Late translational research: putting forward a new model for developing new anti-cancer treatments that addresses the needs of patients and society.

ABSTRACT

Bringing therapeutic innovation and the latest science to routine patient care, while safeguarding principles of affordability and equality, is a challenging mission in the current complex multi-stakeholder environment. Precision oncology and new approaches to clinical trials (methods and clinical setting) have dramatically changed clinical research and the clinical development of new treatments. Improved understanding of molecular biology and immunology paves the way for innovative pharmacological approaches. However, we argue that the evidence generated during the clinical development of these new products for the purpose of obtaining marketing authorisations often does not address fundamental questions concerning the impact of these new interventions on the most relevant clinical outcomes: namely, quality of life and patient survival. Similarly, patient populations (for example defined by biomarkers), treatment duration, and sequence and combination of treatments within current treatment pathways are often poorly defined by clinical developments for regulatory purposes. Finally, the lack of integrated translational research within the pathway of development is a major limiting factor to delivering cost-effective and affordable, evidence-based care to clinical practice. This leaves many gaps in the knowledge on the efficacy and therapeutic use of medicines, which can impose a significant financial burden on healthcare systems, possibly to the detriment of more cost-effective interventions. We argue that policy changes are required to integrate clinical research and healthcare to inform clinical practice. New routes toward optimising the integration of drug development and care are being proposed to achieve this ultimate goal.