Treatment of HER2-postive breast cancer
November 22, 2016
By Amy Yip, BioSpace.com Contributor
The National Institute for Clinical Excellence (NICE), the UK’s drug watchdog, has finally given its backing for the National Health Service’s (NHS’s) approval of Roche (RHHBY)’s Perjeta (pertuzumab) for the treatment of human epidermal growth factor receptor 2 (HER2)-positive breast cancer.
Three years after its initial approval by both the FDA and the European Medicines Agency (EMA), the HER2-targeting monoclonal antibody is being recommended by NICE for use in combination with Genentech (RHHBY)’s Herceptin (trastuzumab) + docetaxel for the neoadjuvant treatment of HER2-postive breast cancer. Notably, this will be the first new breast cancer drug to be approved by NICE for routine NHS funding in almost a decade.
During the last ten years, the cost of Perjeta has been covered by the recently remodelled Cancer Drugs Fund (CDF). In most Western markets, the combination of Perjeta + Herceptin + docetaxel has become the new standard of care for the first-line treatment of HER2-postive breast cancer. NICE initially declined to support Perjeta’s approval in 2013 for the first-line treatment of metastatic HER2-positive breast cancer, and then declined again in May 2016 for Perjeta’s use in the neoadjuvant setting, based on a lack of proven cost-effectiveness due to the high cost of treatment and lack of data on the long-term patient benefit.
At the time, NICE issued a statement that said due to its combination with Herceptin in the first-line metastatic setting and the resulting increased duration of treatment, Perjeta, even when priced at $0, was not cost-effective enough to necessitate approval.
This refusal has since gained notoriety as an example of the drawbacks of health technology assessment and cost-effectiveness analyses in the assessment of new cancer drugs. NICE’s cost-effectiveness assessment thresholds have been criticised as being archaic and poorly adapted to the challenge of fairly and accurately assessing the benefits of modern cancer therapies.
Despite mounting controversy regarding its decision about Perjeta, NICE continued to resist the drug’s approval, stating that the uncertainties surrounding Perjeta use on long-term, real-world survival meant that the agency could not recommend it as a cost-effective use of state funds. However, with Roche’s new pledge to provide the drug to the NHS at an undisclosed discount for patients in the neoadjuvant setting and to share the long–term financial risks, the two parties have finally come to an agreement.
Perjeta’s widespread adoption has been based on the results of the Phase III CLEOPATRA study released in 2013, where treatment with the triple combination extended patient progression free survival by more than six months, compared to Herceptin + docetaxel alone (18.5 months versus 12.4 months). Overall survival data also indicated that the triple combination reduced the risk of death by 34% compared to Herceptin + docetaxel alone.
