Colorectal cancer: Aifa green light for therapeutic mix doubling survival

Italia is the first country in Europe to grant first-line access through the National Health Service to a new therapeutic combination for patients with metastatic colorectal cancer with BRAF mutation, one of the most serious and inauspicious forms of the disease. The approval by Aifa was announced at the Goim (Gruppo Oncologico Italia Meridionale) congress in Bari, Italy.

The combination of two molecularly targeted drugs, encorafenib and cetuximab, combined with traditional chemotherapy, has gone from being 'last resort' to first choice therapy. Approximately 800 people a year will benefit from this treatment in Italy, i.e. 8-10% of patients with metastatic disease who carry the BRAF mutation, as part of a tumour that in Italia registers around 48,000 new diagnoses every year. While Europe is waiting for the formal go-ahead from the Ema, at the instigation of the Goim, the Italian drug agency has shortened the timeframe by activating an early access pathway to therapy through the procedure envisaged by law 648/96. This tool allows innovative drugs to be made available before the conclusion of the European bureaucratic process when there is an urgent clinical need and overwhelming scientific evidence.

"Aifa's decision is based on data from the randomised phase III Breakwater study," explains Fortunato Ciardiello, one of the authors of the study as well as professor of medical oncology at the University of Campania Luigi Vanvitelli in Naples, Italy, "and represents a turning point for a patient population that, until a few years ago, had very limited treatment options and extremely rapid disease progression.

The results, which were published in the New England Journal of Medicine, showed that the new combination doubles patients' overall survival from a median of around 15-16 months to more than 30. 'This treatment represents a turning point for a patient population that, until a few years ago, had very limited treatment options and extremely rapid disease progression,' Ciardiello emphasises. 'Historically, around half of these patients did not even get to receive a second line of treatment. The use of molecular targeted therapy only in advanced stages did not allow the clinical advantage observed in the trials to be regained. Whereas the early introduction of molecular targeted therapy, in combination with chemotherapy, radically changes the clinical history of the disease, making it more controllable and doubling life expectancy'.

In the United States, based on the results of the BREAKWATER study, this approach has already entered clinical practice last year as a new therapeutic reference. "As Gruppo Oncologico dell'Italia Meridionale (Southern Italy Oncology Group), we have contributed to speeding up Italy's access to this therapeutic opportunity," concludes Roberto Bordonaro, GOIM president and director of the Oncology Department and Complex Structure of Medical Oncology at the Garibaldi Hospital in Catania. The procedure envisaged by law 648/96 demonstrates that the National Health Service has the tools at its disposal to make innovative therapies available when there is robust scientific evidence and a high clinical need, avoiding delays that, at such an advanced stage of the disease, can directly affect survival.