FDA Finalizes First of Four PFDD Industry Guidances

By Marina Ness, MA

On June 16th, the FDA released the final version of “Patient-Focused Drug Development: Collecting Comprehensive and Representative Input.” This document, referred to as Guidance 1, advises industry on methodologies they can use to collect patient input that will be considered scientifically valid for the drug and device regulatory approval process — including guidance on using input from social media. It is the first finalized version of the set of four planned guidances for making the patient perspective part of FDA regulatory approval. All of the documents consist of nonbinding recommendations.

In the introduction, the Guidance makes it plain that “patient voice” is not just a checkbox that needs to be added to a clinical trial. The FDA defines the patient experience as inclusive of the entire journey.

“The patient experience in a medical product development context incorporates their journey throughout the course of their disease or condition, including patient views, feelings, needs, actions, preferences, and interactions (e.g., clinical trials, home life, social life) with respect to their disease and its treatment.”1

Guidance 1 also makes it clear that the FDA considers patient experience data collected using approved methods as valuable for processes outside of drug approval: “Patient experience data may be collected throughout medical product development, beginning at the launch of a discovery program, or may be independent of any specific medical product development program.” 1 When defining the research objectives and methods, input from non-patients is also noted as helpful: “You should examine previously conducted studies and other relevant research literature and consult subject matter experts (e.g., clinicians, social scientists, patients, advocates, caregivers)….”1

After presenting the broad definition of patient experience data, Guidance 1 discusses the types of methods that may apply to collecting it, including qualitative, quantitative, and mixed methodologies. For example, a mixed-methods approach gathering patient input might use a survey instrument consisting of closed-ended questions along with interviews using open-ended questions.

Section II discusses general considerations for collecting patient experience data, including defining the research objectives, the target population, the study design and setting (including via social media), sampling methods, and sample size. They note that, for some purposes, input can come from subject matter experts, including caregivers and patient support organizations. Section III discusses considerations related to collection and management of the data.

Regarding use of social media as a data source, the Guidance is encouraging.

“Targeted social media searches may be useful during the preliminary stages of a study to complement literature review findings, inform the development of research tools (e.g., qualitative study discussion guides), or as a supplement to traditional research approaches (e.g., literature, one-on-one interviews, focus groups, or expert opinion).”1

While acknowledging that data sourced from social media shares the limitations of other social methodologies, such as self-selection bias and the necessary respect of privacy laws, the Guidance notes their strengths including, “…cost and time saving for researchers, participant convenience and comfort,” as well as “allow[ing] access to hard-to-reach populations.”1

Guidance 1 demonstrates that the FDA supports using social media as part of patient-focused drug development. While the guidance points out the concerns for representativeness and privacy when using social media data, it encourages its use for study design and cites multiple benefits of using mixed methodologies as part of the evidence presented for drug approval.

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1 https://www.fda.gov/regulatory-information/search-fda-guidance-documents/patient-focused-drug-development-collecting-comprehensive-and-representative-input