The U.S. Food and Drug Administration (FDA) wants sites and sponsors to feel emboldened when it comes to measuring patient experiences during drug development—and at the same time, FDA wants to see some common sense, too.
The agency has just issued a guidance on “Patient-Focused Drug Development: Collecting Comprehensive and Representative Input” that represents the first in a series of four methodological, patient-focused drug development (PFDD) guidance documents projected to come down the pike. FDA is developing the series to address, in a “stepwise” manner, how stakeholders (patients, researchers, medical product developers, and others) can collect and submit patient experience data and other relevant information from patients and caregivers for medical product development and regulatory decision making.
Here’s the line-up and how the agency describes the first PFDD guidance and those yet to come:
Guidance 1: This discusses sampling methods that could be used when planning to collect patient input. It also provides a general overview of the relationship between potential research questions and methods when deciding from whom to get input (including defining the target population and development of the sampling strategy).
Guidance 2: This will discuss methods for eliciting information from individuals identified in Guidance 1, gathering information about what aspects of symptoms, impacts of their disease, and other issues are important to patients. It will discuss best practices for how to do qualitative research, including conducting interviews, development of interview guides, selection of types of survey questions, and considerations for collecting demographics and survey information. It will also discuss survey methods and qualitative research topics to help avoid misleading results, such as inadvertently priming patients in ways that can lead to results that poorly represent what is important to them.
Guidance 3: This will address refining the list of important impacts and concepts from patients to develop potential study instruments. Given that not everything identified as important by patients, caregivers, and clinicians can demonstrate change in a specific treatment trial or is measurable, how will you select what to measure in a medical product development program to show clinical benefit? How will you identify or develop fit-for-purpose clinical outcome assessments (COAs) to assess outcomes of importance to patients?
Guidance 4: This will address topics arising from COA-related endpoint development and interpretation, including topics related to instrument administration and meaningful within-patient score changes.
The FDA has not issued a timetable for the rollout schedule of the remaining guidances.
Author: Michael Causey