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  1. Development & Approval Process | Drugs

FDA Patient-Focused Drug Development Guidance Series for Enhancing the Incorporation of the Patient’s Voice in Medical Product Development and Regulatory Decision Making

Patient-Focused Drug Development

FDA is developing a series of four methodological patient-focused drug development (PFDD) guidance documents to address, in a stepwise manner, how stakeholders can collect and submit patient experience data and other relevant information from patients and caregivers for medical product development and regulatory decision making. This series of guidance documents is intended to facilitate the advancement and use of systematic approaches to collect and use robust and meaningful patient and caregiver input that can better inform medical product development and regulatory decision making.

These guidances are part of FDA’s PFDD efforts in accordance with the 21st Century Cures Act and The Food and Drug Administration Reauthorization Act of 2017 Title I.

This webpage contains information and documents related to FDA’s development of the methodological PFDD guidances, including public workshops, draft guidances, and hypothetical scenarios, which are all intended to serve as a basis for dialogue. To supplement the PFDD Guidance Series, FDA issued two technical specifications guidance documents, Submitting Clinical Trial Datasets and Documentation for Clinical Outcome Assessments Using Item Response Theory and Submitting Patient-Reported Outcome Data in Cancer Clinical Trials.

Guidance 1 discusses sampling methods that could be used when planning to collect patient input. It also provides 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 discusses methods for eliciting information from individuals identified in Guidance 1, gathering information about what aspects of symptoms, impacts of their disease, and other issues are important to patients. It discusses 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 also discusses 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 discusses approaches to selecting, modifying, developing, and evaluating clinical outcome assessments to measure outcomes of importance to patients in clinical trials.

Guidance 4 addresses methodologies, standards, and technologies that may be used for the collection, capture, storage, and analysis of clinical outcome assessment (COA) data. The guidance also addresses methods to better incorporate COAs into endpoints that are considered significantly robust for regulatory decision-making. This includes methods to define meaningful change in a COA-based endpoint and interpretation of results. The guidance includes information on the format and content required for regulatory submissions incorporating patient experience, in particular COA data.

To contact FDA’s CDER Patient-Focused Drug Development Program Staff, please email patientfocused@fda.hhs.gov.

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