Off-Label Uses

There is a debate going on in Arizona relating to off-label use.

Off-Label Use
However, while marketing a drug for off-label uses is illegal in the US, the practice of prescribing drugs for off-label uses has long been legal (FDA does not regulate the practice of medicine), and is encouraged by FDA.

"Good medical practice and the best interests of the patient require that physicians use legally available drugs, biologics and devices according to their best knowledge and judgment," FDA explains on its website. "If physicians use a product for an indication not in the approved labeling, they have the responsibility to be well informed about the product, to base its use on firm scientific rationale and on sound medical evidence, and to maintain records of the product's use and effects."

In other words, while FDA might not have approved a product, it recognizes that evidence may exist to support its use in patients. In fact, off-label use often leads to the eventual approval of existing products for new indications.

Legislators to Ban Off-Label Use

But Arizona legislators, apparently hoping to limit access to abortion services, passed a law in 2012 (House Bill 2036) that would restrict access to RU-486 in cases where it would be used for an off-label indication.

The key passage of the law is as follows:

"The director [of the Arizona Department of Health Services] shall adopt rules relating to the abortion procedure. At a minimum these rules shall require . . . [t]hat any medication, drug or other substance used to induce an abortion is administered in compliance with the protocol that is authorized by the FDA and that is outlined in the final printing labeling instructions for that medication, drug or substance." (Emphasis Added)

Eventually, Planned Parenthood filed a lawsuit against the state, which resulted in a hearing before the California 9th Circuit Court of Appeals in the case.

Off Label: Allowed or Tolerated?
A central point of debate in the case is whether FDA permits the off-label prescribing of drugs, or simply tolerates it. The court found that the agency has actively permitted the practice since at least 1982, noting that the agency sees it as an essential part of the ecosystem of medical innovation.

"Such ‘unapproved’ or, more precisely, ‘unlabeled’ uses may be appropriate and rational in certain circumstances, and may, in fact, reflect approaches to drug therapy that have been extensively reported in medical literature," FDA wrote in a 1982 drug bulletin.

In June 2014, California's 9th Circuit Court of Appeals ruled in favor of Planned Parenthood's attempt to limit the implementation of the law, finding that Arizona lacked any compelling reasons to ban off-label use of the drug. The limits, the court found, would have unduly burdened patients without offering a legitimate benefit.

"Arizona argues that the law prohibits not just safe evidence-based regimens for medication abortion but also other, dangerous off-label regimens," the court explained in its ruling. "But the record contains no evidence that any such dangerous regimen exists or has ever been used by any abortion provider. Therefore, on the current record, the Arizona law appears wholly 'unnecessary as a matter of women's health.'"

On-label use of the drug, the court noted, actually involved a recommended dose three times higher than that given under an off-label "evidence-based" regimen.

Broader Off-Label Limits?

But the court notably avoided ruling on another central point of the case—one with major regulatory implications.
That question: Does federal law preempt Arizona's attempt to ban off-label prescriptions?

The issue has recently been at the forefront of other cases, such as the prescribing of painkillers. If Arizona is permitted to limit off-label prescribing of medicines, the thinking goes, might other states be permitted to limit off-label prescribing of other drugs as well?

For its part, FDA has called efforts to limit the prescribing of FDA-approved painkillers "extremely troubling," but has thus far declined to weigh in on off-label cases.