“Off Label” Uses of Medical Products and Services

“Off Label” Uses of Medical Products and Services

By Vernon Sumwalt, Esq.

Under most workers’ compensation programs, coverage for medical treatment differs from the coverage afforded by other plans. For example, state workers’ compensation programs often cover “reasonable and necessary” medical expenses related to an injury, while group health policies limit the quantity of appointments, the network of available providers, or even the total amount payable. Most workers’ compensation programs do not contain the same exclusions from coverage as those in contractual or governmental plans of coverage.

These other plans often exclude coverage for “experimental” treatment. The defense that proposed treatment is “experimental” and not covered through workers’ compensation has increased in popularity in all jurisdictions over the past decade. The trend corresponds to, among other things, the new clinical treatments for pain and new surgical procedures that the United States Food and Drug Administration (FDA) has yet to approve, even though the medical community accepts their use. E.g., State ex rel. Bax Global, Inc. v. Indus. Comm., 2007 Ohio 695, 2007 Ohio App. LEXIS 627 (2007) (Charite lumbar disk replacement surgery); Tarraferro v. Wyoming Med. Comm., 2005 Wy. 155 (2005) (prescription drug Marinol or “medical marijuana”); State ex rel. Sugardale Foods, Inc. v. Indus. Comm., 90 Ohio.St.3d 383, 2000 Ohio 185, 738 N.E.2d 1238 (2000) (Steffee plating surgery); cf., e.g., Magee v. Thompson Creek Mining Co., 142 Idaho 761, 133 P.3d 1226, 2006 Ida. LEXIS 33 (2006) (declining to address reasonableness of Colchicine injections and Prolo therapy on procedural grounds).

Indeed, the vague adjective “experimental” suggests in its most general sense that the treatment has not yet been validated as effective against a particular diagnosis. But the legal argument used to substantiate this allegation is that “experimental” treatment—that is, “off label” use of medical products—is not reasonable or necessary given the lack of FDA approval. “Off label” use refers to “the prescription of a medication in a manner different from that approved by the FDA.” R. Stafford, “Regulating Off-Label Drug Use—Rethinking the Role of the FDA,” 358 N. Eng. J. Med. 1427 (Apr. 3, 2008), available at www.nejm.org/doi/full/10.1056/NEJMp0802107. To evaluate this argument, therefore, one must know what exactly the FDA’s approval means.

What’s in a Label? Labels for a medical product never contain a full list of the contexts for which medical professionals may prescribe the product. The FDA’s approval concerns only how manufacturers may market a medical product, not its potential uses in clinical practice. For example, the FDA’s Center for Drug Evaluation and Research “provides doctors and patients the information they need to use medicines wisely.” www.fda.gov/Drugs/DevelopmentApprovalProcess/default.htm. Marketing literature thus describes the possible risks and benefits in order to come to an informed decision on whether to use a drug in a certain way. Once the FDA issues approval, a manufacturer can market or “label” its drug or device for use within specific medical contexts, but the approval does not limit health care providers from prescribing the product outside of the approved contexts. See 21 U.S.C. § 396. This is “off label” use. 

The FDA does not play doctor. The distinction between “marketing approval” and “use approval” draws the boundary between “on label” and “off label” uses. The Federal Food, Drug, and Cosmetic Act does not prohibit “off label” uses of medical products, even if that use is outside of the contexts for which the FDA has approved them to be marketed:

"Nothing in this Act [21 U.S.C. § 301 et seq.] shall be construed to limit or interfere with the authority of a health care practitioner to prescribe or administer any legally marketed device to a patient for any condition or disease within a legitimate health care practitioner-patient relationship."

21 U.S.C. § 396. The FDA’s Physician Desk Reference (PDR) likewise explains that there is no prohibition against “off label” use of pharmaceuticals:

"The FDA has always recognized that the [Food, Drug, and Cosmetics] Act does not, however, limit the manner in which a physician may use an approved drug. Once a product has been approved for marketing, a physician may choose to prescribe it for uses or in treatment regimens or patient populations that are not included in approved labeling. The FDA also observes that accepted medical practice often included [sic] drug use that is not reflected in approved drug labeling."

