Non-clinical requirements for severely debilitating or life-threatening diseases
HumanScientific guidelines
Seriously debilitating or life-threatening conditions are those associated with morbidity that has substantial impact on patients’ day-to-day functioning and will progress if left untreated, or those associated with a high likelihood of mortality. A reflection paper would facilitate development of medicinal products in this area, help harmonise non-clinical development, support marketing authorisation applications and earlier patient access to potentially life altering or saving therapies, particularly in those indications where there is an unmet medical need.
Keywords: Non-clinical safety; severely debilitating or life-threatening diseases; tailored non-clinical programmes; 3Rs; NAMs; weight of evidence approaches