Abstract 

The new substance notification (NSN) process, administered by Environment and Climate Change Canada (ECCC), is designed to assess new substances before their introduction into Canada. While this is often seen as a technical requirement to fulfill before clinical development can proceed, the NSN plays a much broader and more strategic role, particularly in advancing innovative therapies for rare diseases. 

ThermoFisher recently applied this process to support the importation of a promising investigational drug product (IP) for Angelman syndrome, a rare neurogenetic disorder. In such cases, the NSN serves as a critical link between scientific discovery and patient access, ensuring that every new therapy introduced into Canada meets the highest standards of human and environmental safety. 

Drawing on professional experience in preparing an NSN submission for a gene therapy in Angelman syndrome, this article explores both the clinical and technical dimensions of the process. It also highlights Canada’s distinctive emphasis on environmental risk assessment under the Canadian Environmental Protection Act (CEPA) and contrasts it with the US Food and Drug Administration (FDA) and European Medicines Authority (EMA) frameworks. Ultimately, the NSN is not just an administrative requirement; it is also a strategic enabler of innovation, essential to Canada’s global competitiveness in rare disease research and its commitment to patient care.