Making Knowledge Hereditary: Public–Private Partnership Drives Progress in Rare Disease Community

Abstract

Background: Hemophilia is a rare and complex inherited bleeding disorder in which people may experience prolonged, painful, and debilitating bleeds due to an inability of their blood to clot properly. Although there has been great progress in the management of the disease over the past 50+ years, many important scientific questions have gone unanswered. Further, despite decades-long interest and desire by medical providers and scientists, the U.S. government and the hemophilia community to conduct comprehensive genetic testing to better understand the disorder, challenges such as cost, access, and privacy concerns served as ongoing obstacles. Focus of the Article: To overcome these challenges and address this unmet need, four community organizations and a health communications and social marketing agency came together in 2011 to create a first-of-its kind public–private partnership (PPP) called My Life, Our Future (MLOF) to provide people with hemophilia information about their disorder while also furthering scientific understanding. Importance to the Social Marketing Field: This article will explain how MLOF utilized social marketing to drive broad community engagement, ultimately genotyping more than one third of the U.S. male hemophilia population through the program and opening the MLOF Research Repository, the largest research repository of its kind. It will also discuss the key learnings gleaned through the establishment and implementation of this program, notably the strength of a PPP in health care.