Morlino v. Medical Ctr. of Ocean County, 152 N.J. 563, 581, 706 A.2d 721, 733 (1998) (quoting PDR, Forward (51st ed. 1997)) (also holding that the PDR, which is approved by the FDA, does not establish a standard of care because “[s]uch an approach... would be inconsistent with the FDA’s position that physicians are not bound by PDR recommendations”).  Consistent with these authorities, the United States Supreme Court has observed that

"...'off-label' usage of medical devices (use of a device for some other purpose than that for which it has been approved by the FDA) is an accepted and necessary corollary of the FDA’s mission to regulate in th[e] area [of medical marketing] without directly interfering with the practice of medicine.... Thus, the FDA is charged with the substantial task of regulating the marketing and distribution of medical devices without intruding upon decisions statutorily committed to the discretion of health care professionals."

Buckman Co. v. Plaintiff’s Legal Committee, 531 U.S. 341, 350 (2001) (quoting Beck & Azari, “FDA, Off-Label Use, and Informed Consent: Debunking Myths & Misconceptions,” 53 Food & Drug L.J. 76-77 (1998), and “noting that courts, several States, and the ‘FDA itself recognize the value and propriety of off-label use.’”). These authorities represent the majority view on “off label” use. 

What is so “experimental”? So if FDA approval does not prohibit “off label” use, then why all the litigation? The answer is pretty straightforward. Just because medical professionals can prescribe something does not always imply that they should prescribe it. The efficacy of “off label” use is not always certain or appreciated, and in many instances it is years before academic literature and journal research catch up to generally accepted clinical practice. “Off label” use is common and accepted within the medical community, and it prevails in some fields of medicine more so than others. The bottom line is that there are few guarantees in medicine, like anything else in life, but if doctors find something that works, then they use it and others eventually will, too. The lag in research and literature is only natural; to insist on the contrary would be to assume omniscience about pharmaceuticals, including their risks, by the academics before their use becomes popular clinically, and everyone knows this is not the case. But see R. Stafford, 358 N. Engl. J. Med. 1427 (discussing both the advantages and shortcomings of “off label” use in light of the FDA’s diminishing role in regulating medical products). Real life and discoveries in clinical practice demonstrate the risks and benefits of many medical products, and that the benefit of many drugs are only discovered well after they are introduced into the medical community, even for other purposes. This trial-and-error is most likely why courts facing this issue have resorted to a case-by-case analysis using local standards of medical necessity and reasonableness instead of disqualifying all “off label” from coverage in workers’ compensation contexts.

Risky business. The problem with litigating “off label” use lies in the fact that all litigation is uncertain and stressful, and these factors often lead parties to settle. Worse yet, some attorneys recommend settlement without disclosing the differences in coverage between workers’ compensation and other plans. All settlements shift risks, for better or for worse, and these risks include future medical treatment and its cost. When the more flexible coverage afforded by workers’ compensation programs stops, there is no guarantee that a new plan will supply the same coverage for “off label” uses. The transition thus runs the risk of legal malpractice or just giving bad advice when a client assumes that future coverage will be like it has always been—or could have been—under workers’ compensation.

Medicare is the perfect example, since the Centers for Medicare & Medicaid Services (CMS) has not endorsed the medical community’s comfort with off-label uses. This is reflected in limited or no coverage for uses beyond the scope of FDA-approved contexts. Only recently has CMS entertained the coverage of off-label uses of some pharmaceuticals, mainly those used to treat cancer, but only because oncologists’ “off label” use of some drugs is the dominant standard in that field of practice. See www.medicareadvocacy.org/2010/09/cma-report-medicare-coverage-for-off-label-drug-use/. CMS has not been so willing to extend coverage in other medical contexts so quickly. If the availability of “off label” uses is limited after a final settlement of the workers’ compensation claim, and parties should account for the shift in costs through the settlement. Medicare set-aside proposals (MSAs) will itemize Medicare-covered treatment in a claim, particularly under Part D, but there are many items of medical compensation that Medicare does not cover. Attorneys can get experts to estimate the anticipated costs outside of the MSA for non-covered expenses. The MSA cannot sponsor these expenses, since MSAs cover only future medical items and services otherwise covered by Medicare.

Of course, all of this assumes that our Congress does not emasculate Medicare in a decade or so, but that topic is for another day and another forum. Nevertheless, most contractual plans of health coverage have similar exclusions against “off label” use. Even if Medicare disappears, the issue will linger.

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