Price cut brings ‘breakthrough’ NICE approval of Perjeta

Breast cancer drug Perjeta has been approved by England’s healthcare cost effectiveness watchdog NICE, a breakthrough achieved after Roche offered a substantial price cut.

The decision has been hailed by a cancer charity as a ‘huge leap forward’ for breast cancer patients – and may signal a shift in UK pharma industry pricing.

The draft ruling is the third positive NICE decision for a cancer drug in as many weeks -a trend enabled by more companies offering major price cuts reductions. However as the price offers made to NICE remain confidential, it’s not clear just how big the discounts are.

Roche has welcomed the decision, but says it wasn’t just simply because it backed down on the price, noting that NICE had also recognised a key clinical trial endpoint for the first time.

The approval is for Perjeta in combination with Herceptin (trastuzumab) and chemotherapy for routine NHS funding as pre-surgery (neoadjuvant) treatment for women with HER2-positive, locally advanced, inflammatory or early-stage breast cancer at high risk of recurrence.

NICE’s committee was persuaded that Perjeta added to Herceptin and chemotherapy was more successful at getting rid of cancer in the breast and lymph nodes before surgery. However, it still insisted that there was a lack of long-term evidence, and said it remained “very uncertain" about how much it could reduce the risk of the disease returning, and its overall impact on survival.

Nevertheless, Perjeta has avoided being put through the ‘managed access’ system of the new Cancer Drugs Fund, and will be funded without any further demands for cost effectiveness data.

This means 1,380 women per year in England and Wales with this aggressive type of breast cancer will be eligible for treatment.

Professor Carole Longson, director of NICE’s centre for health technology assessment said Perjeta’s early approval had made proving its value difficult.

She says the price cut was the decisive change. Patients will receive no more than four cycles of treatment, which has a list price cost of £9,580 ($12,287). The NHS will pay substantially less than this through the agreed confidential discount.

Longson said NICE welcomed the company’s offer to "share the long-term financial risks of a positive recommendation for the NHS" by providing a discount.

"The price discount means that, even with the uncertainties in the evidence highlighted by the committee, pertuzumab represents a cost effective use of NHS money,” she concluded.

Roche’s UK managing director Richard Erwin said the company was ‘delighted’ by NICE’s decision. However he insisted that the breakthrough wasn’t simply down to it cutting Perjeta’s price tag.

"This was not simply about price - the medicine in this setting met the cost effectiveness threshold. This was about NICE accepting that one of the clinical trial endpoints; pathological complete response (pCR), was more likely than not to have an association with longer term survival."

NICE, Roche and charity Breast Cancer Now have remarked on the fact that this decision is the third positive NICE decision on breast cancer medicines in three weeks.

This brings to an end a nine-year period in which no new medicines for breast cancer patients had been approved by NICE.

Richard Erwin welcomed the positive trend, and hoped there was more to come.

"It is critical that this momentum continues so that all patients can access new, innovative treatments in development,” he said.

Mia Rosenblatt, assistant director of Policy and Campaigns at Breast Cancer Now, said:

“This is a huge leap forward. Perjeta is the first addition to primary treatment to be approved by NICE since 2006 and marks the introduction of a new type of breast cancer medicine, to be used before surgery."

One of the biggest rows over NICE guidance in recent years has been between the Institute and Roche over its other breast cancer drug, Kadcyla.

NICE will be meeting to decide on the future of Kadcyla (trastuzumab emtansine) in the metastatic setting in two weeks’ time, and hopes are rising that a similar solution could be found for the drug. NICE first rejected the drug in April 2014, and the two sides have been deadlocked over its future since then. The drug has been available via the CDF, but is among the 31 drugs being re-appraised.

"We very much hope for another positive decision to ensure breast cancer patients in England and Wales have long-term access to this treatment option,” added Richard Erwin.

NICE has also highlighted today that seven of the nine drugs it has reappraised from the ‘legacy’ CDF have gained its approval. These include Novartis’ Afinitor and BMS’ Sprycel, with these companies also providing additional discounts.