On June 13, 2018, the United States Food and Drug Administration (FDA) issued draft guidance, “Patient-Focused Drug Development: Collecting Comprehensive and Representative Input,” a guidance that is the first in a series of four methodological guidance documents that the FDA expects to address “in a stepwise manner how to collect and submit information from patients and caregivers for medical product development and regulatory decision making.”
“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 four-part guidance series is required under the patient-focused drug development component of the 21st Century Cures Act and is meant to provide 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 Prescription Drug User Fee Act (PDUFA VI).
The purpose of Guidance 1 is to present methods for collecting information on the patient experience that is representative of the intended population to inform the development and evaluation of medical products throughout the medical product lifecycle. In addition, the Guidance discusses methods on how to operationalize and standardize the collection, analysis, and dissemination of patient experience data. Guidance 1 also includes a glossary of terms that will be used in one or more of the series of four guidance documents.
The draft guidance includes recommendations for drug makers looking to incorporate patient experience data and other patient-focused information into their drug development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 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The FDA asks that all electronic or written comments on the draft guidance are submitted by September 11, 2018, to ensure that the Agency is able to consider your comment before it begins work on the final version of the guidance.
Next Guidance Documents
The forthcoming 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.
On October 15th and 16th, 2018, the FDA is hosting a two-day public workshop to convene a discussion that will help form the basis for Guidance 2, “Methods to Identify What is Important to Patients,” and Guidance 3, “Selecting, Developing or Modifying Fit-for-Purpose Clinical Outcomes Assessments.”