USFDA is requesting feedback/comments on draft Guidance from Industry, Food and Drug Administration Staff, and Other Stakeholders

On June 12, 2018, USFDA released the draft guidance on 'Patient-Focused Drug Development: Collecting Comprehensive and Representative Input30' for Industry, Food and Drug Administration Staff, and Other Stakeholders. This guidance is the first of four methodological Patient-Focused Drug Development (PFDD) guidance documents that FDA is developing to address in a stepwise manner, how stakeholders (patients, researchers, medical product developers and others) can collect and submit patient experience data and other relevant information from patients and caregivers for medical product development and regulatory decision making. Focusing on practical approaches and methods, this series will inform stakeholders of FDA's current thinking about methods that could be used as a bridge from important early-stage efforts to gain patients' narrative perspectives on the clinical context (e.g., meetings with patients), to development and use of methodologically-sound data collection tools in clinical trials.

These guidance documents will also address agency expectations regarding what sort of analyses might be conducted as part of this work and what sort of documents might be produced, and when appropriate, submitted to FDA.

The topics and questions that each guidance document will address are described below.

Guidance 1: Whom do you get input from, and why? How do you collect the information?

Guidance 1 will discuss sampling methods that could be used when planning to collect patient input. It will also provide a general overview of the relationship between potential research question(s) and method(s) when deciding from whom to get input (including defining the target population and development of the sampling strategy).

Guidance 2: What do you ask, and why? How do you ask non-leading questions that are well understood by a wide range of patients and others?

Guidance 2 will discuss methods for eliciting information from individuals identified in Guidance 1, gathering information about which aspects of symptoms, impacts of their disease, and other issues important to patients. It will discuss best practices in how to do qualitative research including conducting interviews, development of interview guides, selection of types of survey questions, and considerations for collecting demographics and survey information. It will also discuss survey methods and qualitative research topics to help avoid misleading results such as inadvertently priming patients in ways that can lead to results that poorly represent what is important to patients.

Guidance 3: How do you decide what to measure in a clinical trial and select or develop fit-for purpose clinical outcome assessments (COAs)?

Guidance 3 will address refining the list of important impacts and concepts from patients to develop potential study instruments. Given that not everything identified as important by patients, caregivers, and clinicians can demonstrate change in a specific treatment trial or is measurable, how to select what to measure in a medical product development program to show clinical benefit? How to identify or develop fit-for-purpose COAs to assess outcomes of importance to patients?

Guidance 4: Once you have a COA measurement tool and a way to collect data using it, what is an appropriate clinical trial endpoint?

Guidance 4 will address topics related to COA-related endpoint development and interpretation, including topics related to instrument administration and meaningful within 81 patient score changes.

Note - The draft guidance 1 contains nonbinding recommendations31. The four guidances are part of the FDA's PFDD efforts in accordance with the 21st Century Cures Act and the Food and Drug Administration Reauthorization Act of 2017 Title I. Through the PFDD initiative, started in the Prescription Drug User Fee Act V, the FDA has been addressing the need to better enable patients to provide meaningful inputs to drug and biologic development.

Footnotes

30. https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM610442.pdf

31. https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM610442.pdf

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