Complex Drugs: What Are They and Why Do They Need Special Attention?

Abstract

The rise of bioand nano-technologies has accelerated the development of complex medicines, and at the same time it has revealed new hurdles in regulatory science and accelerating patient access to new therapies. Complex medicines include biologics (e.g. vaccines, gene therapies, recombinant proteins) and non-biological complex drug products (NBCDs) Their diverse nature poses challenges for the development of regulatory guidelines. While complexity is not new in medicines, our technical capacity to measure and analyze data has increased. This requires a determination of which measurements are relevant to demonstrate therapeutic efficacy and safety. Further, many obstacles remain in regulatory harmonization across global authorities, given their different approaches and legal frameworks. To advance research and build consensus, it is necessary to engage together key stakeholders from academia, regulatory bodies, industry, and drug manufacturing. The Non Biological Complex Drugs Working Group hosted at the not-for-profit Foundation Lygature was established to do exactly that. Three action lines were defined: – Create awareness of the intricacies of complex drug products – Build understanding of the challenges with characterization of complex drug products, including the determination of Critical Quality Attributes – Aim for alignment across the globe on rules and regulations for complex drug products. Over the years, many scientific publications have been published with the involvement from scientists of the NBCD WG. The papers have identified the best scientific approaches for complex medicines development and regulation, have outlined outstanding challenges in the assessment of equivalence, and touched upon how to improve timely patient access for new medicines. The discussions continue to facilitate the translation of scientific findings into advancements in medicine for the benefit of patients.