Maximizing the Impact of Patient-centric Data: Meaningful Interactions Informing Regulatory Decision-making.

Patient Focused Drug Development (PFDD) has gained growing support from patients, regulators, health technology assessment (HTA) bodies and industry who are seeking to systematically incorporate patient perspectives into drug development, regulatory decisions and value assessments. To maximize the impact of this patient-centric data, it is critical that Sponsors and FDA collaborate and align early in the development process to ensure that the Sponsor’s program will deliver data that is fit for purpose to inform the specific regulatory decision(s) being targeted. Because patient experience data (PED) as a source of evidence in regulatory decision making is important, both regulators and industry benefit from interactions throughout the process of PED development when PED is intended to support a regulatory decision. This session will incorporate FDA, EMA, and industry perspectives and experience to help support meaningful FDA-Sponsor interactions, specific to the design, collection, and analysis of PED, while balancing needs of both parties. The session will feature a series of brief presentations that will provide an introduction to PED and its potential use as evidence in regulatory decisions, discuss examples that demonstrate key elements that resulted in a successful Regulator-Sponsor interaction. The session will highlight learnings and opportunities where a modified approach to Regulator-Sponsor interactions could be even more beneficial. Lastly, a panel discussion will focus on how Regulators and Sponsors can align and work collaboratively on next steps that will have the greatest impact on increasing the efficiency and productivity of interactions and use of the resulting PED to inform regulatory decisions.