer Koc, MD

EquivocalBFor the off-label use: 5 of 5 No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

VemurafenibNon-small cell lung cancer, refractory (with BRAF V600 mutation)2016.05Diedra Bragalone, PharmD, BCOP, BCPS

Stephanie Minich, PharmD, BCOP

Corey Carter, MD

Polly Kintzel, PharmD, BCOP, BCPS

Omer Koc, MD

EquivocalCFor the off-label use: 5 of 5 No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

NivolumabHead and neck cancer, squamous cell (recurrent or metastatic)2016.06Diedra Bragalone, PharmD, BCOP, BCPS

Stephanie Minich, PharmD, BCOP

Polly Kintzel, PharmD, BCOP, BCPS

Omer Koc, MD

Nick Link, PharmD, BCOP

StrongAFor the off-label use: 5 of 5 No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

AxitinibThyroid cancer (advanced, differentiated)2016.06Diedra Bragalone, PharmD, BCOP, BCPS

Stephanie Minich, PharmD, BCOP

Corey Carter, MD

Mark Holdsworth, PharmD

Nick Link, PharmD, BCOP

EquivocalBFor the off-label use: 5 of 5 No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

CarfilzomibWaldenström macroglobulinemia2016.07Diedra Bragalone, PharmD, BCOP, BCPS

Stephanie Minich, PharmD, BCOP

Polly Kintzel, PharmD, BCOP, BCPS

Omer Koc, MD

Nick Link, PharmD, BCOP

EquivocalBFor the off-label use: 5 of 5 No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

RegorafenibHepatocellular cancer (intermediate or advanced)2016.07Diedra Bragalone, PharmD, BCOP, BCPS

Stephanie Minich, PharmD, BCOP

Corey Carter, MD

Mark Holdsworth, PharmD

Omer Koc, MD

StrongAFor the off-label use: 5 of 5 No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

PembrolizumabMerkel cell carcinoma (advanced)2016.08Diedra Bragalone, PharmD, BCOP, BCPS

Stephanie Minich, PharmD, BCOP

Corey Carter, MD

Polly Kintzel, PharmD, BCOP, BCPS

Omer Koc, MD

EquivocalBFor the off-label use: 5 of 5 No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

DabrafenibNon-small cell lung cancer, metastatic, relapsed or refractory (with BRAF V660E mutation)2016.10Diedra Bragalone, PharmD, BCOP, BCPS

Stephanie Minich, PharmD, BCOP

Corey Carter, MD

Polly Kintzel, PharmD, BCOP, BCPS

Omer Koc, MD

EquivocalBFor the off-label use: 5 of 5 No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

TrametinibNon-small cell lung cancer, metastatic, relapsed or refractory (with BRAF V660E mutation)2016.10Diedra Bragalone, PharmD, BCOP, BCPS

Stephanie Minich, PharmD, BCOP

Corey Carter, MD

Polly Kintzel, PharmD, BCOP, BCPS

Omer Koc, MD

EquivocalBFor the off-label use: 5 of 5 No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

NivolumabSmall cell lung cancer (progressive)2016.10Diedra Bragalone, PharmD, BCOP, BCPS

Stephanie Minich, PharmD, BCOP

Mark Holdsworth, PharmD

Omer Koc, MD

Nick Link, PharmD, BCOP

EquivocalBFor the off-label use: 4 of 5 No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

MitomycinVulvar cancer, advanced2016.11Stephanie Minich, PharmD, BCOP

Diedra Bragalone, PharmD, BCOP, BCPS

Corey Carter, MD

Omer Koc, MD

Nick Link, PharmD, BCOP

EquivocalCFor the off-label use: 5 of 5 No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

CarboplatinAnal cancer2016.12Stephanie Minich, PharmD, BCOP

Diedra Bragalone, PharmD, BCOP, BCPS

Corey Carter, MD

Omer Koc, MD

Nick Link, PharmD, BCOP

EquivocalCFor the off-label use: 4 of 5 No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

CarboplatinGastric cancer2016.12Stephanie Minich, PharmD, BCOP

Diedra Bragalone, PharmD, BCOP, BCPS

Corey Carter, MD

Omer Koc, MD

Nick Link, PharmD, BCOP

StrongAFor the off-label use: 5 of 5 No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

CarboplatinThyroid Cancer2016.12Stephanie Minich, PharmD, BCOP

Diedra Bragalone, PharmD, BCOP, BCPS

Corey Carter, MD

Omer Koc, MD

Nick Link, PharmD, BCOP

EquivocalB, GFor the off-label use: 5 of 5 No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

CarboplatinWilms Tumor2016.12Stephanie Minich, PharmD, BCOP

Diedra Bragalone, PharmD, BCOP, BCPS

Omer Koc, MD

Mark Holdsworth, PharmD

Heidi Trinkman, PharmD

EquivocalCFor the off-label use: 5 of 5 No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

