Archived Policies - Prescription Drugs


FDA-Approved Drugs and Biologicals

Number:RX501.087

Effective Date:06-15-2018

End Date:03-31-2019

Coverage:

*CAREFULLY CHECK STATE REGULATIONS AND/OR THE MEMBER CONTRACT*

Medical policies are a set of written guidelines that support current standards of practice. They are based on current peer-reviewed scientific literature. A requested therapy must be proven effective for the relevant diagnosis or procedure. For drug therapy, the proposed dose, frequency and duration of therapy must be consistent with recommendations in at least one authoritative source. This medical policy is supported by FDA-approved labeling and nationally recognized authoritative references. These references include, but are not limited to: MCG care guidelines, DrugDex (IIb level of evidence or higher), NCCN Guidelines (IIb level of evidence or higher), NCCN Compendia (IIb level of evidence or higher), professional society guidelines, and CMS coverage policy.

NOTE 1: This policy is only applicable to those drugs and biologicals that are processed under the Medical Benefit (non self-administered drugs and biologicals). Refer to the applicable pharmacy benefit plan for self-administered drugs and biologicals approved by the FDA (Food and Drug Administration).

NOTE 2: This policy document will apply only when there is no current, active drug/biological-specific Medical Policy in place. Upon development of a drug/biological-specific policy, the coverage in this policy document will no longer be applicable.

FDA-approved drugs and biologicals are considered medically necessary based on the FDA-labeled indications only when the safety, efficacy and established clinical benefit have been identified. In the event the FDA labeling indicates that clinical benefit has NOT been established, the drug or biological will be considered not medically necessary.

The need for genetic testing, scans or other services that might be included in the FDA label as being part of the criteria as a prerequisite to utilize the drug may be considered medically necessary providing that the clinical benefit of the drug or biological has been established. In the event the FDA labeling indicates that clinical benefit has NOT been established, genetic testing, scans or other related services for the drug or biological will be considered not medically necessary.

Coverage of a drug or biological that meets the coverage criteria shall also include medically necessary services associated with the administration of the drug.

HCSC reserves the right to request clinical documentation pertaining to the requested therapy or treatment in question, including but not limited to progress notes, laboratory tests and/or diagnostic studies. A request may be considered medically necessary based on the inclusion and exclusion criteria utilized in pivotal clinical trials that subsequently lead to the FDA approval of the product.

FDA-approved drugs or biologicals are considered medically necessary for an indication when any of the following apply:

The drug or biological is listed in one of the following standard reference compendia as safe and effective for the prescribed indication:

o Thompson Micromedex Drug Dex Compendium (Drug Dex);

o American Hospital Formulary Service Drug Information (AHFS DI);

o National Comprehensive Cancer Network's Drugs and Biologics Compendium.

OR:

The drug or biological is supported by clinical research that appears in peer-reviewed scientific literature specific for the indication in question. NOTE: Peer-reviewed literature means a published scientific study including a trial that is preferably large, multi-centered and prospective, double blinded and randomized. Peer-reviewed medical literature does not include publications or research studies that are sponsored to a significant extent by a pharmaceutical company or financially interested parties.

Orphan Drugs

Use of an orphan drug may be considered medically necessary when used to treat rare diseases and disorders as defined by the Orphan Drug Act of the U.S. Food and Drug Administration.

In the event the FDA labeling for an orphan drug indicates that clinical benefit has NOT been established, the drug or biological will be considered not medically necessary.

The need for any genetic testing, scans or other services that might be included in the FDA label as being part of the criteria as a prerequisite to utilize the orphan drug may be considered medically necessary providing that the clinical benefit of the drug or biological has been established. In the event the FDA labeling indicates that clinical benefit has NOT been established, any genetic testing, scans or other related services for the orphan drug or biological will be considered not medically necessary.

Coverage of a drug or biological that meets the coverage criteria shall also include medically necessary services associated with the administration of the drug.

NOTE 3: An Orphan Drug is defined in the 1984 amendments of the Orphan Drug Act as "a drug intended to treat a condition affecting fewer than 200,000 persons in the United States or will not recover development cost, plus a reasonable profit, within seven years following FDA approval. The Orphan Drug Act was signed into law on January 4, 1983."

NOTE 4: If there is an HCSC medical policy regarding a specific drug or biological, such medical policy supersedes use of this FDA Approved Drugs and Biologicals policy.

NOTE 5: This policy does not address the use of a drug off-label. Refer to RX503.001 Off-Label Use of Drugs without a Medical Policy for coverage on drugs used off-label and no current Medical Policy is in place.

NOTE 6: This policy does not address compounded drugs. Refer to RX501.063 Compounded Drug Products.

Description:

Food and Drug Administration (FDA)

The FDA is a U.S. (Federal) government agency that regulates the following items:

  • Food (human and animal, other than meat and poultry);
  • Dietary Supplements (human and animal);
  • Drugs (human and animal);
  • Cosmetics;
  • Medical and/or Occupational Devices, Equipment, Supplies (human and animal);
  • Radiation Emitting Products (human and animal);
  • Biologics; and
  • Blood Products.

