What is an Actual Use Study?

Whereas randomized clinical trials (RCT) can establish the efficacy and safety of a drug at a specific dose, in a specific population for a specific indication, a key objective in many OTC switch programs is to show whether potential consumers can correctly and safely use a new OTC product in a naturalistic setting without medical supervision. This is a question that can only be answered by an OTC actual use study (AUS).

Design principles for naturalistic, observational AUSs are quite different from those for traditional RCTs (for additional information, see Bradford, et al., SelfCare 2010;1(4):117-123, available from URL: https://selfcarejournal.com/wp-content/uploads/2015/09/Bradford-1.4.117-123.pdf). For example:

• AUSs should recruit and enroll participants in settings where they really go to find and purchase their non-prescription medicines. This usually means using OTC-like recruitment advertising and naturalistic study sites such as community pharmacies, or even e-commerce for some decentralized studies. PEGUS has a network of more than 50 retail pharmacies throughout the country. A subset of those sites is typically selected to meet the needs of a given AUS.
• AUSs are all-comers, meaning participants should be allowed to make the choice about participating in the study in much the same way they would make the decision about purchasing the product in a non-research setting. Exclusion criteria are generally limited to a minimum set required for ethically conducting the study.
• AUSs are generally uncontrolled and non-randomized, meaning they consist of a single treatment – the proposed OTC medication.
• AUSs are open label (non-blinded), meaning participants know what the investigational product is. Generally, the package and labeling should be as close to the intended future OTC product materials as possible.
• Finally, because AUSs are intended to carefully observe and measure real-world behaviors, data collection tools and procedures should minimize obtrusiveness and reactivity (the tendency for the evaluation to affect behaviors of interest) to the extent possible. Simultaneously, follow-up procedures are tailored to ensure collected data will be as complete and reliable as possible.

These principles may seem obvious in the abstract, but the real challenge is how to apply them to the candidate OTC product. We are happy to visit with you and discuss how the science of AUSs can be adapted to the context of your specific program.

The Actual Use Study can be thought of as the culmination of the OTC switch program. It is the trial to demonstrate that consumers can understand the DFL, apply it to their own situations, use the medication safely, and discontinue use when appropriate, all without the help of a physician. The measures of interest vary depending on the indication, the dosing, and areas of specific concern by the FDA. Sample size is largely dependent on factors related to the measures of interest. The recruitment cost and time to enroll are highly variable based on sample size and characteristics of the population(s) to be recruited. Total length of an AUS from initiation to completion varies widely based on the speed of enrollment and the length of the medication use period.

Actual Use Studies for technology mediated “ACNU” products may involve additional procedures that are specific to the technology that will be used for encouraging correct product use. See our “Additional Conditions for Nonprescription Use” page for more information.

If you have questions about how PEGUS can assist with actual use research for your product, please click here or call us at 801-990-6126.