FDA issues last guidance in patient-focused drug development series

The US Food and Drug Administration (FDA) has published its fourth and final guidance in a series of patient-focused drug development (PFDD) guidances meant to help sponsors collect and incorporate patient experience information that can factor into regulatory decision making. The latest guidance focuses on how clinical outcomes assessments (COA) can be used as endpoints to support a product.

In line with recent legislation, FDA has published several guidances covering various aspects of patient-focused drug development, from collecting patient input to choosing and developing “fit-for-purpose” COAs. The latest guidance describes how stakeholders, including healthcare providers, researchers and sponsors can select and construct COA-based endpoints for use in clinical trials, as well as recommendations for evaluating the meaningfulness of results of COA-based endpoints and other study design considerations.

FDA said sponsors should talk to its staff early about the type of patient experience data they plan on collecting related to the burden of disease and the benefits, burdens and harms of treatment. The agency also said sponsors should talk to stakeholders, including patients and subject matter experts when designing their trials to understand the burden of the disease and the benefits and risks of a treatment.

“This guidance focuses on COA issues associated with clinical trial (study) endpoints, design, conduct, and analysis and will be of most relevance for those designing and conducting trials using COAs as well as analyzing and interpreting the trial data,” said FDA.

“Some COAs provide direct insight on how patients feel or function (e.g., a patient-reported outcome (PRO) instrument measuring pain intensity),” the agency added. “Other COAs, however, may provide more indirect information to evaluate clinical benefit (e.g., clinician-reported outcome (ClinRO) instruments measuring extent or activity of disease such as psoriasis area and severity). In these situations, it is important to understand how the COA-based endpoint corresponds to changes relevant to patients (e.g., the type and extent of change that is meaningful to patients).”

The guidance further goes into detail about how sponsors can align the COA-based endpoints they pick with their study design, other endpoints, and analyses with the clinical study objective to improve study planning and interpretation of the analyses. It also discusses methods sponsors can use to interpret the treatment effects on COA-based endpoints. Furthermore, the guidance includes a list of considerations when sponsors are developing COA-based endpoints and how to submit that information properly in their product application.

The first in the series of FDA’s PFDD guidances was finalized in June 2020 and discusses what constitutes patient experience data and general considerations for patient experience data collection. The second guidance in the series was finalized in February 2022 and details how sponsors can collect and submit PFDD data from patients and caregivers for their product application. (RELATED: FDA’s first patient-focused drug development guidance now final, Regulatory Focus 17 June 2020; PFDD: FDA finalizes guidance on identifying what’s important to patients, Regulatory Focus 28 February 2022)

FDA also published draft guidance last year on selecting, developing or modifying COA in clinical trials. Since then, stakeholders have asked the agency to clarify how new terminology introduced in the guidance would fit in with its overall PFDD program. (RELATED: Stakeholders want more clarity on concepts introduced in third PFDD draft guidance, Regulatory Focus 3 October 2022)

Draft Guidance

FDA issues last guidance in patient-focused drug development series

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