Long regarded as a neglected backwater within the drug discovery landscape and the preserve of only a handful of niche players, the rare disease space has been undergoing an extraordinary turnaround in fortunes of late.

This special report delves into the flurry of activity taking place across the rare disease and orphan drug sector – including in investment, M&A, patient advocacy, clinical trials, regulatory reform and much more.

In typical PharmaBoardroom style, InFocus: Rare Diseases brings a truly global perspective, with contributions and reportage from the USA, Canada, the UK, and China, among other regions.

It features the exclusive insights of researchers, global pharma CEOs and country managers, consultants, investment managers, and patient group heads to give a well-rounded and meaningful picture of the rare disease space in 2019.