Therapeutic and research opportunities in refractory, rare, and neglected cancers
2 Feb , 2026
Oncology has made great strides against the most common cancers, but some neoplasms remain difficult to treat.
Refractory, rare, and neglected tumors represent an ongoing challenge: in Italy alone, there are about 90,000 new diagnoses each year, accounting for 20–25% of the total.
These diseases, though diverse, share similar hurdles.
Refractory tumors resist currently available treatments and have a five-year survival rate below 50%, such as pancreatic cancer and glioblastoma. In the United States, the 2012 Recalcitrant Cancer Research Act recognized these neoplasms as a national priority, mandating targeted research plans. Rare tumors, defined by the European Union (EU) as those with an incidence below 6 cases per 100,000 people per year, receive limited research funding.
Neglected tumors, even when not rare, attract little interest from the pharmaceutical industry.
For all of them, the common problem is the difficulty in gathering sufficient data to conduct randomized controlled trials (RCTs), thus delaying the arrival of new therapies.
A major breakthrough comes from molecular medicine.
Today, it is possible to classify tumors not only by the affected organ but also by the genetic alterations that cause them. This approach allows testing targeted drugs on patients with the same mutation, even if they have different tumors. New DNA analysis technologies, such as next-generation sequencing (NGS), also enable the identification of previously unknown therapeutic targets.
These innovations are complemented by new strategies: more effective immunotherapies, the use of clinical data collected in everyday practice (real-world evidence, RWE), and artificial intelligence (AI) to discover new uses for existing drugs.
To translate these opportunities into concrete treatments, however, international collaboration, specialized networks like the National Rare Tumors Network, and shared data registries are essential.
Investing in this sector is both an ethical duty and an opportunity to advance the entire field of experimental, surgical, and clinical oncology.
Credits: This article was originally written in Italian for ilmattino.it
