Abstract

Significant challenges are associated with the availability of and access to treatments for rare diseases. In this issue, Sirrs et al. (2023) frame challenges in terms of evidence, economics and ethics and describe how they manifest in the Canadian context. This short response paper argues that although interesting initiatives exist internationally to deal with some of these challenges, a plug-and-play approach will not suffice given the particularities of the Canadian system. Rather than seeking international lessons on how to deal with Canadian challenges, the emerging interdisciplinary framework of social pharmaceutical innovation is advanced here as a whole-systems approach that stands to address interconnected components of the rare disease ecosystem, and in doing so, a made-in-Canada approach is advocated for.