FluorouracilVulvar cancer, advanced2016.12Diedra Bragalone, PharmD, BCOP, BCPS

Stephanie Minich, PharmD, BCOP

Corey Carter, MD

Omer Koc, MD

Nick Link, PharmD, BCOP

EquivocalCFor the off-label use: 5 of 5 No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

BevacizumabMalignant pleural mesothelioma (unresectable)2016.12Diedra Bragalone, PharmD, BCOP, BCPS

Stephanie Minich, PharmD, BCOP

Corey Carter, MD

Omer Koc, MD

Nick Link, PharmD, BCOP

StrongAFor the off-label use: 5 of 5 No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

BevacizumabHereditary hemorrhagic telangiectasia2016.12Diedra Bragalone, PharmD, BCOP, BCPS

Stephanie Minich, PharmD, BCOP

Mark Holdsworth, PharmD

Nick Link, PharmD, BCOP

Heidi Trinkman, PharmD

EquivocalCFor the off-label use: 5 of 5 No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

TemsirolimusEndometrial cancer (locally advanced, recurrent and/or metastatic)2017.02Diedra Bragalone, PharmD, BCOP, BCPS

Stephanie Minich, PharmD, BCOP

Corey Carter, MD

Polly Kintzel, PharmD, BCOP, BCPS

Nick Link, PharmD, BCOP

EquivocalBFor the off-label use: 5 of 5 No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

PembrolizumabUrothelial carcinoma (advanced)2017.02Diedra Bragalone, PharmD, BCOP, BCPS

Stephanie Minich, PharmD, BCOP

Corey Carter, MD

Polly Kintzel, PharmD, BCOP, BCPS

Omer Koc, MD

StrongAFor the off-label use: 5 of 5 No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

Off-Label Uses Removed From Database

Monograph Off-Label Use Removed Date Participating Panel Member(s) Strength of Recommendation Off-Label Use Removal Vote Record Conflict of Interest Statement
Ixabepilone Endometrial cancer (second-line treatment) 2014.11 Diedra Bragalone, PharmD, BCOP, BCPS

Stephanie Minich, PharmD, BCOP

Polly Kintzel, PharmD, BCOP, BCPS

Nick Link, PharmD, BCOP

Mark Holdsworth, PharmD
Against off-label use: Evidence suggests a lack of support for this off-label use; additional studies are needed to further define the role of ixabepilone in the management of this condition Remove the Off-Label Use: 4 of 5 (add to unsupported use) No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

Bevacizumab Ovarian cancer, advanced, recurrent (platinum sensitive) 2014.11 Diedra Bragalone, PharmD, BCOP, BCPS

Stephanie Minich, PharmD, BCOP
FDA-approved (no panel vote needed - removed from off-label uses due to approval by FDA) N/A N/A
Alemtuzumab Multiple sclerosis 2014.11 Diedra Bragalone, PharmD, BCOP, BCPS

Stephanie Minich, PharmD, BCOP
FDA-approved (no panel vote needed - removed from off-label uses due to approval by FDA) N/A N/A
Medroxyprogesterone injection Renal Carcinoma (labeled use, add to Use: Unsupported) 2015.02 Diedra Bragalone, PharmD, BCOP, BCPS

Stephanie Minich, PharmD, BCOP

Corey Cater, MD

Omer Koc, MD

Polly Kintzel, PharmD, BCOP, BCPS
Against Off-Label Use Add to Use: Unsupported: 5 of 5 (add to unsupported use) No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

Decitabine Sickle Cell Anemia 2015.05 Diedra Bragalone, PharmD, BCOP, BCPS

Stephanie Minich, PharmD, BCOP

Mark Holdsworth, PharmD

Polly Kintzel, PharmD, BCOP, BCPS

Heidi Trinkman, PharmD
Against off-label use: Evidence suggests a lack of support for this off-label use; evidence-based guidelines do not mention decitabine as a therapeutic option for management of sickle cell anemia. Additional studies are needed to further define the role of decitabine in the management of this condition. Remove the Off-Label Use: 4 of 5 (add to unsupported use)No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

Topotecan Merkel cell carcinoma 2015.06 Stephanie Minich, PharmD, BCOP

Diedra Bragalone, PharmD, BCOP, BCPS

Corey Carter, MD

Omer Koc, MD

Nick Link, PharmD, BCOP

Against off-label use: Limited evidence exists for the use of topotecan in the management of merkel cell carcinoma; additional trials are needed to further define the role of topotecan in the treatment of this condition. Remove the Off-Label Use: 5 of 5 (add to unsupported use) No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

Octreotide Meningioma2015.09 Diedra Bragalone, PharmD, BCOP, BCPS

Stephanie Minich, PharmD, BCOP

Corey Carter, MD

Mark Holdsworth, PharmD

Polly Kintzel, PharmD, BCOP, BCPS

Omer Koc, MD
Add to Use: Unsupported: While results from a small study of octreotide LAR for recurrent meningioma demonstrated partial response or stable disease in some patients, subsequent studies in recurrent/progressive meningioma have not demonstrated a benefit. Given the unknown natural history of meningioma, the role of somatostatin analogues in meningioma is unclear.For the Use: 3 of 6 (Off-label use not added [no consensus]); Add to Use: UnsupportedNo Conflict of Interest

