Compendia and Medically Accepted Indications: How Off-Label Uses Reach Paid Claims

Off-label drug compendia false claims can arise when misleading scientific evidence helps a manufacturer obtain government payment for an uncovered use. A compendium entry can turn a journal article into a reimbursement argument. If you work in medical information, publications or market access, you may know what went into that entry and what the payer never saw. The payment question reaches back into the work that made the drug’s use look supported.

What Is Considered Off-Label Promotion?

Off-label promotion means promoting a drug for a use outside the labeling approved by the Food and Drug Administration. That can mean a different condition, patient group or dose.

The Centers for Medicare & Medicaid Services explained the payment connection in its October 2015 fact sheet on off-label pharmaceutical marketing. A manufacturer promotes a use that Medicaid does not cover, and that promotion causes a pharmacy to seek Medicaid payment. The manufacturer can face a false claims case even though the pharmacy submitted the bill.

For someone inside the business, the useful connection is between what the company said about the use, what the prescriber did and what the payer received. A medical-information response, a reimbursement presentation and a pharmacy claim can be parts of the same commercial effort. We examine how those parts fit together in a healthcare fraud case.

What Makes an Off-Label Use Medically Accepted?

For Medicaid covered outpatient drugs, a medically accepted indication is a use approved by the Food and Drug Administration or a use supported by citations included, or approved for inclusion, in specified drug compendia. A compendium is a drug reference work that can describe recommended uses as well as the drug’s characteristics and dosage.

Drug approval and compendium support therefore offer different ways for a use to meet the medical-acceptance definition. For an off-label use, the reference entry can carry substantial financial weight. The evidence behind that entry becomes part of the explanation for why public money should pay for the treatment.

The Medicaid definition points to citations in the specified compendia. A manufacturer seeking that support has an interest in which studies an editor sees, how their results are described and which use the eventual entry endorses. That is why the evidence submitted to a publisher belongs in the same investigation as the reimbursement message sent to a payer.

Amgen and the Route From Article to Reimbursement

Amgen’s Aranesp allegations show why a compendium entry can sit at the center of a pharmaceutical false claims case. The Justice Department’s December 2012 announcement described allegations that Amgen used journal articles insufficient to support the safety and efficacy of off-label uses and improperly obtained compendium listings to make those uses eligible for coverage by federal health care programs.

A whistleblower’s complaint put the alleged scientific work this way:

The same complaint alleged that tainted studies supplied the basis for listing Aranesp for anemia of cancer in Thomson-owned drug compendia, and that the government then used those listings in reimbursement decisions. The alleged sequence ran from scientific article to reference entry to payment. A statement originating with the manufacturer could reach a payer carrying the authority of a separate publication. Taxpayers bear the cost when misleading science turns an uncovered use into a paid claim.

Another complaint alleged that Amgen trained representatives to start a disease discussion designed to provoke an unsolicited question about Aranesp. The representative would then use study data to promote the off-label use. That allegation makes the instructions behind a medical-information exchange as relevant as the exchange itself.

Amgen agreed to a $612 million civil settlement resolving broader allegations concerning the promotion and sale of its drugs, including the compendium allegations. The company allegedly helped create the scientific support that public programs then relied on to pay for its products.

Why Medicaid, Part B and Part D Need Separate Answers

Drug coverage depends on the program, the benefit and the particular use. Medicaid’s covered outpatient drug rules, Medicare Part B’s drug categories and Medicare Part D’s plan benefit each supply a different part of the payment inquiry.

Medicaid and the Covered Outpatient Drug

Medicaid excludes uses that are not medically accepted from its covered outpatient drug benefit. The law names American Hospital Formulary Service Drug Information, United States Pharmacopeia-Drug Information or the publications that replaced it, and the DRUGDEX Information System.

Those names explain why a reference publisher can matter to a manufacturer well beyond the medical library. A supported use can help establish the medical-acceptance basis for payment. A scientific submission can become part of a manufacturer’s effort to obtain payment, well before the pharmacy sends its claim.

State Medicaid programs can also require prior authorization for covered outpatient drugs. That gives the reimbursement file another layer: the clinical information supplied to obtain approval under the state’s requirements. We connect the indication and reference support to the actual coverage decision and claim. The question is what the program was told about the use it was being asked to fund.

Medicare Part B and Anti-Cancer Compendia

Medicare’s compendium regulation for anti-cancer chemotherapy sits within the Part B drug-payment rules. It addresses how the Centers for Medicare & Medicaid Services recognizes compendia used to determine medically accepted off-label uses in an anti-cancer regimen. We connect the listing to the cancer treatment being billed.

Medicare Part D and the Plan’s Coverage Decision

Medicare Part D delivers prescription coverage through private plans. Its covered-drug definition draws on Medicaid’s covered outpatient drug definition, with additional provisions and exclusions of its own.

