The Italian Association of Medical Oncology (Aiom) has formally requested that Revolution Medicines, a US-based biotech company founded in 2019, establish a compassionate-use program in Italy for daraxonrasib, its targeted therapy for metastatic pancreatic cancer.
The request highlights a regulatory gap between the United States and Europe. While daraxonrasib has already received approval from the US Food and Drug Administration, the drug has not yet been approved by the European Medicines Agency. Notably, Revolution Medicines has not submitted a registration dossier to the EMA, leaving European patients without access to the therapy.
Demonstrable Clinical Benefit
Daraxonrasib has shown significant promise in clinical trials, with data indicating it has doubled survival in patients with metastatic pancreatic cancer. This substantial efficacy gain has prompted Italian oncologists to argue that patients should not be denied access while awaiting formal European regulatory processes.
Massimo Di Maio, President of Aiom, articulated the association’s position: “We ask the company to create a compassionate-use program so that daraxonrasib is available to Italian patients for free while we await regulatory approval, as has already happened in the United States.”
Access and Cost Concerns
The Italian medical community has identified two primary obstacles to patient access. First, the absence of an established compassionate-use pathway means that patients with metastatic pancreatic cancer currently have no legal mechanism to receive daraxonrasib in Italy before EMA approval. Second, importing the drug through private channels would impose significant financial burdens on patients and healthcare systems, creating an ethical barrier to treatment.
Compassionate-use programs, also referred to as expanded access programs, allow patients with serious or life-threatening conditions to receive investigational or unapproved drugs outside of clinical trials. These programs are designed to bridge the gap between regulatory approval in one jurisdiction and availability in another, particularly when standard treatment options are exhausted.
European Ecosystem Implications
The Aiom request reflects broader challenges within the European healthcare landscape regarding timely access to innovative oncology treatments. While the European Medicines Agency maintains rigorous approval standards to ensure drug safety and efficacy, the regulatory timeline often extends significantly beyond approvals in other major markets. This creates situations where European patients face delayed access to potentially life-saving therapies.
The case of daraxonrasib demonstrates how the absence of harmonized early-access mechanisms across European countries can disadvantage patients. Unlike some biotech companies that proactively establish compassionate-use programs to serve patients in regions awaiting regulatory approval, Revolution Medicines has not yet committed to such an initiative in Europe.
As the European startup and biotech ecosystem continues to mature, questions about equitable patient access and the balance between regulatory caution and therapeutic urgency remain central to ongoing policy discussions among European health authorities and industry stakeholders.