Daniel Coyle, author of the New York Times best-seller The Talent Code, describes the innate human reaction to failure in a 2016 blog post: “We wince. We close our eyes. We slowly look to see if anybody noticed us. Then we ignore it, or, better, pretend it didn’t happen. It’s basically a full-brain allergic response—minimize it, keep quiet, and move on.” What he’s getting at is a missed opportunity to learn in the moment, a notion that those in the rare disease community know firsthand.
The rare disease community has defaulted to a so-called psychological “allergic reaction” to failure for far too long. While recent gains within the space are celebrated publicly by rare disease advocacy organizations, government agencies and pharmaceutical companies, there’s little work being done to examine the systemic failures that are limiting the degree of progress in successfully researching and bringing to market therapies for rare disease patients.
Misaligned incentive structures, trust issues, inconsistent funding, archaic approval systems and differing views on the ownership of data are fundamentally failing rare disease patients—and their families and caregivers—who are already dealing with the physical and emotional burden of disease.