A pioneering life-extending cancer drug trialled in the North East has not been recommended for use on the NHS, a decision which has shocked and baffled its backers.
Draft guidance published yesterday by the National Institute for Health and Clinical Excellence (Nice) has gone against the use of Perjeta.
Newcastle’s Freeman Hospital was one of the key establishments involved in trials of the drug, which has been shown to boost survival for previously untreated HER2-positive breast cancer patients.
Health campaigners and experts hope that the draft decision will be reversed before officials make a final determination on Perjeta, also known as pertuzumab, as a treatment for an advanced form of the disease.
Dr Mark Verrill, consultant medical oncologist at Newcastle Hospitals NHS Foundation Trust said: “Nice have missed the point in their draft guidance on the use of Perjeta.
“They have said that the drug is not recommended because ‘clinical trial data could not predict how long the drug might extend people’s lives for’. This is based on fewer patients who received the trial treatment dying than would be required to calculate the ‘average’ benefit Perjeta produces.
“In effect the treatment is too good for Nice to be able to calculate financial cost of the benefit it provides.
“Perjeta builds on current Nice approved standards of care and there is absolutely no doubt that it substantially improves the outcome of treatment in HER-2 positive patients with metastatic breast cancer.
“Currently we are able to use Perjeta in appropriate patients because of the Cancer Drugs Fund which provides treatments that Nice have refused or not considered.
“It is hugely frustrating to look towards 2014 not knowing if the Cancer Drugs Fund will continue and so whether this innovative and effective drug will be available for patients.”
Perjeta was widely welcomed when first introduced by manufacturers Roche. It targets the HER-2 gene found in 25% of breast cancer patients. Data from a Phase III Cleopatra study revealed that the drug reduces the risk of death by 34%.
Patients were also seen to live on average 6.1 months longer without their cancer getting worse, compared with those receiving Herceptin and chemotherapy alone.
After one year on treatment, 94% of patients in the Perjeta arm were still alive, compared with 89% in the comparator arm.
Every year, around 2,000 women are diagnosed with breast cancer in the North East. At present, patients can access the drug through the Cancer Drugs Fund but it is not routinely available through the health service.
But Nice said that it cannot recommend it for widespread use because “clinical trial data could not predict how long the drug might extend people’s lives for, yet it costs much more than current NHS treatments”, a spokeswoman said.
The organisation has now launched a consultation to hear the views of charities, patient groups and drug manufacturers.
Nice chief executive, Sir Andrew Dillon, said: “The appraisal committee couldn’t be sure of the benefits of pertuzumab.
“The main clinical trial did not reflect current medical practice in the UK and despite the research data suggesting the treatment could help delay the growth and spread of the disease, the evidence was not robust enough to confirm for how long pertuzumab may actually extend people’s lives.
“The committee also noted that even the manufacturer estimated that the treatment would not be considered cost-effective for the NHS. We have now launched a consultation to gather comments from interested parties to develop the guidance further.”
Mia Rosenblatt, head of policy and campaigns at Breast Cancer Campaign, said: “It’s disappointing that a drug that has shown real benefit to women with advanced HER-2 positive breast cancer has not received Nice approval.
“However this is an interim decision and we hope that appropriate evidence to show its benefit can be provided and an agreement on cost made so that the NHS see this as a cost-effective treatment that could extend lives.”