Earlier this week, the D.C. Circuit upheld the FDA’s authority to regulate stem cells (for a good news report see here). The company in question, Regenerative Sciences, had received a warning letter from FDA, which the company challenged claiming that its use of stem cells as therapy was not prohibited by existing federal law and that the FDA lacked authority to regulate it. They lost before the district court and appealed to the D.C. Circuit.
In a unanimous decision (by judge Griffith for himself, Judge Srinivasan and Edwards) the D.C. Circuit affirmed this decision. Here are some key passages:
Appellants’ principal argument is that the Mixture is not subject to regulation under the FDCA or PHSA because it is neither a drug nor a biological product but is, rather, a medical procedure. The text of those statutes forecloses this argument.
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Alternatively, appellants contend that the Mixture is exempt from the FDCA’s manufacturing and labeling requirements because it is a compounded drug. See 21 U.S.C. § 353a(a). A compounded drug must be produced using certain types of “bulk drug substances,” one of which is “bulk drug substances . . . that . . . are components of drugs approved by the [government].” Id. § 353a(b)(1)(A). Appellants assert that the Mixture meets this definition because cultured MSCs are a component of the FDA-approved drug Carticel. But even if that were the case—and the affidavits appellants cite only suggest that it might be—it would not be enough to bring the Mixture within § 353a. To qualify as a “bulk drug substance,” an item must be
“represented for use in a drug,” 21 C.F.R. § 207.3(a)(4), and appellants point to no evidence in the record even suggesting that MSCs are held out for use in Carticel, or any other drug for that matter. Appellants therefore fail to establish that the Mixture is exempt from the FDCA’s manufacturing and labeling requirements, and we proceed to consider whether the Mixture violated them.
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Appellants nevertheless argue that it is inappropriate to hold them liable for not providing adequate directions because they produced the Mixture only for their own use. This argument, however, misunderstands how the FDCA’s labeling scheme applies to prescription drugs.
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Here, there is no doubt that the Mixture qualifies as a prescription drug. Before the Mixture can be injected into a patient, a physician must review the cultured MSCs to ensure that there are no visible signs of bacterial contamination or genetic mutation. Then, if the MSCs are safe, appellants inject the Mixture using sophisticated imaging devices to ensure that it reaches the right spot on a patient’s bone or tissue so that it has the intended therapeutic effect. Because the Mixture can be safely administered only under a physician’s supervision, the question for us is whether the Mixture qualifies for either § 352(f) exemption. The answer is clear. Both exemptions require that the label bear the symbol “Rx only,” see 21 U.S.C. § 353(b)(4)(A); 21 C.F.R. § 201.l00(b)(1), and it is undisputed that the Mixture’s label does not. Because its label fails to provide the minimum information necessary to qualify for either exemption from § 352(f), the Mixture is misbranded.
The end result is that unless overturned by the en banc D.C. Circuit or the Supreme Court the company will be permanently enjoined “from committing future violations of the FDCA’s manufacturing and labeling provisions.” This is a big win for FDA and should also make those who think these therapies are problematic, 21st Century snake-oil, happy since it will give FDA more levers to get compliance from the industry. As I write about in my forthcoming book on medical tourism, though, stricter FDA regulation of stem cell therapies may increase the outflow of patients to China and other places for these therapies. The audacity of hope for patients and especially parents seeking to help their children may outstrip the regulatory powers of even the FDA…