MP 5.01.01

Off-Label Drug Use

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Medical Policy
Section Prescription Drug	Original Policy Date 3:2008	Last Review Status/Date Local Policy/3:2008
Issue 3:2003		Return to Medical Policy Index

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Disclaimer

Our medical policies are designed for informational purposes only and are not an authorization, or an explanation of benefits, or a contract.  Receipt of benefits is subject to satisfaction of all terms and conditions of the coverage.  Medical technology is constantly changing, and we reserve the right to review and update our policies periodically.

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Description

Policy

Rationale

1. FDA-approved drugs for the treatment of a specific condition, other than a condition for which the drug was approved by the FDA, may be considered medically appropriate when the drug:

• Is recognized for use as effective for the type of condition under treatment; AND
• Is stated in DrugDex OR has documented scientific evidence
• The majority of the scientific evidence indicates that the drug is effective for the off-label indiciation.  The evidence must:
  • Consist of at least one well-designed study with sufficient numbers of patients in relation to the incidence of the disease.
  • Appear in at least one peer-reviewed journal.Studies must appear in journals or other publications that publish original manuscripts critically reviewed by unbiased independent experts for scientific accuracy, validity and reliability.

Note: Internal publications or promotional materials from pharmaceutical manufacturers are not peer-reviewed literature.

Hematology/Oncology Coverage:

1. Blue Cross of Idaho member policies specifically provide coverage for prescribed drugs approved by the FDA for the treatment of certain types of cancer.  We will cover, pending benefits and eligibility, use of a drug not specifically approved for treatment of a specific type of cancer if the drug is recognized for treatment of the specific type of cancer in one of the following references (A provider must include these references with a request for prior authorization):

• National Comprehensive Cancer Network Practice Guidelines in Oncology and Drugs & Biologics Compendium® OR
• Association of Community Cancer Centers Cancer Program Guidelines and Drug Bulletin OR
• DrugDex OR
• Documented Scientific Evidence - The majority of the scientific evidence indicates that the drug is effective for the off-label indication.

The evidence must:

1. Consist of at least one well-designed phase II or Phase III study wth sufficient numbers of patients in relation to the incidence of the disease.
2. Appear in peer-reviewed journals.  Studies must appear in journals or other publications that publish original manuscripts, critically reviewed by unbiased independent experts for scientific accuracy, validity and reliability.

Note: Internal publications or promotional materials from pharmaceutical manufacturers are not peer-reviewed literature.

• Published studies submitted for review must include data indicating the clinical benefit claimed and the toxicities encountered to allow for evaluation of the safety of the agent in a specific situation
• Published phase III studies submitted for review should address the use of the requested agent in the indication requested

1. When a clinical trial is open for accrual at the facility that provides the drug under consideration for the indication requested and when the patient meets the eligibility requirements for that study, please enroll the patient in the study instead of seeking coverage from Blue Cross of Idaho.

Reference:

1. BlueCross of Idaho Medical Policy Reference Manual Policy # 5.01.01. 2003 Oct 9.
2. Choonara I, et al. Unlicensed and off-label drug use in children: implications for safety. Drug Saf 2002; 25(1):1-5
3. Conroy S. Unlicensed and off–label drug use: issues and recommendations. Paediatr Drugs 2002; 4(6):353-9.
4. Roberts R. Adequacy of current laws governing research in children. The view from the FDA. [http://www.fda.gov/ cder/pediatric/presentation/sfethics/index.htm]
5. U.S. Congress. Best pharmaceuticals for children act. Public Law 107-109, S. 1789. [http://www.fda.gov/cder/ pediatric/pl107-109.pdf].
6. U.S. Food and Drug Administration. Improving regulation of drugs. Modernization Act of 1997, Title I. [http://www.fda.gov/modact.htm].
7. U.S. Food and Drug Administration. Off-label and investigational use of marketed drugs, biologicals, and medical devices. Guidance for institutional review boards and clinical investigators. 2001 Apr. [http://www.fda.gov/oc/ ohrt/irbs/offlabel.html] accessed 7/31/03.
8. U.S. Food and Drug Administration. The pediatric exclusivity provision. Status report to Congress. 2001 Jan. [http://www.fda.gov/cder/pediatric/reportcong01.pdf].

