FDA’s first patient-focused drug development guidance now final

Regulatory NewsRegulatory News
| 17 June 2020 | By Kari Oakes 

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.
Data collection and management are also addressed in the guidance, with an acknowledgement that the exact format for submissions may vary depending on what kind of patient experience data are collected and how the data are to be used in developing the product. Those leading studies should plan to include the intended use of patient experience data – “how the data are intended for use in supporting medical product development and regulatory decision-making,” said the guidance – and to submit a study report and protocol from the study along with the patient experience data.
The guidance includes an appendix listing standards and requirements for submission of data, a glossary of terms, and hypothetical case examples. Information can also be found on the PFDD webpage maintained by the Center for Drug Evaluation and Research.
The new guidance on comprehensive and representative patient input, together with others in its series, is meant to facilitate systematic collection of robust input from both patients and caregivers, said the agency.  

The agency said that this first guidance in the series of four answers the questions, “From whom do you get input, and why? How do you collect the information?”
Guidance 2 will answer the questions “What do you ask, and why? How do you ask non-leading questions that are well understood by a wide range of patients and other stakeholders?” Planned content for this guidance includes methods to elicit information from the groups identified by the processes in Guidance 1, including guidance for best practices in qualitative research.
The third guidance will answer the question, “How do you decide what to measure in a clinical trial and select or develop fit-for-purpose COAs [Clinical Outcome Assessments]?” This guidance will give methodologies for refining concepts of interest that are important for patients and will address such issues as deciding what should be measured in the product development program.
The fourth guidance will answer the question “How do you incorporate a given COA tool or set of measures into a defined clinical study endpoint? How would you define a meaningful change in that endpoint?” Questions to be addressed here will include how COAs can be incorporated into regulatory decision-making, including details such as what within-patient changes in score are meaningful, and just how data should be collected, analyzed, interpreted and submitted.


© 2022 Regulatory Affairs Professionals Society.

Tags: FDA, patients, US

Discover more of what matters to you

No taxonomy