Our client was at the beginning of the approval process with its orphan medicinal product for the treatment of an extremely rare metabolic disease. This was a medicinal product that was to be used in an extremely small paediatric patient population; the disease was only known to highly specialised paediatricians. As a result, a definitive diagnosis of the disease was often made at a very late stage of the disease, with negative consequences for the affected patients.
As part of a campaign to increase awareness of the disease, we prepared a medical review in English in close cooperation with clinical experts. In the course of this review, the very positive study data of the new medicinal product were, of course, also presented and acknowledged in a convincing way.
The work was published in a recognised journal with peer review, and supported our client in raising awareness in the target group of the disease and the future therapy option with the medicinal product – even before market launch.