Clinical UM Guideline


Subject:Oncology Drug Treatment Regimens for Adults **NOTE: At this time, this guideline is not implemented for medical benefit determinations
Guideline #:  CG-DRUG-31Current Effective Date:  01/14/2014
Status:NewLast Review Date:  11/14/2013

Description

NOTE: At this time, this guideline is not implemented for medical benefit determinations.

This document addresses oncology drug treatment regimens for adults which may include (whether oral, injectable, or infusable) chemotherapy, biologics and other targeted therapies utilized to treat cancer (collectively, "agents").  A regimen may consist of a single agent or include two or more agents.  The agent(s) in a treatment regimen may be utilized in an off-label indication.  This guideline does not address agents used to treat complications (for example, nausea), toxicities (for example, cytopenias), or adverse events (for example, infection) that occur as a result of the treatment regimen. 

The U.S. Food and Drug Administration (FDA) approves drugs for specific use(s) that are listed in the drug's product information label.  Off-label or "unlabeled" drug use is the utilization of an FDA approved drug for uses other than those listed in the FDA approved labeling or in treatment regimens or populations that are not included in approved labeling.  Many off-label uses are effective, well documented in the peer-reviewed literature and widely used. 

Note: For additional information, please see:

Clinical Indications

Medically Necessary:

An oncology treatment regimen may be medical necessary when all of the following criteria are met:

  1. The agents(s) in the regimen is(are) approved by the FDA for use in humans; and
  2. The treatment regimen is being prescribed to treat an adult (age greater than 18) with cancer for whom treatment is medically appropriate; and
  3. The oncology treatment regimen is supported by one or more of the following for the specific clinical situation under review (for example, but not limited to, stage of disease, prior treatment, performance status, comorbid conditions, or absence of contraindications):
    1. Category 1 or 2A designation found in the most recent edition of the National Comprehensive Cancer Network® (NCCN®) Drugs & Biologics Compendium (NCCN Compendium®) or NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®); or
    2. Two articles are identified which meet all of the following for the specific regimen:
      1. The articles were published in major scientific or medical peer-reviewed journals including, but not limited to: Journal of American Medical Association, New England Journal of Medicine, Journal of Clinical Oncology or Lancet; and
      2. The articles used appropriate study designs including, but are not limited to, randomized, double blind, placebo-controlled clinical trials; and
      3. The articles are not case reports, letters, posters or abstracts; and
      4. The data published in the articles supports the proposed oncology treatment regimen for the specific cancer and clinical situation as safe and effective.

If the off-label use of a drug within a regimen is determined to be medically necessary, its use shall also be determined to be "non-investigational" for the purposes of benefit determination.

Not Medically Necessary: 

An oncology treatment regimen is considered not medically necessary when the above criteria are not met.

Discussion/General Information

The U.S. Food and Drug Administration (FDA) approves drugs for specific use(s) that are listed in the drug's product information label.  Off-label or "unlabeled" drug use is the utilization of an FDA approved drug for uses other than those listed in the FDA approved labeling or in treatment regimens or populations that are not included in approved labeling.  Many off-label uses are effective, well documented in the peer-reviewed literature and widely used.

The drug label approved by the FDA "is the official description of a drug product which includes indication (what the drug is used for); who should take it; adverse events (side effects); instructions for uses in pregnancy, children, and other populations; and safety information for the individual.  Labels are often found inside drug product packaging" (FDA, 2012).  The terms "off-label drug" or "unlabeled drug" refer to the prescription and use of a drug for an indication that is not stated in the approved FDA labeling.

The American Cancer Society (2013) notes in the Chemotherapy Principles:

In some cases, the best choice of doses and schedules for giving each drug are clear, and most oncologists would recommend the same treatment. In other cases, less may be known about the single best way to treat people with certain types and stages of cancer.  In these situations different cancer doctors might choose different drug combinations with different schedules.

