The primary objective of this forum is to describe the Key Project, proposed by the ISPOR Rare Disease Special Interest Group (RD SIG), on Evidence-Based Patient Advocacy (EBPA). A key secondary objective of this forum is to present the future direction of the Rare Disease SIG based on additional planned activities.

The RD SIG believes that the inclusion of the patient’s voice is needed throughout a product’s lifecycle. As such, the RD SIG is aiming to define a framework for EBPA, such that tangible metrics are recognized that optimize integration of the patient’s voice, to support the development and uptake of medical products. The first key task of this project will be to catalogue the activities that Patient Advocacy Organizations are typically conducting in rare diseases. These activities will be mapped against key challenges identified in previous ISPOR RD SIG research, such that any relevant gaps are identified. Next, interviews with key members of such Organizations will be conducted in order to gain a better understanding of the hurdles and successes in capturing and integrating the patient’s voice, and how these initiatives inform the decision-making process. As a final outcome of this project, a consensus will be sought, across a multidisciplinary group of key relevant decision makers, on the appropriate EBPA framework for rare diseases.

In parallel with the Key Project on EBPA, the RD SIG will develop a series of educational webinars in rare diseases, based on feedback from its members. Specifically, we are aiming to cover topics around the key challenges associated with developing evidence supporting rare disease therapies, and how ISPOR can contribute to addressing these. The insights from these webinars will be culminated in an Issue Panel discussion.