Nanotechnology-based drug products: Science and regulatory considerations

Abstract A sponsor must submit data for a review to US Food and Drug Administration (FDA) in order to market nanotechnology-based prescription drug products. An application to submit data for a brand and generic version of drug product are NDA (new drug application) and ANDA (abbreviated NDA), respectively. FDA basic requirements of safety and efficacy for approval have not changed, even for nanomedicines; the onus rests on the sponsor to provide evidence of safety and efficacy. Oftentimes, a sponsor must provide additional safety and efficacy information of nanomedicines due to presence of nanoscale materials in them which are responsible for high reactivity, and unique mechanical and magnetic properties. For example, safety evaluation of nanotechnology-based drug products may include environment and toxicological assessment. In terms of pharmaceutical quality, particle size characterization is a major challenge as there is no consensus on sizing technique. To ensure consistency in product quality, hence clinical performance, it is also important to understand the product and process variables that can affect the quality of products, especially particle size. Quality by design approach provides a mean to link material attributes and process variables to critical quality attributes of the nanomedicines. This chapter provides scientific and regulatory aspects of nanotechnology-based drug products.