Policy History

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Attachment A (Member Contract)

Medically Necessary (or Medical Necessity) — covered services or supplies required to identify or treat a member’s condition, disease, illness or accidental injury that are:

1. The most appropriate supply or level of service, considering potential benefits and harms to the member
2. Proven to be effective in improving health outcomes;
   a. For new treatments, effectiveness is determined by scientific evidence;
   b. For existing treatments, effectiveness is determined first by scientific evidence, then by professional standards, then by expert opinion.
3. Not primarily for the convenience of the member or covered  provider.
4. Cost-effective for the condition when, compared to alternative treatments, including no treatment.

Cost-effectiveness does not necessarily mean lowest price.

When applied to the care of an inpatient, medically necessary further means that the member’s medical symptoms or condition are such that a provider cannot safely and effectively provide services to themember as an outpatient.

The fact that a provider may prescribe, order, recommend or approve a service or supply does not, in and of itself, necessarily establish that service or supply as medically necessary under this policy.

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Attachment B (Member Contract)

Prior Authorization Section

Prior authorization

Notice: To establish eligibility for benefits under the terms of this policy, a provider must get prior authorization to determine the medical necessity of the covered services listed below.  If a provider does not get prior authorization to determine medical necessity, Blue Cross may deny coverage of services.  A provider must resolve disputed decisions using the Blue Cross of Idaho appeals process outlined in the general provisions section.

If a contracting provider performs non-medically necessary services without prior authorization by Blue Cross of Idaho, and Blue Cross denies the claim, the member is no financially responsible for the cost of services.

A member is financially responsible for non-medically necessary services provided by a non-contracting provider.

Prior authorization is a request by the member's contracting provider to Blue Cross of Idaho, or delegated entity, for authorization of a proposed treatment. Blue Cross may review medical records, test results and other sources of information to ensure a service is covered and determine medical necessity or alternative treatments.

A member is responsible for obtaining prior authorization when seeking treatment from a non-contracting provider.

Please refer to Attachment A of the Benefits Outline, check the Blue Cross of Idaho Website at www.bcidaho.com, or call customer service at the telephone number on the member's identification card to determine if the proposed services require prior authorization. To request prior authorization, a contracting provider must notify Blue Cross of the member's intent to receive services that require prior authorization.

A member can notify Blue Cross by telephone call or in writing, and the notification must include the information necessary to establish that the proposed services are medicall necessary and covered under the member's policy. Blue Cross will respond to prior authorization requests from providers or members within two business days of receipt of the medical information necessary to make a determination.

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Attachment A of the Benefits Outline

 

NON-EMERGENCY SERVICES REQUIRING PRIOR AUTHORIZATION ANNUAL NOTICE

EFFECTIVE: January 1, 2007

 

Notice: To establish eligbility for benefits under the terms of this policy, a provider must get prior authorization to determine the medical necessity of covered services listed below. If a provider does not get prior authorization to determine medical necessity, Blue Cross may deny coverage of services.  A provider must resolve disputed decisions using the Blue Cross of Idaho appeals process outlined in the general provisions section.

If a contracting provider performs non-medically necessary services without prior authorization by Blue Cross of Idaho, and Blue Cross of Idaho denies the claim, the member is not financially responsible for the cost of services.

A member is financially responsible for non-medically necessary services provided by a non-contracting provider.

Blue Cross of Idaho will respond to prior authorization requests from providers or members within two business days of the receipt of the medical information necessary to make a determination.  For additional information, please check with your provider, call customer service at the telephone number listed on the member's indentification card or check the Blue Cross of Idaho Website at www.bcidaho.com.

 

Surgical Service - Inpatient or Outpatient

• Organ and tissue transplants
• Gallbladder surgery
• Arthroscopic surgery of the knee, hip, shoulder, wrist or jaw
• Nasal and sinus procedures
• Eyelid surgery
• Spinal surgery
• Hysterectomy
• Gastric reflux procedures
• Plastic and reconstructive surgery
• Surgery for snoring or sleep problems
• Invasive treatment of lower extremity veins (including but not limited to varicose veins)
• Advanced imaging services (not applicable for inpatient services):
  • Magnetic Resonance Imaging (MRI)
  • Magnetic Resonance Angiography (MRA)
  • Computed Tomography Scans (CT Scan)
  • Positron Emission Tomography (PET)