The FDA is responsible for:

Protecting the public health by assuring that foods (except for meat from livestock, poultry and some egg products which are regulated by the U.S. Department of Agriculture) are safe, wholesome, sanitary and properly labeled; ensuring that human and veterinary drugs, and vaccines and other biological products and medical devices intended for human use are safe and effective;

Protecting the public from electronic product radiation;

Assuring cosmetics and dietary supplements are safe and properly labeled;

Regulating tobacco products; and

Advancing the public health by helping to speed product innovations.

The FDA's responsibilities extend to the 50 United States, the District of Columbia, Puerto Rico, Guam, the Virgin Islands, American Samoa, and other U.S. territories and possessions.

The mandate of the FDA is to regulate, by approving, labeling, and monitoring, the numerous medical products in an expeditious, yet thorough evaluation of the product’s safety and effectiveness in meeting the labeling claims. The process is handled by FDA review teams to recommend approval of a product. Following approval, safety teams determine whether the product is hurting an individual. Under current law, the FDA must approve all drugs and devices before they can be marketed. Although the process may be known as the FDA testing program, the FDA does very little of the testing. The developer of a new drug or device uses its own lab or hires another private company to conduct animal tests on the product for safety before proceeding to clinical trials. Generally these tests are often conducted by a medical school department or consulting firm. When each phase of the testing is completed, the developer submits details of the testing process, evidence of adherence to FDA protocols, and the test results to the FDA.

The FDA requires clinical testing for most products, such as drugs, devices, biologics, and blood products. However, dietary products are not pre-approved for safety and efficacy. Only after the dietary supplement has been marketed, the FDA monitor for unsafe products. Certain foods, such as infant formulas or medical foods are regulated more strictly than other dietary supplements as they are consumed by highly vulnerable individuals.

Neither the FDA nor the federal government regulates the practice of medicine. The FDA’s role is to mandate that products should be labeled with their ingredients, be safe, and be effective. A licensed provider may choose to use an FDA approved product for other indications than those stated on the product label. Off-label use is not illegal. (Refer to Off-Label Use of FDA Approved Drugs policy for coverage criteria.) There is an obligation by providers to report any adverse events, including events resulting in hospitalization or death of a patient, especially those not previously described on the product label.

Authority for the FDA comes from laws from the Legislative branch, and regulations, which are the interpretations of laws, from the Executive branch of the Federal government. The basic law is found in the FD&C Act, and the regulations are found in the Code of Federal regulations.

It is important to recognize that it is never a drug or other product, such as a device, that is approved or not approved, but a claim about the use of the drug or other product. The claim is granted a marketing license, which is, in effect, a product label.

FDA approval process for drugs or biologicals

The drug approval process from a laboratory to the market shelves is usually long with varied routes of exploration. Often a drug is developed to target a specific disease, while others are discovered by accident. Most drugs undergo pre-clinical animal laboratory testing or trials, never advancing to human clinical trials or review by the FDA. If a drug does advance to the FDA, a rigorous evaluation begins by scrutinizing the clinical trial design, the severity of adverse effects, the manufacturing conditions, the labeling instructions, and the drug distribution and regulations. The following stages list the process to drug approval:

Stage One – Drug manufacturer or sponsor applies to the FDA by submitting an Investigational New Drug (IND) Application. The FDA must be shown the results of pre-clinical testing and the proposals for human testing. The FDA determines whether it is safe to move forward with drug testing on humans.

Stage Two – Clinical Trials begin once the IND is reviewed by the FDA and a local institutional review board (IRB). The IRB is responsible for approving the clinical trial protocols, to mandate the participants have given consent and are fully informed of the risks, and to direct researchers take appropriate steps to protect participants from harm.

Stage Three – The formal step is for the drug sponsor to request the FDA’s consideration in approving a new drug for marketing in the U.S., via a New Drug Application (NDA). The NDA includes all the animal and human testing data and analysis of data as well as information about the drug’s behavior in the participant’s body, and how the drug will be manufactured. A review team of physicians, chemists, statisticians, microbiologists, pharmacologists, and other experts evaluates the studies the sponsor submitted showing the drug is safe and effective for its proposed use. The FDA may call on an advisory committee of outside experts to review the NDA and make their recommendations. Traditional approval requires that clinical benefit be shown before approval can be granted. Accelerated approval is given to some new drugs for serious and life-threatening illnesses that lack satisfactory treatments. This allows an NDA to be approved before the required measures of effectiveness are available.

There are a number of reasons the FDA may not approve a product claim, including:

Insufficient evidence to support the claim,

The risks are considered unacceptable, or

The FDA and the sponsor cannot come to an agreement about the scope or wording of the claim.

For example, the majority of oncology drugs that are not approved on the basis of the first initial application are subsequently approved upon resubmission of study data or revision of the claim.

510(k) clearance process

From 1976 going forward, any product that was found to be essentially similar to one that was marketed prior to May 28, 1976 was basically “grandfathered in.” This is known as the 510(k) clearance process, and the manufacturer is only required to demonstrate the new product is essentially similar to a product that was marketed prior to 1976. When a product passes through the 510(k) clearance process, the FDA states that it has “cleared” the product for marketing, not approved it, but this distinction is not always made in everyday practice.

