Considerations in View of the Lived Experience of Affected Individuals: Compliance in the Rare Disease Space

Over 10,000 rare diseases are currently affecting more than 30 million Americans. This article, written in partnership with members of the Rare Disease Compliance Consortium, focuses on the role of Patient Advocacy Organizations (“PAOs”) and patient advocates as critical stakeholders in the experience of people affected by rare diseases. PAOs engage in a variety of activities related to the awareness and treatment of medical conditions, with the goal of taking actions to help patients affected by those medical conditions, their families, and caregivers. Patient advocates educate, empower, and support others living with, or caring for those with, a disease by which they are affected in a meaningful way. This article discusses the value of PAO and patient advocate expertise in the lived experience of individuals affected by rare diseases and explores the existing framework under which PAOs and patient advocates can interact with rare disease companies.