Decoding FDA’s Statement on DIY Gene Therapies

Cross-posted on Stanford’s Law and the Biosciences Blog

In late November, FDA posted a statement on its website about the “self-administration of gene therapy”—which various media outlets interpreted as a reaction to some companies recently posting videos of consumers “self-experimenting” with gene therapy. One question that arose in some of the reporting—and in an exchange on twitter—is, what is FDA’s authority to regulate do-it-yourself (DIY) gene therapy?

In short, FDA did not say that consumers who engage in self-experimentation with gene therapy, themselves, are violating the law. Instead, the agency said the “sale” of gene therapy products intended for self-administration violates the law. Since 1986, FDA has said that human gene therapy products are biological drugs (see 51 Fed. Reg. 23309 (June 26, 1986)). So, FDA’s stance on gene therapy products intended for self-experimentation—that companies and other entities cannot market them without FDA’s authorization—is the same as for any unapproved new drug. And a company cannot escape FDA’s requirements for drugs by marketing a product directly to consumers for their own experimental use, instead of marketing them for prescription use.

A related question is whether giving away a gene therapy product for free gets a company out from under FDA requirements.   Probably not, in many cases anyway. Although FDA used the word “sale” to describe the impermissible conduct in its November 2017 statement, most of the prohibited in acts in the FDCA use broader language—i.e., it violates the law to introduce into interstate commerce an adulterated, misbranded, or unapproved drug (or cause the introduction into interstate commerce of such a product).   In other words, so long as the product (or one of its components) crosses state, or national, boundaries at some point, that is probably sufficient to give FDA jurisdiction.

One possibly relevant violation of the FDCA—in section 301(k) (21 U.S.C. § 331(k))—does use the word “sale.” Under that provision, it violates the FDCA to adulterate or misbrand a drug while it is “held for sale” after shipment in interstate commerce. In United States v. Geborde, in which the government prosecuted a man who gave homemade GHB to his friends free of charge, the Ninth Circuit concluded that the GHB was not “held for sale” in such wholly noncommercial circumstances. But that rationale may not extend to commercial entities that give away their products in specific instances (after all, give-aways are a fairly common practice for companies, and a later Ninth Circuit case—United States v. Kaplan—suggests that the holding in Geborde is limited). Moreover, it may be unlikely that FDA would need to rely on a violation of section 301(k) to reach companies marketing DIY gene therapy products—those companies are likely shipping their product across state or national lines to consumers.

So I don’t think there is much doubt about FDA’s authority to regulate gene therapy products or kits marketed for “self-experimentation.” But it isn’t at all clear to me that there is much FDA could do about truly DIY gene therapies—where the user manufacturers and self-administers the therapy herself, without purchasing products marketed for human use. And how to handle such truly DIY conduct is an issue that is not unique to gene therapy. There are other product areas, such as fecal microbiota transplantation or transcranial direct current stimulation, that may be “easy” areas for DIY approaches, and recently there has even been interest in DIY manufacturing of traditional pharmaceuticals. This general DIY movement seems like something to keep our eyes on.

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