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FDA Issues Guidance on Patient Experience Data Collection

Scott Gottlieb

Scott Gottlieb, FDA Commissioner, in November 2017 (A. Kotok)

13 June 2018. The Food and Drug Administration issued draft regulatory guidance outlining methods for collecting data from patients that the agency says will help improve evaluations of more precisely targeted drugs. FDA released the document for comment yesterday on the agency’s web site.

FDA Commissioner Scott Gottlieb announced the document’s release yesterday in an agency statement. Gottlieb says the more precisely targeted drugs addressing specific molecular sub-types call for new evaluation techniques, for which many current methods are not up to the task. He specifically cited tools designed for clinical trials of chemotherapy drugs for treating cancer that provide modest clinical outcomes, while presenting patients with serious side effects. Those tools are designed to decide on the value of clinical outcomes balanced against the significant risks posed to patients.

Today’s more precisely targeted drugs, notes Gottlieb, work differently and require more relevant measurement techniques. “There are more opportunities to demonstrate significantly higher rates of benefit in carefully selected patients—and often show reduced toxicity—in smaller cohorts of patients who might express a marker that predicts clinical response,” says Gottlieb. “In these cases, surrogate endpoints—or measures of intermediate clinical benefit—can be both clinically relevant and highly valued by patients.”

Experiences of patients, says Gottlieb, are driving a series of 4 new regulatory texts, with sampling techniques for collecting data that capture those experiences the focus of the first document released yesterday. The document aims to standardize the terms used to describe sampling methods and identify techniques most appropriate for the clinical and regulatory analysis, reporting, and decision making. These data cover symptoms of conditions, impact on functioning and quality of life, experience with treatments, patients’ input on outcomes they consider important, and patient preferences for treatments and outcomes. In addition, the data may be provided by caregivers and patient advocates, as well as patients themselves.

Specific sampling techniques cover quantitative and qualitative methods, as well as combinations of those techniques. Quantitative data provide more statistical reliability and show relationships among variables, while qualitative data, such as from in-depth interviews, offer more perspective, context, and nuance in the findings. For quantitative findings, the document outlines several sampling methods ranging from simple random to multi-stage probability samples, which would depend on the complexity of the research questions. Not all samples are probability samples, however. In many clinical trials, volunteer participants are self-selected, purposefully recruited to reflect specific characteristics, or enrolled by referrals from other trial participants.

In addition, the document discusses data collection technologies using social media for organizing focus groups, mobile systems such as wearable devices and telemedicine, as well as audio-visual technologies to capture real-time reactions of individuals or groups. The document also notes protections for patients taking part in these studies, spelled out in Department of HHS regulations and legislation, such as the Health Insurance Portability and Accountability Act, or HIPAA.

The guidance document is part of FDA’s patient-focused drug development initiative, “to help ensure that patients’ experiences, perspectives, needs, and priorities are captured and meaningfully incorporated into drug development and evaluation.” The draft document will be available for comment for 90 days. Three other documents are anticipated in this series covering the kinds of questions asked of patients, measurements of clinical outcomes, and appropriate end-points measuring objectives of clinical trials.

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