No Conflict of Interest

No Conflict of Interest

No Conflict of Interest

No Conflict of Interest

No Conflict of Interest
PegfilgrastimPrevention of chemotherapy-induced neutropenia in children 2015.12 Stephanie Minich, PharmD, BCOP

Diedra Bragalone, PharmD, BCOP, BCPS
FDA approved (no panel vote needed - removed from off-label uses due to pediatric dosing addition to FDA labeling).n/a n/a
Nivolumab Renal cell cancer, clear cell (advanced)2015.12 Stephanie Minich, PharmD, BCOP

Diedra Bragalone, PharmD, BCOP, BCPS

FDA approved (no panel vote needed - removed from off-label uses due to approval by FDA).n/an/a
Nivolumab Melanoma, unresectable or metastatic (without BRAF mutation), first-line, single-agent therapy2015.12 Stephanie Minich, PharmD, BCOP

Diedra Bragalone, PharmD, BCOP, BCPS

FDA approved (no panel vote needed - removed from off-label uses due to approval by FDA).n/an/a
Uridine Triacetate Fluorouracil overdose/overexposure2015.12 Diedra Bragalone, PharmD, BCOP, BCPS

Stephanie Minich, PharmD, BCOP

FDA approved (no panel vote needed - removed from off-label uses due to approval by FDA).n/an/a
DocetaxelCervical Cancer (recurrent)2016.02 Diedra Bragalone, PharmD, BCOP, BCPS

Stephanie Minich, PharmD, BCOP

Corey Carter, MD

Omer Koc, MD

Polly Kintzel, PharmD, BCOP, BCPS
Against off-label use: Evidence suggests a lack of support for this off-label use; additional studies are needed to further define the role of docetaxel in the management of this condition.Remove the off-label use: 3 of 5 (Add to Use: Unsupported) No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

PalbociclibBreast cancer, advanced (second-line endocrine-based therapy)2016.03 Stephanie Minich, PharmD, BCOP

Diedra Bragalone, PharmD, BCOP, BCPS
FDA approved (no panel vote needed - removed from off-label uses due to approval by FDA).n/an/a
FulvestrantBreast cancer, advanced (second-line endocrine-based therapy)2016.02 Diedra Bragalone, PharmD, BCOP, BCPS

Stephanie Minich, PharmD, BCOP
FDA approved (no panel vote needed - removed from off-label uses due to approval by FDA). n/an/a
CabozantinibRenal cell carcinoma, advanced2016.05 Stephanie Minich, PharmD, BCOP

Diedra Bragalone, PharmD, BCOP, BCPS
FDA approved (no panel vote needed – removed from off-label uses due to approval by FDA).n/an/a
IfosfamideHead and neck cancer2016.09 Stephanie Minich, PharmD, BCOP

Diedra Bragalone, PharmD, BCOP, BCPS

Corey Carter, MD

Omer Koc, MD

Nick Link, PharmD, BCOP
Against off-label use: Evidence suggests a lack of support for this off-label use; other contemporary therapies have replaced the use of ifosfamide for the treatment of head and neck cancer.Remove the off-label use: 5 of 5 No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

IfosfamideSmall cell lung cancer2016.09 Stephanie Minich, PharmD, BCOP

Diedra Bragalone, PharmD, BCOP, BCPS

Corey Carter, MD

Omer Koc, MD

Nick Link, PharmD, BCOP
Against off-label use: Evidence suggests a lack of support for this off-label use; other contemporary therapies have replaced the use of ifosfamide for the treatment of small cell lung cancer.Remove the off-label use: 5 of 5 (Add to Use: Unsupported) No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

NivolumabHead and neck cancer, squamous cell (recurrent or metastatic)2016.11 Stephanie Minich, PharmD, BCOP

Diedra Bragalone, PharmD, BCOP, BCPS
FDA approved (no panel vote needed - removed from off-label uses due to approval by FDA).n/an/a
MitomycinNon-small cell lung cancer2016.11 Stephanie Minich, PharmD, BCOP

Diedra Bragalone, PharmD, BCOP, BCPS

Polly Kintzel, PharmD, BCOP, BCPS

Omer Koc, MD

Nick Link, PharmD, BCOP
Against off-label use: Evidence suggests a lack of support for this off-label use; other contemporary therapies have replaced the use of mitomycin for the treatment of non-small cell lung cancer.Remove the off-label use: 5 of 5 No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

FluorouracilThymic cancers2017.01Diedra Bragalone, PharmD, BCOP, BCPS

Stephanie Minich, PharmD, BCOP

Mark Holdsworth, PharmD

Polly Kintzel, PharmD, BCOP, BCPS

Nick Link, PharmD. BCOP
Against off-label use: A lack of evidence suggests a lack of support for this off-label use.Remove the off-label use: 5 of 5 No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

No conflict of interest

Last update March 3, 2017.