Medicare Off-Label Approval Checklist
A statement from the physician on the accepted standard of medical practice for the diagnosis
and the medical circumstances of this case that the physician believes justifies the use of the drug
Clinical research literature that appears in at least two Phase III clinical trials that definitively
demonstrates the safety and effectiveness; OR, at least two Phase II clinical trials with reasonably
large patient samples showing consistent results of safety and efficacy.
Clinical research that appears in peer reviewed medical literature.
Published recommendations from specialty societies or in other authoritative evidencebased guidelines supporting a use that is an accepted standard of medical practice.
For support in determining coverage the following will be
considered:
 the adequacy of the number of subjects
 response rate (Ex. evidence of significant decrease in tumor size or reduction in tumor
related symptoms. Stabilization is not considered a response to therapy.)
effect on key status and survival indications,
 appropriateness of study design

the prevalence and life history of disease when evaluating the adequacy of the number of
subjects and the response rate.
Attach any supporting literature from the following publications:
American Journal of Medicine;
 Annals of Internal Medicine;

Annals of Oncology;
Annals of Surgical Oncology;
Biology of Blood and Marrow Transplantation;
Blood;
Bone Marrow Transplantation;
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
British Journal of Cancer; British Journal of Hematology;

British Medical Journal;
Cancer;
Clinical Cancer Research;

Drugs;

European Journal of Cancer (formerly the European Journal of Cancer and Clinical Oncology);
Gynecologic Oncology;
International Journal of Radiation, Oncology, Biology, and Physics;

The Journal of the American Medical Association;
Journal of Clinical Oncology;
Journal of the National Cancer Institute;
Journal of the National Comprehensive Cancer Network (NCCN);

Journal of Urology;
 Lancet;
Lancet Oncology;
Leukemia;
 The New England Journal of Medicine;

Radiation Oncology
After such evidence is received, Medicare will, with help of specialty specific consultants as
indicated, make a coverage determination for the non-FDA approved indication (off-label use)
of the drug or biological.
In those cases when Medicare does not approve off-label use of the drug or biological in question
an ABN may be necessary. (http://www.cms.hhs.gov/BNI/02_ABNGABNL.asp)
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Once the checklist and relevant data is gathered please forward the
information to :
Outpatient Registration Manager, YNHHS at the PCC, Clinical Registration, Room 16E.
YNHHS will follow the below submission information to:
Medicare: Janet Lawrence,
MD Medical Director,
MAC J-13
National Government Services
P.O. Box 4767
Syracuse, NY 13221-4767
Email: janet.lawrence3@wellpoint.com
Source: CMS Local Coverage Determination (National Government Services) Drugs and
Biologicals, coverage of, for Label and Off-Label uses (L25820).
Please also forward a copy of the information being submitted to YNHHS Outpatient Registration
Manager and the response from NGS to the YMG Medical Billing Compliance Officer
(judy.harris@yale.edu)
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