The FDA is creating 4 methodological Patient-Focused Drug Development (PFDD) guidance documents to describe how to collect and submit patient experience data in clinical research. The guidance series is responsive to the mandates of the 21st Century Cures Act and other efforts to include patient experience data in support of regulatory decision-making and medical product development. Patient experience data include information about patients’ symptoms, the effects the disease has on patients over time, and patients’ experience with and views about treatments. It also includes information about patients’ views about the disease, treatment, and outcomes, and the relationship amongst those things. It includes information about the impact of the disease and treatment on the patient, patients’ thoughts about potential and current treatments, and the patients’ enhanced understanding of the progression, severity, and chronicity of the disease.

The guidance series consists of 4 documents:

Guidance 1: Collecting Comprehensive and Representative Input

Guidance 1 was released in June 2020 and is designed for those planning a study and deciding on sampling methods for the collection of patient input. Approaches for patient selection and input collection depend on the research question, and FDA recommends examining previous studies and relevant literature and consulting subject matter experts regarding decisions on methodology and study materials.

Guidance 1 describes the steps in the process of collecting input, which include defining a research question, describing the target population and who will be providing the data (patients, caregivers, or clinicians), choosing a data collection methodology, and collecting, storing and managing the data. Of note, when choosing data collection methodology and setting, ensure the data collected are representative of the population and consider the inclusion of diverse sites to better enable a representative sample.

Read the full guidance.

Guidance 2: Methods to Identify What Is Important to Patients

This draft guidance describes what to ask patients and why. It describes qualitative, quantitative, and mixed methods approaches and provides best practices for eliciting information that is important to patients, including special populations (children, the cognitively impaired, and those with rare diseases) and diverse populations.

Read the draft guidance.

Guidance 3: Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcomes

This guidance is intended to help investigators decide what to measure and how to select or develop clinical outcome assessments (COAs) that are fit for the purpose of assessing outcomes that are important to patients. It “describes how stakeholders (patients, caregivers, researchers, medical product developers, and others) can collect and submit patient experience data and other relevant information from patients and caregivers to be used for medical product development and regulatory decision-making.”

Read the draft guidance.

Guidance 4: Incorporating Clinical Outcome Assessments Into Endpoints for Regulatory Decision-Making

This guidance is forthcoming and will describe how to incorporate a given clinical outcome assessment tool or a set of measures into a clinical research study. The guidance will include information about defining meaningful change, and the collection, analysis, interpretation, and submission of data.