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NICE Expands Approval Range for Enhertu in Advanced Breast Cancer

The National Institute for Health and Care Excellence (NICE) today issued final draft guidance expanding the use of trastuzumab deruxtecan (Enhertu, Daiichi Sankyo UK) to more people with HER2‑positive breast cancer. The drug is already recommended for use later in the treatment pathway and NICE said its decision would extend use to approximately 600 more people per year, earlier in the pathway. The decision was hailed as "fantastic news" by one cancer charity.

Trastuzumab deruxtecan is an antibody-drug conjugate containing a humanised anti-HER2 IgG1 monoclonal antibody with the same amino acid sequence as trastuzumab, produced by Chinese Hamster Ovary cells, covalently linked to DXd, an exatecan derivative and a topoisomerase I inhibitor, via a tetrapeptide-based cleavable linker. Approximately 8 molecules of deruxtecan are attached to each antibody molecule.

Requirement for Prior Anti-HER2 Therapy Cut

The drug is licensed as monotherapy for the treatment of adult patients with unresectable or metastatic HER2-positive breast cancer who have received one or more prior anti-HER2-based regimens. Until now NICE has recommended it within a managed access arrangement (the Cancer Drugs Fund) for adults only after they've had two or more anti-HER2 therapies – such as pertuzumab with trastuzumab and docetaxel, or trastuzumab with paclitaxel. Today’s decision means it will now be available after only one anti-HER2 treatment.

NICE said that clinical trial evidence showed that trastuzumab deruxtecan increased time to progression compared with standard care with trastuzumab emtansine (Kadcyla, Roche). However there was not enough evidence yet on comparative survival because the clinical trial comparing trastuzumab deruxtecan and trastuzumab emtansine (DESTINY-Breast03) is still ongoing. "This means the cost-effectiveness estimates are highly uncertain and Enhertu cannot be recommended for routine use in the NHS," NICE said.

Use Approved Only Within Managed Access Arrangements

Therefore the drug was recommended for use within a managed access arrangement. "This means people can have the treatment while more evidence about its effectiveness is generated. NICE will then use this data to recommend whether the medicine should be made routinely available on the NHS."

The independent appraisal committee concluded that trastuzumab deruxtecan "could be cost-effective" if further evidence from the trial and from NHS practice showed a survival advantage.

Helen Knight, director of medicines evaluation at NICE, said: "Today's draft guidance is good news for people with this type of advanced breast cancer, who often experience severe and debilitating symptoms.

"It also means NICE has made positive recommendations in all 18 of its appraisals of breast cancer medicines since March 2018. These are all now available for clinicians to use in the treatment of thousands of NHS patients, and demonstrate how NICE works successfully at the interface of health and care and the life sciences industry to enable early access to innovation."

'Cutting Edge Drug' Offers Hope

NHS England Cancer Drugs Fund clinical lead Professor Peter Clark said: "This cutting-edge drug will give hundreds of patients with secondary incurable breast cancer hope, increasing the amount of time people have before their cancer gets worse, and allowing them to live normal, healthy lives for longer.

"The NHS is committed to providing the very best treatments for its patients and trastuzumab deruxtecan is just the latest of more than 100 cancer treatments that have been fast-tracked for use on the NHS through the Cancer Drugs Fund, benefitting more than 80,000 patients."

Patients 'Desperately Need New Effective Treatments'

In response to the announcement, Baroness Delyth Morgan, chief executive of Breast Cancer Now, said: "Today's approval of trastuzumab deruxtecan (Enhertu) for use on the Cancer Drugs Fund is fantastic news for certain patients with HER2 positive incurable secondary breast cancer who desperately need new effective treatments.

"This targeted treatment can significantly slow the spread of the disease compared with the current standard treatment, giving people more time to continue doing the things that matter to them. Crucially, this treatment brings people living with incurable secondary breast cancer the hope of more time to live, and we look forward to further results demonstrating its full potential.  

"This decision highlights the continued importance of the Cancer Drugs Fund in enabling promising treatments to reach patients on the NHS quickly, and it shows what is possible when NHS England, NICE and the pharmaceutical industry work together."

However, she added: "Now, the drug company and the Scottish Medicines Consortium must work quickly to ensure women in Scotland have the chance to benefit too."

Enhertu costs £1,455 per vial containing 100 mg powder for concentrate for solution for infusion. The average cost of a course of treatment at list price is £118,000. The company has a confidential commercial arrangement making trastuzumab deruxtecan available to the NHS at a discount.

Final guidance on trastuzumab deruxtecan is expected to be published in February 2023.