Utilising Nanotechnology and Nanosystems for Treatment of Rare Diseases

Background: Rare, or ‘orphan’, diseases are defined as lifethreatening or chronic debilitating conditions affecting a small number of patients, and present a significant public health challenge in terms of the lack of scientific knowledge, clinical expertise, and available therapies for these complex pathologies. Indeed, the difficulties involved in generating new treatments for a limited patient population, and the likely financial loss in bringing such products to market, has deterred research and development by the pharmaceutical industry. To address these issues, sponsors of new therapies can apply to regulatory authorities for ‘orphan designation’, which confers incentives for development such as reduced fees for scientific advice, tax incentives, and market exclusivity for an approved product; although the qualifying criteria vary between regions. Focus: This review provides a global overview of orphan diseases and designation, and focuses on the potential of the rapidly expanding and exciting field of pharmaceutical nanotechnology and fabricated nanosystems (nanomaterials and nanodevices), including nanomedicines and nanosimilars for the therapeutic, diagnostic, or theranostic (development of more specific, individualised therapies for various diseases, and combining diagnostic and therapeutic capabilities into a single system) applications for rare diseases.