Patient-Focused Drug Development: Collecting Comprehensive and Representative Input

The U.S. Food and Drug Administration has issued a  methodological guidance (Guidance 1) for industry, FDA staff, and other stakeholders entitled “Patient-Focused Drug Development: Collecting Comprehensive and Representative Input”.

To support patient-focused drug development, this guidance addresses both a statutory requirement under the 21st Century Cures Act of 2016 (hereafter referred to as “Cures Act”) Section 3002 (c) and a commitment made under the Prescription Drug User Fee Act (PDUFA) VI (authorized under the FDA Reauthorization Act of 2017 (FDARA), Title I).

This guidance finalizes the first of a series of four guidance documents that FDA committed to develop to address, in a stepwise manner, how stakeholders can collect and submit information on the patient experience for medical product development and regulatory decision making under 

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 more consistently inform medical product development and regulatory decision making under the Cures Act and Section I.J of the PDUFA VI Reauthorization Performance Goals for “Enhancing the Incorporation of the Patient’s Voice in Drug Development and Decision-Making,” and the first of the series (referred to as “Guidance 1”).

The term “patient experience data” is defined as follows under the Cures Act.

(1) are collected by any persons (including patients, family members and caregivers of patients, patient advocacy organizations, disease research foundations, researchers and drug manufacturers); and (2) are intended to provide information about patients’ experiences with a disease or condition, including (A) the ‘impact (including physical and psychosocial impacts) of such disease or condition or a related therapy or clinical investigation; and (B) patient preferences with respect to treatment of the disease or condition.

The following list of patient experience data would be considered to fit within the Cures Act. 

  • patient registry data;
  • natural history study data;
  • patient focus group or meeting reports;
  • patient survey data;
  • clinical outcome assessment (COA) data collected during clinical trials; and
  • elicited patient preference data.

This Guidance 1 presents a general overview of methods and approaches for collecting patient experience data and discusses sampling methods that could be used when planning a study to collect patient input.

Download FDA guidance in PDF here >>

Last Updated: 2020-06-16

Comments are closed.