Competing Values, Competing Claims: Diversity among Patient Advocates Who Intervene to Shape Policies on Drugs for Rare Diseases

Abstract

Patient advocacy groups can push regulators to approve and pay for expensive drugs despite weak evidence of efficacy and/or safety. Advocacy organizations that critique high prices for rare diseases are less publicized but can also influence policy. The funding and relationships many groups have with the pharmaceutical industry may contribute to patient advocates' differing perspectives, but the leaders' values and experiences are an overlooked factor. We need to understand the dominant public-private partnership model of patient advocacy, its historical roots, justification and how key advocacy actors respond to it if we are to advance policies that will contain expensive drugs for rare diseases.