FDA Draft Guidance on Patient Centric Drug Development Focuses on Sampling Methods

June 18, 2018

Sites and sponsors should have a lot of leeway in determining how to measure patient experiences during drug development, but researchers should take care that whatever sampling methods they provide are a fair representation of that target population, the FDA said in a new draft guidance issued June 12.

The guidance focuses on sampling methods for patient experiences. It lays out broad suggestions for the kinds of patient experiences that might be relevant, including a disease or treatment’s impact on a patient, patients’ own feelings about potential or current treatments, “unmet medical needs and currently available treatment options” and an “enhanced understanding of the natural history of the disease or condition, including progression, severity, chronicity.”

The draft guidance, mandated under the 21st Century Cures Act, is meant to facilitate the advancement and use of systematic approaches to collect meaningful patient and caregiver data that can better inform medical product development.

The agency suggests it is probably best to let the patients themselves directly report their experience, “unless the patient cannot reasonably be expected to reliably self-report” such as children or individuals who are cognitively impaired.

The 65-page June 12 draft guidance may be an important step in getting patient’s voices into drug development, said Lindsay McNair, chief medical officer at WIRB-Copernicus Group.

“Over the last several years we’ve seen many biopharma companies create programs intended to involve the patient voice in their development programs, and the increasing strength of patient advocacy groups engaging in drug development, but patient input has been largely regarded as a ‘nice to have,’” McNair said.

“This guidance represents a concrete step in the FDA saying that they intend patient input to be part of regulatory decision making. Sponsors of medical product development programs are going to have to think about the collection of information and data from patients in a more structured and formal way than most have been doing so far, and this guidance provides information about how to do that.”

McNair said she was particularly struck by language in the draft guidance that encourages the use of social media as a way of gauging patient attitudes.

“While in other areas the FDA has not provided much formal advice related to social media—for example, we’ve not seen guidance about the use of social media for clinical study recruitment, despite its explosion in use over the last decade—FDA did specifically recognize this community and the strengths and limitations of data collected through interactions with online patient fora,” McNair said.

The guidance is the first of a planned four documents designed to lay out rules for patient-focused drug development. Future guidances will focus on the structure of patient experience questions, how to decide what clinical trials should measure and how to develop fit-for-purpose clinical outcome assessments and—once those outcome assessments are in place—how to determine “an appropriate clinical trial endpoint.”

“We learn through scientific advances, but also by listening to patients,” FDA Commissioner Scott Gottlieb said in a statement released in support of the draft guidance. “Our work demands that we must continue to reflect on how we can make the science of drug development and review more modern and more patient-centered, so that approved products impact the metrics that real-world patients and families value most. This requires ongoing engagement with the patient community.”

You can read the June 12 draft guidance here:

-By William Myers