Patient Advocacy & Patient Experience Data

Patient experience can shape drug and biologic development long before an application is filed. FDA's patient-focused drug development framework recognizes that patient and caregiver input can meaningfully inform disease understanding, unmet need, endpoint selection, clinical outcome assessment strategy, trial design, benefit-risk assessment, and regulatory decision-making.

We help sponsors and patient organizations bring patient experience, disease burden, treatment burden, patient preference, and meaningful outcomes into development strategy, FDA meetings, advisory committee preparation, and review strategy. This work is especially important in rare disease and serious-condition programs, where patient experience can help frame unmet need, acceptable uncertainty, clinically meaningful benefit, and the tradeoffs patients are willing to accept.

We advise on:

• Patient-focused drug development strategy
• Patient organization and rare-disease group engagement with FDA
• Disease-burden, unmet-need, and treatment-burden presentations
• Patient experience data and patient preference information in FDA-facing strategy
• Endpoint strategy and clinically meaningful outcomes
• Benefit-risk framing for FDA meetings, submissions, and advisory committee preparation
• White papers, meeting materials, and other FDA-facing advocacy materials

Selected Experience

• Assisted patient advocacy group on drafting a white paper integrating patient experience data and unmet need in support of a filed marketing application.
• Prepared a patient advocacy group to deliver public testimony at an FDA advisory committee and advised on additional engagement with FDA.
• Leveraged patient preference surveys in the context of a marketing application to reframe benefit-risk and overcome requests for an additional adequate, well-controlled study.