USA -First patient-focused drug development guidance released

Final guidance for drugmakers on gathering comprehensive and representative input from patients is now available from the US Food and Drug Administration (FDA).
 
The document is the first in a series of four that will outline patient-focused drug development (PFDD) guidance “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,” said the FDA. (RELATED: Patient focused drug development: FDA issues guidance on gathering patient input, Regulatory Focus 12 June 2018)
 
The 21st Century Cures Act includes requirements for PFDD, and the guidance also meets one of the commitments made by the agency under the most recent Prescription Drug User Fee Act (PDUFA VI).
 
The guidance outlines what constitutes patient experience data and reviews general considerations for patient experience data collection. Some specific topics presented are questions that can be put to stakeholders to define which research questions are of interest, what the study population should be, and what to consider in designing a study.
 
Also, the guidance suggests specific quantitative and qualitative methods that can be used to collect patient experience data, taking into account that study aims and the approach to collecting patient experience data will vary from study to study. Presenting options and guidance for a variety of data collection methods in the guidance “meets a statutory requirement to address representative data collection in the context of both quantitative and qualitative methods,” according to the guidance…