For example, the common over-the-counter (OTC) drug, Aspirin, originated in Germany prior to World War I. After the war, the U.S. and other countries did not officially recognize the patent held by the German manufacturer on Aspirin; therefore, this allowed U.S. manufacturers to make Aspirin under various brand names. Any drug marketed before 1938 received “grandfather” status as the manufacturers did not have to show efficacy. Then again, prior to 1976, any drug that was similar did not require testing and was cleared by the FDA under the 501(k) clearance process and “grandfathered”. Of note, there is not one true “brand” of Aspirin. If any manufacturer had applied to market Aspirin for use with new indications in 1976 or after, it may have been designated as a prescription only drug.

Premarket Approval (PMA) process

In the PMA process, the manufacturer is required to provide the FDA with clinical data regarding the safety and effectiveness of the product. Typically, this data is generated through an investigational device exemption (IDE) trial that has been reviewed by the FDA. The manufacturer presents the data to an FDA advisory committee. The committee votes on a recommendation for further action to the FDA. If the advisory committee recommends for approval, the FDA takes the recommendation under advisement and issues their final determination sometime after the advisory committee meeting. The time frame is unpredictable, ranging from a month to over a year. Frequently, there is publicity when the advisory committee recommends approval, but final FDA approval, if given, occurs later.

Orphan Product Designation (OPD)

An Orphan Drug is defined in the 1984 amendments of the Orphan Drug Act as "a drug intended to treat a condition affecting fewer than 200,000 persons in the United States or will not recover development cost, plus a reasonable profit, within seven years following FDA approval. The Orphan Drug Act was signed into law on January 4, 1983."

Rationale:

While there may be exceptions, most benefit plans or health contracts specifically exclude coverage for the patient’s use of drug(s) when used:

During a clinical trial or study, and/or

For purposes or in a manner other than the service was intended, and/or

For cosmetic or not medically necessary indications or conditions, and/or

In instances considered to be experimental, investigational and/or unproven.

Contract:

Each benefit plan, summary plan description or contract defines which services are covered, which services are excluded, and which services are subject to dollar caps or other limitations, conditions or exclusions. Members and their providers have the responsibility for consulting the member's benefit plan, summary plan description or contract to determine if there are any exclusions or other benefit limitations applicable to this service or supply. If there is a discrepancy between a Medical Policy and a member's benefit plan, summary plan description or contract, the benefit plan, summary plan description or contract will govern.

Coding:

CODING:

Disclaimer for coding information on Medical Policies

Procedure and diagnosis codes on Medical Policy documents are included only as a general reference tool for each policy. They may not be all-inclusive.

The presence or absence of procedure, service, supply, device or diagnosis codes in a Medical Policy document has no relevance for determination of benefit coverage for members or reimbursement for providers. Only the written coverage position in a medical policy should be used for such determinations.

Benefit coverage determinations based on written Medical Policy coverage positions must include review of the member’s benefit contract or Summary Plan Description (SPD) for defined coverage vs. non-coverage, benefit exclusions, and benefit limitations such as dollar or duration caps.

CPT/HCPCS/ICD-9/ICD-10 Codes

The following codes may be applicable to this Medical policy and may not be all inclusive.

CPT Codes

None

HCPCS Codes

C9399, J3490, J3590, J9999

ICD-9 Diagnosis Codes

Refer to the ICD-9-CM manual

ICD-9 Procedure Codes

Refer to the ICD-9-CM manual

ICD-10 Diagnosis Codes

Refer to the ICD-10-CM manual

ICD-10 Procedure Codes

Refer to the ICD-10-CM manual


Medicare Coverage:

The information contained in this section is for informational purposes only. HCSC makes no representation as to the accuracy of this information. It is not to be used for claims adjudication for HCSC Plans.

The Centers for Medicare and Medicaid Services (CMS) does not have a national Medicare coverage position. Coverage may be subject to local carrier discretion.

A national coverage position for Medicare may have been developed since this medical policy document was written. See Medicare's National Coverage at <http://www.cms.hhs.gov>.

References:

1. FDA.gov – Is It Really FDA Approved? U.S. Food and Drug Administration. Available at <http://www. fda.gov> (accessed 2017 February 6).

2. FDA.gov – The FDA’s Drug Review Process: Ensuring Drugs are Safe and Effective. U.S. Food and Drug Administration. Available at <http://www.fda.gov> (accessed 2017 February 6).

3. Novitas Solutions, Inc. Local Coverage Article: Approved DRUGS and Biologicals; Includes Cancer Chemotherapeutic Agents (A53049). Eff 01/01/2017. Available at <http://www.cms.gov> (accessed 2017 February 6).

Policy History:

Date Reason
6/15/2018 Reviewed. No changes.
8/9/2017 New Medical Policy document. Coverage addresses FDA-approved drugs and biologicals when no specific drug/biological policy is available.

Archived Document(s):

Title:Effective Date:End Date:
FDA-Approved Drugs and Biologicals06-15-201803-31-2019
FDA-Approved Drugs and Biologicals08-09-201706-14-2018
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