Stunning results for Roche’s Perjeta in extending lives
Trials of Roche's Perjeta show it could help extend the lives of women with advanced breast cancer by over a year compared to existing treatments.
The results from the CLEOPATRA study are a stunning success, and Perjeta (pertuzumab) looks set to become the standard of care in these patients with HER2 positive tumours.
The data, presented at Europe's annual cancer congress ESMO, showed that Perjeta, when added to Herceptin and chemotherapy, extended the lives of people with previously untreated metastatic breast cancer by 15.7 months compared to Herceptin and chemotherapy alone. Overall, this meant those taking the Perjeta combination lived a median of 56.5 months, compared to 40.8 months on the existing regimen.
This survival rate is the longest observed to date in people with advanced HER2 positive cancer. Specialists in oncology and patients have become accustomed to seeing a few months of extra life added by new cancer treatments, which means the CLEOPATRA results are truly exceptional.
The breakthrough only helps those with the HER2 positive form of breast cancer, however, who account for around 20 per cent of patients. Remarkably, Roche's advances with Herceptin and Perjeta means that patients with HER2, a more aggressive sub-type, actually now have a better prognosis than those who are HER negative.
Roche is now seeking regulatory approval for use of the drug at a far earlier stage, in the neoadjuvant setting (before surgery) in early breast cancer.
Pricing controversy
Despite the understandable excitement about the drug's results, there remains one major obstacle to its wider uptake: its very high price. In the US, Perjeta is priced at $71,000, but as the drug does not replace Herceptin but is added to it, the overall price for both drugs is around £115,000 for a year's treatment. However the combination is likely to be used by many patients for over a year, meaning its cumulative price will be even higher.
While there are fewer restrictions on use of new drugs because of price in the US, European healthcare systems and other regions frequently restrict access on this basis. In the UK, cost-effectiveness body NICE rejected the drug in draft guidance published in August 2013, saying the main clinical trial presented at the time did not reflect UK medical practice, and that the evidence was not robust enough.
However it was the drug's cost that was the overwhelming block to its approval, and this will be clearer now, since questions about the robustness of the data have been answered.
Reaction to the results were swift at the ESMO conference, and on social media. Industry analyst Sally Church tweeted: "Anyone from NICE here at #ESMO14? I dare them to decline Perjeta based on #CLEOPATRA it is now the 1L SOC [first line standard of care] whether they like it or not!"
In contrast, Caitlin Palframan, senior policy manager at the UK charity Breakthrough Breast Cancer reiterated her organisation's demand for lower prices. "Join @BreakthroughBC to #demandafairprice."
Following objections to NICE's draft decision on Perjeta last year, the organisation has delayed its final ruling on the drug, with discussions about price and affordability ongoing. However, patients in England have access to Perjeta and other treatments not approved by NICE through the Cancer Drugs Fund.
