Changing the paradigm for therapeutic progress for rare tumors: SPECTArare “Optimizing molecular profiling technologies through partnerships”

Denis Lacombe1

1EORTC, Brussels, Belgium

Abstract

Building on the pan European EORTC SPECTA (Screening Patients for Efficient Clinical Trial Access) already up and running in frequent tumors, the program is being customized for rare tumors. Molecular profiling technologies able to detect multiple gene alterations convey promises to develop new treatments based on robust biological information for unknown and scarce tumor types.  Well-structured prospective programs based on high quality data collection can not only help structuring for systematic collection of rare cases, but also allow for the evolution of new system development for research. There is an absolute necessity to improve the quality of diagnostic, treatment and care for rare diseases. A current challenge is to bring latest and fast developing technologies to the patients ensuring therapeutic progress based on knowledge development platform. Integrating research and care is an even greater need for rare tumors as few therapeutic options are sound, at a time when expertise is still scattered across centers, health care providers and researchers. New forms of platforms such as screening models allow optimal access to new treatments.

SPECTArare is a unique pan European standardized quality assured molecular screening platform for tumor characterization. It allows long term patient follow up and enables continued acquisition of clinical, biological and imaging data after all lines of treatment as part of routine clinical care to understand patterns of resistance and relapses. Access to new treatments is subject to revisited development methods currently being addressed by regulators and the pharmaceutical industry sector. For instance adaptive licensing may evolve alongside new forms of clinical studies such as pre-approval bench marking studies and post approval observational studies for clinical situations where the classical drug development models are no longer applicable. Therefore, new methodological advances are needed and can be best developed through multi-stakeholder partnerships and collaboration through molecular screening platforms.