The National Institute for Health and Care Excellence (NICE) is very disappointed that Roche, the manufacturer of Kadcyla (trastuzumab emtansine) has decided not to offer its new treatment at a price that would enable it to be available for routine use in the NHS.
Kadcyla is currently only available (in England) through the Cancer Drugs Fund. NICE wanted to secure the treatment's long-term future in the NHS by making a recommendation for routine funding from NHS England's specialised commissioning budget.
Kadcyla treats people with HER2-positive breast cancer that has spread to other parts of the body, cannot be surgically removed and has stopped responding to initial treatment.
The treatment is estimated to cost more than £90,000 per patient at its full list price.
In April, NICE published draft guidance that did not recommend trastuzumab emtansine. It concluded that its high price made it impossible for it to recommend despite evidence of its benefits to patients.
NICE has now issued final draft guidance reiterating that view.
Sir Andrew Dillon, NICE Chief Executive said: "Although Roche proposed a discount to the full list price of Kadcyla, it made little difference to its value for money, leaving it well above the top of our specially extended range of cost effectiveness for cancer drugs.
"We are really disappointed that Roche were not able to demonstrate more flexibility to help us make a positive recommendation. The company is well aware that we could not have recommended Kadcyla at the price it proposed."
Sir Andrew continued: "Roche considers that the 2014 Pharmaceutical Price Regulation Scheme (PPRS) agreement includes an expectation that NICE will ignore the price a company asks for its product, because the agreement provides for a rebate to be paid by the pharmaceutical industry to the NHS where expenditure on branded drugs rises above an agreed level.
"The agreement does not contain this expectation because both the Association of the British Pharmaceutical Industry and the Department of Health agree that obtaining value for money from new drugs is in the interests of the industry and the NHS."
Women in England and Wales denied life-extending breast cancer drug by NICE
Roche will appeal today's decision by the National Institute for Health and Care Excellence (NICE) to block routine NHS access to the targeted breast cancer drug, Kadcyla. NICE's rejection comes despite Roche offering to cut the price of the drug, by submitting a Patient Access Scheme (PAS).
Kadcyla is the eighth consecutive therapy for advanced breast cancer to be rejected by NICE since 2011, despite the fact it can extend the lives of women with advanced HER2-positive breast cancer by an additional 5.8 months compared with the currently licensed treatment combination, lapatinib plus capecitabine (overall survival 30.9 months vs. 25.1 months, HR=0.68, p=0.0006).
This decision means that women living in England must rely on their clinicians' successful application to the Cancer Drugs Fund (CDF) - a temporary funding solution available only until 2016 - in order to receive Kadcyla. No such fund exists in Wales, Scotland and Northern Ireland, meaning that many patients in the UK may not receive Kadcyla at all.
Today's decision sees the UK continuing to lag behind other countries across Europe with comparable purchasing power in terms of access to innovative cancer medicines. Kadcyla is currently available and routinely reimbursed without restriction, in many European countries including Finland, Denmark, Austria, Norway, Sweden and Switzerland.
"NICE's rejection of Kadcyla demonstrates quite simply that their current system is broken, not fit for purpose and in need of a complete overhaul when it comes to advanced cancer," said Dr Jayson Dallas, General Manager, Roche Products Limited. "Despite Roche offering a significant discount we are once again disappointed that NICE has not shown any flexibility on access to Kadcyla - a drug with qualities that NICE Chief Executive, Sir Andrew Dillon, described on live television as "impressive". Kadcyla's linker technology has taken more than 30 years to develop and the drug itself is a product of more than 15 years of research and development. Refusing patients access to this drug is an incredible injustice and tantamount to turning the clock back in cancer research and development. We plan to appeal this decision."
NICE's decision also underlines the systemic failure to implement the recently negotiated Pharmaceutical Pricing Regulation Scheme (PPRS), agreed between the pharmaceutical industry and Department of Health, to cap the overall drugs budget in the NHS so that patients get access to the latest treatments. NICE has declined to factor in the cost assurance guaranteed by the PPRS in its calculations which would mean the future drug bill cannot be overspent and would remove the burden of additional cost to the NHS.
Under NICE's current process Kadcyla would need to be discounted by 60% to meet the NICE cost-effectiveness threshold. This and other recent rejections highlight that the NICE process is no longer fit for purpose to provide patients access to new cancer medicines.
Professor Paul Ellis, Consultant Oncologist, King's College, London, said: "Kadcyla represents a significant step forward in the fight against a particularly aggressive and notoriously difficult-to-treat form of the disease. NICE's decision not to fund the drug for routine NHS use serves as a real blow for all those involved. Due to the innovative way that Kadcyla works, (by linking a targeted antibody with a highly potent chemotherapy using 'stable linker' technology), Kadcyla is able to deliver significantly increased efficacy to patients without the toxicity associated with potent chemotherapy drugs; ultimately meaning that patients can live for longer with a better quality of life. While patients in England can access the drug via the CDF, we need to work towards a solution to ensure long-term access to this truly revolutionary drug."
Kadcyla is a first of its kind in breast cancer. Kadcyla consists of the HER2-targeted antibody, contained in the medicine Herceptin® (trastuzumab), linked with a potent chemotherapy agent and initiates a unique, double attack on cancer cells: first, by seeking out the faulty cells and blocking their growth signals and second, by penetrating the cell's outer surface and releasing a payload of potent chemotherapy to destroy it from within. This targeted approach means that many patients are spared some of the traditional side effects of chemotherapy, such as hair loss.
Kadcyla is licensed for the treatment of women with advanced HER2-positive breast cancer who have previously failed treatment with Herceptin® (trastuzumab) and a taxane chemotherapy, separately or in combination. Patients who received Kadcyla experienced fewer severe side effects than those who received lapatinib plus capecitabine, allowing patients to live a better quality of life during treatment. In the EMILIA study, the most common grade 3 (severe) or higher side effects for those patients receiving lapatinib plus capecitabine chemotherapy, compared to Kadcyla were diarrhoea (20.7% vs. 1.6%), hand-foot syndrome (16.4% vs. 0%) and vomiting (4.5% vs. 0.8%).8f In the study, the most common grade 3 side effects associated with Kadcyla versus lapatinib plus capecitabine, were low platelet count (12.9% vs. 0.2%), increased levels of enzymes released by the liver (aspartate aminotransferase or 'AST': 4.3% vs. 0.8%; alanine aminotransferase or 'ALT': 2.9% vs. 1.4%) and anaemia (2.7% vs. 1.6%).8g Like other treatments that target HER2, heart monitoring is required before and during treatment.