Roche's Perjeta long-term benefits "too uncertain," says NICE in draft guidance

UK medicines cost-effectiveness watchdog the National Institute for Health and Care Excellence (NICE) has today issued draft guidance not recommending Perjeta (pertuzumab), from Swiss pharma giant Roche (ROG: SIX), to treat HER2-positive breast cancer because it is uncertain how the responses to treatment seen in the clinical trials translate into long term benefits for patients.

Pertuzumab is licensed for use in combination with trastuzumab (Roche’s Herceptin) and docetaxel (a type of chemotherapy) before breast cancer surgery to shrink the cancer so that it becomes operable. The draft guidance looks at pertuzumab used in this way to treat HER2-positive breast cancer that is locally advanced, inflammatory, or early-stage and at high risk of coming.

The NICE Appraisal Committee concluded that there was a lack of long-term evidence comparing pertuzumab with other treatments used before breast cancer surgery. There was evidence that adding pertuzumab to trastuzumab and docetaxel was more successful at getting rid of cancer in the breast and lymph nodes before surgery, but it was very uncertain about the extent to which this would reduce the risk of the disease recurring and result in longer survival.

Pertuzumab costs £2,395 ($3,437) per 420mg vial (excluding VAT). The total cost of treatment with pertuzumab ranges from £7,185 (three cycles of treatment) to £16,765 (six cycles of treatment).

“In order to be able to recommend pertuzumab as an addition to trastuzumab and chemotherapy, the committee needed to have more evidence of its long-term clinical benefits, particularly its impact on overall survival. On the basis of the evidence presented, the committee was very unsure about the extent of these benefits,” said Sir Andrew Dillon, chief executive of the NICE. “Taking all the uncertainties around the clinical effectiveness of pertuzumab into account, as well as uncertainties with the economic data presented by the company, the committee concluded that it could not recommend the drug for the treatment of HER2-positive breast cancer before surgery as a good use of NHS resources,” Sir Andrew added.

Roche reiterates concerns about NICE methodology

Richard Erwin, general manager, Roche UK, said:  “It is a real concern for cancer patients that Perjeta, the neoadjuvant treatment, may not be available for patients in the UK when its clinical efficacy has been proven and it is within the cost effectiveness range for this indication. This again demonstrates why we are concerned that NICE’s methodology does not work for some medicines and therapy areas.  We need to create a more sustainable and flexible approval system that makes the assessment of cancer medicines as streamlined as possible. Roche will play its part in this.”

This provisional recommendation is a tangible example of the fears expressed by 15 leading cancer charities earlier this week that nothing has been done to update NICE’s inflexible assessment methods, said Roche, noting that Cancer charities wrote to UK Prime Minister, David Cameron, asking for the government’s urgent intervention to review NICE’s “outdated mechanisms,” meaning that “many clinically-effective treatments will now struggle to gain approval.”