1657P - The new mutational model in oncology. What changes in welfare, clinical practice, research, and regulatory procedures?

Abstract

BackgroundThe diffusion of next-generation sequencing (NGS)-based tests for comprehensive genomic profiling (CGP), the development of new drugs matching specific genetic alterations, and the recent “agnostic approval” processes of anti-cancer agents represent an important innovation that favors the development of Precision Oncology. This “mutational model” supports and integrates the traditional approach to cancer therapy based on histology. Nevertheless, an uncontrolled use of CGP tests and of mutation-driven drugs can compromise their appropriateness, and put the sustainability of the health system at risk. MethodsA group of experts in pathology, molecular biology, medical oncology, organization of health systems and health economics has developed an organizational model for precision oncology based on multidisciplinarity, on the careful selection of patients to be submitted to CGP tests and on access to off-label drugs in the context of a regulatory framework. ResultsA network of Molecular Tumor Board (MTB) accredited by the Italian Drug Agency (AIFA) according to transparent criteria with regard to composition, activities, traceability, and processing of data can represent the very new tool for managing the mutational model in clinical practice. The multidisciplinary MTBs meet regularly to select patients to be analyzed with CGP tests in order to ensure the appropriateness of the test. The MTBs also discuss therapeutic options on information deriving from NGS analyses. When no clinical trials matching the genetic alterations are available and an off-label drug is recommended, this therapy will be authorized by the Italian Drug Agency and data on the outcome of the patients will be stored in a national registry. ConclusionsThe increasing availability of CGP tests and of possibly active targeted-based agents represents an important opportunity of cancer patients to access to new drugs. The proposed model will allow access of Italian patients to possibly active treatments and at the same time will contribute to ensure the sustainability of this new approach and to increase the knowledge on the activity of target-based agents in specific subgroups of patients. Legal entity responsible for the studyThe authors. FundingFondazione Ricerca&Salute. DisclosureAll authors have declared no conflicts of interest.