The National Institute of Health and Care Excellence (NICE) has approved the combined use of nivolumab (Opdivo) and ipilimumab (Yervoy) for patients who have previously been treated metastatic bowel cancer in England.
Around four in 100 metastatic bowel cancers have a lot more DNA mutations than others, as they're unable to repair any mistakes that are made in their DNA. These cancers are said to have high 'microsatellite instability' (MSI) or 'mismatch repair (MMR) deficiency," which can make them more aggressive, and are associated with a poorer outlook and greater risk of death. Despite this, there aren't specific treatments for this type of bowel cancer, so people are offered the same treatment whether or not their cancer has this inability to fix mistakes in its DNA.
Immunotherapies harness the body's immune system to fight cancer. Researchers have found that bowel cancer tumours with high MSI or MMR deficiency have a lot of immune cells within them, but the cancer stops them from working. Nivolumab and ipilimumab block the cancer's ability to do this, allowing the immune cells to attack the tumours.
People with previously treated metastatic bowel cancer are currently offered combination chemotherapy as their second-line treatment. However, following the NICE decision, patients with bowel cancer that has high MSI or an MMR deficiency will now have an option of a combination of immunotherapies.
The clinical evidence for the effectiveness of this treatment came from the single-arm, phase 2 CheckMate 142 study. The trial included 119 people with metastatic colorectal cancer with high MSI or MMR deficiency, previously treated with combination therapies. As a single-arm trial, the study did not directly compare the use of nivolumab plus ipilimumab with other treatments, so the data had to be compared indirectly with data about current treatments.
The NICE committee concluded that despite this uncertainty, the trial showed that the benefits of the treatment, measured by overall survival for patients and the amount of time where their cancer did not get worse, was likely to be greater than current standard care.
Overall, the treatment was considered cost-effective for use in the NHS by NICE and will now be an option on the NHS in England. NICE decisions are usually adopted in Wales and Northern Ireland as well, so the decision is likely to affect patients in all three nations. Scotland has a separate process for reviewing drugs.