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

Abstract

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.

Decentralized Clinical Trial Case Study: Five-stage Process for Recruiting and Completing a Site-less Clinical Study in Less Time and Lower Cost than Traditional Methods

“…This is relevant to consider as many patient advisors often recommend ways to stay connected with the sponsor when the product comes to market. [11][12][13][14][15] As shown above, the numbers illustrate that this campaign was able to locate more patients and deliver more study completions in the least amount of time for a lower cost, thus ensuring the efficiency of the program. Some of the older patients may not be as tech-savvy which may make the program perhaps a bit more challenging for them.…”

A rare partnership: patient community and industry collaboration to shape the impact of real-world evidence on the rare disease ecosystem

“…In drug development, PAGs have significantly contributed to patient recruitment, clinical trial protocol design, identification of endpoints and PROs [ 23 – 27 ]. For instance, after the inception of the International Pompe Association (IPA) in 1999, members could engage with and directly fund preliminary research into enzyme replacement therapies (ERTs).…”