The Lynx Group

Health Plans Need Policies for Dealing with Off-Label Drug Use

October 2012, Vol 3, No 7

Houston, TX—To eliminate coverage inconsistencies and enhance relationships with providers, health plans should have specific policies for dealing with off-label use of oncologic drugs, said Kristen M. Reimers, RPh, Specialty Pharmacy Director and Clinical Operations Manager for Excellus Health Plans.

At the Second Annual Association for Value-Based Cancer Care Con­ference, Ms Reimers described the rationale for and the process of development and benefits of the off-label drug policy that she helped to develop at Excellus.

More than 60% of cancer therapies are the result of off-label prescribing, and drugs that are used off label often become part of the standard of care for some tumor types, she noted.

“Many off-label uses are effective, well documented in peer-reviewed literature, and widely used,” Ms Reimers said, but coverage determinations are inconsistent.

As noted by the National Com­prehensive Cancer Network on its website, “The introduction and utilization of many innovative drugs and biologics in oncology practice, combined with their significant expense, has focused the attention of payers and other involved constituencies on processes and programs that facilitate the appropriate, effective, and efficient use of such agents,” she explained.

Issues with Off-Label Drug Use

Ms Reimers cited a number of issues of concern with off-label drug use:

  • High risk for variation in treatment of relatively rare and complex cancers
  • Inconsistent application of policy (if there even is one)
  • Increased medical costs that result from inappropriate prescribing
  • Concerns about efficacy, safety, and ethics
  • Frustration among providers
  • Mandated coverage (by 39 states) when listed in compendia.

“These issues have led to prescriber frustrations and inconsistency in our determinations. In the absence of clear criteria, emotional responses often guide determinations,” Ms Reimers pointed out. “We needed to do something differently, which is why we developed our off-label drug use policy.”

Off-Label Use Policy.
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Developing the Policy

Ms Reimers and her colleagues developed a policy that emphasized evidence-based decisions. “In making determinations, we wanted to weigh the evidence and make clear and concise determinations on efficacy, safety, and value,” she said (Figure).

The policy acknowledges the New York state mandate that requires health plans to cover any US Food and Drug Administration–approved drug that is acceptable for the treatment of the malignancy in question by a recognized or authoritative compendium, or by a review article or editorial comment published in a major peer-reviewed professional journal.

In addition, the reviewers look at the strength of the recommendations. Class I and IIa recommendations are always acceptable, whereas class IIb recommendations are acceptable in some, but not all, cases. These situations require a full literature search, with an emphasis on studies that are appropriately sized, that conclude that the drug used off label is generally safe in relation to the severity of the disease being treated and other existing treatment options, and that the study outcomes represent clinically meaningful outcomes experienced by patients, Ms Reimers stated.

“If the request is not supported by compendia or articles (abstracts and poster presentations are not acceptable), as listed above, then the request is not considered medically appropriate and will be denied as experimental or investigational,” she said.

Off-Label Use Policy Process

“We also needed to change our process for reviewing these cases,” Ms Reimers noted. “Our current process was problematic. Our reviewers did not have time to devote to these unusual cases. We needed a dedicated team to review off-label requests."

A collaborative team of reviewers was developed for policy implementation, including specially trained nurses, pharmacists, and medical directors. Under the new process, nurses receive the off-label requests, then they contact the requesting physician’s office for more information in the form of progress notes, previous treatments and outcomes, laboratory and scan results, and so forth.

“The reviewers are looking for the doctor’s thought processes about this recommendation, and any literature to support it,” Ms Reimers commented.

In the next step, pharmacists initiate a systematic review process that weighs and documents the evidence; this report is then sent to the medical director. After the medical director’s review, the case is discussed at a joint meeting of all team members, who consider the merits of the case “coming from 3 different perspectives” and make a coverage determination.

These individual cases are discussed again during subsequent team conferences, where they are used as training experiences. “And we monitor our results, track appeals, and change policies, if we need to, as standards of care change,” she said.

“We developed an off-label drug use policy that uses a stepwise approach to tackling cases. Our goal was to improve consistency and we were able to achieve that,” Ms Reimers reported.

Implementation of the new process has allowed Excellus to keep its individual drug policies up to date, she added. “Because we are combing through the literature more frequently, we are always proactive, tracking the pipeline, looking at what is happening at national meetings, and maintaining a dialogue with community experts….We wanted to eliminate emotion in the decision-making process, and we were able to do so.”

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