Needs

Nanotherapeutics are regulated under the legislative-regulatory frameworks of medicinal products or medical devices requiring the provision of data for quality, safety and efficacy as any other product class when applying for clinical application. To be successfully translated into the market nanomedicines, they must meet the same safety, efficacy and pharmaceutical quality criteria applied to all drug products. However, due to their unique and hybrid nature, the quality assessment of nanomedicine formulations pose substantial analytical challenges when compared to small molecular or biological drugs (e.g. antibodies), due to their structural complexity.

The European Medical Agency (EMA) states that, with the approval of innovative nanotherapeutics, we need to «develop and standardise new testing methods related to the quality and safety assessment of nanomedicines», to reach an understanding of «the critical quality attributes of a given product and the relationship between those and its biological activity and in-vivo behaviour».

The REFINE project, under the lead of the European Commission’s Joint Research Centre (JRC) identified priorities for method development and standardisation, including methods to measure: (i) surface properties, (ii) stability and kinetic properties in biological media, and (iii) uptake, absorption, and distribution in cells and tissues.

The project industrial partners confirmed these measurement needs for different classes of nanotherapeutics as a global industry requirement.

• nanoPET, for contrast agents based on iron oxide and gadolinium- nanoparticles, states «the need for understanding the definition of particle size; feasibility, and improved methodologies to determine amount, homogeneity, and functionalisation of the nanoparticle surface and for the functionality and detection of nanoparticles in vivo».
• Curadigm who develop liposome-based nanotherapeutics, highlighted «the lack of standardised methods to ensure the integrity of lipid-based products, to characterize their physico-chemical properties, and their behaviour in complex biological medium» and the need «to follow nanoparticle biodistribution and bio-transformation in vivo».
• Nanobiotix,  a global company designing clinically approved radio-enhancers for the treatment of soft tissue sarcoma, based on hafnium oxide nanoparticles, underlines the critical «absence of reference materials and standardized methods for metal oxide nanoparticles» that are needed «for method validation of fit-for–purpose characterisation techniques following regulatory agency recommendations».