Diagnostic and therapeutic odyssey : essays in health economics.

Summary This doctoral thesis focuses on the challenges faced by rare disease patients. It is structured in three parts, each of which is dedicated to the issues of an actor at the heart of the diagnostic and therapeutic Odyssey of rare disease patients. The first part of the thesis focuses on the patient and his social network. Chapter 1 considers the sources of delay in access to diagnosis, and explores in particular the effect of social capital on the delay in obtaining a diagnosis. Chapter 2 discusses the negative externalities on maternal health of a diagnosis of a chronic disease in children. The second part of the thesis is dedicated to the pharmaceutical industry and focuses on R&D investment decisions targeting rare diseases. Chapter 3 assesses the causal effect of the Orphan Drug Policy on research effort, and Chapter 4 considers the inequalities in the allocation of R&D investments between rare diseases. Part 3 is dedicated to public policy makers and discusses the issues of evaluating the benefits of therapeutic innovation and defining the conditions of access to this innovation. Chapter 5 assesses the causal effect of therapeutic innovation on the longevity of rare disease patients. Chapter 6 is a critical discussion of the use of tools.