Value based healthcare for rare diseases: efficiency, efficacy, equity

Abstract

The paper reviews the literature available on value based health care and relates it to rare diseases. Starting from the economic definition of value and healthcare evaluation, efficacy and efficiency, it includes the equity dimension to define value-based healthcare. It embraces also the cultural framework associated to the concepts of health and disease, normal and pathological, right or wrong for the patient. The paper highlights that a prevention and recovery view and global evaluation of costs/benefits ratio for rare diseases make difficult and limited the applicability of the value-based approach to rare diseases. Since epidemiology of rare diseases identified a series of difficulties in applying value-based public health strategies to rare diseases, the paper underlines the necessity of new culture of health and well-being, radically re-examining how to organise the delivery of prevention, and healthcare services, and finding alternative ways of empowering and giving voice to vulnerable and marginalised groups.