ICON has provided due diligence services to a large institutional investment company for more than five years, to support them in funding of development opportunities for both drugs and medical devices. For this client, ICON has performed technical evaluations of more than sixty assets across a range of therapeutic areas and modalities. Technical evaluations of assets include an assessment of development risks and the probability of achieving registration and commercial success.

This cell-based therapy for an acute immunological condition leveraged a historical control trial design instead of using a control arm within the clinical study. The technical review of the asset had a specific focus on the use of historical controls and data from an open-label single arm phase 3 study in paediatrics. The sponsor intended to use data from adults and paediatric patients as a means of securing labelling for paediatric populations.

ICON assembled a bespoke due diligence team including expert statisticians and therapeutic experts with experience in clinical trial design to advise on the regulatory approval process for the cell-based therapy, including the use of a historical control. In this capacity, ICON provided support for the investor as well as the sponsor developing the therapy. The ICON diligence team reviewed all available technical information and discussed development logistics and commercialisation with the sponsor and client.

An evaluation of development risk and commercial success was provided, highlighting specific regulatory concerns the therapy might encounter. As a consequence of diligence, ICON recommended that an additional study be conducted. The client proceeded with investment in the opportunity cognizant of the risks that ICON had highlighted. When the sponsor consulted with the FDA, FDA feedback was consistent with the position of the ICON team. ICON was engaged by the sponsor as a development consultant

In this case, the client requested that ICON evaluate the probability of achieving regulatory approval of a stem cell therapy for the treatment of an acute immunological condition in adult and paediatric patients. The asset had orphan drug designation and was in phase 3 development with a first filing planned in paediatric patients. ICON conducted thorough due diligence over approximately six weeks and provided additional consultation for the client