Factors to consider in choosing which drug to use for a chemotherapy regimen include:

The National Comprehensive Cancer Network® (NCCN) Drug & Biologics Compendium® is a listing of appropriate uses of agents as defined in and derived from the NCCN Clinical Guidelines in Oncology®.  The compendium lists both FDA-approved uses and NCCN designated off-label indications.  According to the NCCN, the identified off-label indications are based upon evaluation of evidence from scientific literature integrated with expert judgment in an evidence-based process.  Indications are categorized in a systematic approach that describes the type of evidence available for and the degree of consensus underlying each recommendation.  For purposes of this clinical UM guideline, any indication listed in the NCCN Drug & Biologics Compendium with a Category of Evidence and Consensus 1 or 2A is considered an off-label indication and is determined to be medically necessary.  See Attachment A for NCCN Categories of Evidence and Consensus.

NCCN Categories of Evidence and Consensus

Category 1: Based upon high-level evidence, there is uniform NCCN consensus that the intervention is appropriate.

Category 2A: Based upon lower-level evidence, there is uniform NCCN consensus that the intervention is appropriate.

Category 2B: Based upon lower-level evidence, there is NCCN consensus that the intervention is appropriate.

Category 3: Based upon any level of evidence, there is major NCCN disagreement that the intervention is appropriate.

© National Comprehensive Cancer Network, Inc. 2013

The Company takes into account credible scientific evidence published in peer-reviewed medical literature generally recognized by the relevant medical community.  "Peer-reviewed medical literature" does not include publications or supplements to publications that are sponsored to a significant extent by a pharmaceutical manufacturing company or health carrier.

Definitions

Drug: A substance intended for use in the diagnosis, cure, mitigation, treatment, or prevention of disease; a substance (other than food) intended to affect the structure or any function of the body; biological products are included within this definition and are generally covered by the same laws and regulations, but differences exist regarding their manufacturing processes (chemical process versus biological process).

ECOG (Eastern Cooperative Oncology Group) Performance Status: A scale used to determine the individual's level of functioning.  This scale may also be referred to as the WHO (World Health Organization) or Zubrod score based on the following scale:

0  Fully active, able to carry on all pre-disease performance without restriction
1  Restricted in physically strenuous activity but ambulatory and able to carry out work of a light or sedentary nature, e.g., light house work, office work
2  Ambulatory and capable of all self-care but unable to carry out any work activities. Up and about more than 50% of waking hours
3  Capable of only limited self-care, confined to bed or chair more than 50% of waking hours
4  Completely disabled. Cannot carry on any self-care. Totally confined to bed or chair
5  Dead

Karnofsky Score: A measure of the individual's overall physical health, judged by their level of activity; the score uses the following scale:

100%  Normal, no complaints, no signs of disease
90%    Capable of normal activity, few symptoms or signs of disease
80%    Normal activity with some difficulty, some symptoms or signs
70%    Caring for self, not capable of normal activity or work
60%    Requiring some help, can take care of most personal requirements
50%    Requires help often, requires frequent medical care
40%    Disabled, requires special care and help
30%    severely disabled, hospital admission indicated but no risk of death
20%    Very ill, urgently requiring admission, requires supportive measures or treatment
10%    Moribund, rapidly progressive fatal disease processes
0%      Death

References

Government Agency, Medical Society, and Other Authoritative Publications: 

  1. National Comprehensive Cancer Network®. NCCN Drugs & Biologic Compendium™ (electronic version). For additional information visit the NCCN website: http://www.nccn.org/index.asp. Accessed on September 26, 2013.
Web Sites for Additional Information
  1. American Cancer Society. Chemotherapy Principles. An in-depth discussion of the techniques and its role in cancer treatment. Revised February 7, 2013. Available at: http://www.cancer.org/acs/groups/cid/documents/webcontent/002995-pdf.pdf. Accessed on September 26, 2013.
  2. American Cancer Society. Targeted Therapy. Revised June 20, 2012. Available at: http://www.cancer.org/acs/groups/cid/documents/webcontent/003024-pdf.pdf.  Accessed on September 26, 2013.
Index

Biologic agents
Chemotherapy
Prescription Drugs, Unlabeled Use of
Targeted therapy
Unlabeled Use of Drugs

History

Status

Date

Action

New11/14/2013Medical Policy & Technology Assessment Committee (MPTAC) review.
New

11/13/2013

 

Hematology/Oncology Subcommittee review.  Initial document development.