 

Other services

• Inpatient stays that originate from an outpatient service
• Diabetes self management education
• Home intravenous therapy
• Non-emergent ambulance
• Certain prescription drugs (including drugs that cost five hundred dollars ($500) or more)
• Restorative dental services following accidental injury to sound natural teeth
• Hospice services
• Growth hormone therapy
• Genetic testing services
• Home health skilled nursing services

The following services require Prior Authorization when the expected charges exceed ($300):

• Rental or purchase of durable medical equipment
• Prosthetic appliances
• Orthotic devices

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Attachment C

 

Investigational — Any technology (service, supply, procedure, treatment, drug, device, facility, equipment or biological product) that is in a developmental stage or has not been proven to improve health outcomes such as length of life, quality of life and functional ability.

A technology is investigational if, as determined by Blue Cross of Idaho, it fails to meet any one of the following criteria:

• The technology must have failed final approval from the appropriate government regulatory body. This applies to drugs, biological products, devices, and other products/procedures that must have approval from the U.S. Food and Drug Administration (FDA) or another federal authority before marketing them. Interim approval is not sufficient. The condition for which the technology is approved must be the same condition that Blue Cross is evaluating.
• The scientific evidence must permit conclusions concerning the effect of the technology on health outcomes. The evidence should consist of current published medical literature and investigations published in peer-reviewed journals. Evaluation of evidence will take into consideration the quality of the studies and consistency of results. The evidence should demonstrate that the technology can measure or alter physiological changes related to a disease, injury, illness or condition. In addition, there should be evidence that such measurement or alteration affects health outcomes.
• The technology must improve the net health outcome. The technology’s beneficial effects on health outcomes should outweigh any harmful effects on health outcomes.
• The technology must be as beneficial as any established alternatives.
• The technology must show improvement that is attainable outside the investigational setting.  Use under usual conditions of medical practice must demonstrate the improvements.

 

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Attachment D (Provider Contract)

 

 

Medically Necessary (Medical Necessity)-

Covered services or supplies required o identify or treat a member's condition, disease, illness or accidenal injury that are:

1. The most appropriate supply or level of service, considering potential benefits and harms to the member;
2. Proven to be effective in improving health outcomes;
   • For new treatments, effectiveness is determined by scientific evidence;
   • For existing treatments, effectiveness is determined first by scientific evidence, then by professional standards, then by expert opinion;
   • Not primarily for the convenience of the member or covered provider; and
3. Cost-effective for the condition when compared to alternative treatments, including no treatment. Cost-effectiveness does not necessarily mean lowest price.

When applied to the care of an inpatient, medically necessary further means that the member's medical symptoms or condition are such that a provider cannot safely and effectively provide services for the member as an outpatient.

The fact that a covered provider may prescribe, order, recommend or approve a service or supply does not in and of itself necessarily establish that such service or supply is medically necessary under the applicable member policy and this agreement.

The term medically necessary as defined and used in this agreement is strictly limited to the application and interpretation of this agreement, and any determination as to whether a service is medically necessary hereunder we make solely for determining whether we provide coverage for the services rendered.

 

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Attachment E

 

NICC PREAUTHORIZATION REQUEST

 

 

 

 

Date:__________________                    

 

Member Name:________________________________

 

Member DOB:________________________________________

 

BCI Member Identification Number: _________________________________

                                               

Diagnosis Code/s (ICD-9):__________________________________________

 

Physician Name:____________________

Physician Phone:____________________

 

 

 

  

              

DRUG	DOSE	FREQ	CPT CODE

 

SOURCE of DOCUMENTATION for OFF LABEL USE (circle one)

 

DrugDex

 

NCCN               Guidelines          Compendium

 

ACCC

 

OR:

 

Peer reviewed journal:    _______________________________   ______     _________   ________

                                    Journal name                                            Vol            Pages          Year

 

Peer reviewed journal:    _______________________________   ______     _________   ________

                                    Journal name                                            Vol            Pages          Year

 

Peer reviewed journal:    _______________________________   ______     _________   ________

                                    Journal name                                            Vol            Pages          Year

 

                

 

 

 

KCC Preauthorization staff name and phone number:__________________________________

                                                                             

       ___________________________________   

 

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