Leveraging Prior Knowledge to Support Early Phase Clinical Trial Applications: Regulatory CMC Considerations and Case Studies

Prior knowledge was successfully used to support the clinical trial applications of two small interfering RNA (siRNA) investigational medicinal products (IMPs). A comparative, risk-based approach was used to introduce supportive stability data from three other siRNA IMPs to establish an initial 36 month drug product shelf life. The structuring of supportive data from other clinical programs into Module 3 Quality of the Common Technical Document and response from health authorities is additionally presented.