FDA Issues First of Four Draft Guidances Related to Patient-Focused Drug Development


Today, the U.S. Food and Drug Administration (FDA) released guidance titled, Patient-Focused Drug Development: Collecting Comprehensive and Representative Input (Guidance 1), to address how stakeholders can collect and submit patient experience data from patients, caregivers, clinicians and patient advocacy groups for product development and regulatory decision-making. “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 (i) symptoms of a condition and its natural history; (ii) impact of a condition on functionality and quality of life; (iii) experience with treatments; (iv) input on which outcomes are important; (v) patient preferences for outcomes and treatments; and (vi) relative importance of any issue as defined by patients. Guidance 1 is provided by the FDA in response to Affordable Care Act (ACA) and PDUFA IV requirements and incorporates findings from the disease-specific patient-focused drug development (PFDD) meetings previously held by FDA.

Guidance 1 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 inform clinical trial design, trial endpoint selection, and regulatory reviews, including potential labeling. 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.

FDA intends to supplement this Guidance 1 with three additional guidance documents:

  • Guidance 2 will discuss methods for eliciting information from patients and gathering information about issues important to such patients.
  • Guidance 3 will discuss the development of fit-for-purpose clinical outcome assessments (COAs) to appropriately measure patient experience data.
  • Guidance 4 will discuss COA-related endpoint development and data interpretation.

The FDA has not provided a timeline for the release of these supplemental guidances. We will continue to monitor developments on this topic and update the blog accordingly. 



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