An Evolved Approach to Advisory Boards in Rare Disease Drug Development: 5-Step Model to Finding and Engaging Patient Advisors.

Nearly all new product development teams at pharmaceutical companies will routinely conduct patient advisory boards. These board meetings will help collect and document the experience of patients and caregivers for medical product development and regulatory decision-making. Recently, in June 2020, The US Food and Drug Administration (FDA) published a final guidance on methodological patient-focused drug development (PFDD) to address, in a stepwise manner, how stakeholders (patients, researchers, medical product developers, and others) can successfully use these patient forums. In the process of developing this guidance, the FDA acknowledged that leading its own PFDD meetings, especially when limited to organized disease advocacy groups, cannot address the gaps in information on the patient perspective. So, it has expressed support for advancing the science and utilization of patient input other means. Because traditional methods of conducting patient advisory boards often do not achieve the full potential of patient centricity, the authors of this article share an approach to consider when selecting patient advisors, in order to gain the most actionable input to a product development team.