Keeping Safe and Effective Medications Accessible

The U.S. Food and Drug Administration (FDA) was established and delegated the responsibility of approval and regulation of medications by acts of Congress. As part of the drug approval process, experts at the FDA evaluate evidence from studies to determine the safety and efficacy of the drug for its intended use. FDA will approve the medication if a drug meets these criteria, and if the drug’s benefits outweigh any potential risk [1]. In addition, the FDA conducts ongoing post marketing surveillance after a medication has been approved to identify adverse effects that may not have been detected during pre-approval trials. If there is a signal that an approved drug may be unsafe, the FDA has an established process to evaluate the supporting data and, if indicated, withdraw the drug from the market [2]. FDA’s regulatory process is considered one of the most robust in the world and has prevented unsafe medications from being distributed, such as in the case of the thalidomide disaster in the 1960s.

Recently, there have been efforts to reverse the FDA approval of mifepristone, an agent frequently used for medication abortion. A lawsuit filed by anti-abortion advocates against the FDA and U.S. Health and Human Services in November 2022 challenged the approval of mifepristone, claiming the drug was not safe. On April 7, 2023, a federal district court in Texas, under Judge Matthew Kacsmaryk, issued a ruling in favor of the plaintiffs. In his decision, he stated that the FDA approved mifepristone despite “legitimate safety concerns” [3]. Since the initial ruling, an appeal has been filed and on April 14, 2023, the Supreme Court issued a temporary stay that allows access to the medication pending further appeals [4].

Mifepristone is a progesterone antagonist that was approved by the FDA over two decades ago for medication abortion and for the treatment of Cushing’s syndrome [5]. It is also being increasingly used in conjunction with misoprostol for the medical management of early pregnancy loss, as it has demonstrated superior efficacy to misoprostol alone [6]. While mifepristone was approved with an FDA Risk Evaluation and Mitigation Strategy (REMS), this was intended to prevent misdiagnosis of ectopic pregnancy, not due to inherent toxicity of the drug itself [5, 7]. Mifepristone is well tolerated in both therapeutic use and in high dose studies. Adverse effects are mild, including vaginal bleeding, pelvic cramping, gastrointestinal discomfort, headache, and dizziness. Review of years of post-marketing data further supports the safety of mifepristone [8].

Revoking mifepristone’s approval will limit access to the medication, which can have unintended and harmful consequences for patients [9]. The ongoing debate aside, abortion remains a legal procedure in many states. Limiting access to mifepristone would force women seeking legal medication abortion to use less effective misoprostol only regimens, resulting in complications or an increase in unnecessary surgical procedures. There would be a similar impact on women with early pregnancy loss, who are not seeking termination [6]. Patients should not be exposed to unnecessary risk of harm when there is a safe and effective therapeutic agent available. While there are risks associated with any approved medication, this should be part of an informed discussion between the patient and their physician, in keeping with the principle of patient autonomy.

The case at hand is not just about access to mifepristone. The current legal challenge to mifepristone’s approval undermines the robust FDA approval process and would overturn decades of precedent on the scientific regulation of drugs. In addition, there is a regulatory framework for removal of a drug from the market when there are safety concerns, which post-marketing surveillance has not identified for mifepristone. The determination if a medication is safe should be based on science and remain in the hands of medical experts. This process should not be influenced by political agendas, because in the end, patients may suffer.

Sources of Funding

No funding support was provided for this manuscript.

Declarations

Conflicts of Interest

None.

Disclosures

Dr. Amirshahi has served on advisory panels for the FDA. She is also on the ACMT Board of Directors and an editorial board member for JMT. This work does not represent the views of any of these organizations. Dr. Stolbach is on the Board of Directors for ACMT, and his contribution does not reflect the views of ACMT.