A Broad Challenge to the FDA's Regulations: Botox Manufacturer Sues Asserting First Amendment Protection for Off Label Drug Claims

In a move that could undermine the FDA's premarket approval process for all health care products, the maker of Botox has filed a legal action against the government claiming first amendment protection for the distribution of “truthful, relevant information” to doctors regarding off-label uses of its drugs.

As Natasha Singer of the New York Times reports:

The Food and Drug Administration approves medicines for specific therapeutic indications. Once a drug is approved for a specific use, doctors are then free to use their medical judgment to prescribe the drugs for other unapproved, or off-label, uses. Manufacturers, however, are prohibited from promoting off-label uses to medical providers or advertising such uses directly to the public.

Botox, an injectable drug that can temporarily reduce muscle or gland activity, is approved in the United States to treat crossed eyes, eyelid spasms, excessive sweating, severe neck contortions and wrinkles. But many doctors use the drug for unapproved indications like facial spasms, vocal cord problems and migraine headaches. *** ‘If you could get a drug approved for one narrow use and then market it for everything else, there would be no incentive or motivation for a company to prepare data to ensure that it meets the standard for safety and efficacy,’ said Marc J. Scheineson, a lawyer specializing in food and drug regulation at Alston & Bird in Washington.

It appears that the Botox suit is claiming free speech protections that are similar to those that are currently provided for dietary supplements under the questionable decision of Pearson and Shaw v. Shalala, 1999 U.S. App. LEXIS 464 (Jan. 15, 1999). However, Pearson can be distinguished as dietary supplements are targeted to healthy portions of the population to help maintain or enhance health. Drugs are marketed to unhealthy, vulnerable portions of the population claim to aid in an individual’s struggle to return to normal health. This difference should be outcome determinative in the Botox case.

What the manufacturer of Botox wants to achieve is the ability for drug manufacturers to once again promote drugs while there is scientific uncertainty over their safety and effectiveness. It is taking the first step toward this goal by claiming this free speech in the context of communication to physicians. While the debate starts over this question, it will be prudent to recall the lessons taught by the Thalidomide case which occurred prior to the 1962 Drug Amendments and involved the distribution of drug to thousands of patients by doctors while there was still scientific uncertainty over its safety:

Thalidomide was widely distributed in Germany, Japan and the United Kingdom for sedative purposes and for the treatment of nausea in pregnancy for several years. In 1960, William S. Merrell Company, the manufacturer of the American version of Thalidomide, Kevadon, applied for FDA approval. However, it was clear to Dr. Frances Kelsey, one of the FDA officers examining the application, that the drug had not been adequately tested for safety before distribution. In spite of pressure to approve the drug placed by the manufacturer on both the FDA and Dr. Kelsey individually, Dr. Kelsey insisted that the drug needed additional testing to prove safety before FDA approval could be granted. In 1961, Dr.Kelsey learned of a possible connection between nerve damage in adults and Thalidomide. [These babies were born with phocomelia, which is a Greek word thatcombines the words ‘seal’ and ‘limb’ … where the long bones of the infants’ arms had almost completely failed to grow; their arms were so short that their hands extended almost directly from their shoulders. Their legs were less affected but showed signs of a similar distortion of growth . . . .”]. She requested that Merrell provide studies on the use of its Thalidomide product on pregnant women. In 1962, it was discovered that Thalidomide was causing serious birth defects in children. While the application for Thalidomide was pending for FDA approval,hundreds of severely deformed babies were being born in Germany. [Worldwide, it is estimated that 8,000 severely deformed babies were born because of thalidomide].Without the FDA’s knowledge, Thalidomide had already been provided to 20,000 patients in the United States as part of an 'investigational study.' Slaying the Hydra.