Abstract
New nanomedicine formulations and novel applications of nanomedicinal drugs are reported on an almost daily basis. While academic progress and societal promise continue to shoot for the stars, industrial acceptance and clinical translation are being looked at increasingly critically. We here discuss five key challenges that need to be considered when aiming to promote the clinical translation of nanomedicines. We take the perspective of the end-stage users and consequently address the developmental path in a top-down manner. We start off by addressing central and more general issues related to practical and clinical feasibility, followed by more specific preclinical, clinical, and pharmaceutical aspects that nanomedicinal product development entails. We believe that being more aware of the end user’s perspective already early on in the nanomedicine development path will help to better oversee the efforts and investments needed, and to take optimally informed decisions with regard to market opportunities, target disease indication, clinical trial design, therapeutic endpoints, preclinical models, and formulation specifications. Critical reflections on and careful route planning in nanomedicine translation will help to promote the success of nanomedicinal drugs.Graphical abstract
Highlights
Nanomedicines are a highly diverse group of drug products
Many thousands of different nanomedicine formulations have been designed and evaluated over the years, for various different types of diseases. Fifty of these formulations are currently approved for clinical use, and there are several hundreds of trials ongoing in which nanomedicines are being evaluated in patients [4, 5]
With the exception of nucleic acid–based nanotherapeutics, such as the lipid-based small-interfering RNA formulation patisiran, nanomedicinal drug products are typically reformulations of active pharmaceutical ingredients (API) which have already been approved for clinical use
Summary
Nanomedicines are a highly diverse group of drug products. They encompass polymer-drug conjugates, polymer-protein conjugates, protein-based nanoparticles, polymeric micelles, inorganic nanoparticles, and a range of lipid-based nanoparticulate drugs, of which PEGylated liposomes are the prime example [1,2,3]. With the exception of nucleic acid–based nanotherapeutics, such as the lipid-based small-interfering RNA formulation patisiran, nanomedicinal drug products are typically reformulations of active pharmaceutical ingredients (API) which have already been approved for clinical use This makes sense, as improving the in vivo performance, efficacy, or safety of an established API is a less venturesome enterprise as compared with developing a completely new chemical entity (NCE), which has unknown pharmacological and toxicological behavior in human beings. It should preferably be done according to quality-by-design (QbD) principles, meaning that adequate in-process controls should be implemented that enable the monitoring of key quality attributes during compounding, giving the manufacturer time to adjust critical process parameters, such as temperature and pressure, to safely steer the final formulation within the set specifications With this in mind, it makes sense to carefully assess composition, excipients, and key quality attributes (including quality control assays) as early on as possible in the design of a nanomedicine drug, preferably even before preclinical testing in animal models. Carefully and comprehensively considering chemistry, manufacturing, and control aspects as soon as possible in nanomedicine development crucially contributes to translational success [11]
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