How many Italian patients could be treated with precision oncology? That is, with drugs that specifically target mutations that contribute to tumor development? 45%, that is about half. This is what the data from the Italian Register of Mutations for which there are drugs (The Italian Register of Actionable Mutations - RATIONAL) tells us, which involved over 730 men and women, affected by various forms of cancer, being treated in 44 different centers of the peninsula. In total, 45% of patients have specific mutations for which there are drugs already approved by the Italian regulatory body, or in the experimental phase. The study was conducted under the aegis of FICOG (Federation of Italian Cooperative Oncology Groups) and the results were published in the scientific journal European Journal of Cancer.
Tumors, a national program for free access to the most advanced genomic tests is underway
by Tiziana Moriconi
The diffusion of NGS tests
To understand if patients have these mutations it is necessary to use next generation genetic sequencing tests (Next Generation Sequencing or NGS). The Registry also made it possible to photograph the Italian situation with respect to their use. “Their use is still low, especially that of the tests with "wide panels" that we instead offered to some patients enrolled in our study. The collected data are in line with those of other European countries and reaffirm the extreme biological heterogeneity of some pathologies such as lung or breast, pancreatic or biliary tract carcinoma", underlines Nicola Normanno, Director of the Department of Translational Research of the 'National Cancer Institute G. Pascale Foundation of Naples and scientific director of the RATIONAL study. “It is also very interesting to have identified the presence of mutations which may tend to be associated with a risk of certain neoplasms. From these it is possible to start a preventive activity by subjecting the patient's family members to genetic tests capable of highlighting the possible development of an oncological pathology”.
Tumors, multidisciplinary teams and genomic profiling tests are needed for precision medicine
by Irma D'Aria
Access to therapies
Some problems also emerge from the data collected for RATIONAL: of all the patients who have obtained in-depth genomic profiling, only 11% are able to receive the targeted therapies available. “This focuses on an increasingly relevant problem today: guaranteeing access to off-label drugs on the basis of both an actionable molecular alteration, highlighted in NGS, and the subsequent evaluation of the institutional Molecular Tumor Board (MTB) which gave indication to the prescription of the drug”, states Carmine Pinto, President of FICOG. In fact, none of the currently usable methods, such as Law 648/96, Law 326/2003 art. 48 (5% fund), the DM 08/05/2003 (compassionate use), the DM 07/09/2017 (therapeutic use) and the Class Cnn (class C drugs, not negotiated) - can govern off use -label of drugs as required by genomic profiling. “With these methods of access to off-label medicines, the current times are not compatible with clinical practice and important differences in behavior and regional inequalities are determined for access to medicines. A reference indication from AIFA is needed”, continues Pinto. "The draft ministerial decree on MTB currently under discussion intervenes on this issue, but requires better definition and clarity on the levels of evidence and methods of access to medicines".
Cancer, genomic profiling changes treatment for one in 3 patients
by Dario Rubino
A uniform path
“For all these reasons, it is necessary to establish a structured and uniform path for all patients, which facilitates access to medicines as much as possible - adds Normanno. "Also for this reason we want to follow up on the study with more in-depth research and the Registry must be expanded by involving more patients, collecting new data and also offering the possibility of access to drugs to patients with mutations for which therapeutic intervention is possible". “The creation of RATIONAL is a positive example of the collaboration that can be established between public academic centers and private companies - concludes Pinto -. Precision oncology is now a consolidated and essential reality in the fight against many tumours: from some of the most common to rare ones. The Italian national health system may also be at the forefront in this sector but new rules are needed as soon as possible to improve the prospects of the sick".