In a draft guidance issued Tuesday, the US Food and Drug Administration (FDA) details how drugmakers can gather comprehensive and representative input from patients.
"FDA recognizes the need to engage the wider stakeholder community and provide guidance on approaches to bridge early-stage efforts…to more systematic, methodologically-sound approaches to collect patient input so that it becomes data that can further inform regulatory decision-making," FDA Commissioner Scott Gottlieb said in a statement.
The guidance is the first of a four-part series of guidance documents required under the patient-focused drug development component of the 21st Century Cures Act
and is meant to provide methodological recommendations for how to collect and submit information gathered from patients and caregivers as part of regulatory submissions.
The guidance also fulfills one of FDA's commitments
under the recently reauthorized Prescription Drug User Fee Act
In addition to developing guidance on patient-focused drug development, FDA also committed to creating a repository of patient-focused drug development tools and hosting a public workshop aimed at gathering input from the patient and caregiver community on approaches to enhance patient engagement in clinical trials.
The agency has also hosted more than 20 disease-specific patient-focused drug development meetings as part of its commitments under PDUFA V. FDA says those meetings helped it to gain a "deeper appreciation for the expertise that patients and caregivers can bring to the process and the value of incorporating their voice" in the product development and regulatory decision-making.
The 65-page draft guidance includes recommendations for drugmakers looking to incorporate patient experience data and other patient-focused information into their drug development programs, focusing specifically on who such information should be gathered from, and how that information is collected.
The guidance also provides recommendations for both qualitative and quantitative research methods for analyzing patient experience data, as well as advice on how to operationalize and standardize data collection and data management.
The next guidances in the series will focus on what questions to ask patients and caregivers and how to ask them; how to decide what patient-focused outcomes to measure in a clinical trial; and how to determine an appropriate clinical trial endpoint using a patient-focused clinical outcome assessment.
Patient-Focused Drug Development: Collecting Comprehensive and Representative Input
; Federal Register Notice