The sponsor requested assistance with two issues; one in the United States and one in Europe. The first involved obtaining US FDA approval to start a long term Phase III clinical study for a biological drug administered intravenously (IV). In response to the initial Clinical Trial Application, the FDA requested that the sponsor conduct a chronic toxicological study in advance of the trial. The client also required assistance designing non-clinical toxicological studies necessary to conduct clinical trials by the inhalation route in Phase I-III in Europe. For this product, the inhalation route offered several potential benefits; including high exposure to the target organ (lung) and lower systemic uptake, thereby minimizing for side effects.
The primary challenge was to present valid and compelling scientific and regulatory data that supported waiving the costly and time-consuming conduct of chronic non-clinical studies by the intravenous route.
The second challenge was to design, conduct and evaluate inhalation toxicological studies and get regulatory approval for the planned trials by inhalation.