The shift of a medication from prescription-only to over the counter (OTC) is a major milestone, often signaling broader public access, improved health outcomes, and commercial opportunity. But the U.S. Food and Drug Administration (FDA) does not take this decision lightly. For a drug to be marketed OTC, it must meet strict regulatory criteria to ensure consumers can safely and effectively use the medication without professional supervision.
To begin with, the primary requirement is that the drug must be safe and effective for use without a healthcare provider’s oversight. This means that users must be able to self-diagnose, self-treat, and self-manage the condition the drug addresses. The FDA evaluates whether the layperson can correctly interpret labeling, dosing instructions, and warnings (FDA, 2022). If the condition is too complex or the risks too high without a clinician’s input, the drug is unlikely to qualify for OTC status.
Second, the labeling must be clear, comprehensive, and consumer friendly. The Drug Facts label, mandated by the FDA, plays a central role. It must contain information about active ingredients, uses, directions, warnings, and inactive ingredients in a standard format. The FDA frequently requires consumer studies, such as label comprehension and actual-use trials, to determine if average users understand and follow the instructions correctly (Gandhi et al., 2020). This is critical because a misunderstanding could lead to misuse, overdose, or harmful drug interactions.
The switch from prescription to OTC can happen in two ways: through the OTC Monograph process or via a New Drug Application (NDA). Most new switches use the NDA pathway, where sponsors submit data to support OTC use, including results from safety studies, efficacy trials, and consumer behavior research. A notable example is the switch of loratadine (Claritin) from prescription to OTC, which followed extensive trials demonstrating both efficacy and safe self-use (CDER, 2019).
The FDA also considers the risk of misuse and abuse. For example, certain cough medications containing dextromethorphan remain restricted due to potential abuse. The balance of benefits versus potential harm is a cornerstone of OTC approval. If the drug poses significant risks even when used as directed, or if incorrect use could result in serious health consequences, it likely won’t make the switch (Mauri et al., 2021).
Finally, the FDA’s recent efforts to expand the OTC category through innovations like Additional Conditions for Nonprescription Use (ACNU) signal a more flexible approach. These allow non-traditional methods, like apps or kiosks, to guide consumers through appropriate use criteria, especially useful for more complex drugs (FDA, 2022).
In summary, whether seeking OTC approval or dual-use recognition, the pathway to expanded access for any therapeutic product is grounded in science, safety, and regulatory integrity. Securing over-the-counter status requires sponsors to demonstrate that their product can be used safely and effectively without a healthcare provider’s oversight—supported by strong clinical evidence and consumer-use data. Similarly, gaining recognition for a secondary indication, like the potential dementia-prevention benefit of the shingles vaccine, demands a robust body of research, biological plausibility, and regulatory diligence.
As innovation accelerates and health literacy improves, these pathways offer tremendous potential to broaden access and maximize public health impact. EMMA International stands ready to help you navigate these complex regulatory journeys. Contact us at (248) 987-4497 or info@emmainternational.com to learn how we can support your strategy from bench to bedside.
References:
CDER (2019) Guidance for Industry: Labeling OTC Human Drug Products. Center for Drug Evaluation and Research, U.S. FDA.
FDA (2022) Final Rule: Nonprescription Drug Product with an Additional Condition for Nonprescription Use. U.S. Food and Drug Administration.
Gandhi, M., Kesselheim, A.S. and Avorn, J. (2020) ‘From Prescription to Over-the-Counter: Reformulating the Regulatory Framework’, New England Journal of Medicine, 382(12), pp. 1093–1097.
Mauri, M.C., Paletta, S., and Di Pace, C. (2021) ‘OTC Drug Use and Misuse: A Review of the Literature’, Frontiers in Psychiatry, 12, 635217.