The plan’s pharmacy and therapeutics committee develops and reviews its formulary, the list of drugs it covers. The committee also reviews clinical prior-authorization criteria, step therapy and quantity limits. For market-access staff, this is where a scientific account of a drug can meet a plan’s conditions for payment.

There is also a benefit boundary. When Part A or Part B payment is available for the drug as prescribed and dispensed or administered to that individual, the drug falls outside the Part D covered-drug definition for that situation. We connect the company’s message to the plan, the use and the claim it was meant to support.

What Must a Medicare Cancer Drug Compendium Show?

The Medicare cancer-drug compendium rule requires a transparent process for evaluating therapies and identifying potential conflicts of interest. It reaches behind the published recommendation into the work that produced it.

The public review information includes the request for a therapy listing, the criteria used to evaluate it, the evidence considered, the people who substantively participated, and meeting minutes and voting records.

The conflict information reaches direct and indirect financial relationships with the drug’s manufacturer or seller. The rule identifies compensation arrangements, grants, contracts, collaborations and ownership interests. These details give an insider’s knowledge of who funded or participated in a review a concrete place in the published process.

For an employee who worked on a submission, the comparison can be revealing: the evidence the company possessed, the evidence the publisher considered and the recommendation a payer later relied on. The entry is the end of an editorial process. The employee may know the beginning.

When Reimbursement Support Reaches the Claim

Payer-facing communications can carry the same disputed science into a coverage request. The Medicare and Medicaid agency’s fact sheet identifies practices including promoting favorable studies while suppressing unfavorable ones, making false representations directly to Medicaid, and advising prescribers how to code claims and document records to obtain payment for uncovered off-label uses.

That puts publications, medical information and reimbursement staff close to the same question from different directions. One person may know which findings were left out of a summary. Another may know how that summary became a coverage argument. A third may have seen the instructions sent to the practice submitting the claim.

Allergan’s $225 million civil settlement over Botox supplies a parallel at the billing end. The Justice Department’s September 2010 announcement described allegations of promotion for noncovered uses, misleading safety and efficacy statements, and instructions to use inappropriate diagnosis codes. The allegations against Amgen concern the science behind the listing. Those against Allergan show how the diagnosis sent to the payer could serve the same effort to get an uncovered use paid.

Medical necessity brings that inquiry back to the patient. A compendium entry describes support for a use. The clinical record and reimbursement submission explain the treatment for the person whose care generated the bill. We examine whether the patient information sent to the payer matched the care described in the underlying records, and who supplied any conflicting account.

What an Insider’s Records Can Show

The strongest account connects the scientific evidence, the reference entry, the payer’s decision and the resulting claims. It also identifies what the people preparing those materials knew. Under the False Claims Act, a company can be responsible for knowingly causing false claims or using false statements that matter to those claims.

The Study and the Submission

Study reports, article drafts and the materials sent to the compendium can show how the evidence changed as it moved toward publication. The Amgen allegations make that comparison concrete. We examine the findings behind a claimed benefit, the description sent to the publisher and the use the resulting entry supported.

The Medicare cancer-compendium rule supplies another useful comparison through its required record of evidence considered and review decisions. Correspondence about the listing can connect an insider’s knowledge to that process. The useful question is who knew about an omitted or misstated finding when the company presented the evidence as support for payment.

The Indication and the Payment Data

A payment record for a drug may need to be connected to clinical information to show why the patient received the drug. The trade article Indication-Specific Drug Pricing–Simple in Theory, Complex in Reality, published in Managed Care in May 2018, describes a practical obstacle to pricing drugs by their use: indication information may sit in an electronic medical record without being captured when the pharmacy dispenses the drug.

That observation matters to a compendium inquiry. A sales or dispensing total can identify the product, while the patient record supplies the reason it was used. We connect those records to the indication the company promoted and the coverage representation the payer received. The missing connection may be in a different system or with a different participant in the transaction.

The Instructions Behind the Submission

Training materials and reimbursement communications can show how staff were told to describe the use, the supporting evidence or the patient’s condition. The account becomes more specific when it connects those instructions to the material a prescriber or payer actually received.

Medical-information, publications, market-access and reimbursement employees may each hold a different part of that account. We build the case with the insider and pharmaceutical fraud whistleblower attorneys who understand how scientific submissions reach government claims. A whistleblower case under the False Claims Act can pay 15% to 30% of what the government recovers.

When You Know How the Entry Was Made

If you worked on a compendium submission or reimbursement message that concealed what the science actually showed, speak with our whistleblower experts about the evidence behind the paid claims. We build the case with you and bring in pharmaceutical fraud whistleblower lawyers who understand off-label coverage and the scientific record. Bring us what you know about how the entry was made, and we will work beside you to build your strongest compendium whistleblower case.