The U.S. Food and Drug Administration (FDA) released a draft guidance entitled Patient-Focused Drug Development: Collecting Comprehensive and Representative Input, to address how stakeholders can collect and submit patient experience data from patients, caregivers, clinicians and patient advocacy groups for medical product development and regulatory decision-making. The draft guidance is open for comment until September 11, 2018.
“Patient experience data” is defined as data that is “collected by any persons and intended to provide information about patients’ experiences with a disease or condition” including patients’ experiences, perspectives, needs, and priorities related to
The 65-pages draft guidance (Guidance 1) on patient-focused drug development (PFDD) provides instruction on methodologically-sound data collection tools and sampling methods to gather patient experience data that is relevant, objective, accurate and representative of the target population. Qualitative and/or quantitative patient experience data can be collected through various research methods, including clinical trials, observational studies, advisory boards, public meetings, and other novel settings. The data can be used to help in clinical trial design, trial endpoint selection, and regulatory reviews including benefit-risk assessments as well as potential labeling (or other communications). The FDA makes it clear that the level of rigor needed to generate patient experience data will vary across studies and depend on the intended use of a product and, as such, FDA should be consulted early to obtain feedback. Guidance 1 also includes a glossary of terms.
FDA recommends that data management be addressed in the early stages of a research study. "Before initiating data collection, you should formulate a data management plan (DMP)". In regard of monitoring and quality assurance FDA expects "that external stakeholders will be responsible for monitoring the study, ensuring data integrity, and performing the data analysis".
FDA intends to supplement Guidance 1 with three additional guidance documents. The topics and questions that each guidance document will address are the following:
The FDA has not provided a timeline for the release of these three supplemental guidances.