Introduction

The FDA’s job is to protect the public health by ensuring the safety, effectiveness, and security of biological products, devices, foods, cosmetics, dietary supplements, and certain products that give out radiation. To accomplish this mission, the FDA evaluates products to ensure that only those that are safe, effective, and secure will be able to reach patients.

Selling a product for an unapproved indication would violate the law. However, once it’s been approved, health care providers can lawfully use and prescribe the product for treatments that are not included in the approved labeling. The FDA recognizes that these off-label uses or treatment regimens may be important therapeutic options and may actually even be the standard of care. However, despite this recognition, the FDA seems to be blurring the boundary of its jurisdiction with the use of Risk Evaluation and Mitigation Strategies (REMS).

W

hat is R

EMS?

The Food and Drug Administration Amendments Act (FDAAA) created REMS and allowed the FDA to use it as a tool to monitor a drug and ensure that its benefits outweigh its risks. REMS has the potential to interfere with the practice of medicine if it prevents physicians from exercising their own independent medical judgment or if it prevents patients from getting the medication that they need.

REMS Levels

REMS may be enacted in multiple ways, depending on the risk profile of the drug in question. The FDA may choose between the use of med-guides, additional studies and data points, timetables to submit results, and other elements to assure safe use (ETASUs). ETASUs might consist of additional training and registration requirements for patients and health care providers. Specifically, ETASUs may require that a product only be prescribed by certain prescribers and only if the patient qualifies for the product for the indication approved by the FDA. To meet these requirements, pharmaceutical companies will often contract with health care providers and hospitals only on the condition that they promise not to use the medication for anything other than the approved indication, with the threat of a discontinued supply of medication if the agreement is violated.

Impact

By limiting the supply of a medication, REMS effectively withholds drugs from prescribers despite the FDA’s admission that there may be appropriate off-label uses of certain drugs. In doing so, the FDA has moved beyond merely approving drugs and is now limiting independent medical judgment. Congress has never authorized the FDA to hinder the prescription of appropriately approved drugs. As a result, the FDA has opened itself up to challenge on the grounds that its use of REMS constitutes an overreach.