When Makena, a drug intended to prevent premature birth, came onto the market in 2011 for $1,500 per dose, it received a lot of backlash. The drug was based on an active ingredient that had been available for years at a much lower cost. Faced with the public outcry, the FDA took an unusual step: Pharmacies could continue making their own copies of the drug through compounding, selling it for a fraction of Makena’s price.
Compounding plays a crucial role by allowing pharmacies to tailor medications to the specific needs of individual patients when commercially available medications are insufficient, for example by blending flavored elixirs for children, removing allergens for sensitive individuals or creating alternative formulations, such as converting of pills in liquids. or topical ointment.
The FDA had granted Makena approval under a policy designed to incentivize companies to conduct the costly studies needed to prove that older, unapproved drugs were safe and effective. Many of these older medications were already in use when the new drug approval process was introduced in the 1960s, so they had never undergone clinical trials to gain FDA approval. By allowing compounding companies to compete with Makena instead of enforcing its exclusivity, the FDA sent a clear message: The agency opened the door to competition by compounding pharmacies as a rebuke to the high price of a brand-name drug .
The episode reflects the current debate over the FDA’s permissive stance on the composition of GLP-1 drugs used for weight loss. Initially, the agency cited shortages of semaglutide and tirzepatide — the active ingredients in the popular medications Wegovy and Zepbound — and allowed pharmacies to compound these drugs under a provision to address drug shortages. In October, the FDA announced that the shortage of tirzepatide injections had been resolved and removed it from the agency’s drug shortage list. The agency then quickly backtracked and allowed compounding to continue, citing the high cost of the branded drug in explaining its decision to the press. By doing so, the FDA risks setting a precedent that, like Makena, could undermine its broader public health obligations.
Pharmacy compounding provides essential medical options for certain patients, but the practice was never intended to create price competition for brand-name drugs. That role is primarily reserved for generic medicines, and for policies that ensure strong competition once legitimate intellectual property protection has lapsed. The rules for modern regulation of compounding are set out in the Drug Quality and Security Act (DQSA), which was signed into law in 2013. The law was in response to the tragedy surrounding the New England Compounding Center, where contaminated compound drugs caused an outbreak of meningitis. , which killed more than 100 patients and sickened 800. Implementing the provisions of DQSA was one of the most challenging political tasks I faced as FDA Commissioner.
The FDA has long harbored safety concerns when drugs are prepared in bulk, especially when distributed through the mail or through doctors’ offices, where they can sit on a shelf for long periods of time. Under certain conditions, the law allows compounding pharmacies to qualify as “outsourcing facilities” by agreeing to meet FDA standards for good manufacturing practices to ensure the quality and sterility of a drug product. In return, these pharmacies can prepare medications for storage in doctors’ offices or later shipment to patients. Because the medications from outsourcing facilities are produced under careful supervision to ensure their sterility, the risk of contamination while in storage is generally lower.
In many cases, however, GLP-1 medicines are prepared in pharmacies that do not have to meet these good production requirements. These are often smaller pharmacies that are only allowed to prepare medicines for individual patients, with the expectation that the medicines will be used shortly after production. Yet they prepare and ship large quantities of drugs across the country. Even in cases where outsourcing facilities produce these drugs, there are pressing questions about how pharmacies can obtain large quantities of the same active ingredients used in the brand-name drugs, raising concerns about the quality of the compounded versions. Doctors often do not have full knowledge of what these products contain, and patients may misunderstand what they are receiving because they often assume that compounded medications are the same as brand-name alternatives.
The FDA’s efforts to implement the provisions of the DQSA have always been politically charged. We faced continued opposition from some members of Congress, who were concerned about the economic impact of the bill on small pharmacies. Many of these pharmacies play a critical role in rural communities, and for some, compounding bulk medications for use in doctors’ offices or distribution to patients is an important source of income. We took steps to reduce the financial burden of the law, but implementing the core elements of the DQSA remained a high priority to ensure the safety of patients dependent on compounded medicines.
The DQSA was not in effect during the Makena action, and the new law never intended for the FDA to weigh drug prices when authorizing compounded drugs. High-quality manufactured medications should not become products that only certain patients can afford, while others must rely on compounded medications that are not made to the same standards.
If the FDA demonstrates that its regulations can be selectively enforced to align with drug pricing goals, it will likely face even more pressure to relax its oversight of other compounded drugs. The precedent it set with Makena, and possibly now tirzepatide, could make it difficult for the agency to maintain control over this crucial policy in the future.
Scott Gottlieb is a physician, senior fellow at the American Enterprise Institute, and served as commissioner of the Food and Drug Administration from 2017 to 2019. He serves on the Pfizer board of directors and is a partner at New Enterprise Associates.