Page 69 - Medicare Benefit Policy Manual

Radiation Oncology

D. Generally

FDA-approved drugs and biologicals may also be considered for use in the determination of
medically accepted indications for off-label use if determined by the contractor to be
reasonable and necessary.

If a use is identified as not indicated by the Centers for Medicare and Medicaid Services
(CMS) or the FDA, or if a use is specifically identified as not indicated in one or more of the
compendia listed, or if the contractor determines, based on peer-reviewed medical literature,
that a particular use of a drug is not safe and effective, the off-label usage is not supported
and, therefore, the drug is not covered.

50.4.5.1 - Process for Amending the List of Compendia for
Determination of Medically-Accepted Indications for Off-Label Uses of
Drugs and Biologicals in an Anti-Cancer Chemotherapeutic Regimen
(Rev. 120, Issued: 01-29-10, Effective: 01-01-10, Implementation: 03-01-10)

A. Background

In the Physician Fee Schedule final rule for calendar year (CY) 2008, the CMS
established a process for revising the list of compendia, as authorized under section
1861(t)(2) of the Social Security Act, and also established a definition for
“compendium.” At 42 CFR 414.930(a), a compendium is defined “as a comprehensive
listing of FDA-approved drugs and biologicals or a comprehensive listing of a specific
subset of drugs and biologicals in a specialty compendium, for example, a compendium
of anti-cancer treatment.” A compendium: (1) includes a summary of the pharmacologic
characteristics of each drug or biological and may include information on dosage, as well
as recommended or endorsed uses in specific diseases; (2) is indexed by drug or
biological, and, (3) effective January 1, 2010, pursuant to section 182(b) of the Medicare
Improvements for Patients and Providers Act (MIPPA), has a publicly transparent
process for evaluating therapies and for identifying potential conflicts of interests. See 42
CFR 414.930(a); 72 FR 66222, 66404, and 74 FR 61901.

B. Desirable Characteristics of Compendia

CMS increased the transparency of the process by incorporating a list of desirable
compendium characteristics outlined by the Medicare Evidence Development and
Coverage Advisory Committee (MedCAC) as criteria for decision-making. The list of
desirable compendium characteristics was developed by the MedCAC during a public
session on March 30, 2006. The goal of this session was to review the evidence and
advise CMS on the desirable characteristics of compendia for use in the determination of
medically accepted indications of drugs and biologicals in anti-cancer therapy. As a
result of this meeting, the MedCAC generated the following list of desirable
characteristics: