The US Food and Drug Administration (FDA) has released the fourth and final chapter in a series of guidance documents designed to support patient-focused drug development. In this 2023 update, the FDA reiterated its stance on traditional randomised clinical trial designs, whilst also incorporating technological advances such as computerized adaptive testing (CAT) to carry out clinical research in the most effective way.

The draft guidance, released on March 5, is open for comments until May 7, after which it will replace an earlier 2009 guidance for the industry. The ”Patient-Focused Drug Development: Incorporating Clinical Outcome Assessments Into Endpoints for Regulatory Decision-Making” document outlines how to collect and analyse clinical outcome assessment (COA) data. These recommendations suggest ways to minimise patient burden and ensure that clinical trials are designed efficiently to collect the necessary data in a clinical trial. These guidelines have been produced to help stakeholders collect and submit patient data from patients and caregivers during drug development for regulatory decision making.

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Key takeaways for patient-centred research

In the guidance, the FDA backed the industry standard of doing clinical trials in a randomised controlled setting when possible, as best practice. Furthermore, the agency highlighted a preference that masking is maintained when comparing different treatment regimens. Pertaining to this, the FDA encouraged “early engagement with patient communities and the involvement of patient representatives” when planning a clinical trial, to reduce participant burden.

The guidance also touched upon clinical trial design elements that must be explained, described, or rationalised to the FDA. For example, the agency wants sponsors to “prespecify the method(s) used to interpret COA-based treatment effects and to convey the uncertainty around guides for score interpretation”. Furthermore, The FDA explains that companies must describe their rationale for the chosen trial endpoints to ensure that data is being taken necessarily.

One notable update over the previous guidance is the guideline on the use of CAT in clinical trials. The agency suggests that CAT could be used to collect patient scores in clinical trials. CAT uses an algorithm to select and deliver items from a data bank based on the participant’s previous responses. Participants’ answers are entered into the system to generate an updated estimate of the participant’s profile for an item of interest, for example symptom severity. The CAT algorithm then uses this information to best match the current status on a specific endpoint, providing data for later estimation. According to the guidance, the CAT method allows clinical trials to be more individualised for each patient.

However, the guidance asserted that CAT should only be used when necessary, stating “sponsors should consider whether administering items from an item bank via CAT will be more advantageous than administering a short form consisting of the same set of items to every patient in the trial”.

The contents of the document are currently non-binding, but once finalised will represent the current thinking of the agency.