Rare diseases are uncommon for all, especially for those suffering the disease, and research in these conditions provides hope to patients and their families. Contributing to the development of potential cures for these rare diseases is an attractive challenge from a medical perspective and an unavoidable responsibility from an ethical viewpoint. Finding patients and experienced sites and investigators, and even activating and conducting clinical trials, might be defying but highly rewarding as it helps to bring remedies to affected subjects.
At Pivotal, we have always opted for investigating rare diseases despite all concerns and issues related to the trials that they may bring along. More than half a hundred projects later, in quite diverse conditions and phases, we remain hungry for more of this, aware of what the results of these investigations will bring to the society in general and the affected subjects in particular.