Session (CTI)

ISSUE: Sponsors routinely collect patient experience data (PED) to inform their understanding of a disease area and ensure that they are meeting the needs of patients. To advance patient-focused drug development, alignment between industry, patient advocacy groups, and regulators needs to start early in the development process to support identification of what is clinically meaningful to patients, ensure that the sponsor’s program deliver fit-for-purpose data, inform regulatory decisions, and inclusion of PED into labelling and patient-facing resources. Although the regulators encourage Industry to engage early and often regarding the collection and use of PED in development programs, Industry is often unclear regarding when to engage and what information is needed to support PED discussions to elicit actionable feedback. To facilitate strategic and timely discussions on the use of PED in product development and regulatory decision-making, resources including frameworks, best practices documents are being developed by stakeholders, and there continues to be a need for regulatory feedback and guidance. In this session, panelists will discuss the realities of early regulatory engagement on topics related to patient experience data and present frameworks that can help sponsors as they develop their regulatory engagement strategy. OVERVIEW: In this session, the moderator will provide background on current practices and recommendations. FDA representative will discuss regulatory considerations for engagement with regulators related to PED (e.g., qualitative PED, COAs, patient preference information). Industry panelists will present case examples and discuss frameworks (E.g., BIO’s FDA-Sponsor Engagement Framework for Patient Preference Information) that are being developed to support regulators call for “early engagement” and help support regulatory discussions.